Track market-moving stories before they get noisy

Martin Shkreli, known as Pharma Bro, said Ultragenyx Pharmaceutical (NASDAQ:RARE) is undervalued and a potential takeover target for rare-disease peers like BioMarin Pharmaceuticals (NASDAQ:BMRN) after a trial setback.

He said "$RARE is very cheap" and urged BioMarin and other rare-disease companies to explore buying the company, adding that "everyone and their brother with 3 months of med school/shkreli training knew this trial would fail."

Ultragenyx’s Cost-Cutting Measures Ultragenyx is planning significant expense reductions after its Phase 3 Aspire study for the Angelman syndrome therapy, apazunersen, failed to meet primary endpoints. The trial’s failure has prompted the company to review the program’s future and implement cost-cutting measures. Analyst Downgrades for Ultragenyx In a Thursday note, analysts downgraded Ultragenyx’s stock, with Baird’s Joel Beatty cutting the price target from $40 to $16.53. Evercore ISI Group’s Liisa Bayko also downgraded the stock and reduced the target price to $16.53, reflecting the impact of the trial results. These downgrades were part of a broader analyst review, which also included changes for Moderna, Inc. Technical AnalysisRARE’s RSI(14) is at 18.99, indicating an oversold condition. The stock trades 47.39% below its 50-day SMA of $27.99 and 43.54% below its 200-day SMA of $26.08. The 50-day SMA remains above the 200-day SMA.

RARE Stock Price Activity: Ultragenyx Pharmaceutical shares were down 43.95% at $14.87 on Thursday, according to Benzinga Pro data.

Read Next

Image: Shutterstock

Read Next

This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.

Market News and Data brought to you by Benzinga APIs

© 2026 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

To add Benzinga News as your preferred source on Google, click here.

It has been about a month since the last earnings report for Ultragenyx (RARE - Free Report) . Shares have added about 6.4% in that time frame, outperforming the S&P 500.

But investors have to be wondering, will the recent positive trend continue leading up to its next earnings release, or is Ultragenyx due for a pullback? Well, first let's take a quick look at its latest earnings report in order to get a better handle on the recent drivers for Ultragenyx Pharmaceutical Inc. before we dive into how investors and analysts have reacted as of late.

Ultragenyx's Q2 Earnings Beat Estimates, Revenues Jump Y/YUltragenyxreported second-quarter 2026 loss of 90 cents per share, which was narrower than the Zacks Consensus Estimate of a loss of $1.27. The company had incurred a loss of $1.17 per share in the year-ago quarter.

Total revenues in the second quarter were $214 million, which surged 28.1% year over year due to higher product sales. The top line also beat the Zacks Consensus Estimate of $181 million.

Management stated that second-quarter 2026 revenues were the highest quarterly revenues ever reported by the company.

RARE’s Q2 Results in DetailCrysvita’s total revenues were $156 million, up 28.9% year over year. Management noted that Crysvita sales were consistent with expected seasonality in the United States and Canada and ordering patterns in Latin America. Crysvita’s net product revenues in the second quarter of 2026 included $94 million from North America, $54 million from Latin America and Turkey, and $8 million from Europe.

Mepsevii product revenues increased 11.1% year over year to $10 million in the reported quarter. Dojolvi product revenues were $27 million, up 17.4%, driven by strong demand. Evkeeza recorded sales of $21 million in the second quarter, up 50%, driven by increased demand from new country launches and early access.

Operating expenses of $289 million in the quarter rose 5.1% year over year due to increased investments in multiple late-stage pipeline programs and marketing costs for approved drugs. Operating expenses included research and development (R&D) expenses of $167 million (up 1.2%), selling, general and administrative (SG&A) expenses of $88 million (up 1.1%) and cost of sales of $34 million (up 47.8%).

Cash, cash equivalents and marketable securities amounted to $436 million as of June 30, 2026, compared with $534 million as of March 31, 2026.

RARE Reiterates 2026 Financial GuidanceUltragenyx continues to expect total revenues in 2026, excluding potential revenues from new product launches, between $730 million and $760 million.

Crysvita revenues in 2026 are expected to be in the range of $500-$520 million, reflecting growing underlying global demand. Meanwhile, Dojolvi revenues are expected to be between $100 million and $110 million in 2026.

How Have Estimates Been Moving Since Then?It turns out, estimates revision have trended downward during the past month.

The consensus estimate has shifted 25.84% due to these changes.

VGM ScoresCurrently, Ultragenyx has a average Growth Score of C, however its Momentum Score is doing a lot better with an A. However, the stock was allocated a score of F on the value side, putting it in the lowest quintile for value investors.

Overall, the stock has an aggregate VGM Score of C. If you aren't focused on one strategy, this score is the one you should be interested in.

OutlookEstimates have been trending downward for the stock, and the magnitude of these revisions looks promising. Notably, Ultragenyx has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months.

Performance of an Industry PlayerUltragenyx belongs to the Zacks Medical - Biomedical and Genetics industry. Another stock from the same industry, CRISPR Therapeutics AG (CRSP - Free Report) , has gained 8.5% over the past month. More than a month has passed since the company reported results for the quarter ended June 2026.

CRISPR Therapeutics reported revenues of $10.18 million in the last reported quarter, representing a year-over-year change of +1043.8%. EPS of -$0.94 for the same period compares with -$1.29 a year ago.

CRISPR Therapeutics is expected to post a loss of $1.06 per share for the current quarter, representing a year-over-year change of +9.4%. Over the last 30 days, the Zacks Consensus Estimate has changed +5.9%.

CRISPR Therapeutics has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of F.

CompaniesSept 3 (Reuters) - Shares of Ultragenyx Pharmaceutical (RARE.O), opens new tab plunged more than 40% in premarket on Thursday after its neurodevelopmental disorder treatment failed a closely watched ​late-stage trial, dealing a major blow to one of ‌its biggest growth bets.

The drug, apazunersen, failed to meet both its main goal of improving cognitive skills and its secondary goals of overall patient response in ​participants with Angelman syndrome, a rare condition that affects the ​nervous system and impairs typical brain development in childhood.

Sign up here.

Ultragenyx ⁠said there were no differences between patients receiving the treatment and those ​on placebo, prompting a review of the program's future.

The trial outcome ​is a major blow to the company and the patient community, as there are currently no approved disease-modifying treatments for the condition.

It also marks Ultragenyx's second consecutive late-stage ​trial failure, a setback Jefferies analyst Maury Raycroft called "unambiguously negative" ​and likely to "further erode street confidence".

Following the trial results, at least five brokerages slashed ‌their ⁠price targets on the stock.

TD Cowen analysts said the failure "removes a key growth driver," while Cantor Fitzgerald analyst Kristen Kluska warned that Ultragenyx "truly has to lower its spend substantially for investors to want to ​even consider building ​a position".

Ultragenyx ⁠said it will implement "significant expense reductions" to manage its high operating costs.

Jefferies noted Ultragenyx's trial failure "raises risk ​for competitors" like Ionis Pharmaceuticals (IONS.O), opens new tab and Oak Hill Bio ​who ⁠are developing similar treatments.

"The investment case has changed shape," Leerink Partners analyst Joseph Schwartz said, arguing that Ultragenyx is now "a commercial and expense story ⁠rather ​than a pipeline execution story."

As of Wednesday's ​close, Ultragenyx stock was up 15.4% year-to-date, with a market value of $2.62 billion, according ​to LSEG data.

Reporting by Kamal Choudhury in Bengaluru; Editing by Vijay Kishore

Our Standards: The Thomson Reuters Trust Principles., opens new tab

 | Source: Ultragenyx Pharmaceutical Inc.

Phase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI)

NOVATO, Calif., Sept. 02, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome. The study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI). The safety profile observed in Aspire was consistent with Phase 1/2.

“Based on everything we observed in the robust Phase 1/2 clinical development program and long-term extension study, we are disappointed by the Aspire result,” said Emil Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “Even more, we are disappointed for the global patient community who has invested so much in early-stage research, working to bring a first-ever treatment to their children.”

In Aspire, the randomized groups were comparable at baseline and consistent with the patients studied in Phase 2. There were no differences between the treated and control groups that could support efficacy in the Bayley Cognition raw scores nor in the MDRI when looking at net response or mean changes of the individual five endpoints included in the MDRI.

The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.

Dr. Kakkis continued: “We will maintain focus on our growing commercial business, which continues to create meaningful value, including new sources of revenue from the recent approval of GENGLYCOS for glycogen storage disease type Ia, the potential approval of UX111 for Sanfillipo syndrome, and the expansion of existing products to new territories. This strong commercial foundation will support our pipeline, while continuing toward profitability in 2027.”

About apazunersen (GTX-102)
Apazunersen (GTX-102) is an investigational antisense oligonucleotide (ASO) therapy delivered via intrathecal administration and designed to target and inhibit expression of the UBE3A-AS to prevent silencing of the paternally inherited allele of the UBE3A gene and reactivate expression of the deficient protein. Apazunersen has been granted Breakthrough Therapy Designation, Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the FDA and Orphan Designation and PRIME designation from the EMA.

About Angelman Syndrome
Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system (CNS), a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A-AS, the intended target of apazunersen. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is generally not inherited but instead occurs spontaneously. It is estimated to affect approximately 60,000 people in commercially accessible geographies.

Angelman syndrome is a lifelong neurodevelopmental disorder that causes cognitive impairment, motor impairment, balance issues and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. Although individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome, suggesting that improvement of symptoms can potentially be achieved at any age.

About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx’s plans to evaluate its operations and implement significant expense reductions, the Company’s expectations for profitability in 2027, the expected scope, timing, benefits and impact of those actions, its future operating results and financial performance, its business plans and objectives for GTX-102 following the Aspire results, the future development and regulatory path for GTX-102, the growth and importance of its commercial business, and the potential approval and commercialization of UX111 are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the company’s ability to accurately analyze and interpret the Aspire results and determine an appropriate path forward for GTX-102, the uncertainty of clinical drug development and the unpredictability and lengthy process for obtaining regulatory approvals, the risk that results from earlier studies may not be predictive of future study results, the company’s ability to define and implement expense reductions and realize anticipated savings and benefits, the risk that expense reductions may disrupt the company’s operations, adversely affect its workforce or impair its ability to execute its business plans, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect the sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 5, 2026 and its subsequent periodic reports filed with the SEC. 

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Ultragenyx Contacts

Investors
Joshua Higa
[email protected]

Media
Jess Rowlands
[email protected]

At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50%

Complete elimination of nighttime cornstarch dosing observed in 33% of DTX401 treatment group and 42% of crossover group, while maintaining glycemic control

Analyses at Week 48 showed that 83% of DTX401-treated participants met or exceeded their own expectations for meaningful cornstarch reduction

NOVATO, Calif., Sept. 01, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the publication of 96-week data from its Phase 3 study of GENGLYCOS™ AAV gene therapy (pariglasgene brecaparvovec-opnr), also known as DTX401, for the treatment of glycogen storage disease type Ia (GSDIa) in The Journal of Inherited Metabolic Disease. GENGLYCOS was recently approved by the U.S. Food and Drug Administration (FDA) in patients ages eight and older with GSDIa.

