Ultragenyx Pharm Stock Price, News & Analysis

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.

Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced the grant of 48,731 restricted stock units (RSUs) to 26 newly hired non-executive employees. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan, effective June 16, 2025. The RSUs will vest over a four-year period, with 25% of shares vesting annually on each grant date anniversary. This grant was made in accordance with Nasdaq Listing Rule 5635(c)(4) as an employment inducement for new hires. Ultragenyx, which focuses on developing and commercializing therapies for rare and ultra-rare diseases, uses these equity awards to attract and retain talent.

Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in novel therapies for rare and ultra-rare genetic diseases, announced its upcoming participation in the Goldman Sachs 46th Annual Global Healthcare Conference. CEO Emil Kakkis, M.D., Ph.D., will engage in a fireside chat on Tuesday, June 10, 2025, at 10:00 a.m. ET. Investors and interested parties can access both the live and archived webcast of the presentation through the company's investor relations website at ir.ultragenyx.com/events-presentations.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of 44,805 restricted stock units (RSUs) to 20 newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan, with a grant date of May 16, 2025. The RSUs were granted as employment inducements in compliance with Nasdaq Listing Rule 5635(c)(4). The vesting schedule spans four years, with 25% of shares vesting annually on the grant date anniversary, contingent on continuous employment.

Ultragenyx Pharmaceutical (NASDAQ: RARE) has released its 2024 Impact Report, highlighting the company's commitment to rare disease innovation and corporate responsibility. The report is structured around six key pillars: Innovation, Patients, People, Communities, Planet, and Governance. Key achievements include: successful transfer of DTX401 gene therapy manufacturing to their Bedford facility, supporting over 650 patients in 50 countries through access programs, maintaining 88% employee engagement, and approving $3M+ in charitable donations. The company advanced several clinical programs for rare diseases including osteogenesis imperfecta and Angelman syndrome. Ultragenyx received multiple recognitions in 2024, including being named among Barron's 100 Most Sustainable Companies and Top Places to Work in the USA. The company demonstrated environmental commitment by purchasing 100% renewable electricity for its headquarters and achieving My Green Lab Green certification.