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Key Takeaways Ultragenyx will receive $210M from selling a Rare Pediatric Disease Priority Review Voucher.The deal adds non-dilutive cash to support research, development and commercialization efforts.About $168M remains before other costs after a $42M NIH payment tied to a prior patent license. Ultragenyx Pharmaceutical (RARE - Free Report) has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million. The transaction is expected to provide the rare-disease drugmaker with a meaningful influx of non-dilutive cash, strengthening its financial position as it advances therapies for rare and ultra-rare diseases. The proceeds could also support Ultragenyx’s efforts to move toward profitability without requiring an equity financing. The deal remains subject to the satisfaction of certain regulatory and customary closing conditions.

The PRV was awarded to Ultragenyx following the FDA approval of Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401. Under the agency’s Rare Pediatric Disease PRV program, an eligible sponsor can receive a voucher after gaining approval for a qualifying rare pediatric disease therapy. The voucher can then be redeemed to obtain priority review for a future marketing application for a different eligible product. Importantly, the voucher can also be sold or transferred, allowing drugmakers to monetize the regulatory asset and generate capital.

Recent FDA Approval of Genglycos Earns RARE a Valuable PRVIn August, the FDA granted accelerated approval to Ultragenyx’s Genglycos to treat glycogen storage disease type Ia (GSDIa), making it the first treatment designed to address the underlying cause of the rare metabolic disorder. The therapy is indicated as an adjunct to nutritional management to reduce daily cornstarch intake in adults and pediatric patients aged eight years and older. The approval represents another important regulatory milestone for Ultragenyx as the company expands its portfolio of therapies targeting serious rare diseases.

Year to date, RARE shares have plunged 35.3% against the industry’s 1.3% growth.

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Ultragenyx’s Genglycos is an AAV8 gene therapy developed to target the underlying disease biology in GSDIa. By focusing on the root cause rather than solely managing symptoms through dietary measures, the therapy broadens treatment options for patients with a condition associated with significant unmet medical need. The approval marked Ultragenyx’s first gene therapy product and fifth FDA approval overall, strengthening its rare-disease franchise and expanding its presence in genetic and metabolic disorders.

The approval was strategically important because the resulting PRV gives Ultragenyx immediate financial flexibility beyond Genglycos’ commercial opportunity. The $210 million sale will provide non-dilutive capital to support research, development and commercialization. However, 20% ($42 million) of the proceeds is payable to the NIH under a prior patent license agreement, leaving about $168 million before other costs. Ultragenyx has previously benefited from PRV monetization to fund Genglycos’ development, highlighting the program’s role as a source of non-dilutive funding for rare-disease innovation.

Ultragenyx’s marketed portfolio spans several rare metabolic and genetic disorders. Crysvita (burosumab-twza) is approved for X-linked hypophosphatemia in adults and pediatric patients and for tumor-induced osteomalacia, while Mepsevii (vestronidase alfa-vjbk) treats mucopolysaccharidosis type VII. Dojolvi (triheptanoin) is indicated for long-chain fatty acid oxidation disorders, and Evkeeza (evinacumab-dgnb) is approved for homozygous familial hypercholesterolemia. Evkeeza is a partnered drug, with Ultragenyx holding rights to develop, commercialize and distribute it outside the United States, while Regeneron Pharmaceuticals commercializes it in the United States.

In September, the FDA also approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time gene therapy for the neurologic manifestations of mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome Type A, in pediatric patients with preserved neurodevelopmental function. The approval marked Ultragenyx’s sixth FDA approval overall and second gene therapy approval, further expanding its presence in rare and ultra-rare diseases.

RARE’s Zacks Rank & Stocks to ConsiderUltragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Alnylam Pharmaceuticals (ALNY - Free Report) , currently sporting a Zacks Rank #1 (Strong Buy), and CRISPR Therapeutics (CRSP - Free Report) and Aldeyra Therapeutics (ALDX - Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Alnylam’s 2026 earnings per share have decreased from $8.65 to $8.63. Over the same period, earnings estimates for 2027 have decreased from $12.13 to $12.04. ALNY shares have plunged 43.5% year to date.

Alnylam’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 27.58%.

Over the past 60 days, estimates for CRISPR Therapeutics’ 2026 loss per share have narrowed from $4.53 to $4.47. Over the same period, earnings estimates for 2027 have narrowed from $3.63 to $3.61. CRSP shares have lost 0.1% year to date.

CRISPR Therapeutics’ earnings beat estimates in two of the trailing four quarters and missed on the remaining two occasions, with the average negative surprise being 1.38%.

Over the past 60 days, loss per share estimates for Aldeyra Therapeutics have narrowed from 43 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 22 cents to 16 cents. ALDX shares have plunged 81.5% year to date.

Aldeyra Therapeutics’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 29.25%.

Key Takeaways Ultragenyx's MPS IIIA gene therapy gained EMA validation, advancing its application toward formal review.Clinical data showed a durable efficacy nearly 8 years after treatment with an acceptable safety profile.Gene therapy-treated patients showed a 23.5-point greater mean Bayley-III Cognitive score change. Ultragenyx Pharmaceutical (RARE - Free Report) announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for the investigational gene therapy rebisufligene etisparvovec (also known as UX111) to treat mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A. The validation marks a key regulatory milestone as the therapy advances toward formal review in Europe.

EMA validation confirms that the application is sufficiently complete for the agency to begin its scientific assessment. However, the validation does not indicate the therapy’s safety or efficacy, nor does it guarantee that the MAA will ultimately be approved. As an investigational advanced therapy, the MAA will undergo the EU’s Joint Clinical Assessment, which supports a coordinated clinical evaluation across EU member states as part of the broader pathway to patient access.