“These results demonstrate the potential of gene therapy to provide greater stability in day-to-day life for patients with GSDIa and may help guard against the risk of severe hypoglycemia associated with missed doses of raw cornstarch," said Dr. John Mitchell, scientist in the Child Health and Human Development Program at the Research Institute of the McGill University Health Centre (The Institute), pediatric endocrinologist at the Montreal Children’s Hospital, lead author of the publication and an investigator on the study. “For me, the reduction in overnight cornstarch dosing will have the most meaningful impact by reducing sleep disruption, with patient-reported outcomes included in the publication underscoring the profound impact that cornstarch reductions may have on daily life. I view long-term outcomes from the 96-week period showing continued improvements as particularly important, offering valuable insight into how post-treatment management may continue to evolve and improve as clinical experience grows."

“The complete results from this Phase 3 study more fully capture the benefits of this gene therapy and the importance of providing patients the ability to breakdown glycogen to provide a source of glucose during fasting or times of increased metabolic demands,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “Most patients achieved cornstarch reductions that met or exceeded their own expectations, with substantially less overnight treatment burden and reduced dependence on the around-the-clock cornstarch need that defines life with this disease. Importantly, reducing cornstarch dependence while maintaining glycemic control indicates the establishment of the liver’s ability to regulate glucose production on its own, giving us confidence that the therapy is directly addressing the underlying cause of disease and offering protection from the risk of life-threatening hypoglycemia.”

Authors emphasize statistically significant and clinically meaningful reductions in cornstarch while maintaining glycemic control

As previously reported, the study met its primary endpoint with patients treated with DTX401 (n=20) experiencing a mean reduction in cornstarch of 41% at Week 48 compared to 10% reduction in the placebo group (n=24) (p < 0.0001). Data at Week 96 showed even greater improvements, with both the DTX401 group (n=20) and the crossover group (n=19) achieving a mean reduction in daily cornstarch intake of 61% from baseline. Participants in both groups also experienced statistically significant improvements in other cornstarch-related endpoints.

Additionally, 72% of participants in the crossover-DTX401 group and 67% of participants in the original DTX401 group achieved reductions of at least 50% in daily cornstarch intake by Week 96. In the manuscript, the authors noted that reductions within the crossover period are particularly meaningful, as that period more closely approximates anticipated patient management in a real-world setting.

Importantly, participants maintained low levels of hypoglycemia and improved levels in euglycemic range (70-120 mg/dL) throughout the second year of the study despite substantial reductions in daily cornstarch intake. Participants dosed with DTX401 also experienced improved normalized fasting tolerance in a controlled fasting challenge (CFC) through year 2 of the study, supporting the potential for protection from severe hypoglycemia (< 54 mg/dL).

Publication offers additional insights into nighttime treatment burden

The publication provides detailed analyses of nighttime cornstarch use, one of the most burdensome aspects of current GSDIa management.

Among participants requiring nighttime cornstarch at baseline:

At Week 48, 50% of DTX401-treated participants eliminated at least one nighttime cornstarch dose compared with 7% of placebo-treated participants (p=0.031).At Week 96, 67% of participants in both treatment groups eliminated at least one nighttime cornstarch dose.By Week 96, 33% of original DTX401 participants and 42% of crossover-DTX401 participants had completely eliminated nighttime cornstarch dosing. Despite substantial reductions in daily and nighttime cornstarch use, participants maintained glycemic control throughout the study, without inducing severe hypoglycemic episodes. These findings build upon previously reported reductions in nighttime cornstarch requirements and provide additional insight into the impact of DTX401 on overnight disease management.

Patient-reported outcomes support treatment effect as clinically meaningful

Authors detailed findings of a patient-centered analysis that showed the average reduction in daily cornstarch intake considered meaningful by participants at baseline was 45%. At Week 48, 83% of DTX401-treated participants met or exceeded their own baseline expectations for meaningful reduction in cornstarch use, with continued improvements through Week 96.

At Week 48, 79% of DTX401-treated participants reported improvement in GSDIa on the Patient Global Impression of Change compared with 52% of placebo-treated participants (p=0.131); at Week 96, improvement was reported by 95% of crossover-DTX401 participants and 83% of original DTX401 participants.

The publication further characterizes how reducing cornstarch requirements affected overall nutritional management. At baseline, cornstarch accounted for nearly 50% of study participants’ total caloric intake. Following treatment with DTX401, participants were able to transition to a more balanced, food-based diet closer to the U.S. Dietary Guidelines for the general population.

DTX401 was generally well tolerated with an acceptable safety profile

Consistent with previously reported findings, the authors concluded that DTX401 demonstrated an acceptable and manageable safety profile. The most common treatment-related adverse events were transient elevations in liver enzymes, which were generally nonserious and managed with prophylactic corticosteroids.

No AAV8 class effects of dorsal root ganglion toxicity, malignancy, or thrombotic microangiopathy were observed in the study through Week 96. Hypertriglyceridemia was observed in all study groups but more frequently following DTX401 treatment.

INDICATION

GENGLYCOS (pariglasgene brecaparvovec-opnr) is indicated to reduce daily cornstarch intake as an adjunct to nutritional management in adult and pediatric patients 8 years of age and older with glycogen storage disease type Ia (GSDIa).

This indication is approved under accelerated approval based on reduction in daily cornstarch intake. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS
GENGLYCOS is contraindicated in patients with known severe hepatic fibrosis or cirrhosis.

WARNINGS AND PRECAUTIONS

Hypersensitivity and Infusion Reactions (IRs)

Hypersensitivity reactions including anaphylaxis and IRs have occurred with GENGLYCOS treatment. Severe reactions have been reported. Monitor for signs and symptoms of hypersensitivity and IRs, including urticaria, flushing, hypotension, bronchospasm, dyspnea, chest tightness, nausea, vomiting, headache, abdominal pain, lightheadedness, flu-like symptoms, shivering, rash, and hypertension.Premedicate with acetaminophen and non-sedating antihistamines and administer GENGLYCOS according to recommended infusion rates. Monitor patients during and after completion of GENGLYCOS infusion as clinically indicated. If anaphylaxis or severe IR occurs, pause GENGLYCOS infusion immediately and initiate medical treatment as clinically indicated, monitoring as needed. For mild to moderate IRs, consider slowing or temporarily interrupting the infusion, and administer symptomatic treatment as clinically indicated. The infusion may be restarted at half the prior rate upon resolution of symptoms.Medical support measures, including cardiopulmonary resuscitation equipment and medications for the treatment of anaphylaxis (e.g., epinephrine, antihistamines, corticosteroids), should be available during GENGLYCOS administration. Hepatotoxicity

Immune-mediated hepatotoxicity, with elevated alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels, has occurred with GENGLYCOS. Avoid use in patients with preexisting hepatic impairment or acute hepatic viral infection.Prior to GENGLYCOS infusion, evaluate liver-related medical history and assess liver function by clinical examination and laboratory testing. Advise patients to immediately report signs and symptoms of hepatotoxicity, including fatigue, jaundice, dark urine, nausea, vomiting, and right upper quadrant pain. Administer corticosteroids to all patients after GENGLYCOS infusion in order to mitigate hepatic reactions. Elevated transaminases may require adjustment of the corticosteroid treatment regimen, including increased dose or prolongation of the corticosteroid taper.Monitor transaminase levels for the first 6 months after GENGLYCOS administration. Continue to monitor transaminases in all patients who develop transaminase elevations, until transaminases return to baseline or as clinically indicated. Adrenal Insufficiency

Adrenal insufficiency, including serious events, has been reported in patients receiving GENGLYCOS during corticosteroid use and tapering.Signs and symptoms of adrenal insufficiency include fatigue, weakness, anorexia, nausea, vomiting, hypotension, hyponatremia, and hypoglycemia. Adrenal crisis may present as severe hypotension, acute abdominal pain, or loss of consciousness.Monitor patients for signs and symptoms of adrenal insufficiency and adrenal crisis after GENGLYCOS administration during and after corticosteroid therapy and tapering. Taper corticosteroid therapy gradually. Do not abruptly discontinue corticosteroid therapy. AAV Vector Integration and Risk of Tumorigenicity

There is a theoretical risk of tumorigenicity due to integration of AAV vector DNA into the genome.GENGLYCOS is composed of a recombinant, non-replicating AAV8 vector whose DNA persists largely in episomal form. Random integration of recombinant AAV-vector DNA into human DNA has been reported with AAV gene therapies. The clinical relevance of individual integration events is unknown, but it is acknowledged that individual integration events could potentially contribute to a risk of tumorigenicity. If a tumor develops in a patient receiving GENGLYCOS, health care providers should contact and report the tumor to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. Adverse Reactions

Seven serious adverse events were observed in the Primary Efficacy Analysis Period (PEAP) of Study 1 (Weeks 1-48), including anaphylaxis/infusion reaction (2), adrenal insufficiency (2), high lactate level (2) and hypoglycemia (1).The most common adverse reactions during the PEAP of Study 1 (occurring in ≥10% of patients) with higher frequency in GENGLYCOS compared to placebo were ALT/AST Enzyme elevated (71%), Nausea (38%), Headache (24%), Hypertriglyceridemia (29%), Adrenal Insufficiency (24%), Constipation (19%), Hyperglycemia (14%), Acne/Dermatitis Acneiform (19%), Cushingoid Features (14%), and Anaphylaxis (10%). DRUG INTERACTIONS

Vaccinations

Vaccine schedules may need to be adjusted for immunosuppressive therapy, and vaccines should be avoided 1 month prior to GENGLYCOS administration. USE IN SPECIFIC POPULATIONS

Pregnancy

GENGLYCOS should not be used during pregnancy. There are no data on the use of GENGLYCOS in pregnant women. It is unknown whether GENGLYCOS can cause fetal harm when administered to a pregnant woman or can affect reproductive capacity. Contraception

Women of childbearing potential should use effective contraception for at least 12 months after administration of GENGLYCOS.For 6 months after administration of GENGLYCOS, men must not donate semen, and men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception. ADDITIONAL PATIENT COUNSELING INFORMATION
Vector Shedding

Inform patients/caregivers that vector distribution in blood and vector shedding in urine, stool, and saliva can occur after GENGLYCOS infusion. Advise patients/caregivers on proper hygiene when handling patient body waste. These precautions should be followed for 3 months after GENGLYCOS infusion. Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. 

Please see the full Prescribing Information for GENGLYCOS.

