Track market-moving stories before they get noisy

The market expects BioMarin Pharmaceutical (BMRN - Free Report) to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended September 2026. This widely-known consensus outlook is important in assessing the company's earnings picture, but a powerful factor that might influence its near-term stock price is how the actual results compare to these estimates.

The stock might move higher if these key numbers top expectations in the upcoming earnings report. On the other hand, if they miss, the stock may move lower.

While management's discussion of business conditions on the earnings call will mostly determine the sustainability of the immediate price change and future earnings expectations, it's worth having a handicapping insight into the odds of a positive EPS surprise.

Zacks Consensus EstimateThis rare disease biopharmaceutical is expected to post quarterly earnings of $1.35 per share in its upcoming report, which represents a year-over-year change of +1025%.

Revenues are expected to be $1.03 billion, up 32.7% from the year-ago quarter.

Estimate Revisions TrendThe consensus EPS estimate for the quarter has been revised 0.1% higher over the last 30 days to the current level. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period.

Investors should keep in mind that the direction of estimate revisions by each of the covering analysts may not always get reflected in the aggregate change.

Price, Consensus and EPS Surprise

Earnings WhisperEstimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. Our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction) -- has this insight at its core.

The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier.

Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only.

A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP.

Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell).

How Have the Numbers Shaped Up for BioMarin?For BioMarin, the Most Accurate Estimate is higher than the Zacks Consensus Estimate, suggesting that analysts have recently become bullish on the company's earnings prospects. This has resulted in an Earnings ESP of +9.40%.

On the other hand, the stock currently carries a Zacks Rank of #3.

So, this combination indicates that BioMarin will most likely beat the consensus EPS estimate.

Does Earnings Surprise History Hold Any Clue?Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number.

For the last reported quarter, it was expected that BioMarin would post earnings of $0.96 per share when it actually produced earnings of $1.20, delivering a surprise of +25.00%.

Over the last four quarters, the company has beaten consensus EPS estimates three times.

Bottom LineAn earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss.

That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported.

BioMarin appears a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release.

Stay on top of upcoming earnings announcements with the Zacks Earnings Calendar.

For new and old investors, taking full advantage of the stock market and investing with confidence are common goals. Zacks Premium provides lots of different ways to do both.

The research service features daily updates of the Zacks Rank and Zacks Industry Rank, full access to the Zacks #1 Rank List, Equity Research reports, and Premium stock screens, all of which will help you become a smarter, more confident investor.

It also includes access to the Zacks Style Scores.

What are the Zacks Style Scores? The Zacks Style Scores, developed alongside the Zacks Rank, are complementary indicators that rate stocks based on three widely-followed investing methodologies; they also help investors pick stocks with the best chances of beating the market over the next 30 days.

Each stock is assigned a rating of A, B, C, D, or F based on their value, growth, and momentum characteristics. Just like in school, an A is better than a B, a B is better than a C, and so on -- that means the better the score, the better chance the stock will outperform.

The Style Scores are broken down into four categories:

Value ScoreValue investors love finding good stocks at good prices, especially before the broader market catches on to a stock's true value. Utilizing ratios like P/E, PEG, Price/Sales, Price/Cash Flow, and many other multiples, the Value Style Score identifies the most attractive and most discounted stocks.

Growth ScoreGrowth investors are more concerned with a stock's future prospects, and the overall financial health and strength of a company. Thus, the Growth Style Score analyzes characteristics like projected and historic earnings, sales, and cash flow to find stocks that will see sustainable growth over time.

Momentum ScoreMomentum investors, who live by the saying "the trend is your friend," are most interested in taking advantage of upward or downward trends in a stock's price or earnings outlook. Utilizing one-week price change and the monthly percentage change in earnings estimates, among other factors, the Momentum Style Score can help determine favorable times to buy high-momentum stocks.

VGM ScoreIf you want a combination of all three Style Scores, then the VGM Score will be your friend. It rates each stock on their combined weighted styles, helping you find the companies with the most attractive value, best growth forecast, and most promising momentum. It's also one of the best indicators to use with the Zacks Rank.

How Style Scores Work with the Zacks Rank The Zacks Rank, which is a proprietary stock-rating model, employs earnings estimate revisions, or changes to a company's earnings expectations, to make building a winning portfolio easier.

It's highly successful, with #1 (Strong Buy) stocks producing an unmatched +23.86% average annual return since 1988. That's more than double the S&P 500. But because of the large number of stocks we rate, there are over 200 companies with a Strong Buy rank, plus another 600 with a #2 (Buy) rank, on any given day.

But it can feel overwhelming to pick the right stocks for you and your investing goals with over 800 top-rated stocks to choose from.

That's where the Style Scores come in.

To have the best chance of big returns, you'll want to always consider stocks with a Zacks Rank #1 or #2 that also have Style Scores of A or B, which will give you the highest probability of success. If you're looking at stocks with a #3 (Hold) rank, it's important they have Scores of A or B as well to ensure as much upside potential as possible.

The direction of a stock's earnings estimate revisions should always be a key factor when choosing which stocks to buy, since the Scores were created to work together with the Zacks Rank.

For instance, a stock with a #4 (Sell) or #5 (Strong Sell) rating, even one that boasts Scores of A and B, still has a downward-trending earnings forecast, and a much greater likelihood its share price will decline as well.

Thus, the more stocks you own with a #1 or #2 Rank and Scores of A or B, the better.

Stock to Watch: BioMarin Pharmaceutical (BMRN - Free Report) San Rafael, CA-based BioMarin Pharmaceutical Inc. focuses on the development and commercialization of treatments for life-threatening severe medical conditions, mainly for children.

BMRN is a #3 (Hold) on the Zacks Rank, with a VGM Score of B.

It also boasts a Value Style Score of B thanks to attractive valuation metrics like a forward P/E ratio of 11.13; value investors should take notice.

Three analysts revised their earnings estimate upwards in the last 60 days for fiscal 2026. The Zacks Consensus Estimate has increased $0.03 to $4.98 per share. BMRN boasts an average earnings surprise of +67.5%.

With a solid Zacks Rank and top-tier Value and VGM Style Scores, BMRN should be on investors' short list.

New analysis showcases impact of VOXZOGO® (vosoritide) in foramen magnum and craniocervical junction growth in children with achondroplasia, including infants

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced it will present new data from 13 studies at the American Society for Bone and Mineral Research 2026 Annual Meeting (ASBMR) in Boston, including results from studies of VOXZOGO® (vosoritide) in children with achondroplasia and hypochondroplasia. The company will also share data from studies of the investigational long-acting medicine BMN 333.

The data include encouraging new imaging results from a post-hoc analysis of a randomized, double-blind, placebo-controlled trial evaluating the effect of VOXZOGO on the growth of the foramen magnum and craniocervical junction in children with achondroplasia, including infants.

"These findings provide important new insight into the potential effect of VOXZOGO on growth-related dimensions of the foramen magnum and craniocervical junction, regions of the skull implicated in cervicomedullary compression and serious complications in children with achondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "The results also underscore the potential VOXZOGO treatment may offer when started as early as possible, when it can have the greatest impact. Together with the additional data we're presenting on bone strength, limb alignment, and our emerging work in hypochondroplasia and next-generation medicines such as BMN 333, these findings reflect the depth and breadth of our commitment to understanding and improving outcomes for children living with skeletal dysplasias."

BioMarin recently submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for VOXZOGO for the treatment of hypochondroplasia. The company is also on track with its submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.

Additional data to be presented at the meeting include research in hypophosphatasia, as well as results from the Phase 3 ENERGY 3 study of BMN 401 in children ages 1 to 12 years with ENPP1 deficiency.

Below are key presentations at ASBMR, with all times listed in Eastern Daylight Time:

C-Type Natriuretic Peptide (CNP) Agonist CNP38 Promotes Longitudinal Bone Growth by Growth Plate Expansion in the Hyp Mouse Model of XLH
Oral Presentation #1138
Monday, Oct. 12, 11:30 – 11:42 a.m.

BMN 333, a Long-Acting Vosoritide Investigational Product, Promotes Endochondral Bone Growth and Skeletal Microarchitecture in Juvenile Mice and Nonhuman Primates
Poster Presentation #092
Friday, Oct. 9, 5:30 – 7 p.m.
Sunday, Oct. 11, 2 – 3:30 p.m.

Examining the Effect of Vosoritide Treatment on Bone Strength in Children With Achondroplasia
Poster Presentation #486
Saturday, Oct. 10, 2 – 3:30 p.m.

Efficacy and Safety of BMN 401 in Children With ENPP1 Deficiency: 52-Week Results From the Pivotal ENERGY 3 Trial
Poster Presentation #554
Saturday, Oct. 10, 2 – 3:30 p.m.

Economic Burden of Hypophosphatasia: A Systematic Literature Review
Poster Presentation #473
Saturday, Oct. 10, 2 – 3:30 p.m.

Development of BMN 333, a Long-Acting Version of Vosoritide for FGFR3-Related Dwarfism and Other Skeletal Dysplasias
Poster Presentation #115
Sunday, Oct. 11, 2 – 3:30 p.m.

Comparison of Quantitative Computed Tomography Bone Mineral Density Measurements of Vosoritide-Treated Children With Achondroplasia to an Untreated Achondroplasia Reference Population
Poster Presentation #474
Sunday, Oct. 11, 2 – 3:30 p.m.

Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results
Poster Presentation #552
Sunday, Oct. 11, 2 – 3:30 p.m.

The Effect of Vosoritide on Growth of the Foramen Magnum and Craniocervical Junction in Children With Achondroplasia
Poster Presentation #483
Sunday, Oct. 11, 2 – 3:30 p.m.

Molecular Insights into Vosoritide's Role in Enhancing Bone Growth and Mineralization
Poster Presentation #362
Sunday, Oct. 11, 2 – 3:30 p.m.

Understanding the Impact of Hypophosphatasia (HPP) on Health-Related Quality-of-Life and Activities of Daily Living: A Patient-Based Conceptual Model
Poster Presentation #011
Sunday, Oct. 11, 2 – 3:30 p.m.

Prospective, Observational Study of a Cohort of Hypophosphatasia Patients' Phenotypes Over Time and the Utility of Digital Outcomes Measures in This Population
Poster Presentation #521
Sunday, Oct. 11, 2 – 3:30 p.m.

ENPP1 Inhibition Improves the Bone Phenotype, Mobility, Muscle Strength and Endurance in Later-Onset HPP Mice
Oral Presentation #1139
Monday, Oct. 12, 11:42 – 11:54 a.m.

About Achondroplasia

Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.

More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.

For more information about our clinical trials in achondroplasia, hypochondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.

About Hypochondroplasia 

Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings. BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO. 

There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia. 

For more information about our clinical trials in hypochondroplasia, achondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com. 

About VOXZOGO 

In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. 

VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.  

VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). An sNDA with long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth such as body proportionality and arm span, is under review with an FDA Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.

The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency. 

Patient Support Accessing VOXZOGO

BioMarin's robust support services have ensured a seamless treatment experience, spearheaded by Clinical Coordinators, who have conducted hundreds of trainings for families with achondroplasia since approval. BioMarin provides resources to support families navigating achondroplasia, including a caregiver mentorship program that connects parents with other caregivers, and a U.S. doctor directory that helps families and healthcare professionals identify clinicians experienced in achondroplasia care.

To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or email [email protected]. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at [email protected]. 

VOXZOGO U.S. Important Safety Information 

What is VOXZOGO used for? 

VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses).  VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.  What is the most important safety information about VOXZOGO? 

VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO.  What are the most common side effects of VOXZOGO? 

The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away.  How is VOXZOGO taken? 

VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made.  Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual.  The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups.  Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider.  What should you tell the doctor before or during taking VOXZOGO? 

Tell your doctor about all of the patient's medical conditions including  If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine).  If the patient has kidney problems or renal impairment.  If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby.  If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk.  Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.  You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. 

Please see additional safety information in the full Prescribing Information and Patient Information. 

About BioMarin 

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.  

