Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm stock.
Ultragenyx Pharmaceutical Inc. develops and commercializes therapies for serious rare and ultra-rare genetic diseases. Company news commonly covers revenue from approved medicines such as Crysvita, Dojolvi, and Mepsevii, financial guidance, and updates to a portfolio of approved therapies and product candidates for diseases with high unmet medical need and clear biology.
Recurring developments include FDA interactions and clinical data for investigational programs such as UX111 for Sanfilippo syndrome type A, GTX-102 for Angelman syndrome, UX016 for GNE myopathy, and AAV gene therapy candidates for rare metabolic disorders. Updates also include healthcare conference participation, employment inducement equity grants under Nasdaq rules, and corporate restructuring or expense-management actions when disclosed.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has initiated a rolling submission of a Biologics License Application (BLA) to the FDA for DTX401, their AAV gene therapy treatment for Glycogen Storage Disease Type Ia (GSDIa). The company has submitted the non-clinical and clinical modules and plans to complete the full BLA with the chemistry, manufacturing and controls (CMC) module in Q4 2025.
The BLA includes 96-week Phase 3 study data showing significant cornstarch reduction in patients: 60% reduction in the ongoing DTX401 group and 64% reduction in the Crossover Placebo to DTX401 group. The submission will address FDA observations from the UX111 complete response letter regarding CMC and manufacturing facilities.
Ultragenyx (NASDAQ: RARE) reported Q2 2025 financial results with total revenue of $166 million, representing 13% growth year-over-year. Key revenue drivers include Crysvita at $120 million and Dojolvi at $23 million.
The company reaffirmed its 2025 guidance, expecting total revenue between $640-670 million. Despite a net loss of $115 million ($1.17 per share), Ultragenyx maintains its path to profitability target for 2027. Cash position stands at $539 million as of June 30, 2025.
Notable pipeline updates include: UX143 Phase 3 data for osteogenesis imperfecta expected by year-end, GTX-102 receiving FDA Breakthrough Therapy Designation for Angelman syndrome with Phase 3 fully enrolled, and planned BLA submission for DTX401 in Q4 2025.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has completed enrollment for its Phase 3 Aspire study of GTX-102 (apazunersen) for Angelman Syndrome treatment. The study successfully enrolled 129 participants aged 4-17 with confirmed maternal UBE3A gene deletion in just seven months.
The trial design includes a 1:1 randomization between GTX-102 intrathecal injection and sham control, with active treatment consisting of three monthly 8 mg loading doses followed by maintenance doses up to 14 mg quarterly. The study's primary endpoint focuses on cognitive improvement measured by Bayley-4 cognitive raw score, with completion expected in second half of 2026.
Additionally, Ultragenyx plans to initiate the Aurora study of GTX-102 in additional ages and genotypes in the second half of 2025.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in treatments for rare and ultra-rare genetic diseases, has scheduled a conference call for Tuesday, August 5, 2025, at 5:00 p.m. ET. The call will cover the company's financial performance and corporate updates for Q2 2025, ending June 30, 2025.
Investors can access both the live and replay versions of the webcast through the company's investor relations website. The replay will remain available for three months following the presentation.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultra-rare disease therapies, has announced the grant of 45,292 restricted stock units (RSUs) to 29 newly hired non-executive officers. The grants were approved by the company's compensation committee and issued under the Ultragenyx Employment Inducement Plan on July 16, 2025.
The RSUs will vest over a four-year period, with 25% of the shares vesting annually on each grant date anniversary, contingent on continued employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for UX111, a gene therapy treatment for Sanfilippo syndrome type A. The FDA cited specific chemistry, manufacturing and controls (CMC) related observations that require resolution.
While the FDA acknowledged the robustness of clinical data and supportive biomarker evidence, this delay pushes the potential approval timeline to 2026. The company believes the CMC observations are addressable and primarily relate to facilities and processes, not product quality. After resolving these issues, Ultragenyx plans to resubmit the BLA, which will be followed by an estimated 6-month review period.
Ultragenyx Pharmaceutical (NASDAQ: RARE) and Mereo BioPharma announced that their Phase 3 Orbit study of UX143 (setrusumab) for osteogenesis imperfecta (OI) will continue to final analysis, expected around year-end. The Data Monitoring Committee confirmed UX143's acceptable safety profile.
The study evaluates UX143 in pediatric and young adult OI patients. The parallel Cosmic study in younger patients is also progressing well, with safety consistent with other studies. Both trials will conclude after patients complete 18 months of therapy, with significance thresholds set at p<0.04 for Orbit and p<0.05 for Cosmic.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has received FDA Breakthrough Therapy Designation for GTX-102 (apazunersen) in treating Angelman syndrome. The designation is based on promising Phase 1/2 study results involving 74 patients aged 4-17 years, showing consistent developmental improvements across multiple symptom domains for up to 3 years.
The company's Phase 3 Aspire study, which began in December 2024, aims to enroll approximately 120 children with Angelman syndrome. Additionally, the Aurora study, evaluating GTX-102 in other Angelman syndrome genotypes and ages, is expected to start in the second half of 2025.