About the Phase 3 GlucoGene study
The 48-week randomized, double-blind, placebo-controlled study treated 46 participants aged eight years and older with DTX401 (1.0 x 10^13 GC/kg dose measured by ddPCR) or placebo. There were 44 participants in the modified intention-to-treat (mITT) population providing efficacy data within the Week 48 analysis period following treatment with DTX401 (n=20) or placebo (n=24). At Week 48, eligible participants crossed over and received the alternate treatment. After crossover, participants continued to be followed with analyses conducted at Week 96 and Week 144. After study completion, participants will be offered enrollment into the GSDIa Disease Monitoring Program (DMP) where they will be followed for 10 years post-DTX401 infusion.

About Glycogen Storage Disease Type Ia (GSDIa)
GSDIa is an ultra-rare, serious, and life-threatening disease due to an inborn error of carbohydrate metabolism caused by pathogenic variants of the G6PC gene, which encodes G6Pase, an enzyme that is critical for the release of glucose from glycogen and other metabolic sources. Deficiency of G6Pase activity results in severe hypoglycemia during periods of fasting between meals and during the night along with excess hepatic glycogen storage, metabolic derangements and other disease related complications. Cornstarch is critical in the management of GSDIa throughout the day and night in providing an exogenous source of glucose to help avoid sudden and severe drops in plasma glucose levels; however, current management strategies carry a significant burden to patients and families. GSDIa affects approximately 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide within commercially accessible geographies.

About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding the interpretation, significance and potential implications of the published 96-week Phase 3 data and analyses for GENGLYCOS (also known as DTX401); the clinical meaningfulness and durability of reductions in daily and nighttime cornstarch requirements; the ability of patients to maintain glycemic control and improve fasting tolerance following treatment; the potential for GENGLYCOS to protect against severe hypoglycemia, reduce treatment burden, improve nutritional management and provide other patient benefits; the safety and tolerability of GENGLYCOS; expectations regarding continued follow-up of study participants and the design, enrollment, timing, conduct and results of the GSDIa Disease Monitoring Program and other post-marketing requirements; Ultragenyx’s ability to confirm clinical benefit, satisfy FDA requirements and maintain accelerated approval for GENGLYCOS; and estimates of the prevalence of GSDIa and the potential patient population for GENGLYCOS, are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause actual results to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the risk that results from a limited number of study participants, including crossover and other analyses, may not be replicated or predictive of future or real-world results; the risk that longer-term follow-up may not demonstrate sustained efficacy, durability, safety or patient benefit; risks related to serious or undesirable side effects, including risks associated with AAV gene therapy; Ultragenyx’s ability to complete post-marketing requirements within required timeframes and confirm clinical benefit; the risk that the FDA may modify the approved indication or impose additional requirements, or may withdraw accelerated approval if clinical benefit is not confirmed or post-marketing requirements are not satisfied; and other matters that could affect the availability or commercial potential of Ultragenyx’s products and product candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 5, 2026, and its subsequent periodic reports filed with the SEC.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Ultragenyx Contacts

Investors
Joshua Higa
[email protected]

Media
Jess Rowlands
[email protected]

 | Source: Ultragenyx Pharmaceutical Inc.

NOVATO, Calif., Aug. 25, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 45,984 restricted stock units of the company’s common stock to 23 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of August 16, 2026, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

The restricted stock units vest over four years, with 25% of the underlying shares vesting on each anniversary of the grant date, subject to the employee being continuously employed by the company as of such vesting dates.

About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Contact Ultragenyx
Investors & Media
Joshua Higa
(415) 475-6370

Ultragenyx Pharmaceutical Inc. (RARE) Discusses FDA Approval of Gene Therapy for Glycogen Storage Disease Type 1a Transcript

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are up during Thursday’s premarket session on the recent U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr, or DTX401).

Genglycos Approved For Ultra-Rare Genetic DisorderThis marks a significant milestone as it is the first-ever FDA-approved treatment for Glycogen Storage Disease Type Ia (GSDIa).

The approval of Genglycos represents Ultragenyx’s first gene therapy and fifth FDA approval.

GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream.

The deficiency reduces the liver’s ability to control glucose levels and is associated with potentially life-threatening hypoglycemia episodes and other serious complications.

GSDIa affects 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide.

Read Next

Phase 3 Data Supports FDA ApprovalThe approval of GENGLYCOS is based on data from the 48-week Phase 3 GlucoGene study, which treated 46 participants aged eight years and older with DTX401 (1.0 x 10^13 GC/kg dose) or placebo, showing a reduction in the cornstarch requirements in the treated group.

As part of accelerated approval, Ultragenyx has agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patients through enhancement of its existing GSDIa Disease Monitoring Program (DMP).

The DMP will also evaluate previously treated clinical trial participants as well as these new commercial patients for a total of 10 years.  

Analyst BullishCiting a conference call, William Blair noted the wholesale acquisition cost for Genglycos will be $2.7 million per patient.

Analyst Sami Corwin said, “We are encouraged by the approval of Genglycos, which we believe is incrementally positive to Ultragenyx’s stock.”

Analyst Corwin resumed coverage of Ultragenyx with an Outperform rating and currently models $362 million in peak sales for Genglycos.

RARE Stock Price Activity: Ultragenyx Pharmaceutical shares were up 11.28% at $29.20 during premarket trading on Thursday, according to Benzinga Pro data.

Read Next

Photo: Shutterstock

Market News and Data brought to you by Benzinga APIs

© 2026 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

To add Benzinga News as your preferred source on Google, click here.

Ultragenyx Pharmaceutical (RARE.O) said on Wednesday that its gene therapy became the ​first to receive the U.S. Food and Drug Administration's approval to ‌treat a rare metabolic disorder, sending its shares up 7.5% in after-hours trading.

The drug, branded as Genglycos, has been approved for glycogen storage disease type Ia (GSDIa), also known ​as Von Gierke disease, in adults and pediatric patients.

It is an ​ultra-rare genetic disorder that is caused due to deficiency of ⁠an enzyme that helps release glucose from the liver into the bloodstream. ​GSDIa affects 1,500 to 2,500 patients in the U.S., the company estimates.

The deficiency ​reduces the liver's ability to control glucose levels and is associated with potentially life-threatening low blood sugar levels.

Day-to-day management of the condition requires a relentless regimen of raw cornstarch ​as an oral glucose replacement therapy.

Ultragenyx's Genglycos addresses the underlying cause of ​the disorder by aiding the normal breakdown of glycogen to produce glucose, which helps ‌in regulating ⁠blood sugar levels and reducing reliance on cornstarch.

Genglycos will have a U.S. per-patient list price of $2.7 million and be available through qualified treatment centers within 30 to 60 days, the company said.

The regulator's authorization is based on ​a 48-week late-stage ​trial that found ⁠patients treated with Genglycos showed a reduction in the cornstarch requirements compared to placebo.

Reducing cornstarch intake is, however, the ​surrogate endpoint, and the manufacturer must complete additional clinical trials ​to ⁠confirm Genglycos's effectiveness, the FDA said.

The company said it has agreed to provide two years of safety and efficacy clinical data from patients.

Several experimental treatments for ⁠GSDIa are ​also in development, including those from Moderna (MRNA.O) ​and Beam Therapeutics (BEAM.O), though the candidates remain in early stages of clinical testing.

Posted by Defense World Staff on Aug 13th, 2026

Assenagon Asset Management S.A. lowered its stake in shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Free Report) by 94.6% during the 2nd quarter, according to the company in its most recent 13F filing with the SEC. The institutional investor owned 120,864 shares of the biopharmaceutical company’s stock after selling 2,136,807 shares during the quarter. Assenagon Asset Management S.A. owned 0.12% of Ultragenyx Pharmaceutical worth $4,036,000 at the end of the most recent quarter.

A number of other hedge funds also recently made changes to their positions in the stock. Parallel Advisors LLC increased its stake in Ultragenyx Pharmaceutical by 778.4% in the first quarter. Parallel Advisors LLC now owns 1,625 shares of the biopharmaceutical company’s stock valued at $34,000 after acquiring an additional 1,440 shares during the last quarter. Leonteq Securities AG lifted its stake in Ultragenyx Pharmaceutical by 288.5% during the first quarter. Leonteq Securities AG now owns 1,795 shares of the biopharmaceutical company’s stock worth $38,000 after purchasing an additional 1,333 shares during the last quarter. Versant Capital Management Inc lifted its stake in Ultragenyx Pharmaceutical by 662.9% during the second quarter. Versant Capital Management Inc now owns 1,152 shares of the biopharmaceutical company’s stock worth $38,000 after purchasing an additional 1,001 shares during the last quarter. Danske Bank A S bought a new stake in Ultragenyx Pharmaceutical during the third quarter worth about $39,000. Finally, EverSource Wealth Advisors LLC boosted its holdings in shares of Ultragenyx Pharmaceutical by 573.0% in the 1st quarter. EverSource Wealth Advisors LLC now owns 2,167 shares of the biopharmaceutical company’s stock worth $45,000 after purchasing an additional 1,845 shares in the last quarter. 97.67% of the stock is owned by institutional investors.

Insiders Place Their Bets In other Ultragenyx Pharmaceutical news, Director Corazon (Corsee) D. Sanders sold 2,000 shares of the stock in a transaction that occurred on Monday, June 15th. The stock was sold at an average price of $25.05, for a total value of $50,100.00. Following the completion of the sale, the director owned 21,095 shares in the company, valued at approximately $528,429.75. This trade represents a 8.66% decrease in their position. The transaction was disclosed in a filing with the SEC, which can be accessed through the SEC website. Also, EVP Karah Herdman Parschauer sold 1,899 shares of the firm’s stock in a transaction on Monday, June 15th. The stock was sold at an average price of $24.62, for a total value of $46,753.38. Following the completion of the sale, the executive vice president directly owned 94,462 shares of the company’s stock, valued at $2,325,654.44. The trade was a 1.97% decrease in their position. Additional details regarding this sale are available in the official SEC disclosure. Over the last 90 days, insiders sold 14,292 shares of company stock worth $351,644. 5.20% of the stock is currently owned by company insiders.

Ultragenyx Pharmaceutical Stock Down 0.4% Shares of Ultragenyx Pharmaceutical stock opened at $26.86 on Thursday. The firm has a market cap of $2.65 billion, a PE ratio of -4.60 and a beta of 0.35. Ultragenyx Pharmaceutical Inc. has a 12-month low of $18.29 and a 12-month high of $39.89. The company’s fifty day simple moving average is $28.16 and its two-hundred day simple moving average is $24.90.

Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) last issued its quarterly earnings data on Tuesday, August 4th. The biopharmaceutical company reported ($0.90) EPS for the quarter, beating the consensus estimate of ($1.22) by $0.32. The firm had revenue of $214.00 million during the quarter, compared to the consensus estimate of $183.08 million. Ultragenyx Pharmaceutical had a negative net margin of 81.79% and a negative return on equity of 1,024.42%. The company’s quarterly revenue was up 28.5% compared to the same quarter last year. During the same period in the prior year, the firm posted ($1.17) earnings per share. As a group, sell-side analysts expect that Ultragenyx Pharmaceutical Inc. will post -4.08 earnings per share for the current fiscal year.