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at the American Society for Bone and Mineral Research 2026 Annual Meeting (ASBMR), including the potential impact and safety of VOXZOGO in children with achondroplasia and hypochondroplasia; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including plans for submissions to the European Medicines Agency (EMA) and other regional health authorities as well as the expectation that, if approved by the U.S. Food and Drug Administration (FDA), VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia with an anticipated launch in 2027; BioMarin's expectations regarding its supplemental New Drug Application (sNDA) for VOXZOGO for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; BioMarin's plans and expectations for the development of BMN 333; and BioMarin's estimate regarding incidence and total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including achondroplasia and hypochondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. 

BioMarin®, BioMarin RareConnections® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Katherine Powell

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 827-2968

SOURCE BioMarin Pharmaceutical Inc.

Ms. Bauer Brings More Than 25 Years of Experience in Human Resources, Across Biotechnology and Technology Industries

Appointment Effective Oct. 5, 2026

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that Cara Bauer, Ph.D., has been named Executive Vice President, Chief People Officer. Ms. Bauer will assume responsibility for the company's Human Resources organization, effective Oct. 5, succeeding Amy Wireman, who is retiring.

Ms. Bauer joins BioMarin from Kyverna Therapeutics, Inc., where she served as Chief Human Resources Officer, bringing more than 25 years of experience as a leader in human resources across the biopharmaceutical and technology industries, and a track record of building and scaling organizations through periods of rapid growth and change. Prior to joining Kyverna in October 2024, she served as global head of human resources at Kite, a Gilead company. Earlier in her career, Ms. Bauer led human resources for the entertainment division at Riot Games and held HR leadership roles at Netflix, Amgen and Novo Nordisk.

"Cara is an experienced business leader who brings to the role a significant breadth of knowledge in human resources across multiple sectors at companies of varying size," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Throughout her career, she has demonstrated the ability to drive large-scale transformation, taking an innovative approach to building high-performing organizations and developing talent. She will provide valuable perspective as we continue to deliver for patients with rare conditions, and I am delighted to welcome her to BioMarin."

Ms. Bauer holds a bachelor's degree in industrial and organizational psychology from DePaul University and a doctorate in the same field from Wayne State University.

"For nearly 30 years, BioMarin has worked to advance breakthrough treatments for people with rare genetic diseases and that shapes how I think about growing teams, developing talent and strengthening organizational capabilities," said Ms. Bauer. "I am excited to join BioMarin and look forward to partnering with the organization to continue building a culture that puts patients at the center of our work and fosters scientific innovation."

Ms. Wireman will remain at BioMarin in an advisory capacity to support the transition. She joined BioMarin in 2013 to lead Total Rewards, HR Systems and Operations, and was appointed Chief People Officer in 2019. During her tenure, she helped grow the organization to support the launch and commercialization of five medicines. More recently, she has played a pivotal role in embedding the company's corporate strategy and the integration of Amicus Therapeutics following the acquisition early this year.

"Amy has been an extraordinary partner to me and to the organization, providing strong leadership and counsel to all of us at BioMarin through key business and cultural milestones," said Mr. Hardy. "For nearly 14 years, she has helped shape BioMarin's culture and all of us are deeply grateful for her many contributions."

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the appointment of Cara Bauer as Chief People Officer, including expectations regarding her contributions to BioMarin and the anticipated benefits to BioMarin of her appointment; and Amy Wireman's plans to remain at BioMarin in an advisory capacity to support the transition. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, Ms. Bauer commencing and continuing employment as Chief People Officer as anticipated, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc. 

Contacts:

Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558 

 Media
Erin Rau
BioMarin Pharmaceutical Inc.
(925) 683-9622

SOURCE BioMarin Pharmaceutical Inc.

Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) in late-onset Pompe disease (LOPD) were presented at the 31st Annual Congress of the World Muscle Society (WMS), in Hiroshima, Japan.

These results from the PROPEL open-label extension (OLE) study of POMBILITI + OPFOLDA, which were shared in a late-breaking presentation, demonstrated durable motor function outcomes and relative stabilization of pulmonary function over long-term follow-up. The analysis included data from 82 individuals who received POMBILITI + OPFOLDA continuously from baseline observed in the initial PROPEL study (PROPEL baseline) to year five. For the enzyme replacement therapy (ERT)-experienced cohort, the mean duration of prior ERT at baseline was 7.6 years. These data represent long-term benefits for people with LOPD followed in the study, including both ERT-experienced and ERT-naïve cohorts. 

"These five-year results demonstrate that POMBILITI and OPFOLDA can provide durable, long-term benefits for people living with late-onset Pompe disease, including those who had been on enzyme replacement therapy for years before enrolling in the study," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Sustaining motor function and limiting pulmonary decline for up to five years in a progressive disease like LOPD is clinically meaningful, and we remain committed to continuing to share long-term evidence to inform the care of people with this rare genetic condition."

Table 1. Efficacy Measures With POMBILITI + OPFOLDA From PROPEL Baseline (BL) for up to Five Years

ERT-Experienced

to Year 5
(n=62 at BL)

ERT-Naïve

to Year 4.5

(n=20 at BL)

Mean (standard deviation,
SD) change from baseline
in % predicted six-minute
walk distance (pp6MWD)     

0.7% (7.76)

10.5% (11.44)

Mean (SD) change from
baseline in % predicted
forced vital capacity
(ppFVC)

-2.8% (8.55)

-4.4% (6.89)

No new safety signals were identified. 51.2% of participants (n=42) experienced treatment-related adverse events, the majority of which were manageable. Five individuals discontinued treatment for safety reasons, including two due to serious treatment-related adverse events (chills and hypoxia [n=1], anaphylactoid reaction [n=1]).

Phase 1/2 Data Show Positive Dystrophin Expression and Favorable Tolerability With Nivudirsen (BMN 351)

Additional presentations at WMS included data evaluating nivudirsen (BMN 351), an investigational antisense oligonucleotide for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Included in these presentations was an update from the ongoing Phase 1/2 351-201 study, which was previously shared at the 19th International Congress on Neuromuscular Diseases in July. Results from 351-201 showed treatment with nivudirsen was generally well tolerated and led to consistently positive outcomes across multiple measures of therapeutic benefit, including expression of near full-length dystrophin.

Below are key presentations at WMS, with all times listed in Japan Standard Time:

Phase 1/2 Results of BMN 351, an Antisense Oligonucleotide for Exon 51 Skip-Amenable Duchenne Muscular Dystrophy
Poster Presentation #2.66P
Wednesday, Sept. 30, 5:15 – 6:15 p.m.

Comprehensive Panel of Pharmacokinetic, Pharmacodynamic and Biomarker Measures Drives Clinical Development of BMN 351 for Duchenne Muscular Dystrophy
E-Poster Presentation #2.43eP
Wednesday, Sept. 30, 5:15 – 6:15 p.m.

Muscle Composition and Muscle Injury Biomarkers Improve With BMN 351 Treatment
E-Poster Presentation #2.59eP
Wednesday, Sept. 30, 5:15 – 6:15 p.m.

Long-Term (Five-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline
E-Poster Presentation #3.081LBeP
Friday, Oct. 2, 2:15 – 3:15 p.m. 

Long-Term (Four-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline
Poster Presentation #3.56P
Friday, Oct. 2, 2:15 – 3:15 p.m.

Baseline Disease Burden and Real-World Monitoring Practices: Global Insights From the followME Pompe Journey Registry
Poster Presentation #3.57P
Friday, Oct. 2, 2:15 – 3:15 p.m.

ATB200-03/07 Danish Subpopulation: Four-Year Efficacy and Safety of Cipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease
E-Poster Presentation #3.35eP
Friday, Oct. 2, 2:15 – 3:15 p.m.

About the PROPEL Open-Label Extension Study

The Phase 3 open-label extension (OLE) study (ATB200-07; NCT04138277) is a multicenter, international study designed to evaluate the long-term safety and efficacy of POMBILITI + OPFOLDA in adults with late-onset Pompe disease (LOPD) who completed the Phase 3 PROPEL study (ATB200-03; NCT03729362). Of the 118 people treated in the OLE, 82 continued POMBILITI + OPFOLDA treatment from PROPEL (62 ERT-experienced prior to PROPEL; 20 ERT-naïve) and 37 switched from the comparator arm to POMBILITI + OPFOLDA (29 ERT-experienced; eight ERT-naïve).

The study evaluates long-term changes from PROPEL baseline in six-minute walk distance (6MWD, the primary endpoint in PROPEL), forced vital capacity (FVC), creatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, and patient-reported outcomes, as well as long-term safety.

About Study 351-201

The 351-201 trial (NCT06280209) is a Phase 1/2 multicenter open-label dose-escalation study evaluating BMN 351 for the treatment of boys aged 4 to 10 with Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping. The primary endpoints examine the safety and tolerability of BMN 351, the secondary endpoints assess plasma, urine and muscle pharmacokinetics, and key exploratory endpoints include exon-skipping efficiency, dystrophin expression, immunogenicity and functional assessments. 

The study is being conducted in three cohorts, with Cohort 1 being divided into Cohort 1A and Cohort 1B. In Cohort 1A, three boys received increasing doses once every two weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351. In part two, the participants in Cohort 1A transitioned to once-weekly dosing. The participants in Cohort 1B, 2 and 3 will receive low (6 mg/kg), medium (9 mg/kg) and high (12 mg/kg) doses of BMN 351 and continue once-weekly dosing at that same dose.

For more information, please visit clinicaltrials.biomarin.com.

About Late-Onset Pompe Disease

Late-onset Pompe disease (LOPD) is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. LOPD can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly in skeletal muscles and muscles that control breathing.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe neuromuscular condition that leads to progressive muscle weakness and premature mortality. DMD is caused by genetic changes that result in the absence or dysfunction of dystrophin, an essential protein that is necessary for preserving muscle function.

As an X-linked disorder, DMD primarily affects young boys, with onset of symptoms occurring around 2 to 3 years of age. The incidence of DMD is up to 1 in 3,600 to 6,000 live male births.

Individuals with DMD experience serious functional impairments, including loss of limb strength and ambulation, and respiratory and cardiovascular impacts that require lifelong care.

About POMBILITI + OPFOLDA

POMBILITI + OPFOLDA is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that is designed to reduce loss of enzyme activity in the blood.

Patient Support Accessing POMBILITI + OPFOLDA

To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-866-906-6100 or email [email protected]. For more information about POMBILITI + OPFOLDA, please visit www.pombilitiopfolda.com. For additional information regarding these products, please contact BioMarin Medical Information at [email protected].

POMBILITI + OPFOLDA U.S. Indication and Important Safety Information

POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).

SAFETY INFORMATION

HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See the full U.S. Prescribing Information for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with OPFOLDA is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see U.S. full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) and full PRESCRIBING INFORMATION for OPFOLDA (miglustat).

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at the 31st Annual Congress of the World Muscle Society (WMS), including the potential benefits and safety of POMBILITI + OPFOLDA in late-onset Pompe disease as well as of nivudirsen (BMN 351) for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping; BioMarin's plans and expectations for the development of POMBILITI + OPFOLDA and nivudirsen; and BioMarin's estimate regarding the incidence of DMD. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, BioMarin RareConnections®, POMBILITI® and OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc. or its affiliates.

Contacts:

Investors

Media

Traci McCarty

Katherine Powell

BioMarin Pharmaceutical Inc.     

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 827-2968

SOURCE BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical (NASDAQ:BMRN – Get Free Report) and Metagenomi (NASDAQ:MGX – Get Free Report) are both healthcare companies, but which is the better stock? We will compare the two businesses based on the strength of their risk, profitability, institutional ownership, dividends, earnings, analyst recommendations and valuation.

Earnings and Valuation This table compares BioMarin Pharmaceutical and Metagenomi”s revenue, earnings per share (EPS) and valuation.