Last month, Ultragenyx received standard full FDA approval for rebisufligene etisparvovec to be marketed in the United States as Fayuvi for treating the neurologic manifestations of MPS IIIA in pediatric patients with preserved neurodevelopmental function. The approval made Fayuvi the first FDA-approved treatment for the rare and fatal neurodegenerative disorder. The approval marked Ultragenyx’s second gene therapy approval and sixth FDA approval overall.

Ultragenyx in-licensed the drug from Abeona Therapeutics in 2022 under an exclusive agreement for ABO-102, which RARE later developed as UX111. Under the deal, Ultragenyx assumed responsibility for the program and obtained exclusive rights to develop, manufacture and commercialize the therapy worldwide. In return, Abeona is eligible for tiered royalties ranging from mid-single-digit percentages to 8% of net sales, along with up to $30 million in commercial milestone payments following regulatory approval.

Clinical Data Backs the FDA Approval of RARE’s MPS IIIA DrugThe EMA validation of the MAA for rebisufligene etisparvovec in MPS IIIA is supported by data from the pivotal Transpher A study and long-term follow-up studies. According to Ultragenyx, clinical data now extend to nearly eight years and show a durable treatment effect across multiple clinical assessments and biomarkers, while maintaining an acceptable safety profile. Biochemical efficacy was demonstrated through reductions in accumulated cerebrospinal fluid heparan sulfate levels, reflecting replacement of the deficient enzyme responsible for the disease throughout the study and across all age groups.

Year to date, Ultragenyx shares have plunged 33.8% against the industry’s 4% growth.

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The key efficacy analysis compared 17 rebisufligene etisparvovec-treated patients in the modified intention-to-treat population with 27 untreated patients from an external natural-history cohort. Between 24 and 60 months of age, rebisufligene etisparvovec-treated patients demonstrated a 23.5-point greater mean change in Bayley-III Cognitive raw score compared with the natural-history group. The difference was statistically significant and provided the efficacy basis for the FDA’s standard full approval.

MPS IIIA is an ultra-rare lysosomal storage disorder caused by mutations that result in a deficiency of the sulfamidase enzyme. The resulting accumulation of heparan sulfate primarily damages the central nervous system, leading to progressive developmental delay and eventual loss of cognitive, language and motor abilities. Ultragenyx estimates that the disease affects approximately 3,000 to 5,000 patients across commercially accessible geographies, with a median life expectancy of about 15 years.

Ultragenyx’s rebisufligene etisparvovec is designed as a single-dose intravenous AAV9 gene therapy that delivers a functional copy of the SGSH gene. This enables production of the sulfamidase enzyme that is deficient in patients with MPS IIIA and is intended to address the underlying cause of the disease.

Treatment options for children affected by MPS IIIA remain limited outside the United States, underscoring the significant unmet medical need in this patient population. Rebisufligene etisparvovec has received Priority Medicines (PRIME) and Orphan Drug designations from the EMA, reflecting the rarity and severity of MPS IIIA and the need for new treatment options. These designations support the development and regulatory evaluation of medicines for serious and rare conditions and do not imply that marketing authorization will ultimately be granted.

Ultragenyx is also engaging with health authorities across multiple regions, including initiating discussions with the regulatory bodies in the United Kingdom and Saudi Arabia, as it works toward potential geographic expansion.

RARE’s Zacks Rank & Stocks to ConsiderUltragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Precigen (PGEN - Free Report) , currently sporting a Zacks Rank #1 (Strong Buy), and AC Immune (ACIU - Free Report) and Aldeyra Therapeutics (ALDX - Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Precigen’s 2026 bottom line have improved from a loss of 2 cents to earnings per share of 24 cents. Over the same period, earnings estimates for 2027 have risen from 25 cents to 73 cents. PGEN shares have increased 104.3% year to date.

Precigen’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 108.96%.

Over the past 60 days, estimates for AC Immune’s 2026 loss per share have narrowed from 84 cents to 60 cents. Over the same period, earnings estimates for 2027 remained unchanged at 17 cents. ACIU shares have lost 13.2% year to date.

AC Immune’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 33.25%.

Over the past 60 days, loss per share estimates for Aldeyra Therapeutics have narrowed from 43 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 22 cents to 16 cents. ALDX shares have plunged 77.4% year to date.

Aldeyra Therapeutics’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 29.25%.

, /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.

The investigation concerns whether Ultragenyx and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.

[Click here for information about joining the class action]

On September 2, 2026, Ultragenyx issued a press release "announc[ing] results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome." Per the press release, "[t]he 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)."

On this news, Ultragenyx's stock price fell $11.68 per share, or 44.03%, to close at $14.85 per share on September 3, 2026.

Pomerantz LLP, with offices in New York, Chicago, Los Angeles, London, Paris, and Tel Aviv, is acknowledged as one of the premier firms in the areas of corporate, securities, and antitrust class litigation. Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions. Today, more than 85 years later, Pomerantz continues in the tradition he established, fighting for the rights of the victims of securities fraud, breaches of fiduciary duty, and corporate misconduct. The Firm has recovered numerous multimillion-dollar damages awards on behalf of class members. See www.pomlaw.com.

Attorney advertising. Prior results do not guarantee similar outcomes. 

CONTACT:
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Pomerantz LLP
[email protected]
646-581-9980 ext. 7980

SOURCE Pomerantz LLP

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]

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.