Ultragenyx Pharmaceutical News Roundup Here are the key news stories impacting Ultragenyx Pharmaceutical this week:

Positive Sentiment: HC Wainwright sharply raised its Q3 2026 EPS estimate to $0.75 from a loss of $0.84 per share and increased its Q4 2026 forecast to $0.78 from a loss of $0.35. The changes suggest expectations for a faster-than-anticipated improvement in Ultragenyx’s profitability. Ultragenyx Pharmaceutical Earns Buy Rating from HC Wainwright Neutral Sentiment: HC Wainwright maintained its Buy rating and $50 price target, well above the stock’s recent trading level. Separately, a brokerage consensus cited a price target of approximately $57.82, indicating substantial potential upside if the company executes on its pipeline and reaches profitability. Brokerages Set Ultragenyx Pharmaceutical Price Target Neutral Sentiment: Ultragenyx and Baylor Research Institute reportedly settled a patent lawsuit involving generic competition to DOJOLVI. The settlement removes some litigation uncertainty, but the financial and commercial terms were not provided, limiting the immediate valuation impact. Ultragenyx and Baylor Settle DOJOLVI Patent Lawsuit Negative Sentiment: HC Wainwright lowered its 2027 EPS estimates across the board: Q1 to $0.06 from $0.27, Q2 to $0.91 from $1.18, Q3 to $1.25 from $1.54, Q4 to $2.27 from $2.63, and full-year 2027 to $4.50 from $5.61. These reductions weigh on longer-term earnings expectations, even though the analyst remains bullish. Wall Street Analyst Weigh In RARE has been the topic of a number of research reports. Barclays decreased their price objective on Ultragenyx Pharmaceutical from $44.00 to $43.00 and set an “overweight” rating on the stock in a report on Wednesday, April 29th. Guggenheim cut their target price on Ultragenyx Pharmaceutical from $43.00 to $35.00 and set a “buy” rating for the company in a research note on Tuesday, August 4th. Wells Fargo & Company reduced their price target on shares of Ultragenyx Pharmaceutical from $47.00 to $45.00 and set an “overweight” rating on the stock in a research report on Wednesday, August 5th. Royal Bank Of Canada lifted their price target on shares of Ultragenyx Pharmaceutical from $35.00 to $40.00 and gave the stock an “outperform” rating in a research note on Tuesday, July 7th. Finally, Wall Street Zen upgraded shares of Ultragenyx Pharmaceutical from a “sell” rating to a “hold” rating in a report on Saturday, August 8th. One investment analyst has rated the stock with a Strong Buy rating, fourteen have issued a Buy rating, two have assigned a Hold rating and one has given a Sell rating to the company’s stock. According to MarketBeat.com, the stock presently has a consensus rating of “Moderate Buy” and an average price target of $57.82.

View Our Latest Analysis on Ultragenyx Pharmaceutical

About Ultragenyx Pharmaceutical (Free Report)

Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic disorders. Since its founding in 2010 and headquarters in Novato, California, the company has built expertise in protein replacement therapies, small molecules and gene therapy approaches to address high-unmet medical needs. Ultragenyx applies a precision medicine model, leveraging both in-house research and strategic collaborations to advance its product pipeline from discovery through regulatory approval.

The company’s commercial portfolio includes Crysvita (burosumab-tmyl) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders.

See Also Five stocks we like better than Ultragenyx Pharmaceutical GE Vernova’s AI Power Boom Faces a Profit Test Cardinal Health Earnings: Can Perfection Get Priced In Twice? Nebius’ Q2 Beat Shows the AI Bottleneck Is Capacity, Not Demand Legacy Jet Builders Stall While Embraer Accelerates to New Highs

Receive News & Ratings for Ultragenyx Pharmaceutical Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Ultragenyx Pharmaceutical and related companies with MarketBeat.com's FREE daily email newsletter.

« PREVIOUS HEADLINEGreen Dot Corporation $GDOT Shares Sold by Assenagon Asset Management S.A.

NEXT HEADLINE »Assenagon Asset Management S.A. Acquires New Holdings in Westlake Corporation $WLK

Key Takeaways RARE beat Q2 earnings estimates as revenues rose 28% year over year to a record quarterly high.Crysvita, Dojolvi and Evkeeza sales grew on demand, supporting higher product revenues.Ultragenyx reaffirmed 2026 revenue guidance of $730M-$760M, excluding new product launches. Ultragenyx Pharmaceutical (RARE - Free Report) reported second-quarter 2026 loss of 90 cents per share, which was narrower than the Zacks Consensus Estimate of a loss of $1.27. The company had incurred a loss of $1.17 per share in the year-ago quarter.

Total revenues in the second quarter were $214 million, which surged 28.1% year over year due to higher product sales. The top line also beat the Zacks Consensus Estimate of $181 million.

Management stated that second-quarter 2026 revenues were the highest quarterly revenues ever reported by the company.

Ultragenyx markets four drugs, namely Crysvita, Mepsevii, Dojolvi and Evkeeza. Crysvita is approved for treating X-linked hypophosphatemia, an inherited disorder and tumor-induced osteomalacia, an ultra-rare disease. Mepsevii is approved to treat Mucopolysaccharidosis VII, also known as Sly syndrome. Dojolvi is approved for treating all forms of long-chain fatty acid oxidation disorders. Evkeeza is indicated for homozygous familial hypercholesterolemia (HoFH).

In 2022, Ultragenyx announced a license and collaboration agreement with Regeneron Pharmaceuticals (REGN - Free Report) for Evkeeza, which is approved in multiple geographies as a first-in-class therapy for use together with diet and other low-density lipoprotein-cholesterol-lowering therapies to treat adults and adolescents aged 12 years and older with HoFH.

Per the deal, RARE has obtained the rights to develop, commercialize and distribute Evkeeza outside the United States. The regions include the European Economic Area. The collaboration with Regeneron for Evkeeza gives Ultragenyx a fourth approved product that adds to the top line. However, REGN solely commercializes Evkeeza in the United States.

Year to date, shares of Ultragenyx have gained 12.2% compared with the industry’s 1.6% rise.

Image Source: Zacks Investment Research

RARE’s Q2 Results in DetailCrysvita’s total revenues were $156 million, up 28.9% year over year. Management noted that Crysvita sales were consistent with expected seasonality in the United States and Canada and ordering patterns in Latin America. Crysvita’s net product revenues in the second quarter of 2026 included $94 million from North America, $54 million from Latin America and Turkey, and $8 million from Europe.

Mepsevii product revenues increased 11.1% year over year to $10 million in the reported quarter. Dojolvi product revenues were $27 million, up 17.4%, driven by strong demand. Evkeeza recorded sales of $21 million in the second quarter, up 50%, driven by increased demand from new country launches and early access.

Operating expenses of $289 million in the quarter rose 5.1% year over year due to increased investments in multiple late-stage pipeline programs and marketing costs for approved drugs. Operating expenses included research and development (R&D) expenses of $167 million (up 1.2%), selling, general and administrative (SG&A) expenses of $88 million (up 1.1%) and cost of sales of $34 million (up 47.8%).

Cash, cash equivalents and marketable securities amounted to $436 million as of June 30, 2026, compared with $534 million as of March 31, 2026.

RARE Reiterates 2026 Financial GuidanceUltragenyx continues to expect total revenues in 2026, excluding potential revenues from new product launches, between $730 million and $760 million.

Crysvita revenues in 2026 are expected to be in the range of $500-$520 million, reflecting growing underlying global demand. Meanwhile, Dojolvi revenues are expected to be between $100 million and $110 million in 2026.

RARE’s Key Pipeline UpdatesIn April 2026, the FDA accepted the resubmitted biologics license application (BLA) seeking accelerated approval of UX111 for the treatment of MPS IIIA. The application included extensive long-term data with follow-up of up to eight years. The data showed sustained clinical benefits compared with the decline seen in natural history studies, along with durable treatment effects across multiple clinical measures and biomarkers, while maintaining an acceptable safety profile. A final decision from the regulatory body is expected on Sept. 19, 2026.

The FDA has also accepted for review Ultragenyx’s BLA for its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia. A final decision from the regulatory agency is expected on Aug. 23, 2026.

Ultragenyx is also evaluating UX701, an investigational AAV9 gene therapy, in a phase I/II/III Cyprus2+ study to treat Wilson disease and expects to share top-line data in the fourth quarter of 2026.

Ultragenyx’s GTX-102, an investigational antisense oligonucleotide, is being developed in the pivotal phase III Aspire study for treating Angelman syndrome (AS) patients with a genetically confirmed diagnosis of UBE3A deletion. Top-line data are expected in September or October 2026. Meanwhile, enrollment in the phase II/III Aurora study is currently ongoing to evaluate the safety and efficacy of GTX-102 for treating other AS genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of AS patients. The study is expected to complete enrollment in the second half of 2026.

RARE’s Zacks Rank & Other Stocks to ConsiderUltragenyx currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks in the biotech sector are Repligen (RGEN - Free Report) and Liquidia Corporation (LQDA - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while estimates for 2027 have increased from $2.57 to $2.62 during the same time. RGEN shares have declined 8.6% year to date.

Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.

Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $2.97 to $3.02, while estimates for 2027 have increased from $4.81 to $5.31 during the same time. LQDA shares have surged 156.3% year to date.

Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.

Ultragenyx (RARE - Free Report) came out with a quarterly loss of $0.9 per share versus the Zacks Consensus Estimate of a loss of $1.27. This compares to a loss of $1.17 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of +29.13%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.55 per share when it actually produced a loss of $1.84, delivering a surprise of -18.71%.

Over the last four quarters, the company has surpassed consensus EPS estimates just once.

Ultragenyx, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $214 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 18.23%. This compares to year-ago revenues of $166.5 million. The company has topped consensus revenue estimates two times over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

Ultragenyx shares have added about 8.8% since the beginning of the year versus the S&P 500's gain of 11%.

What's Next for Ultragenyx?While Ultragenyx has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for Ultragenyx was favorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #2 (Buy) for the stock. So, the shares are expected to outperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.85 on $189.98 million in revenues for the coming quarter and -$4.53 on $746.71 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 42% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

One other stock from the same industry, Zealand Pharma A/S (ZLDPF - Free Report) , is yet to report results for the quarter ended June 2026. The results are expected to be released on August 13.

This company is expected to post quarterly loss of $0.42 per share in its upcoming report, which represents a year-over-year change of -102.6%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days.

Zealand Pharma A/S's revenues are expected to be $81.55 million, down 94.1% from the year-ago quarter.