Gross Revenue Price/Sales Ratio Net Income Earnings Per Share Price/Earnings Ratio BioMarin Pharmaceutical $3.41 billion 3.40 $348.90 million $0.37 161.81 Metagenomi $25.21 million 1.69 -$87.87 million ($2.49) -0.45 BioMarin Pharmaceutical has higher revenue and earnings than Metagenomi. Metagenomi is trading at a lower price-to-earnings ratio than BioMarin Pharmaceutical, indicating that it is currently the more affordable of the two stocks. Analyst Recommendations This is a summary of current ratings and recommmendations for BioMarin Pharmaceutical and Metagenomi, as reported by MarketBeat.

Sell Ratings Hold Ratings Buy Ratings Strong Buy Ratings Rating Score BioMarin Pharmaceutical 1 7 15 1 2.67 Metagenomi 1 0 2 1 2.75 BioMarin Pharmaceutical currently has a consensus price target of $89.17, suggesting a potential upside of 48.95%. Metagenomi has a consensus price target of $6.00, suggesting a potential upside of 430.97%. Given Metagenomi’s stronger consensus rating and higher possible upside, analysts clearly believe Metagenomi is more favorable than BioMarin Pharmaceutical.

Institutional and Insider Ownership 98.7% of BioMarin Pharmaceutical shares are owned by institutional investors. 0.7% of BioMarin Pharmaceutical shares are owned by company insiders. Comparatively, 12.5% of Metagenomi shares are owned by company insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a company will outperform the market over the long term.

Profitability This table compares BioMarin Pharmaceutical and Metagenomi’s net margins, return on equity and return on assets.

Net Margins Return on Equity Return on Assets BioMarin Pharmaceutical 2.14% 5.32% 3.69% Metagenomi -384.61% -63.65% -44.52% Risk & Volatility BioMarin Pharmaceutical has a beta of 0.24, meaning that its share price is 76% less volatile than the S&P 500. Comparatively, Metagenomi has a beta of 0.67, meaning that its share price is 33% less volatile than the S&P 500.

Summary BioMarin Pharmaceutical beats Metagenomi on 10 of the 14 factors compared between the two stocks.

(Get Free Report)

BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc. The company was incorporated in 1996 and is headquartered in San Rafael, California.

About Metagenomi (Get Free Report)

Metagenomi, Inc., a genetic medicines company, develops therapeutics for patients using metagenomics-derived genome editing toolbox in the United States. The company's genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems, such as prime editing systems and clustered regularly interspaced short palindromic repeat-associated transposases. It has a strategic collaboration and license agreement with ModernaTX, Inc. focusing on new genome editing system for in vivo human therapeutic applications; a development, option, and license agreement with Affini-T Therapeutics, Inc. to develop and commercialize gene edited T-cell receptor-based therapeutic products in the field of treatment, prevention, or diagnosis of human cancer; and a collaboration and license agreement with Ionis Pharmaceuticals, Inc. to research, develop, and commercialize investigational medicines using genome editing technologies. The company was incorporated in 2016 and is based in Emeryville, California.

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Can BioMarin Stock Live Up to Wall Street’s High Expectations?BioMarin Pharmaceutical NASDAQ: BMRN said it is progressing with the integration of Amicus Therapeutics while pursuing additional business-development opportunities, as executives outlined growth plans for newly acquired rare-disease products, VOXZOGO and several pipeline programs at Morgan Stanley’s Global Healthcare Conference.

President and CEO Alexander Hardy said the company has delivered business growth, margin expansion and efficiency improvements while integrating Amicus. He described the transaction as primarily driven by the opportunity to accelerate revenue from GALAFOLD and POMBILITI + OPFOLDA, as well as realize operating-cost synergies. Diversification of BioMarin’s growth profile was a secondary benefit, he said.

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Amicus Integration and Financial Targets 3 Oversold Stocks with Big RSI Rebound PotentialBioMarin projects peak sales of approximately $1.4 billion for GALAFOLD and $1.2 billion for POMBILITI + OPFOLDA by the mid-2030s. Hardy said the opportunity for GALAFOLD is centered on improving Fabry disease diagnosis and treatment rates, supplemented by geographic expansion. He said the company believes it can more than double the number of U.S. patients on GALAFOLD by the mid-2030s.

For POMBILITI + OPFOLDA, Hardy said the commercial opportunity is more dependent on patients switching from other Pompe disease therapies as evidence and real-world data develop. He characterized that market as a longer and slower opportunity than GALAFOLD’s.

Are Gene Therapy Stocks The Market's Next Big Winners?BioMarin expects $200 million in non-GAAP expense savings from the Amicus transaction. Hardy said Amicus had about 505 employees and BioMarin expects to retain approximately 192 over the long term. The company primarily retained commercial and medical-affairs personnel supporting GALAFOLD and POMBILITI + OPFOLDA, while about 70% of the savings are expected to come from general and administrative expenses, with the remainder from duplicated research and development functions.

Executive Vice President and CFO Brian Mueller said the integration planning has moved beyond high-level targets, with employee decisions communicated and operational cutover plans established across functions. He said integration will continue through this year and into next year because some systems and processes are qualified and regulated. BioMarin expects to realize most synergies in 2027, with 2028 representing the first full year of full synergy realization.

Mueller said BioMarin expects the transaction to be accretive in its first year, with substantial accretion beginning next year. The additional cash flow has allowed the company to advance its deleveraging target. BioMarin now expects leverage to fall below 2.5 times by the middle of 2027, nearly a year earlier than its initial target of within two years of the transaction’s announcement.

Hardy said reaching that target would restore the company’s pre-Amicus acquisition capacity for deals of up to $5 billion. Mueller said that, with continued EBITDA growth, capacity could reach approximately $6 billion by 2028. BioMarin recently announced and closed a deal to add Alesta Therapeutics’ ALE1 program to its pipeline, and Hardy said the company intends to continue pursuing transactions while it deleverages.

VOXZOGO Growth and Competitive Landscape Hardy said VOXZOGO continued to grow despite the entry of a competitor in the U.S. market. He cited 20% quarterly patient growth and said the company raised its full-year outlook. BioMarin expects VOXZOGO to reach $1 billion in revenue this year, which would make it the company’s first blockbuster product, according to Hardy.

He said most new VOXZOGO patient starts have been in the zero-to-two-year-old population, where BioMarin has an indication and data advantage that will remain exclusive for several years. The company is also awaiting a potential FDA approval for VOXZOGO in hypochondroplasia, an opportunity not incorporated into its 2026 outlook.

BioMarin has reached a settlement with Ascendis that provides BioMarin a 20% royalty rate in the U.S. and an 18% royalty rate in Europe, Brazil and South Korea. Hardy said the arrangement allows BioMarin to benefit if Ascendis’ TransCon treatment brings additional patients into the market.

Hardy added that BioMarin markets VOXZOGO in 55 countries and has an 80-country commercial footprint. He said market access outside the U.S. is more complex and requires local expertise, giving BioMarin an advantage over newer entrants.

Established Products and Pipeline BioMarin said PALYNZIQ grew 27% to $135 million, aided by the U.S. adolescent-label launch. Hardy estimated the 12-to-17-year-old population could add roughly 10% to the addressable patient opportunity. He said the therapy’s efficacy may appeal to families preparing adolescents for greater independence, including college.

Hardy also said VIMIZIM has the potential to exceed $1 billion in revenue. He noted that quarterly results can be affected by international ordering patterns and government purchases, but said VIMIZIM grew 7% in 2025 and has historically grown at around that pace. Mueller encouraged investors to focus on full-year results and guidance because of quarterly timing effects.

On pipeline development, Hardy said BioMarin’s BMN 333 showed more than 13-fold higher free CNP exposure than another long-acting CNP agent in a Phase I healthy-volunteer study. The company is conducting an operationally seamless Phase II/III study to determine whether higher free CNP levels translate into improved annualized growth velocity and other health benefits. BioMarin expects to have Phase II findings around the middle of the year.

Regarding ALE1, Hardy said the oral candidate for hypophosphatasia could potentially address bone manifestations as well as symptoms such as muscle pain and fatigue by reducing pyrophosphate at its source. He said approximately 6,000 U.S. patients are diagnosed with the disease, but only an estimated 10% to 30% receive treatment.

About BioMarin Pharmaceutical (NASDAQ:BMRN)BioMarin Pharmaceutical Inc is a biotechnology company focused on developing and commercializing therapies for rare and serious diseases. Founded in 1997 and headquartered in San Rafael, California, the company specializes in treatments for conditions that often have limited or no available therapeutic options, including inherited metabolic disorders, skeletal diseases, and certain bleeding disorders.

BioMarin's marketed products include Vimizim for mucopolysaccharidosis type IV A, Naglazyme for mucopolysaccharidosis type VI, Brineura for late-infantile neuronal ceroid lipofuscinosis type 2, Palynziq and Kuvan for phenylketonuria, and Voxzogo for achondroplasia in children with open growth plates.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].

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BioMarin Pharmaceutical Inc. (BMRN) Morgan Stanley 24th Annual Global Healthcare Conference September 14, 2026 10:00 AM EDT

Company Participants

Alexander Hardy - President, CEO & Director
Brian Mueller - CFO & Executive VP of Finance

Conference Call Participants

Sean Laaman - Morgan Stanley, Research Division

Presentation

Sean Laaman
Morgan Stanley, Research Division

Good morning, everyone. I'm Sean Laaman, Head of U.S. SMID-cap biotech Equity Research here at Morgan Stanley, and welcome to our Global Healthcare Conference. Before we begin, I'll make you aware of some important disclosures. For those disclosures, see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. And if you have any questions, please reach out to your Morgan Stanley sales representative.

I welcome here today, President and CEO, Alexander Hardy; and EVP and CFO, Brian Mueller. Welcome to both of you. Alexander, I believe you wanted to make some introductory remarks before we kick off the formal Q&A part of the session. But thank you for your time today.

Alexander Hardy
President, CEO & Director

Thanks very much, Sean. Thanks very much for having us. Thanks to Morgan Stanley for hosting this. Yes, I just want to say a few things. We mainly want to hear the questions that you have and folks in the room. I'm very, very happy with our execution and how we're delivering over the last several quarters. To be able to perform strongly with the delivery of the business, at the same time, integrate a major acquisition simultaneously is really exciting, and it's a testament to the organization.

I think with the Amicus integration, we've added two additional significant growth drivers, further strengthening the growth profile of the business. We're showing that BioMarin is a rare disease company at scale. We've delivered, obviously, efficiency in profit and growth in profit margins. Now with Amicus, that translates to additional cash flow. We

Amundi lifted its stake in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 37.8% during the 2nd quarter, according to the company in its most recent filing with the Securities & Exchange Commission. The firm owned 259,782 shares of the biotechnology company’s stock after acquiring an additional 71,246 shares during the period. Amundi owned 0.13% of BioMarin Pharmaceutical worth $14,865,000 as of its most recent filing with the Securities & Exchange Commission.

Other institutional investors and hedge funds have also recently modified their holdings of the company. CIBC Private Wealth Group LLC grew its stake in shares of BioMarin Pharmaceutical by 38.2% in the 4th quarter. CIBC Private Wealth Group LLC now owns 648 shares of the biotechnology company’s stock valued at $39,000 after buying an additional 179 shares during the period. Mitsubishi UFJ Asset Management Co. Ltd. purchased a new position in BioMarin Pharmaceutical in the second quarter worth approximately $39,000. Caitong International Asset Management Co. Ltd purchased a new position in BioMarin Pharmaceutical in the third quarter worth approximately $40,000. Parallel Advisors LLC increased its stake in BioMarin Pharmaceutical by 62.2% during the third quarter. Parallel Advisors LLC now owns 769 shares of the biotechnology company’s stock worth $42,000 after acquiring an additional 295 shares during the last quarter. Finally, Keating Financial Advisory Services Inc. purchased a new stake in BioMarin Pharmaceutical in the second quarter valued at $56,000. Institutional investors and hedge funds own 98.71% of the company’s stock.

BioMarin Pharmaceutical Price Performance Shares of BMRN opened at $65.68 on Friday. BioMarin Pharmaceutical Inc. has a fifty-two week low of $49.26 and a fifty-two week high of $70.98. The firm’s 50 day moving average price is $63.30 and its 200 day moving average price is $58.50. The company has a market cap of $12.71 billion, a P/E ratio of 177.51, a PEG ratio of 0.47 and a beta of 0.24. The company has a quick ratio of 1.32, a current ratio of 2.39 and a debt-to-equity ratio of 0.56.

BioMarin Pharmaceutical (NASDAQ:BMRN – Get Free Report) last issued its quarterly earnings results on Thursday, August 6th. The biotechnology company reported $1.20 EPS for the quarter, beating the consensus estimate of $0.95 by $0.25. BioMarin Pharmaceutical had a net margin of 2.14% and a return on equity of 5.32%. The company had revenue of $989.71 million during the quarter, compared to analysts’ expectations of $932.03 million. During the same quarter last year, the company posted $1.44 EPS. The firm’s revenue was up 20.0% compared to the same quarter last year. BioMarin Pharmaceutical has set its FY 2026 guidance at 4.900-5.100 EPS. On average, research analysts forecast that BioMarin Pharmaceutical Inc. will post 4.1 earnings per share for the current fiscal year. Wall Street Analyst Weigh In A number of analysts recently issued reports on BMRN shares. Jefferies Financial Group raised their price objective on BioMarin Pharmaceutical from $104.00 to $105.00 and gave the stock a “buy” rating in a research report on Monday, August 31st. Piper Sandler lifted their price target on shares of BioMarin Pharmaceutical from $88.00 to $90.00 and gave the company an “overweight” rating in a research report on Tuesday, September 1st. BMO Capital Markets assumed coverage on shares of BioMarin Pharmaceutical in a report on Wednesday, August 19th. They issued an “outperform” rating and a $98.00 price objective on the stock. Wall Street Zen raised shares of BioMarin Pharmaceutical from a “hold” rating to a “buy” rating in a research report on Saturday, August 8th. Finally, Canaccord Genuity Group lifted their target price on shares of BioMarin Pharmaceutical from $111.00 to $114.00 and gave the company a “buy” rating in a report on Monday, August 10th. Two analysts have rated the stock with a Strong Buy rating, fourteen have assigned a Buy rating, six have assigned a Hold rating and one has given a Sell rating to the company. Based on data from MarketBeat, the company has an average rating of “Moderate Buy” and an average price target of $89.17.

View Our Latest Analysis on BioMarin Pharmaceutical

BioMarin Pharmaceutical Company Profile (Free Report)

BioMarin Pharmaceutical Inc is a biotechnology company focused on developing and commercializing therapies for rare and serious diseases. Founded in 1997 and headquartered in San Rafael, California, the company specializes in treatments for conditions that often have limited or no available therapeutic options, including inherited metabolic disorders, skeletal diseases, and certain bleeding disorders.

BioMarin’s marketed products include Vimizim for mucopolysaccharidosis type IV A, Naglazyme for mucopolysaccharidosis type VI, Brineura for late-infantile neuronal ceroid lipofuscinosis type 2, Palynziq and Kuvan for phenylketonuria, and Voxzogo for achondroplasia in children with open growth plates.

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BioMarin Pharmaceutical Inc. (BMRN) 12th Annual Cantor Fitzgerald Global Healthcare Conference September 10, 2026 10:20 AM EDT

Company Participants

Cristin Hubbard - Executive VP & Chief Commercial Officer
Gregory Friberg - Executive VP and Chief Research & Development Officer

Conference Call Participants

Olivia Brayer - Cantor Fitzgerald & Co., Research Division

Presentation

Olivia Brayer
Cantor Fitzgerald & Co., Research Division

Okay. Hi. Good morning, everyone. Welcome to day 2 of our Cantor Healthcare Conference. My name is Olivia Brayer. I'm one of the senior biotech analysts here at Cantor. And really excited about this morning's fireside chat with the BioMarin team. We have Cristin Hubbard, who is Chief Commercial Officer; and Greg Friberg, who is Chief R&D Officer.

Thank you both so much for being here and making the trip to New York.

Cristin Hubbard
Executive VP & Chief Commercial Officer

Thank you for having us.

Gregory Friberg
Executive VP and Chief Research & Development Officer

Thank you.

Question-and-Answer Session

Olivia Brayer
Cantor Fitzgerald & Co., Research Division

So it's been a really busy year for BioMarin. A lot has changed. You've done some deals. You've added some new assets. Maybe just talk about the state of the company today, how it's evolved over time? And what, kind of, the vision is for BioMarin going forward from here?

Cristin Hubbard
Executive VP & Chief Commercial Officer

So I'll kick us off. For those of you following us, we're BioMarin. We're a leading rare disease biotechnology company. And we operate specifically in genetically defined conditions. We have 9 commercial products that are across -- that are spread across our 2 business units, so skeletal conditions and metabolic conditions.

Now very recently, we announced the acquisition of Amicus. And with Amicus, that gave us 2 high-growth commercial products. One is Galafold in Fabry disease, which we just announced peak

Detailed Phase 3 CANOPY-HCH-3 data in children living with hypochondroplasia also featured in a late-breaking oral presentation at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting

VOXZOGO demonstrated statistically significant improvements across multiple measures of growth, including annualized growth velocity, standing height, height Z-score and arm span

BioMarin recently submitted a supplemental New Drug Application (sNDA) to the FDA to support expanding treatment with VOXZOGO to include children with hypochondroplasia

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data from the Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO® (vosoritide) in children with hypochondroplasia were published in New England Journal of Medicine (NEJM) Evidence and presented at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting. The data included new results on the magnitude of benefit seen in children receiving VOXZOGO, including statistically significant improvements in annualized growth velocity (AGV), standing height, height Z-score and arm span after 52 weeks, with safety findings consistent with the established profile of VOXZOGO in achondroplasia.

The CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint (least squares [LS] mean difference of 2.33 cm/year; p<0.0001). Children treated with VOXZOGO also showed significant improvements in standing height (LS mean difference of 2.35 cm; p<0.0001), height Z-score (LS mean difference of 0.39 standard deviation score; p<0.0001), and arm span (LS mean difference of 1.03 cm; p=0.0082) compared with placebo. Children who received VOXZOGO also demonstrated numerical improvements in quality of life, and follow-up will continue to assess the impact of treatment over a longer term. The overall safety profile was consistent with previous studies of VOXZOGO, with most adverse events reported as mild and no treatment-related serious adverse events identified.

"These results presented in detail for the first time provide a comprehensive picture of the impact of VOXZOGO across multiple measures of growth in children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Based on this compelling body of evidence, we have submitted these data to the FDA with the goal of securing approval for the first medicine for children with hypochondroplasia."

"Hypochondroplasia can affect a child's growth, physical function and everyday life, with families often navigating unique challenges as they support their children's development," said Andrew Dauber, M.D., lead study investigator and Chief of Endocrinology at Children's National in Washington, D.C. "The changes we observed in annualized growth velocity and arm span provide encouraging evidence of how children with hypochondroplasia responded to treatment throughout the study. These findings deepen our understanding of the condition while reinforcing VOXZOGO's potential as the first targeted medicine developed specifically for children with hypochondroplasia."

BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia and are on track with the submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.

Below are key BioMarin presentations across both achondroplasia and hypochondroplasia at ESPE, with all times listed in Central European Summer Time:

Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results
Oral Presentation #LBA 1067
Wednesday, Sept. 9, 10:48 – 10:56 a.m.

Vosoritide Safety and Effectiveness in Young Children With Achondroplasia Aged ≤3 Years and With up to 36 Months of Follow-Up from the Japanese Post-Marketing Safety Surveillance Study (111-604)
Oral Presentation #FC3.4
Tuesday, Sept. 8, 3:30 – 3:40 p.m.

About Hypochondroplasia 

Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings. BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO. 

There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia. 

For more information about our clinical trials in hypochondroplasia, achondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com. 

About VOXZOGO 

In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. 

VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.

VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). An sNDA with long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth such as body proportionality and arm span is under review with an FDA Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.

The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency. 

VOXZOGO U.S. Important Safety Information 

What is VOXZOGO used for? 

VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses).  VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.  What is the most important safety information about VOXZOGO? 

VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO.  What are the most common side effects of VOXZOGO? 

The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away.  How is VOXZOGO taken? 

VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made.  Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual.  The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups.  Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider.  What should you tell the doctor before or during taking VOXZOGO? 

Tell your doctor about all of the patient's medical conditions including  If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine).  If the patient has kidney problems or renal impairment.  If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby.  If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk.  Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.  You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. 

Please see additional safety information in the full Prescribing Information and Patient Information. 

About BioMarin 

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting, including the safety profile and potential benefits of VOXZOGO for children with hypochondroplasia and achondroplasia; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including the expectation that, if approved by the U.S. Food and Drug Administration (FDA), VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia with a potential 2027 launch; BioMarin's expectations regarding its supplemental New Drug Application (sNDA) for VOXZOGO for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; and BioMarin's estimate regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including hypochondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.

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Media

Traci McCarty

Andrew Villani

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

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SOURCE BioMarin Pharmaceutical Inc.

Wells Fargo 21st Annual Healthcare Conference, September 9 at 3:00 pm EST Cantor Global Healthcare Conference 2026, September 10 at 10:20 am EST Citi's 2026 Biopharma Back to School Conference, September 10 at 1:00 pm EST Morgan Stanley 24th Annual Global Healthcare Conference, September 14 at 10:00 am EST SAN RAFAEL, Calif., Sept. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will present at upcoming investor conferences in September.

Agreement Resolves all Pending Patent-Related Proceedings Between BioMarin and Ascendis

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it has entered into binding terms with Ascendis Pharma A/S, resolving the patent and ancillary disputes pending globally, including before the U.S. International Trade Commission (ITC) concerning Ascendis's Yuviwel. As part of the agreement, Ascendis will pay BioMarin a royalty equal to 20% of net sales of Yuviwel in the U.S., retroactive to the first commercial sale, and 18% of net sales in the European Union, Brazil and South Korea until May 2030. 

"This outcome incentivizes companies like BioMarin to keep investing in the kind of long-term innovation that is critical to bringing breakthrough treatments to the people who need them," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We have spent decades focused on understanding the underlying biology of rare genetic conditions, building the deep scientific expertise that led to our development of six first-in-disease medicines for patients. We look forward to continuing to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on our ongoing momentum for children with achondroplasia."

The scope of the settlement includes a license for BioMarin's patents that relate to Yuviwel for all current and potential indications, including achondroplasia and hypochondroplasia. It also covers the use of Yuviwel in combination with other medicines. Under the terms of the agreement, BioMarin will dismiss the pending Section 337 investigation before the ITC and the parties will resolve all claims relating to the asserted intellectual property, including litigation pending in Brazil, Denmark, Germany, South Korea and the Northern District of California.

Reaching this agreement recognizes the value of BioMarin's pioneering innovations in C-type natriuretic peptide (CNP) technology, including the development of VOXZOGO® (vosoritide), while providing a framework that enables continued access to medicine for children with achondroplasia around the world.

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com. 

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the settlement and license agreement with Ascendis Pharma A/S, including expected benefits of such agreement and anticipated royalty payments, and future commercialization of licensed products and BioMarin's expectations to continue to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on its momentum for children with achondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's ability to enforce the agreement; actual sales of licensed products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Andrew Villani

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558 

(628) 269-7393

SOURCE BioMarin Pharmaceutical Inc.

ADAR1 Capital Management LLC acquired a new stake in BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) during the 2nd quarter, according to the company in its most recent disclosure with the Securities and Exchange Commission. The institutional investor acquired 49,000 shares of the biotechnology company’s stock, valued at approximately $2,804,000.