Analysts Say These 2 Mid-Cap Biotechs Have 2x PotentialUltragenyx Pharmaceutical NASDAQ: RARE reported second-quarter 2026 revenue of $214 million, led by growth across its commercial rare-disease portfolio, while the company prepared for potential regulatory decisions on two gene therapies and a late-stage data readout in Angelman syndrome.

Chief Executive Officer and President Emil Kakkis said the quarter marked the company’s highest quarterly revenue to date and supported its reaffirmed full-year revenue guidance. He also said Ultragenyx sees a path to profitability in 2027 through continued product growth, potential contributions from upcoming launches, expense discipline and possible monetization of priority review vouchers.

Get RARE alerts:

Second-Quarter Financial Results Chief Financial Officer Howard Horn said total second-quarter revenue reached $214 million. Crysvita generated $156 million, including $94 million in North America, $54 million in Latin America and Turkey, and $8 million in Europe. DOJOLVI contributed $27 million, while Evkeeza generated $21 million, up 50% from the second quarter of 2025. MEPSEVII revenue was $10 million.

Total operating expenses were $289 million, including $34 million in cost of sales and $255 million in combined research and development and selling, general and administrative expenses. Operating expenses included $34 million of non-cash stock-based compensation.

Ultragenyx posted a net loss of $92 million, or $0.90 per share, for the quarter. As of June 30, the company had $436 million in cash equivalents and marketable securities. Net cash used in operations was $97 million, which Horn said was significantly lower than in the first quarter and in line with prior expectations.

The company reaffirmed its revenue guidance and its guidance for combined R&D and SG&A expenses. Horn said 2026 combined R&D and SG&A spending is expected to be flat to down low single digits from 2025, while 2027 spending is expected to decline by at least 15%, including planned investments for potential launches.

Commercial Portfolio Expands Patient Reach Chief Commercial Officer Erik Harris said underlying demand remained strong despite seasonal factors that can affect quarterly ordering patterns. In Latin America, approximately 50 patients began commercial Crysvita therapy during the quarter, bringing the regional total to 1,000 patients. More than 100 patients in Turkey are receiving Crysvita through a named-patient program.

For DOJOLVI, Ultragenyx generated about 30 start forms in North America and had approximately 675 patients on reimbursed treatment. About 300 patients in Europe were receiving treatment through named-patient or early-access programs. The company also began treating patients in Japan after the therapy was listed on the country’s National Health Insurance Drug Price List.

Evkeeza was being provided to more than 500 patients in 25 countries outside the U.S., Harris said. He added that Ultragenyx’s commercial infrastructure is designed to support patient identification, reimbursement navigation and field execution across individual markets.

Gene Therapy Decisions Approach Ultragenyx is awaiting FDA action on DTX-401, an investigational gene therapy for glycogen storage disease type Ia, with a Prescription Drug User Fee Act date of Aug. 23. The company’s second anticipated decision is for UX-111, a gene therapy for Sanfilippo syndrome type A, with a PDUFA date of Sept. 19.

Kakkis said DTX-401 clinical studies showed that treated patients could significantly reduce the amount and frequency of cornstarch dosing to a prespecified clinically meaningful degree. He said the therapy is designed to provide expression of the missing G6Pase enzyme and improve glucose regulation during fasting or metabolic stress.

For UX-111, Kakkis said clinical data showed reductions in heparan sulfate and stabilization or retention of cognitive function relative to natural history. He said earlier treatment appeared to produce better results, although patients across the age range studied showed benefits after treatment.

Harris said launch preparations for the two potential gene therapies were progressing, including work with qualified treatment centers and more than 200 payer engagements. He said the company already has commercial experience in relevant treatment settings, with approximately 75% overlap between institutions and healthcare providers involved with the potential gene therapies and those serving DOJOLVI and MEPSEVII patients.

During the question-and-answer session, Kakkis said the FDA review of DTX-401 had proceeded normally, with the company responding to information requests involving clinical and chemistry, manufacturing and controls matters. He said Ultragenyx could not predict the agency’s ultimate action.

Angelman Readout and Setrusumab Regulatory Path Ultragenyx expects to report top-line Phase III ASPIRE data for GTX-102, also known as apazunersen, in Angelman syndrome during September or October. The randomized, double-blind, sham-controlled study enrolled patients with a full maternal UBE3A gene deletion.

Chief Medical Officer Eric Crombez said the study’s statistical alpha is split between the Bayley-4 cognitive raw score, which receives 80%, and the Multi-Domain Responder Index, or MDRI, which receives 20%. The endpoints are tested in parallel rather than hierarchically. A statistically successful study would require a Bayley endpoint p-value of 0.04 or less, or an MDRI p-value of 0.01 or less.

Kakkis said a five- to six-point change on the Bayley assessment would be clinically meaningful and that the study was powered to detect an average change in that range. He also said the company’s Phase I/II program has shown a consistent safety profile, and the Phase III study had not been stopped because of a safety issue.

Separately, Ultragenyx continues discussions with regulators regarding UX143, or setrusumab, for osteogenesis imperfecta. Crombez said neither of the Phase III ORBIT and COSMIC studies met their primary endpoints for annualized fracture reduction, though the studies showed improvements in bone mineral density and patient-reported outcomes, along with fracture reductions in certain bones and patient groups.

The U.K.’s Medicines and Healthcare products Regulatory Agency indicated that a new randomized study may be required before it would consider an application, according to Crombez. The FDA has been open to discussing alternative fracture analyses, but Ultragenyx said further discussions are needed to determine the additional clinical data required for a potential biologics license application.

About Ultragenyx Pharmaceutical (NASDAQ:RARE)Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic disorders. Since its founding in 2010 and headquarters in Novato, California, the company has built expertise in protein replacement therapies, small molecules and gene therapy approaches to address high-unmet medical needs. Ultragenyx applies a precision medicine model, leveraging both in-house research and strategic collaborations to advance its product pipeline from discovery through regulatory approval.

The company's commercial portfolio includes Crysvita (burosumab-tmyl) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII and Dojolvi (triheptanoin) for long-chain fatty acid oxidation 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].

Continue following MarketBeat

Add MarketBeat as your preferred source on Google to see our latest stories in your feed.

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

MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and Ultragenyx Pharmaceutical wasn't on the list.

While Ultragenyx Pharmaceutical currently has a Moderate Buy rating among analysts, top-rated analysts believe these five stocks are better buys.

View The Five Stocks Here

Discover the next wave of investment opportunities with our report, 7 Stocks That Will Be Magnificent in 2026. Explore companies poised to replicate the growth, innovation, and value creation of the tech giants dominating today's markets.

Get This Free Report

NOVATO, Calif., July 28, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, August 4, 2026, to discuss its financial results and corporate update for the quarter ending June 30, 2026.

Posted by Defense World Staff on Jul 27th, 2026

Entropy Technologies LP grew its position in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Free Report) by 80.9% during the first quarter, according to the company in its most recent filing with the Securities and Exchange Commission (SEC). The fund owned 109,727 shares of the biopharmaceutical company’s stock after acquiring an additional 49,086 shares during the quarter. Entropy Technologies LP owned 0.11% of Ultragenyx Pharmaceutical worth $2,299,000 as of its most recent filing with the Securities and Exchange Commission (SEC).

A number of other large investors have also added to or reduced their stakes in the company. Jefferies Financial Group Inc. purchased a new position in shares of Ultragenyx Pharmaceutical in the fourth quarter valued at $10,973,000. BNP Paribas Financial Markets raised its stake in Ultragenyx Pharmaceutical by 57.6% during the fourth quarter. BNP Paribas Financial Markets now owns 903,050 shares of the biopharmaceutical company’s stock valued at $20,770,000 after purchasing an additional 330,031 shares in the last quarter. GW&K Investment Management LLC raised its stake in Ultragenyx Pharmaceutical by 19.6% during the fourth quarter. GW&K Investment Management LLC now owns 1,375,407 shares of the biopharmaceutical company’s stock valued at $31,634,000 after purchasing an additional 225,238 shares in the last quarter. UBS Group AG lifted its position in Ultragenyx Pharmaceutical by 22.3% during the fourth quarter. UBS Group AG now owns 668,811 shares of the biopharmaceutical company’s stock valued at $15,383,000 after purchasing an additional 121,786 shares during the last quarter. Finally, Aberdeen Group plc lifted its position in Ultragenyx Pharmaceutical by 51.0% during the fourth quarter. Aberdeen Group plc now owns 1,469,951 shares of the biopharmaceutical company’s stock valued at $33,809,000 after purchasing an additional 496,295 shares during the last quarter. Institutional investors own 97.67% of the company’s stock.

Ultragenyx Pharmaceutical Stock Performance RARE stock opened at $26.90 on Monday. Ultragenyx Pharmaceutical Inc. has a 12 month low of $18.29 and a 12 month high of $39.89. The company has a 50-day moving average price of $27.45 and a 200 day moving average price of $24.65. The stock has a market cap of $2.65 billion, a PE ratio of -4.40 and a beta of 0.31.

Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) last issued its quarterly earnings results on Tuesday, May 5th. The biopharmaceutical company reported ($1.84) EPS for the quarter, missing the consensus estimate of ($1.49) by ($0.35). Ultragenyx Pharmaceutical had a negative net margin of 91.03% and a negative return on equity of 1,024.42%. The firm had revenue of $136.00 million for the quarter, compared to the consensus estimate of $158.19 million. During the same quarter in the previous year, the company posted ($1.57) earnings per share. The business’s revenue for the quarter was down 2.2% on a year-over-year basis. Sell-side analysts forecast that Ultragenyx Pharmaceutical Inc. will post -4.53 EPS for the current year.

Insiders Place Their Bets In other Ultragenyx Pharmaceutical news, Director Shehnaaz Suliman sold 5,740 shares of the business’s stock in a transaction that occurred on Monday, May 18th. The stock was sold at an average price of $25.12, for a total transaction of $144,188.80. Following the completion of the transaction, the director owned 27,951 shares in the company, valued at $702,129.12. The trade was a 17.04% decrease in their ownership of the stock. The sale was disclosed in a filing with the Securities & Exchange Commission, which is available at this link. Also, Director Corazon (Corsee) D. Sanders sold 2,000 shares of the stock in a transaction that occurred on Monday, June 15th. The stock was sold at an average price of $25.05, for a total value of $50,100.00. Following the completion of the sale, the director owned 21,095 shares in the company, valued at approximately $528,429.75. The trade was a 8.66% decrease in their ownership of the stock. Additional details regarding this sale are available in the official SEC disclosure. Insiders have sold 19,319 shares of company stock worth $476,837 over the last three months. 5.20% of the stock is currently owned by corporate insiders.