Other hedge funds and other institutional investors have also bought and sold shares of the company. NewEdge Advisors LLC boosted its position in BioMarin Pharmaceutical by 11.5% in the 1st quarter. NewEdge Advisors LLC now owns 3,769 shares of the biotechnology company’s stock valued at $266,000 after buying an additional 388 shares during the period. Empowered Funds LLC increased its position in shares of BioMarin Pharmaceutical by 207.4% during the first quarter. Empowered Funds LLC now owns 12,023 shares of the biotechnology company’s stock worth $850,000 after acquiring an additional 8,112 shares during the period. Focus Partners Wealth raised its stake in shares of BioMarin Pharmaceutical by 70.2% during the first quarter. Focus Partners Wealth now owns 11,817 shares of the biotechnology company’s stock valued at $835,000 after acquiring an additional 4,876 shares in the last quarter. Sivia Capital Partners LLC increased its holdings in BioMarin Pharmaceutical by 33.0% during the 2nd quarter. Sivia Capital Partners LLC now owns 4,439 shares of the biotechnology company’s stock worth $244,000 after purchasing an additional 1,102 shares during the period. Finally, Invesco Ltd. raised its position in BioMarin Pharmaceutical by 23.5% during the 2nd quarter. Invesco Ltd. now owns 618,835 shares of the biotechnology company’s stock valued at $34,017,000 after purchasing an additional 117,617 shares in the last quarter. Hedge funds and other institutional investors own 98.71% of the company’s stock.

Analyst Ratings Changes BMRN has been the topic of a number of recent research reports. Sanford C. Bernstein dropped their price target on BioMarin Pharmaceutical from $94.00 to $82.00 and set an “outperform” rating on the stock in a research report on Tuesday, May 5th. Canaccord Genuity Group lifted their price objective on BioMarin Pharmaceutical from $111.00 to $114.00 and gave the company a “buy” rating in a report on Monday, August 10th. Royal Bank Of Canada lowered their price objective on shares of BioMarin Pharmaceutical from $66.00 to $62.00 and set a “sector perform” rating for the company in a research note on Tuesday, July 7th. Bank of America dropped their target price on shares of BioMarin Pharmaceutical from $85.00 to $80.00 and set a “buy” rating on the stock in a report on Tuesday, May 19th. Finally, The Goldman Sachs Group assumed coverage on shares of BioMarin Pharmaceutical in a research report on Monday, May 11th. They set a “neutral” rating and a $69.00 target price on the stock. Two analysts have rated the stock with a Strong Buy rating, fifteen have given a Buy rating, six have given a Hold rating and one has issued a Sell rating to the stock. According to MarketBeat, the company has an average rating of “Moderate Buy” and a consensus price target of $88.83.

Check Out Our Latest Analysis on BMRN BioMarin Pharmaceutical Stock Performance Shares of BMRN stock opened at $66.12 on Wednesday. BioMarin Pharmaceutical Inc. has a 12-month low of $49.26 and a 12-month high of $70.98. The business’s fifty day simple moving average is $61.07 and its 200-day simple moving average is $57.99. The company has a current ratio of 2.39, a quick ratio of 1.32 and a debt-to-equity ratio of 0.56. The company has a market cap of $12.80 billion, a PE ratio of 178.70, a PEG ratio of 0.47 and a beta of 0.24.

BioMarin Pharmaceutical (NASDAQ:BMRN – Get Free Report) last posted its quarterly earnings data on Thursday, August 6th. The biotechnology company reported $1.20 earnings per share for the quarter, topping analysts’ consensus estimates of $0.95 by $0.25. The firm had revenue of $989.71 million during the quarter, compared to analysts’ expectations of $932.03 million. BioMarin Pharmaceutical had a return on equity of 5.32% and a net margin of 2.14%.The business’s quarterly revenue was up 20.0% on a year-over-year basis. During the same quarter last year, the firm earned $1.44 EPS. BioMarin Pharmaceutical has set its FY 2026 guidance at 4.900-5.100 EPS. Analysts expect that BioMarin Pharmaceutical Inc. will post 4.1 earnings per share for the current fiscal year.

BioMarin Pharmaceutical Company Profile (Free Report)

BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.

The company’s commercial portfolio includes several approved therapies targeting inherited disorders.

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BioMarin Pharmaceutical is making more waves on the M&A front. The company announced Tuesday that it will acquire Alesta Therapeutics for $275 million upfront. The deal gives BioMarin access to Alesta's oral treatment for hypophosphatasia, or HPP – a rare genetic bone disease. The drug, ALE1, is currently in early stage clinical trials, but has the potential to be the first oral treatment for HPP. BioMarin will pay up to an additional $215 million if the drug hits certain milestones. 

The move further bolsters BioMarin's unit focused on rare skeletal conditions, a portfolio anchored by its drug Voxzogo. It also marks the company's third major deal in the rare disease space over the past year, headlined by the $4.8 billion deal to buy Amicus Therapeutics announced last December.

"This is exactly the kind of opportunity to address a significant unmet need that lets us compete in larger rare disease markets – adding an asset that has the potential to reach our largest addressable patient population. We plan to continue to seek these kinds of opportunities as we focus on clinical-stage innovation to drive durable growth for BioMarin," BioMarin's president and CEO, Alexander Hardy, said in a statement released yesterday. 

The release also noted that more than 9,000 people in the United States have been diagnosed with HPP, though it is believed that the true number of people impacted by the disease is likely higher. 

Ilan Ganot, Alesta's CEO, said BioMarin's ability to scale the treatment factored into his board's decision to approve the deal. "BioMarin's global reach, scale, and proven expertise in rare disease drug development make it an ideal partner to advance ALE1 and realize its potential as a promising treatment for patients with HPP worldwide."

The deal is expected to close this quarter.

BioMarin to pay $275 million upfront, plus additional payments upon achievement of development and regulatory milestones

Alesta to spin out all non-ALE1 assets to a new entity and Alesta employees to transfer to the spinout entity prior to close

ALE1 has significant potential to help BioMarin expand into larger rare disease markets with a medicine intended to address a high unmet need for patients and offers strong strategic fit for the company

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that BioMarin has entered into a definitive agreement to acquire Alesta Therapeutics to gain Alesta's lead clinical-stage asset, ALE1. Alesta plans to spin out all non-ALE1 assets prior to the close of the transaction, which has been approved by the boards of directors of both companies and is expected to be completed this quarter, subject to customary closing conditions.

ALE1 is an orally active, small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. ALE1 has the potential to be the first oral therapy for HPP and is currently being evaluated in an ongoing Phase 1/2a clinical trial assessing safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP.

HPP is a serious condition that can affect bone and tooth mineralization, resulting in easy or frequent bone breaks, early tooth loss, and, in adults, clinically significant muscle weakness, fatigue and pain. If approved, ALE1 is expected to be the first oral therapy approach that targets the central disease metabolite, PPi (inorganic pyrophosphate), with the potential to impact both skeletal and broader manifestations of HPP through systemic correction of disease biology. The program will become part of BioMarin's Skeletal Conditions Business Unit following close.

"ALE1 is a strong strategic fit for BioMarin, bringing a potential oral alternative to the injectable therapies available today for people living with HPP around the world while meaningfully strengthening our early-stage clinical pipeline," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "This is exactly the kind of opportunity to address a significant unmet need that lets us compete in larger rare disease markets – adding an asset that has the potential to reach our largest addressable patient population. We plan to continue to seek these kinds of opportunities as we focus on clinical-stage innovation to drive durable growth for BioMarin."

Under the terms of the agreement, BioMarin will acquire Alesta and Alesta shareholders will receive an upfront payment of $275 million plus up to $215 million in subsequent payments upon achievement of certain development and regulatory milestones. Additionally, immediately prior to the close of BioMarin's acquisition of Alesta, Alesta will spin out all non-ALE1 assets to a new entity and former Alesta employees will transfer to the spinout entity. As a result, no Alesta employees will become employees of BioMarin in connection with the transaction. BioMarin intends to fund the transaction with cash on hand. BioMarin expects to provide updated full-year 2026 guidance reflecting the acquisition of Alesta following the closing of the transaction. Excluding the upfront consideration, the transaction is expected to have a modestly dilutive impact on 2026 financial results.

"We chose to partner with BioMarin due to their deep commitment to people living with rare diseases," said Ilan Ganot, Chief Executive Officer of Alesta Therapeutics. "BioMarin's global reach, scale, and proven expertise in rare disease drug development make it an ideal partner to advance ALE1 and realize its potential as a promising treatment for patients with HPP worldwide. This acquisition is also a testament to the extraordinary work, scientific expertise, and drug development capabilities of the Alesta team."

Morgan Stanley & Co. LLC is acting as the exclusive financial advisor to BioMarin, and Jones Day is serving as legal counsel in connection with the acquisition. J.P. Morgan Securities LLC is acting as exclusive financial advisor to Alesta, and Goodwin Procter LLP and NautaDutilh N.V. are serving as legal counsel.

About ALE1
ALE1 is designed to inhibit a novel target that regulates levels of inorganic pyrophosphate (PPi), the metabolite at the center of HPP pathology. By lowering excess PPi, ALE1 aims to restore healthier bone and mineral metabolism across the full spectrum of HPP.

More than 9,000 people have been diagnosed with HPP in the U.S., however, the disease is often underdiagnosed due to a broad spectrum of symptoms that can mimic more common conditions.

About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.

To learn more, please visit www.biomarin.com.

About Alesta Therapeutics
Alesta Therapeutics is a clinical-stage biotechnology company committed to developing novel oral small-molecule therapies for underserved diseases. The company's lead asset, ALE1, is being developed for hypophosphatasia (HPP), a rare genetic disorder with significant unmet need.

For more information, visit www.alestatherapeutics.com.  

Forward-Looking Statements
This press release contains forward-looking statements about, among other things, the proposed acquisition of ALE1, the lead clinical-stage asset, of Alesta Therapeutics (Alesta) by BioMarin Pharmaceutical Inc. (BioMarin) and the business prospects of BioMarin, including, without limitation, statements about: the anticipated occurrence, manner, funding and timing of the closing of the proposed acquisition; BioMarin's plans to update financial guidance; the potential impact of the acquisition on BioMarin's financial results and financial guidance; the prospective benefits of the proposed acquisition, including expectations that it will be a strong strategic fit for BioMarin and will meaningfully strengthen BioMarin's early-stage clinical pipeline; expectations regarding ALE1 and its ongoing development, including its potential to be a first-in-class oral therapy for the treatment of hypophosphatasia (HPP) and the potential benefits of ALE1 to patients with HPP around the world; BioMarin's plans to drive durable growth and strengthen its pipeline for the future; BioMarin's ability to compete in larger rare disease markets; BioMarin's expectations regarding unmet need and opportunities in HPP that may potentially be addressed by ALE1, including BioMarin's estimates regarding the prevalence of HPP; and other statements that are not historical facts.

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: consummating the proposed acquisition in the anticipated timeframe, if at all; Alesta's ability to complete the contemplated spinout of non-ALE1 assets prior to closing of the proposed acquisition, if at all; the possibility that competing offers or acquisition proposals will be made; the possibility that various closing conditions for the transaction may not be satisfied or waived, including that a governmental entity may prohibit, delay, or refuse to grant approval for the consummation of the transaction (or only grant approval subject to adverse conditions or limitations); the difficulty of predicting the timing or outcome of regulatory approvals or actions, if any; the effects of the proposed acquisition (or the announcement thereof) on BioMarin's stock price and/or BioMarin's operating results; unknown or inestimable liabilities; the development, launch and commercialization of products and product candidates; BioMarin's ability to realize the anticipated benefits of the proposed acquisition, including the possibility that the expected benefits from the proposed acquisition will not be realized or will not be realized within the expected time period and that integration will not be successful or that such integration may be more difficult, time-consuming or costly than expected; the time-consuming and uncertain regulatory approval process for pharmaceutical product development; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients, including with respect to current and planned future clinical trials; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to BioMarin's business operations and financial results; the sufficiency of BioMarin's cash flows and capital resources; BioMarin's ability to fund the acquisition; BioMarin's evaluation of the potential impact of the transaction on its financial results and financial guidance; BioMarin's ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the effects of the transaction on relationships with key third parties, including employees, customers, suppliers, other business partners or governmental entities; transaction costs; risks that the proposed acquisition disrupts current plans and operations; risks that the proposed transaction diverts management's attention from ongoing business operations; changes in Alesta's business during the period between announcement and closing of the proposed acquisition; any legal proceedings and/or regulatory actions that may be instituted related to the proposed acquisition; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

Contacts:

Investors

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Traci McCarty

Erin Rau

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(925) 683-9622

SOURCE BioMarin Pharmaceutical Inc.

Can BioMarin Stock Live Up to Wall Street’s High Expectations?BioMarin Pharmaceutical NASDAQ: BMRN outlined its growth strategy, integration plans for Amicus Therapeutics and pipeline priorities during a Canaccord discussion with Chief Financial Officer Brian Mueller.

Mueller described BioMarin as one of the largest rare-disease-focused biopharmaceutical companies, with more than 25 years of operating history, six first-in-disease medicines and nine approved therapies. The company operates in more than 80 countries and expects revenue approaching $4 billion this year, he said.

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3 Oversold Stocks with Big RSI Rebound PotentialThe company’s strategy centers on genetically defined rare diseases, where BioMarin seeks to identify the underlying genetic cause of a condition and develop precision medicines targeted at that cause. Mueller said that approach can support smaller clinical studies, regulatory incentives and strong engagement with patient advocacy communities.

Quarterly Performance and Amicus Integration BioMarin reported nearly $1 billion in second-quarter revenue, representing 20% year-over-year growth, Mueller said. The company raised total-revenue guidance to a range of $3.875 billion to $3.925 billion and raised non-GAAP earnings-per-share guidance to $4.90 to $5.10.

Are Gene Therapy Stocks The Market's Next Big Winners?During the quarter, BioMarin completed its acquisition of Amicus Therapeutics, adding two commercial rare-disease products: Galafold for Fabry disease and Pombiliti and Opfolda for Pompe disease. Mueller characterized the products as high-growth assets that can benefit from BioMarin’s larger global infrastructure.

BioMarin estimates peak revenue of $1.4 billion for Galafold and $1.2 billion for Pombiliti and Opfolda, with those opportunities expected in the mid- to late 2030s, respectively. The company also expects to realize synergies equal to roughly 50% of Amicus’ legacy operating-cost base, primarily through general and administrative expenses and other support functions.

Mueller said BioMarin is preserving and investing in the acquired sales and marketing organization rather than pursuing aggressive commercial cost reductions. “These products are in their high-growth phase,” he said, adding that maintaining customer-facing capabilities is important to sustaining growth.

BioMarin originally targeted a leverage ratio below 2.5 times within two years of the acquisition’s closing. Following its assessment of revenue opportunities, operating synergies and the combined company’s EBITDA profile, management now expects to reach that target by the middle of next year, roughly a year earlier than originally planned.

The company raised approximately $3.6 billion in debt for the transaction and also has a $600 million convertible note maturing next May, Mueller said. By 2030, BioMarin expects the Amicus business to operate at an approximately 60% operating margin.

Commercial Opportunities for Galafold and Pompe Franchise Mueller cited geographic expansion as a major growth driver for the acquired portfolio. Amicus had commercialized Galafold in roughly 40 markets and Pombiliti in approximately 15 markets, compared with BioMarin’s presence in more than 80 countries.

BioMarin does not expect to bring both medicines to every market where it operates, but Mueller said its on-the-ground commercial, market-access, medical and regulatory teams could accelerate international expansion in complex markets.

For Galafold, BioMarin sees an opportunity to improve diagnosis of Fabry disease, which Mueller said remains significantly underdiagnosed. The company plans to use efforts including AI-enabled patient identification, newborn screening and familial genetic cascade screening.

For Pombiliti and Opfolda, BioMarin’s strategy includes encouraging treatment switches from existing enzyme therapies. Mueller said there is an emerging body of real-world evidence suggesting improved outcomes with Pombiliti and Opfolda, while the efficacy of some existing therapies can wane over time.

Pipeline, Business Development and VOXZOGO BioMarin’s business-development focus remains centered on rare, genetically defined diseases. The company plans to prioritize opportunities that can use its internal manufacturing, development, regulatory, clinical and commercial infrastructure, Mueller said.

Management will continue to pursue early-stage collaborations and in-licensing opportunities, including a recently announced collaboration with the n-Lorem Foundation for a rare neurologic disease. As BioMarin reduces leverage, it may consider larger mid- to late-stage opportunities, though Mueller did not provide specifics on deal size or timing.

The company also raised revenue guidance for VOXZOGO to at least $1 billion after what Mueller described as strong first-half performance. VOXZOGO revenue grew 14% in the first quarter, and BioMarin added 20% new patients globally, he said.

VOXZOGO is approved for achondroplasia, and BioMarin has submitted a supplemental new drug application to the FDA for hypochondroplasia following positive Phase III results. If the filing is accepted, a potential approval and launch could occur next year, according to Mueller.

BioMarin estimates there are about 24,000 achondroplasia patients worldwide and approximately 14,000 hypochondroplasia patients. Mueller said diagnosis will be a key challenge in hypochondroplasia, but the company believes its established VOXZOGO presence in achondroplasia provides a foundation for the potential label expansion.

Separately, Mueller highlighted BMN 820, formerly DMX-200 at Amicus, as a potentially meaningful development asset. The small-molecule treatment is being studied for focal segmental glomerulosclerosis, or FSGS, a kidney disorder. BioMarin holds U.S. rights, estimates a U.S. population of roughly 30,000 patients and noted that the FDA agreed to proteinuria as the primary endpoint in the fully enrolled two-year study.

About BioMarin Pharmaceutical (NASDAQ:BMRN)BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.

The company's commercial portfolio includes several approved therapies targeting inherited disorders.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].

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Should You Invest $1,000 in BioMarin Pharmaceutical Right Now?Before you consider BioMarin Pharmaceutical, you'll want to hear this.

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Stock to Watch: BioMarin Pharmaceutical (BMRN - Free Report) San Rafael, CA-based BioMarin Pharmaceutical Inc. focuses on the development and commercialization of treatments for life-threatening severe medical conditions, mainly for children.

BMRN is a #2 (Buy) on the Zacks Rank, with a VGM Score of B.

It also boasts a Value Style Score of B thanks to attractive valuation metrics like a forward P/E ratio of 14.02; value investors should take notice.

Two analysts revised their earnings estimate upwards in the last 60 days for fiscal 2026. The Zacks Consensus Estimate has increased $0.01 to $4.96 per share. BMRN boasts an average earnings surprise of +67.5%.

With a solid Zacks Rank and top-tier Value and VGM Style Scores, BMRN should be on investors' short list.

Resources Investor Relations Journalists Agencies Client Login Send a Release News Products Contact , /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced management will present at the Canaccord Genuity 46th Annual Growth Conference on Wednesday, August 12, 2026, at 8:00AM ET in Boston.

An audio webcast of the presentation will be available live. The webcast can be accessed at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the conference.

About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com. 

Contacts:

Investors

Media

Traci McCarty

Marni Kottle

BioMarin Pharmaceutical Inc.         

BioMarin Pharmaceutical Inc.

(415) 455-7558

(650) 374-2803

SOURCE BioMarin Pharmaceutical Inc.

Also from this source

BioMarin Pharmaceutical Inc. (BMRN) Q2 2026 Earnings Call August 6, 2026 4:30 PM EDT

Company Participants

Traci McCarty - Group Vice President
Alexander Hardy - President, CEO & Director
Cristin Hubbard - Executive VP & Chief Commercial Officer
Gregory Friberg - Executive VP and Chief Research & Development Officer
Brian Mueller - CFO & Executive VP of Finance

Conference Call Participants

Christopher Raymond - Raymond James & Associates, Inc., Research Division
Cory Kasimov - Evercore ISI Institutional Equities, Research Division
Jessica Fye - JPMorgan Chase & Co, Research Division
Tommie Reerink - Goldman Sachs Group, Inc., Research Division
Philip Nadeau - TD Cowen, Research Division
Eliana Merle - Barclays Bank PLC, Research Division
Mohit Bansal - Wells Fargo Securities, LLC, Research Division
Phoebe Tan - Jefferies LLC, Research Division
Paul Matteis - Stifel, Nicolaus & Company, Incorporated, Research Division
Sean Laaman - Morgan Stanley, Research Division
Alexandria Hammond - Wolfe Research, LLC

Presentation

Operator

Good afternoon, and welcome, everyone, to the BioMarin Pharmaceutical Second Quarter 2026 Conference Call. Today's conference is being recorded. [Operator Instructions]

At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations.

Traci McCarty
Group Vice President

Thank you, operator, and thank you all for joining us today. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.

In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP

BioMarin Pharmaceutical (BMRN - Free Report) reported $989.71 million in revenue for the quarter ended June 2026, representing a year-over-year increase of 19.9%. EPS of $1.20 for the same period compares to $1.44 a year ago.

The reported revenue represents a surprise of +7.31% over the Zacks Consensus Estimate of $922.33 million. With the consensus EPS estimate being $0.96, the EPS surprise was +25%.

While investors closely watch year-over-year changes in headline numbers -- revenue and earnings -- and how they compare to Wall Street expectations to determine their next course of action, some key metrics always provide a better insight into a company's underlying performance.

As these metrics influence top- and bottom-line performance, comparing them to the year-ago numbers and what analysts estimated helps investors project a stock's price performance more accurately.

Here is how BioMarin performed in the just reported quarter in terms of the metrics most widely monitored and projected by Wall Street analysts:

Revenues- Net product revenues: $984.39 million versus the eight-analyst average estimate of $914.12 million. The reported number represents a year-over-year change of +21.1%.Revenues- Royalty and other revenues: $5.32 million compared to the $8.21 million average estimate based on eight analysts. The reported number represents a change of -57.2% year over year.Revenues- Net Product Revenues- NAGLAZYME: $135 million compared to the $125.91 million average estimate based on seven analysts. The reported number represents a change of +4.7% year over year.Revenues- Net Product Revenues- VIMIZIM: $194 million compared to the $205.82 million average estimate based on seven analysts. The reported number represents a change of -9.8% year over year.Revenues- Net Product Revenues- PALYNZIQ: $135 million versus $112.75 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +27.4% change.Revenues- Net Product Revenues- VOXZOGO: $253 million versus the seven-analyst average estimate of $238.2 million. The reported number represents a year-over-year change of +14.5%.Revenues- Net Product Revenues- KUVAN: $24 million versus the seven-analyst average estimate of $17.25 million. The reported number represents a year-over-year change of -11.1%.Revenues- Net Product Revenues- ALDURAZYME: $44 million versus the seven-analyst average estimate of $52.41 million. The reported number represents a year-over-year change of -21.4%.Revenues- Net Product Revenues- BRINEURA: $51 million versus $50.06 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +4.1% change.View all Key Company Metrics for BioMarin here>>>

Shares of BioMarin have returned +0.3% over the past month versus the Zacks S&P 500 composite's +3.3% change. The stock currently has a Zacks Rank #3 (Hold), indicating that it could perform in line with the broader market in the near term.

Second Quarter 2026 Total Revenues Increased 20% Year-over-year to $990 million

Stronger Growth Expectations Drive Increased Guidance for Full-year 2026 Total Revenues, VOXZOGO®, and Non-GAAP Diluted Earnings Per Share (EPS)

Addition of GALAFOLD® and POMBILITI® + OPFOLDA®, with Cost Synergies, Expected to Accelerate Revenue Growth, Non-GAAP Diluted EPS Accretion, Non-GAAP Operating Margin Expansion, and Operating Cash Flow through the Mid-2030s

Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the second quarter ended June 30, 2026.

"This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin's operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026.  Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations." Mr. Hardy added, "With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability. We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world."