Wall Street Analysts Forecast Growth A number of analysts have recently weighed in on RARE shares. Cantor Fitzgerald boosted their target price on shares of Ultragenyx Pharmaceutical from $84.00 to $96.00 and gave the stock an “overweight” rating in a report on Thursday, May 21st. Barclays lowered their price target on shares of Ultragenyx Pharmaceutical from $44.00 to $43.00 and set an “overweight” rating for the company in a report on Wednesday, April 29th. Morgan Stanley lifted their price target on Ultragenyx Pharmaceutical from $50.00 to $67.00 and gave the stock an “overweight” rating in a research report on Thursday, April 16th. Royal Bank Of Canada lifted their price target on Ultragenyx Pharmaceutical from $35.00 to $40.00 and gave the stock an “outperform” rating in a research report on Tuesday, July 7th. Finally, Wedbush reduced their price objective on Ultragenyx Pharmaceutical from $27.00 to $26.00 and set a “neutral” rating on the stock in a research note on Wednesday, May 6th. One analyst has rated the stock with a Strong Buy rating, fourteen have assigned a Buy rating, two have issued a Hold rating and one has assigned a Sell rating to the stock. According to MarketBeat, the company currently has a consensus rating of “Moderate Buy” and a consensus price target of $58.41.

Check Out Our Latest Analysis on RARE

Ultragenyx Pharmaceutical Profile (Free Report)

Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic disorders. Since its founding in 2010 and headquarters in Novato, California, the company has built expertise in protein replacement therapies, small molecules and gene therapy approaches to address high-unmet medical needs. Ultragenyx applies a precision medicine model, leveraging both in-house research and strategic collaborations to advance its product pipeline from discovery through regulatory approval.

The company’s commercial portfolio includes Crysvita (burosumab-tmyl) for X-linked hypophosphatemia, Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII and Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders.

Read More Five stocks we like better than Ultragenyx Pharmaceutical RTX and Lockheed Earnings: Can Strong Guidance Reset the Defense Trade? These 4 Earnings Reports Expose the Market’s Growing Economic Divide Broadcom May Be the Biggest Winner From Alphabet’s Earnings Volatility Is Back and These 3 Market Tollbooths Are Best Positioned to Profit Want to see what other hedge funds are holding RARE? Visit HoldingsChannel.com to get the latest 13F filings and insider trades for Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Free Report).

Receive News & Ratings for Ultragenyx Pharmaceutical Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for Ultragenyx Pharmaceutical and related companies with MarketBeat.com's FREE daily email newsletter.

« PREVIOUS HEADLINERange Resources Corporation $RRC Shares Acquired by Entropy Technologies LP

NEXT HEADLINE »Entropy Technologies LP Purchases Shares of 33,496 W.R. Berkley Corporation $WRB

SYDNEY, July 23, 2026 (GLOBE NEWSWIRE) -- Brazilian Rare Earths Limited (ASX: BRE / OTCQX: BRELY) ('BRE') is pleased to report progress during the quarter ended 30 June 2026. Highlights during and subsequent to the end of the quarter include: VELHINHAS AIRBORNE GEOPHYSICS AND DRILLING UNLOCKS +9 KM RARE EARTH CORRIDOR IN THE MONTE ALTO DISTRICT New District-Scale Growth Corridor: High-resolution airborne geophysics defined more than 9 km of cumulative exploration corridors across the Velhinhas project, beginning ~5 km south of the ultra-high-grade Monte Alto Deposit and extending rare earth mineralisation to over 8 km south of Monte Alto Multiple Parallel Exploration Trends: The survey confirmed four large-scale north-northeast trending mineralised corridors, converting numerous ultra-high-grade surface results into a district-scale exploration model analogous with the Sulista district opportunity Drilling Results Confirm High-Grade Mineralisation: Reconnaissance diamond drilling returned grades of 19.6% TREO, 33,607 ppm NdPr, 1,463 ppm Dy₂O₃, 248 ppm Tb₄O₇, 7,431 ppm Y₂O₃ and 1,087 ppm U₃O₈ Critical Mineral Grades: High-grade assays are accompanied by NdPr, DyTb, yttrium, niobium, scandium, tantalum and uranium, consistent with BRE's high-value REE-Nb-Sc-Ta-U systems at Monte Alto and Sulista Monte Alto District Growth Accelerates: Velhinhas expands the Monte Alto growth story, highlighting the potential for a district-scale mineral system extending south from the ultra-high-grade Monte Alto Deposit EXCEPTIONAL YTTRIUM-RICH HEAVY RARE EARTH DRILL RESULTS EXPAND THE MONTE ALTO DISTRICT Exceptional heavy rare earth grades: MADD0210 returned 2.5 m at 7.5% TREO from 6.0 m, including 1.3 m at 10.9% TREO from 7.2 m, with 58,249 ppm (5.8%) Y2O3, 4,135 ppm Dy2O3, 488 ppm Tb4O7 and 1,588 ppm U3O8 New high-grade, heavy rare earth target remains open: Auger hole STU2478, located ~200 m southwest of the initial diamond drilling, returned 12 m at 8.7% TREO from 18 m to end of hole, including 1,004 ppm DyTb and 0.44% Y2O3.

LEADING EDGE MATERIALS ADVANCES MIDSTREAM PROCESSING STRATEGY FOR THE NORRA KÄRR HEAVY RARE EARTH ELEMENTS PROJECT

Initiative launched to enhance Norra Kärr’s flowsheet with collaboration on new hydrometallurgical research in support of future pilot plant establishment. Swedish Government funding secured: two projects involving the company awarded funding through Vinnova, the Swedish Innovation agency, as part of a SEK 70+ million (CAD 10 million) national push to secure Sweden's critical raw materials supply.Path to concentrate production: Domain sampling now underway to generate high-grade eudialyte concentrate, the essential feedstock for upcoming hydrometallurgical pilot testing.On track to develop the EU’s first heavy rare earth elements mine following the grant of Norra Kärr’s 25-year mining lease in June 2026 Vancouver, July 20,2 026 - Leading Edge Materials Corp. ("Leading Edge Materials" or the "Company") (TSXV: LEM) (Nasdaq First North: LEMSE) (OTCQB: LEMIF) (FRA: 7FL) is pleased to provide an update on its midstream processing strategy for eudialyte concentrate from the Norra Kärr Heavy Rare Earth Elements ("HREE") Project – and the path toward pilot-scale hydrometallurgical testing, a key step in demonstrating the process at commercial scale. This builds on the significant flowsheet development work completed over the last 15 years, and new research into silicate management under the Vinnova-funded SHLENK project.

Eudialyte Processing

Eudialyte is recognised globally as one of the most promising mineral sources of heavy rare earth elements, with projects in Greenland and Australia being developed in addition to Norra Kärr in Sweden.   With eudialyte, the behaviour of silicates during leaching needs to be managed to prevent gel formation. Two well-known approaches are dilution, reducing silicate concentrations through sufficient dilution during leaching and inhibiting gel formation, and pre-treatment, treating the eudialyte concentrate ahead of leaching to prevent silicate release during dissolution, while preserving rare earth element solubility. Studies have demonstrated that under correctly selected pretreatment conditions for a given source material, silicate leaching can be effectively prevented. The pre-treatment option was used in the Company's 2021 Preliminary Economic Assessment ("PEA") and current project development workstreams are focused on performance and cost optimisation, and upscaling studies.

Pilot-Scale Hydrometallurgical Testing

The pre-treatment approach has shown strong results in laboratory-scale testing for Norra Kärr material and an extended pilot is intended to further strengthen hydrometallurgical understanding. To achieve optimal pilot data, a new high-grade eudialyte concentrate will be produced. Over the summer, the Company is conducting a domain sampling programme using the existing drill core library. This will be followed by further mineral processing to produce eudialyte concentrate while also generating valuable nepheline syenite as co-product for customer trials.

Consistent with the design set out in the Company’s 2021 PEA, mineral processing at Norra Kärr will only feature crushing, grinding, and magnetic separation; the eudialyte concentrate would then be transported to a midstream hydrometallurgical facility at a separate, established industrial location – keeping the footprint at Norra Kärr small and placing chemical processing where infrastructure already exists.

New Innovation Research: The SHLENK Project

The pilot work programme will benefit from research into improved processing methods, including through the Company's collaboration with RISE (Research Institutes of Sweden) under the SEK 1.5 million (CAD 0.2 million) funded SHLENK project – "Silicate management in leaching of eudialyte from Norra Kärr”.

SHLENK is one of two research projects involving the Company’s wholly owned Swedish subsidiary, Greenna Mineral AB, that have recently been granted funding by the Swedish Government through Vinnova's Impact Innovation call, "Resilient metals and minerals supply for strengthened preparedness," alongside the NordAL (Nordic Alumina) project.

Together with 17 other projects, these initiatives form part of a national effort to strengthen Sweden's metals and minerals supply chain in peacetime, during heightened preparedness, and in times of crisis. The combined programme represents more than SEK 70 million (approximately CAD 10 million) in funding, of which approximately SEK 42 million (CAD 6 million) is provided by Vinnova.

For the Company, this funding represents strong recognition of the strategic importance of its work at Norra Kärr and its contribution to building a more resilient and secure supply chain for critical raw materials in Sweden and the Nordics.

The work is carried out within Swedish Metals & Minerals, a joint initiative by the Swedish Energy Agency, Formas, and Vinnova under the framework of Impact Innovation.

Kurt Budge, Chief Executive Officer, commented:

“Following the grant of the 25-year mining lease, our work continues towards developing Norra Kärr, the EU's first heavy rare earth elements mine.

Progressing with our Pre-feasibility Study, we continue to de-risk and enhance the processing flowsheet for Norra Kärr. We have a clear plan: working with the best eudialyte concentrate, verifying our pre-treatment approach to silicate management, and generating the data we need to run at pilot scale.

Heavy rare earths like dysprosium and terbium are foundational to the technologies driving electrification and defence readiness across Europe, and Norra Kärr remains one of the continent's richest deposits. The support from Vinnova is a strong validation of the strategic role Norra Kärr can play in strengthening Sweden's and Europe's resilience in critical raw materials."

About the Norra Kärr Project

Norra Kärr is one of Europe's most significant deposits of heavy rare earth elements, hosted in an eudialyte-bearing alkaline rock body in southern Sweden. The project's strategic value is underpinned by its high dysprosium and terbium ("Dy/Tb") content, critical inputs for permanent magnets used in electric vehicles, wind turbines, and defence applications.

On 28 June 2026, an Exploitation Concession – 25-year mining lease - was granted by the Swedish Government following a formal recommendation from the Mining Inspectorate (Sw. Bergsstaten), which submitted the application to the Government for a final decision after all involved agencies had either endorsed the application or recommended approval.

The strategic importance of heavy rare earth elements to Europe's industrial future has never been more apparent. As China's export controls have demonstrated, access to dysprosium, terbium and yttrium cannot be taken for granted — and the consequences of supply disruption are severe. Norra Kärr, now holding an Exploitation Concession, is key to addressing Europe’s critical risk exposure.