2026 Business and Pipeline Highlights

Innovation

BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch. The company plans to provide an update on the application status as part of its third quarter earnings update. In May, the company announced that the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity (AGV) at week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), along with significant improvements in standing height, height Z-score, and the key secondary endpoint of arm span. The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September. In June, at the Endocrine Society Annual Meeting (ENDO 2026), a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through year three, with mean height standard deviation score (SDS) improving 0.72 over the three years. Also at ENDO 2026, the company presented Phase 1 data for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a single-ascending-dose study in healthy adults, BMN 333 demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent, reflecting its potential to become a new standard of care in achondroplasia. The Phase 2/3 study is enrolling, with a data update expected in 2027. In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027. In the second quarter, the European Commission approved PALYNZIQ® for adolescents 12 years and older with phenylketonuria (PKU). PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. During the quarter, BioMarin added BMN 820 (formerly DMX-200) to its portfolio, a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS) for which BioMarin holds exclusive U.S. commercialization rights. BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000. The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028. BMN 351, BioMarin's Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development. The company expects to provide a program update by year-end. Following the pivotal ENERGY 3 trial results, previously announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications. In July, BioMarin and the n-Lorem Foundation entered a collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. ReNU syndrome has an expected global population of approximately 100,000. Growth

BioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s and for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s. BioMarin expects these high growth therapies to benefit from its global scale and proven commercial capabilities. Metabolic Conditions (formerly Enzyme Therapies) revenue grew 25% Y/Y in the second quarter of 2026, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ. The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially. Strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion. The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter. In the U.S., the majority of new patient starts were under two years of age, and the region drove approximately 25% of total VOXZOGO revenue during the quarter. Value Commitment

As part of the acquisition of Amicus, which closed on April 27, 2026, the company identified approximately $280 million of cost reductions on a GAAP basis, and approximately $220 million of cost reductions on a Non-GAAP basis, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. Synergies reflect a reduction of Amicus' legacy labor costs and external spend and are expected to be largely driven by general and administrative functions, with the large majority of sales and marketing capabilities retained to support continued commercial growth. GALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030. The company is targeting gross leverage below 2.5 times by mid-year 2027, an acceleration by approximately one year of prior timeline guidance provided at deal announcement, supported by profitability growth of the combined company. Second Quarter 2026 Financial Highlights

Total Revenues for the second quarter of 2026 were $990 million, an increase of $165 million compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired from Amicus on April 27, 2026, as well as new patients initiating VOXZOGO therapy across all regions and growth in U.S. patients treated with PALYNZIQ. These increases were partially offset by lower VIMIZIM® revenue due to the timing of large government orders outside the U.S. and lower ALDURAZYME® sales volume due to the timing of order fulfillment to Sanofi. GAAP Net Income for the second quarter of 2026 decreased to $45 million compared to $241 million for the same period in 2025. The decrease was primarily driven by the acquisition of Amicus, including integration and restructuring costs, intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, and amortization of inventory fair value step-up. Other drivers included higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO and higher Research and Development (R&D) spend related to BMN 401, which was acquired in the third quarter of 2025, partially offset by higher gross profit driven by revenue growth as described above. Non-GAAP Income for the second quarter of 2026 decreased to $236 million compared to $282 million for the same period in 2025. The decrease was primarily driven by higher interest expense, higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO, and higher R&D spend related to BMN 401, partially offset by higher gross profit driven by revenue growth as described above. Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

% Change

2026

2025

% Change

Total Revenues

$990

$825

20 %

$1,756

$1,571

12 %

Net Product Revenues by Product:

VOXZOGO

$253

$221

14 %

$472

$435

9 %

Metabolic Conditions:

VIMIZIM

$194

$215

(10) %

$405

$404

— %

NAGLAZYME®

135

129

5 %

265

243

9 %

PALYNZIQ

135

106

27 %

225

199

13 %

GALAFOLD

106

—

NM

106

—

NM

BRINEURA®

51

49

4 %

98

89

10 %

ALDURAZYME

44

56

(21) %

80

105

(24) %

POMBILITI + OPFOLDA

30

—

NM

30

—

NM

Total Metabolic Conditions Revenue

$695

$555

25 %

$1,209

$1,040

16 %

KUVAN®

$24

$27

(11) %

$48

$52

(8) %

ROCTAVIAN®(1)

$12

$9

33 %

$14

$20

(30) %

GAAP Net Income

$45

$241

(81) %

$150

$426

(65) %

Non-GAAP Income (2)

$236

$282

(16) %

$385

$502

(23) %

GAAP Operating Margin % (3)

11.2 %

33.5 %

13.7 %

31.9 %

Non-GAAP Operating Margin % (2)

36.4 %

39.9 %

31.0 %

37.9 %

GAAP Diluted EPS

$0.23

$1.23

(81) %

$0.77

$2.19

(65) %

Non-GAAP Diluted EPS (2)

$1.20

$1.44

(17) %

$1.96

$2.57

(24) %

NM

Percentage change is not meaningful for products acquired from Amicus on April 27, 2026.

(1)

In 2026, the company announced that it will no longer market ROCTAVIAN.

(2)

Refer to Non-GAAP Information beginning on page 10 of this press release for definitions of Non-GAAP Income, Non-GAAP Operating Margin percentage and Non-GAAP Diluted EPS along with the related reconciliations to the comparable information reported under U.S. GAAP.

(3)

GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations divided by Total Revenues.

Forward-Looking Non-GAAP Financial Information

BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.

Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts)

Total Revenues, VOXZOGO, and Non-GAAP Diluted EPS guidance raised, reflecting strong first-half 2026 performance and second-half 2026 revenue expectations for both Metabolic Conditions and VOXZOGO. Guidance reflects post-close contributions from Amicus beginning April 27, 2026. BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non‑GAAP financial results. Based on current rates, interest expense associated with the financing is estimated at approximately $200 million on an annualized basis, with Term Loans and Senior Notes scheduled to mature after 2030. Item

Provided on May 4, 2026

Updated August 6, 2026

Midpoint Growth
(Y/Y)

Total Revenues

$3,825

to

$3,925

$3,875

to

$3,925

21 %

Metabolic Conditions

$2,725

to

$2,775

Unchanged

31 %

VOXZOGO

$975

to

$1,025

$1,000

to

$1,050

11 %

Other Revenues(1)

$100

to

$125

Unchanged

Non-GAAP Diluted EPS (2)(3)(4)

$4.85

to

$5.05

$4.90

to

$5.10

59 %

(1)

Other Revenues includes KUVAN, ROCTAVIAN, and royalties.

(2)

Refer to Non-GAAP Information beginning on page 10 of this press release for definition of Non-GAAP Diluted EPS.

(3)

Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.

(4)

Non-GAAP Diluted EPS guidance assumes a combined company tax rate of 22%.

BioMarin will host a conference call and webcast to discuss second quarter 2026 financial results today, Thursday, August 6, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

U.S./Canada Dial-in Number: 800-715-9871

Replay Dial-in Number: 800-770-2030

International Dial-in Number:  646-307-1963

Replay International Dial-in Number: 609-800-9909

Conference ID:  3551298 

Conference ID: 3551298 

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Diluted EPS, Non-GAAP Operating Margin, gross leverage, operating cash flow and revenue compound annual growth rate (CAGR) for, in certain instances, the full-year 2026, fourth quarter and second half of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin's Metabolic Conditions portfolio, including PALYNZIQ, and VOXZOGO, and the expected impact of the acquisition of Amicus Therapeutics, Inc. (Amicus); the anticipated benefits of the acquisition of Amicus, including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA, including BioMarin's plans and expectations to accelerate growth through mid-2030s; BioMarin's plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin's product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin's commercial products and product candidates, including with respect to the potential new indication for VOXZOGO in hypochondroplasia; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products.

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; BioMarin's ability to realize the anticipated benefits of any acquisitions; BioMarin's ability to accurately estimate future financial performance; impacts of macroeconomic and other external factors on BioMarin's operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; geopolitical instability, wars and military conflicts; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin's ability to meet product demand; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN®, ROCTAVIAN®, GALAFOLD®, and POMBILITI® + OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF INCOME

Three and Six Months Ended June 30, 2026 and 2025

(In thousands of U.S. dollars, except per share amounts)

(Unaudited)

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

REVENUES:

Net product revenues

$         984,393

$         812,982

$       1,744,471

$       1,547,626

Royalty and other revenues

5,315

12,428

11,445

22,929

Total revenues

989,708

825,410

1,755,916

1,570,555

OPERATING EXPENSES:

Cost of sales

202,795

150,090

397,794

301,648

Research and development

206,952

161,308

385,748

320,039

Selling, general and administrative

395,549

232,279

653,839

438,395

Intangible asset amortization

73,492

4,846

77,975

9,693

Total operating expenses

878,788

548,523

1,515,356

1,069,775

INCOME FROM OPERATIONS

110,920

276,887

240,560

500,780

Interest income

10,480

18,827

33,040

37,840

Interest expense

(63,295)

(2,679)

(78,253)

(5,542)

Other income, net

3,279

4,833

7,240

2,879

INCOME BEFORE INCOME TAXES

61,384

297,868

202,587

535,957

Provision for income taxes

16,622

57,336

52,298

109,739

NET INCOME

$          44,762

$        240,532

$        150,289

$        426,218

EARNINGS PER SHARE, BASIC

$              0.23

$              1.25

$              0.78

$              2.23

EARNINGS PER SHARE, DILUTED

$              0.23

$              1.23

$              0.77

$              2.19

Weighted average common shares outstanding, basic

193,423

191,907

192,959

191,440

Weighted average common shares outstanding, diluted

194,467

197,091

194,147

196,643

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

June 30, 2026 and December 31, 2025

(In thousands of U.S. dollars, except per share amounts)

(Unaudited)

June 30, 2026

December 31, 2025

ASSETS

Current assets:

Cash and cash equivalents

$              874,005

$            1,311,679

Short-term investments

—

248,930

Accounts receivable, net

1,061,047

908,214

Inventory

1,782,524

1,298,883

Other current assets

254,047

185,784

Total current assets

3,971,623

3,953,490

Noncurrent assets:

Long-term investments

—

492,242

Property, plant and equipment, net

989,994

952,508

Intangible assets, net

4,879,367

213,837

Goodwill

655,745

196,199

Deferred tax assets

888,575

1,508,697

Other assets

338,138

277,049

Total assets

$           11,723,442

$            7,594,022

LIABILITIES AND STOCKHOLDERS' EQUITY

Current liabilities:

Accounts payable and accrued liabilities

$            1,005,793

$              759,031

Current portion of long-term debt, net

658,203

—

Total current liabilities

1,663,996

759,031

Noncurrent liabilities:

Long-term debt, net

3,527,939

597,176

Other long-term liabilities

209,815

150,816

Total liabilities

5,401,750

1,507,023

Stockholders' equity:

Common stock, $0.001 par value: 500,000,000 shares authorized; 193,535,556 and 192,300,101 shares issued and outstanding, respectively

194

192

Additional paid-in capital

6,037,019

5,956,582

Company common stock held by the Nonqualified Deferred Compensation Plan

(11,233)

(10,508)

Accumulated other comprehensive income (loss)

(8,783)

(13,473)

Retained earnings

304,495

154,206

Total stockholders' equity

6,321,692

6,086,999

Total liabilities and stockholders' equity

$           11,723,442

$            7,594,022

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

Six Months Ended June 30, 2026 and 2025

(In thousands of U.S. dollars)

(Unaudited)

Six Months Ended June 30,

2026

2025

CASH FLOWS FROM OPERATING ACTIVITIES:

Net income

$          150,289

$          426,218

Adjustments to reconcile net income to net cash provided by operating activities:

Depreciation and amortization

114,049

40,632

Non-cash interest expense

27,912

1,320

Stock-based compensation

119,152

85,231

Impairment of assets

—

2,967

Deferred income taxes

2,261

61,771

Unrealized foreign exchange gains

(4,046)

(5,306)

Other

(5,534)

(4,633)

Changes in operating assets and liabilities, net of effects of business acquired:

Accounts receivable, net

(46,005)

(156,124)

Inventory

12,334

(72,462)

Other current assets

(23,589)

(15,092)

Other assets

9,005

(13,505)

Accounts payable and accrued liabilities

26,265

3,111

Other long-term liabilities

6,667

5,537

Net cash provided by operating activities

388,760

359,665

CASH FLOWS FROM INVESTING ACTIVITIES:

Purchases of property, plant and equipment

(49,739)

(33,869)

Maturities and sales of investments

767,277

195,738

Purchases of investments

(25,792)

(202,433)

Purchase of intangible assets

(5,433)

(266)

Acquisition of Amicus, net of cash acquired

(5,067,630)

—

Other

4,966

—

Net cash used in investing activities

(4,376,351)

(40,830)

CASH FLOWS FROM FINANCING ACTIVITIES:

Proceeds from exercises of awards under equity incentive plans

6,655

7,707

Taxes paid related to net share settlement of equity awards

(39,504)

(51,089)

Proceeds from borrowings

3,650,000

—

Payments of debt issuance costs

(65,604)

—

Net cash provided by (used in) financing activities

3,551,547

(43,382)

Effect of exchange rate changes on cash

(1,630)

(4,479)

NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS

(437,674)

270,974

Cash and cash equivalents:

Beginning of period

$        1,311,679

$          942,842

End of period

$          874,005

$        1,213,816

Non-GAAP Information
The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Cost of Sales (COS), Non-GAAP R&D expenses and Non-GAAP Selling, General and Administrative (SG&A) expenses are defined by the company as GAAP COS, GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans or convertible debt in periods when they are dilutive under Non-GAAP. Projected Gross Leverage is defined by the company as undiscounted debt (total debt excluding unamortized discount and deferred offering costs) as of the balance sheet date divided by 4 quarter projected Non-GAAP Adjusted EBITDA. Non-GAAP Adjusted EBITDA is defined by the company as GAAP Income (or Loss) from Operations excluding the impact of depreciation, amortization and stock-based compensation expense. Non-GAAP synergies is defined by the company as Amicus' legacy labor and external spend cost reductions, excluding the impact of stock-based compensation.

BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Because these Non-GAAP metrics are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business.

Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:

Reconciliation of GAAP Reported Information to Non-GAAP Information (1)

(In millions of U.S. dollars, except per share data)

(unaudited)

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

GAAP Reported Net Income

$         45

$        241

$        150

$        426

Adjustments

Stock-based compensation expense - COS

5

4

9

6

Stock-based compensation expense - R&D

16

14

28

26

Stock-based compensation expense - SG&A(2)

55

30

83

53

Amortization of intangible assets

73

5

78

10

Amortization of acquired inventory step-up(3)

12

—

12

—

Acquisition-related costs(3)

84

—

84

—

Severance costs (4)

3

—

12

—

Loss on investments (5)

—

—

—

3

Income tax effect of adjustments

(57)

(11)

(70)

(22)

Non-GAAP Income

$        236

$        282

$        385

$        502

Three Months Ended

June 30,

2026

2025

COS

R&D

SG&A

COS

R&D

SG&A

GAAP expenses

$     203

$      207

$      396

$      150

$      161

$       232

Adjustments

Stock-based compensation expense(2)

(5)

(16)

(55)

(4)

(14)

(30)

Amortization of acquired inventory step-up(3)

(12)

—

—

—

—

—

Acquisition-related costs (3)

—

—

(84)

—

—

—

Severance costs (4)

—

—

(3)

—

—

—

Non-GAAP expenses

$     186

$      191

$      253

$      146

$      147

$       203

Six Months Ended

June 30,

2026

2025

COS

R&D

SG&A

COS

R&D

SG&A

GAAP expenses

$      398

$      386

$      654

$      302

$      320

$      438

Adjustments

Stock-based compensation expense(2)

(9)

(28)

(83)

(6)

(26)

(53)

Amortization of acquired inventory step-up(3)

(12)

—

—

—

—

—

Acquisition-related costs (3)

—

—

(84)

—

—

—

Severance costs (4)

—

—

(12)

—

—

—

Non-GAAP expenses

$      378

$      358

$      475

$      295

$      294

$      385

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

Percent
of GAAP
Total
Revenue

2025

Percent
of GAAP
Total
Revenue

2026

Percent
of GAAP
Total
Revenue

2025

Percent
of GAAP
Total
Revenue

GAAP Income from Operations

$      111

11.2 %

$     277

33.5 %

$     241

13.7 %

$     501

31.9 %

Adjustments

Stock-based compensation expense(2)

76

7.7

48

5.8

120

6.8

85

5.4

Amortization of intangible assets

73

7.4

5

0.6

78

4.4

10

0.6

Amortization of acquired inventory step-up(3)

12

1.2

—

—

12

0.7

—

—

Acquisition-related costs (3)

84

8.5

—

—

84

4.8

—

—

Severance costs (4)

3

0.3

—

—

12

0.7

—

—

Non-GAAP Income from Operations

$      360

36.4 %

$     329

39.9 %

$     545

31.0 %

$     596

37.9 %

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

GAAP Diluted EPS

$       0.23

$       1.23

$       0.77

$       2.19

Adjustments

Stock-based compensation expense(2)

$       0.38

$       0.24

0.60

0.43

Amortization of intangible assets

$       0.37

$       0.03

0.39

0.05

Amortization of acquired inventory step-up(3)

$       0.06

$          —

0.06

—

Acquisition-related costs(3)

$       0.42

$          —

0.42

—

Severance costs (4)

$       0.02

$          —

0.06

—

Loss on investments (5)

$          —

$          —

—

0.02

Income tax effect of adjustments

$      (0.29)

$      (0.06)

(0.35)

(0.11)

Non-GAAP Diluted EPS

$       1.20

$       1.44

$       1.96

$       2.57

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

GAAP Weighted-Average Diluted Shares Outstanding

194.5

197.1

194.1

196.6

Adjustments

Common stock issuable under company's convertible debt (6)

4.4

—

4.4

—

Non-GAAP Weighted-Average Diluted Shares Outstanding

198.9

197.1

198.5

196.6

(1)

Certain amounts may not sum or recalculate due to rounding.

(2)

Stock-based compensation expense recorded in SG&A for the three and six months ended June 30, 2026, includes approximately $13 million related to the post-combination service period for unvested Amicus stock options.

(3)

These amounts represent costs resulting from the Amicus acquisition that closed on April 27, 2026. Acquisition-related costs were included in SG&A and consisted of severance, transaction and integration costs. Amortization of acquired inventory step-up was included in COS.

(4)

These amounts were included in SG&A and represent charges for severance in connection with the company's plan to simplify its organizational design and strategic initiatives in the first and second quarters of 2026.

(5)

Represents impairment loss on non-marketable equity securities recorded in Other income, net, in the first quarter of 2025.

(6)

Common stock issuable under the company's convertible debt were excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding for the three and six months ended June 30, 2026 as they were anti-dilutive.

Amicus Therapeutics, Inc. (1)

Reconciliation of Non-GAAP Financial Measures

(in thousands)

(Unaudited)

Twelve Months Ended

December 31, 2025

Total operating expenses - as reported GAAP

$                528,492

Research and development:

Share-based compensation

12,156

Selling, general and administrative:

Share-based compensation

75,254

Loss on impairment of assets

1,702

Depreciation and amortization

7,460

Total operating expense adjustments to reported GAAP

96,572

Total operating expenses - as adjusted

$                431,920

(1)

The above historical reconciliation is reproduced from Amicus' earnings release furnished as Exhibit 99.1 to its Current Report on Form 8-K dated February 20, 2026 and reflects Amicus' historical definitions of the applicable non-GAAP measures.

Contact:

Investors:

Media:

Traci McCarty

Marni Kottle

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(650) 374-2803

SOURCE BioMarin Pharmaceutical Inc.

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Alexander Hardy, President and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, August 6, 2026, at 4:30 p.m. ET to discuss second quarter 2026 financial results and provide a general business update.

Dial-in Number 
U.S. / Canada Dial-in Number: 800-715-9871
International Dial-in Number: 646-307-1963
Conference Call ID: 3551298 

U.S. / Canada Replay Dial-in Number: 800-770-2030
International Replay Dial-in Number: 609-800-9909
Playback ID: 3551298 

Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, https://investors.biomarin.com/. A replay of the call will be archived on the site for one week following the call.

About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com. 

Contacts:

Investors

Media

Traci McCarty

Marni Kottle

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(650) 374-2803

SOURCE BioMarin Pharmaceutical Inc.

ReNU syndrome was discovered in 2024 and there are no medicines approved for the condition

Collaboration combines BioMarin's leadership in genetic medicines with n-Lorem's pioneering antisense expertise

Investigational antisense oligonucleotide (ASO) aims to address the underlying genetic cause of this serious neurodevelopmental condition

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and n-Lorem Foundation, a nonprofit organization, today announced a strategic collaboration and global exclusive license agreement to develop a first-in-disease, antisense oligonucleotide (ASO) medicine for people living with ReNU syndrome, a serious and rare neurodevelopmental condition caused by variants in the RNU4-2 gene.

Under the agreement, BioMarin and n-Lorem will collaborate to advance an investigational ASO candidate targeting the RNU4-2 (n.64_65insT) variant, which is estimated to account for approximately 75% of ReNU syndrome cases. ReNU syndrome was first discovered in 2024 by an international team of geneticists led by Dr. Nicola Whiffin at the University of Oxford's Big Data Institute and Dr. Ernest Turro at the Mt. Sinai Icahn School of Medicine. There are currently no approved medicines that address the underlying cause of disease.

Both BioMarin and n-Lorem will conduct preclinical studies and collaborate to select the lead candidate to move forward in clinical studies.

"ReNU syndrome was identified as a distinct genetic condition in 2024, thanks in large part to the pioneering efforts of families, advocates and researchers who helped raise awareness and accelerate understanding of this condition," said Kevin Eggan, Ph.D., Chief Scientific Officer at BioMarin. "For many families, a ReNU diagnosis can finally provide answers, but currently there are no approved medicines that address the underlying cause of the disease. By combining BioMarin's expertise in genetic medicines with n-Lorem's pioneering antisense capabilities, we aim to bring the first treatment option for people living with ReNU syndrome."

The n-Lorem Foundation typically focuses on conditions with a very small number of individuals (approximately 30 people or less) worldwide. When a program has the potential to reach a broader population, the foundation seeks a partner such as BioMarin to support development. In the case of ReNU syndrome, the foundation began its program and accepted a number of patients with RNU4-2 to initiate individualized clinical trials in the coming months. Through this new collaboration, BioMarin will lead the development of the investigational medicine for the wider ReNU syndrome community.

"We are proud to partner with BioMarin, a company that shares our urgency and has the scientific, clinical, and commercial expertise to bring this innovative new medicine to better help people living with ReNU Syndrome globally," said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO of n-Lorem. "Our commitment is to develop ASO medicines and, when we recognize the opportunity to support even more individuals, identify a partner that can advance our medicines to be commercially approved."

ReNU syndrome is a rare genetic neurodevelopmental condition associated with cognitive, language and adaptive behavioral impairments. ReNU syndrome is projected to be one of the leading monogenetic causes for developmental delay and impairment, with an expected global population of approximately 100,000.

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com. 

About n-Lorem

n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem received over 475 applications for treatment with more than 275 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. For more information, please visit www.nlorem.org.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: plans and expectations regarding the strategic collaboration and global exclusive license agreement between BioMarin and n-Lorem Foundation to develop a first-in-disease, antisense oligonucleotide (ASO) medicine for people living with ReNU syndrome; prospects and timing of actions relating to preclinical and clinical studies and approvals; and BioMarin's estimates regarding global population with ReNU syndrome as well as the prevalence of the RNU4-2 variant. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of planned preclinical and clinical studies; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.

BioMarin Contacts:

Investors 
Traci McCarty 
BioMarin Pharmaceutical Inc. 
(415) 455-7558 

Media
Andrew Villani
BioMarin Pharmaceutical Inc.
(628) 269-7393

n-Lorem Foundation Contact:

Amy Williford
n-Lorem Foundation
(760) 378-8005

SOURCE BioMarin Pharmaceutical Inc.

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