Edison Investment Research

"Leading Edge Materials - Meeting Europe's Rare Earths Challenge", 9 February 2026.
https://www.edisongroup.com/research/meeting-europes-rare-earths-challenge/BM-2766/  

Edison estimates current European dysprosium demand at 180-200 tpa of Dy₂O₃. Norra Kärr's 2021 PEA discloses average annual Dy₂O₃ production of 248t, which Edison notes is "similar to European consumption."

“Leading Edge Materials — Addressing the European REE shortage”, 21 April 2026.
https://www.edisongroup.com/research/addressing-the-european-ree-shortage/BM-2909/

The scientific and technical information contained in this news release relating to the Norra Kärr project, including details of forecast dysprosium, terbium and yttrium production, are set out the National Instrument 43-101 technical report entitled "Preliminary Economic Assessment of Norra Kärr Rare Earth Deposit and Potential By-Products, Sweden", with effective date August 18, 2021, and issue date August 19, 2021, prepared for Leading Edge Materials Corp. by SRK Consulting (UK) Ltd. The report is available on the Company's website at www.leadingedgematerials.com and under its SEDAR profile at www.sedar.ca.

Qualified Person

The scientific and technical information contained in this news release relating to the Norra Kärr project has been reviewed and approved by John Willis of SRK Consulting (UK) Ltd, a Chartered Professional and Member of the Australasian Institute of Mining and Metallurgy, who is an independent Qualified Person under the terms of NI 43-101 for REE deposits. SRK Qualified Persons are all independent as defined under National Instrument 43-101 - Standards of Disclosure for Mineral Projects. 

Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accept responsibility for the adequacy or accuracy of this news release.

On behalf of the Board of Directors,
Leading Edge Materials Corp.

Kurt Budge, CEO

For further information, please contact the Company at:
[email protected]
www.leadingedgematerials.com

Corporate Head Office (Vancouver, Canada): 778-686-5357

About Leading Edge Materials

Leading Edge Materials Corp. is a Canadian-listed company focused on developing critical raw material assets across the European Union. Its primary focus is the wholly owned Norra Kärr Heavy Rare Earth Element project in Sweden — one of the world's most strategically significant heavy rare earth deposits and among the few advanced-stage projects within the EU capable of producing dysprosium, terbium, and yttrium at meaningful scale.

Situated in one of the globe's most politically and regulatory stable mining environments, Norra Kärr is well-positioned to contribute directly to the objectives of the EU's Critical Raw Materials Act, including the bloc's target of sourcing 10% of its critical raw material consumption domestically by 2030. Beyond rare earths, the Company also holds the Woxna Graphite mine in Sweden — a fully constructed and permitted facility — as well as a 90% stake in the Bihor Sud Nickel-Cobalt exploration alliance in Romania.

Additional Information

The information was submitted for publication through the agency of the contact person set out above, on July 20, 2026, at 23:30 Vancouver time.

Leading Edge Materials is listed on the TSXV under the symbol “LEM”, OTCQB under the symbol “LEMIF” and Nasdaq First North Stockholm under the symbol “LEMSE”. Svensk Kapitalmarknadsgranskning (“SKMG”) is the Company’s Certified Adviser for the Nasdaq First North Growth Market (Stockholm) and may be contacted via email [email protected] or by phone +46 (0)8 913 008.

Reader Advisory

This news release may contain statements which constitute “forward-looking information”, including statements regarding the plans, intentions, beliefs and current expectations of the Company, its directors, or its officers with respect to the future business activities of the Company. The words “may”, “would”, “could”, “will”, “intend”, “plan”, “anticipate”, “believe”, “estimate”, “expect” and similar expressions, as they relate to the Company, or its management, are intended to identify such forward-looking statements. Investors are cautioned that any such forward-looking statements are not guarantees of future business activities and involve risks and uncertainties, and that the Company’s future business activities may differ materially from those in the forward-looking statements as a result of various factors, including, but not limited to, fluctuations in market prices, changes in the Company’s intended use of proceeds from the Private Placement, successes of the operations of the Company, continued availability of capital and financing and general economic, market or business conditions. There can be no assurances that such information will prove accurate and, therefore, readers are advised to rely on their own evaluation of such uncertainties. The Company does not assume any obligation to update any forward-looking information except as required under the applicable securities laws.

Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accept responsibility for the adequacy or accuracy of this news release.

1D_20260720 Norra Kärr Processing _CLEAN

July 08, 2026 09:08 ET  | Source: Ultragenyx Pharmaceutical Inc.

NOVATO, Calif., July 08, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2025 Impact Report, detailing the company's work on behalf of patients and families living with rare and ultra-rare diseases. In 2025, Ultragenyx advanced five investigational therapies in pivotal clinical programs with the potential to reach tens of thousands of patients around the world — while continuing to build on years of work to support patients who cannot yet access approved treatments, which have now reached individuals in 50 countries.

“The rare disease community has waited long enough — every program we advance, every trial site we open, and every patient we support through access to approved medicines is a reflection of that belief," said Emil Kakkis, M.D., Ph.D., founder, president, and chief executive officer of Ultragenyx. "Our 2025 Impact Report reflects the progress we are making today to deliver urgently needed, first-ever therapies, as well as our commitment to shaping a more accessible future for rare diseases.”

The report outlines progress across six key pillars — Innovation, Patients, People, Communities, Planet, and Governance — as well as Sustainability Accounting Standards Board (SASB), Global Reporting Initiative (GRI), and Task Force on Climate Related Disclosures (TCFD) indices. Updates reflect Ultragenyx’s ongoing focus on delivering meaningful, long-term impact for patients and families impacted by rare and ultra-rare diseases and cover activities from January 1 through December 31, 2025. For the full report and more on Ultragenyx’s Corporate Responsibility efforts, visit https://www.ultragenyx.com/ultra-committed/corporate-responsibility/.

Highlights from 2025 include:

Innovation
Ultragenyx continued to expand access to its four approved therapies across five indications and advanced its pipeline with a singular focus on addressing significant unmet medical needs for rare disease patients:

Invested approximately 62% of operating expenses in research and development in 2025Drove progress on five clinical-stage investigational therapies with the potential to reach tens of thousands of patientsApplied its Dynamic Development Model (DDM) — centered around direct patient and caregiver input — to inform clinical trial design and accelerate clinical development taking place across 160+ clinical trial sites in 19 countries Patients
Ultragenyx remained committed to improving access, advancing advocacy, and partnering with the rare disease community.

Hosted two Rare Bootcamps, including the first held on the U.S. East Coast, to support patient-led drug development initiatives
Supported more than 700 patients in 50 countries through expanded access and patient assistance programs since 2013
Participated in over 45 global patient advocacy events and engagements in 2025Launched Act for Ultra-Rare, initiating policy discussions to advocate for legislation that supports the development of treatments for ultra-rare diseases to help ensure that these patient communities are not left behind
People
Ultragenyx invested in its global workforce through engagement, inclusion, and professional development.

Maintained strong employee engagement, with an overall engagement score of 86%Delivered more than 80 employee learning and development workshops in 2025Achieved 92% participation in the annual employee engagement survey
Communities
Ultragenyx expanded its philanthropic impact and community engagement efforts globally.

Supported ~180 organizations across ~230 grant programs focused on education, awareness, and patient advocacyContributed more than 2,800 employee volunteer hours supporting local and global initiatives and hosted the third annual Global Days of Service, encompassing nearly 40 volunteer projects worldwideApproved approximately $3.3 million in charitable donations, medical education, and health-related grants across more than 20 countries
Planet
Ultragenyx advanced environmental sustainability initiatives across its operations.

Purchased ~100% renewable electricity for its Novato, CA headquarters campus and Bedford, MA Gene Therapy Manufacturing Facility, and diverted tens of thousands of pounds of waste through recycling and sustainable lab practicesInitiated its second My Green Lab certification in Somerville, MA, building on a ‘Green’ level certification in Novato, CAConducted its inaugural Climate Risk Assessment to evaluate physical and transition risks across operations and supply chain
Governance
Ultragenyx focused on strong corporate governance, compliance, and ethical business practices.

Continued to integrate corporate responsibility oversight through board-level governance structuresMaintained a comprehensive compliance program aligned with global regulatory standardsReported no material data privacy breaches or cybersecurity incidents in 2025
Recognition
Ultragenyx’s achievements in 2025 were recognized through multiple awards and honors.

Named a Top Place to Work in the USA and recognized by The Boston Globe and San Francisco ChronicleReceived multiple Top Workplaces Culture Excellence Awards, including recognition for compensation, well-being, innovation, leadership, and professional developmentHonored for excellence in patient engagement, including recognition from the rare disease community
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media
This press release and the 2025 Ultragenyx Impact Report and other materials cross-referenced in this press release contain statements that are aspirational or reflective of our views about the company’s future performance that constitute “forward looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are generally identified through the inclusion of words such as “aim,” “anticipate,” “aspire,” “believe,” commit,” “endeavor,” “estimate,” “expect,” “goal,” “intend,” “may,” “plan,” “seek,” “strive,” “target,” “will,” vision,” “mission,” “strategy,” “commitment” and “work,” or similar statements or variations of such terms and other similar expressions that predict or indicate future events or trends or that are not statements of historical fact. The forward-looking statements in this document and the materials cross-referenced concern Ultragenyx’s goals, progress or expectations with respect to corporate responsibility, sustainability, patients, products, product candidates, employees, environmental matters, policy and business risks and opportunities and are not intended to create legal rights or obligations. Forward-looking statements inherently involve risks and uncertainties that could cause actual results to differ materially from those predicted in such statements including changes in economic conditions, slowed or insufficient technological developments, stakeholder engagement, changes in corporate strategy, and changes in the legal or regulatory environment. These statements are based on numerous assumptions that the company believes are reasonable but are open to a wide range of uncertainties and business risks. In addition, these statements may be based on standards for measuring progress that are still developing, controls and processes that continue to evolve, and assumptions that are subject to change in the future, and certifications, representations or data reviewed or provided by third parties Consequently, actual results may vary materially from what is contained in a forward-looking statement.

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 6, 2026, and its subsequent periodic reports filed with the SEC. Forward-looking statements are aspirational and are not guarantees or promises that goals or targets will be met. Ultragenyx undertakes no obligation to update any forward-looking or other statements, whether as a result of new information, future events, or otherwise, and notwithstanding any historical practice of doing so. Ultragenyx may determine to adjust any goals and targets or establish new ones to reflect changes in its business. The information included in, and any issues identified as material for purposes of, the 2025 Ultragenyx Impact Report is not an indication that they are considered material to Ultragenyx, its investors or other stakeholders, or required to be disclosed in the company’s filings, in each case under SEC reporting or any other laws or requirements that may apply to the company. In the context of this report, the term “material” is distinct from, and should not be confused with, such term as defined for SEC or other mandatory reporting purposes. Historical clinical trial success rates are not necessarily predictive, and should not be considered a guarantee, of future success rates.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Ultragenyx Contacts

Investors
Joshua Higa
[email protected]

Media
Jess Rowlands
[email protected]

Ultragenyx (RARE) is transitioning from a broad pipeline to a focused rare disease platform, entering a high-catalyst period with four approved products and multiple late-stage assets. Despite a recent Phase 3 failure and Q1 2026 net loss, RARE reaffirmed 2026 revenue guidance of $730–$760 million, supported by a scalable commercial base and cost controls. Key near-term catalysts include FDA reviews for DTX401 and UX111 (PDUFA dates in H2 2026) and pivotal Phase 3 Aspire results for GTX-102 in Angelman syndrome.

Wall Street expects a year-over-year increase in earnings on higher revenues when Ultragenyx (RARE - Free Report) reports results for the quarter ended March 2026. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.

The earnings report might help the stock move higher if these key numbers are better than expectations. 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 biotechnology company is expected to post quarterly loss of $1.55 per share in its upcoming report, which represents a year-over-year change of +1.3%.

Revenues are expected to be $161.26 million, up 15.8% from the year-ago quarter.

Estimate Revisions TrendThe consensus EPS estimate for the quarter has been revised 5.07% 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. This insight is at the core of our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction).

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 Ultragenyx?For Ultragenyx, the Most Accurate Estimate is lower than the Zacks Consensus Estimate, suggesting that analysts have recently become bearish on the company's earnings prospects. This has resulted in an Earnings ESP of -0.05%.

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

So, this combination makes it difficult to conclusively predict that Ultragenyx will 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 Ultragenyx would post a loss of$1.2 per share when it actually produced a loss of -$1.29, delivering a surprise of -7.50%.

Over the last four quarters, the company has beaten consensus EPS estimates just once.

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.

Ultragenyx doesn't appear 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.

An Industry Player's Expected ResultsAmong the stocks in the Zacks Medical - Biomedical and Genetics industry, Grail (GRAL - Free Report) , is soon expected to post loss of $3 per share for the quarter ended March 2026. This estimate indicates a year-over-year change of +3.2%. This quarter's revenue is expected to be $40.47 million, up 27.1% from the year-ago quarter.

Over the last 30 days, the consensus EPS estimate for Grail has remained unchanged. Nevertheless, the company now has an Earnings ESP of -2.67%, reflecting a lower Most Accurate Estimate.

When combined with a Zacks Rank of #3 (Hold), this Earnings ESP makes it difficult to conclusively predict that Grail will beat the consensus EPS estimate. Over the last four quarters, the company surpassed consensus EPS estimates three times.

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

Arcutis Biotherapeutics, Inc. (ARQT - Free Report) is expected to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended March 2026. This widely-known consensus outlook gives a good sense of the company's earnings picture, but how the actual results compare to these estimates is a powerful factor that could impact its near-term stock price.

The earnings report, which is expected to be released on May 6, might help the stock move higher if these key numbers are better than expectations. On the other hand, if they miss, the stock may move lower.

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

Zacks Consensus EstimateThis company is expected to post quarterly loss of $0.02 per share in its upcoming report, which represents a year-over-year change of +90%.

Revenues are expected to be $99.15 million, up 50.6% from the year-ago quarter.

Estimate Revisions TrendThe consensus EPS estimate for the quarter has been revised 40% lower 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. This insight is at the core of our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction).

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 Arcutis Biotherapeutics?For Arcutis Biotherapeutics, the Most Accurate Estimate is lower than the Zacks Consensus Estimate, suggesting that analysts have recently become bearish on the company's earnings prospects. This has resulted in an Earnings ESP of -20.00%.

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

So, this combination makes it difficult to conclusively predict that Arcutis Biotherapeutics will beat the consensus EPS estimate.

Does Earnings Surprise History Hold Any Clue?While calculating estimates for a company's future earnings, analysts often consider to what extent it has been able to match past consensus estimates. 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 Arcutis Biotherapeutics would post earnings of $0.03 per share when it actually produced earnings of $0.13, delivering a surprise of +333.33%.

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

Arcutis Biotherapeutics doesn't appear 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.

An Industry Player's Expected ResultsAmong the stocks in the Zacks Medical - Biomedical and Genetics industry, Ultragenyx (RARE - Free Report) , is soon expected to post loss of $1.55 per share for the quarter ended March 2026. This estimate indicates a year-over-year change of +1.3%. This quarter's revenue is expected to be $161.26 million, up 15.8% from the year-ago quarter.

The consensus EPS estimate for Ultragenyx has been revised 5.1% higher over the last 30 days to the current level. However, a lower Most Accurate Estimate has resulted in an Earnings ESP of -0.05%.

This Earnings ESP, combined with its Zacks Rank #3 (Hold), makes it difficult to conclusively predict that Ultragenyx will beat the consensus EPS estimate. Over the last four quarters, the company surpassed EPS estimates just once.

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

Ultragenyx (RARE - Free Report) came out with a quarterly loss of $1.84 per share versus the Zacks Consensus Estimate of a loss of $1.55. This compares to a loss of $1.57 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of -18.96%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.2 per share when it actually produced a loss of $1.29, delivering a surprise of -7.5%.

Over the last four quarters, the company has surpassed consensus EPS estimates just once.

Ultragenyx, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $136 million for the quarter ended March 2026, missing the Zacks Consensus Estimate by 15.66%. This compares to year-ago revenues of $139.29 million. The company has topped consensus revenue estimates two times over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

Ultragenyx shares have added about 7.9% since the beginning of the year versus the S&P 500's gain of 5.2%.

What's Next for Ultragenyx?While Ultragenyx has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for Ultragenyx was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is -$1.25 on $184.41 million in revenues for the coming quarter and -$4.49 on $750.36 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the bottom 42% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

One other stock from the same industry, Iovance Biotherapeutics (IOVA - Free Report) , is yet to report results for the quarter ended March 2026. The results are expected to be released on May 7.

This biotechnology company is expected to post quarterly loss of $0.19 per share in its upcoming report, which represents a year-over-year change of +47.2%. The consensus EPS estimate for the quarter has been revised 18.2% lower over the last 30 days to the current level.

Iovance Biotherapeutics' revenues are expected to be $77.11 million, up 56.3% from the year-ago quarter.

Ultragenyx Pharmaceutical Inc. (RARE) Q1 2026 Earnings Call Transcript

Tuesday, Rare Earth Americas priced its IPO at $19 per share.

watch now

Developing a new drug can cost a billion dollars and take more than a decade. That makes investing in new treatments in the rare disease space — where patient populations are small, and the chance of earning a return on that investment even smaller — a risky bet for big pharmaceutical companies.

That's a big reason why 95% of the more than 10,000 rare diseases that exist do not have an FDA-approved treatment.

But for parents of children diagnosed with those diseases, doing nothing is not an option. And that's fueling a major trend in the rare disease community: medical innovation and breakthroughs that are being driven by the patient groups themselves.

Last October, Rare As One — a project funded by the Chan Zuckerberg Biohub that supports patient-led research in the rare disease space — released a report that showed of the 20 organizations it funded when the program launched in 2019, half of them were involved in clinical trials within five years.

That's remarkable progress from advocacy groups that are mostly led by the communities they represent – regular parents from varied backgrounds, partnering with researchers and clinicians to develop life saving treatments for their loved ones. But for many of these families, figuring out where to start that process can be incredibly complicated.

Ultragenyx, an established player in rare and ultra-rare drug development, is looking to fill that gap and provide families and patient advocates the tools and resources for discovering a treatment.

Twice a year the company hosts Rare Bootcamp, a multi-day forum that helps families learn about conducting rare disease research and the important steps that go into developing a new drug. It also lets them connect with others going through a similar experience.

The event is free for attendees, and is sponsored by other biotech firms, such as Alexion, BioMarin, GeneDx and BridgeBio, that also send experts to participate in panels.

During its most recent bootcamp in Boston last month, the company held more than 20 sessions covering the full spectrum of drug development, from early science to regulatory approval.

Ultragenyx founder and CEO Emil Kakkis launched the bootcamps nine years ago, inspired by his own difficulties researching new treatments during his early days in the industry.

"There's no book, there's no 'CliffsNotes' on how to develop a drug," Kakkis said.

"I struggled and struggled, and I promised myself at that time that if I ever became in the position that I knew how to do it, I was just going to tell everyone else that needed it," Kakkis said.

Mike and Evelyn Ribadeneyra attended the most recent bootcamp. They are trying to find a treatment for their daughter Abbie, who suffers from hereditary spastic paraplegia type 26, a progressive neurodegenerative disorder that took two decades to diagnose. 

While they said they did not initially start their journey expecting to find a cure for their daughter's condition, in the past year they discovered that a gene therapy existed for a similar disorder called SPG 50.  

"We've been on a long journey, but our new journey begins at rare disease boot camp," Mike Ribadeneyra said.

The Ribadeneyras came to Boston to receive a roadmap for how to convince others to conduct research on behalf of their child, and to develop a proof of concept for the drug.

"I didn't know what to expect, but it has been overwhelming. The access that we've been given to the researchers, specialists, and doctors … I feel like they're really in our community now," Evelyn said.

The event also serves as a gathering place for families and advocates navigating similar circumstances. Through networking sessions at the conference and dinners at the end of the day, attendees share tips and compare notes on their rare disease journeys. 

"We felt so alone for so many years, and my daughter feels so alone … and to know that there are other people going through the exact same process is really encouraging," Mike said.

Laura Wilson attended the event to help her daughter Ellia, who was diagnosed with a rare neurodevelopmental disorder called ReNU2.

Wilson spent about nine years without a formal diagnosis for Ellia, as the gene was only identified in April 2025. She recently founded an organization called ReNU2 United to help families affected by the disease. So far, about 60 families in 17 countries have identified that their child has the disorder.

Wilson attended Ultragenyx's bootcamp to hear from experts in the room about developing treatments, and to learn best practices from other families that have used their organizations to support research. 

"No one in this room has a child with the same disorder that my child has, but we share so much in common," Wilson said in an interview.

"That experience of being a parent to a child with a rare disorder is so unifying that, you know, you sort of get it right away," Wilson said.

The bootcamp, which is co-hosted by the EveryLife Foundation — an advocacy group founded by Kakkis in 2009 — has grown through word of mouth. Roughly two dozen attendees attended last month's event, and since it launched about 235 people from 142 organizations have completed the program.

It's also inspired other similar events, like the RARE Advocate Development Brain Workshop. That workshop focuses on rare diseases that attack the central nervous system, and is hosted by the Rare Epilepsy Network, Mahzi Therapeutics and the advocacy group Global Genes.

Ultimately though, Kakkis says he's looking forward to the day that these types of events will no longer be needed.

"My hope some day is that we'll fix this problem," Kakkis said. "And we'll know that it's fixed, because we won't see any more parents having to develop their own drugs. They'll all be done. That's what I'm hoping for."

For more stories like this, sign up for the CNBC Cures Newsletter.