Ultragenyx Pharm Stock Price, News & Analysis

Ultragenyx Pharmaceutical (NASDAQ: RARE) reported strong financial results for Q4 and full year 2024, with total revenue of $560 million, representing 29% growth compared to 2023. Key revenue drivers included Crysvita at $410 million (+25% YoY) and Dojolvi at $88 million (+25% YoY).

The company provided 2025 guidance projecting total revenue between $640-670 million, with Crysvita revenue of $460-480 million and Dojolvi revenue of $90-100 million. Despite revenue growth, Ultragenyx reported a net loss of $569 million ($6.29 per share) for 2024, though improved from $607 million loss in 2023.

The company ended 2024 with $745 million in cash and equivalents, with net cash used in operations of $414 million for the year. Multiple clinical milestones are expected in 2025, including a PDUFA decision for Sanfilippo syndrome gene therapy and Phase 3 results in osteogenesis imperfecta.

Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in novel products for rare and ultrarare genetic diseases, has announced a conference call scheduled for February 13, 2025, at 5:00 p.m. ET. The call will discuss the company's financial results and provide a corporate update for both the fourth quarter and full year ending December 31, 2024. Investors and interested parties can access the live webcast and replay through the company's investor relations website. The replay will remain available for three months following the call.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced significant clinical improvements in children treated with UX111 AAV gene therapy for Sanfilippo Syndrome Type A. The modified intent-to-treat group showed a +22.7 point treatment effect in Bayley-III cognitive raw scores compared to untreated patients, with a p-value <0.0001.

The treatment demonstrated substantial reductions in heparan sulfate levels in cerebrospinal fluid, with a median reduction of 65% across all patients (N=27) and 66% in the modified intention to treat group (N=17). The mean follow-up duration was 34 months for all patients, with the longest being 77 months.

In older patients with advanced disease, 9 out of 10 children retained abilities in ambulation and self-feeding, while all 10 maintained communication skills. The therapy was generally well-tolerated, with mostly mild to moderate liver enzyme elevations that resolved. A BLA submission was filed with the FDA in December 2024, with a PDUFA decision expected in H2 2025.

Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare disease therapies, has announced the grant of 7,750 restricted stock units to eight newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan on January 16, 2025. The grants serve as employment inducements in compliance with Nasdaq Listing Rule 5635(c)(4). The restricted stock units feature a four-year vesting schedule, with 25% of shares vesting annually on the grant date anniversary, contingent on continuous employment.

Ultragenyx Pharmaceutical (NASDAQ: RARE) reported preliminary 2024 revenue of $555-560 million, exceeding guidance, with Crysvita revenue of $405-410 million and Dojolvi revenue of $87-89 million. The company projects 2025 total revenue of $640-670 million.

Key pipeline updates include: UX143 Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025; GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025; BLA submission for UX111 gene therapy for Sanfilippo syndrome with PDUFA decision expected in second half of 2025; and DTX401 BLA filing for Glycogen Storage Disease Type Ia planned for mid-2025.

The company ended 2024 with approximately $745 million in cash and investments, expecting 14-20% revenue growth in 2025 with decreased net cash usage in operations.

Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference. The company's CEO and president, Emil D. Kakkis, M.D., Ph.D., will deliver a presentation on Monday, January 13, 2025, at 10:30 AM PT.

The presentation will be accessible via live webcast and can be viewed through the company's investor relations website at https://ir.ultragenyx.com/events-presentations. A replay option will remain available for 30 days following the event. Ultragenyx specializes in developing and commercializing novel therapies for serious rare and ultrarare genetic diseases.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that the European Commission has extended the approval of Evkeeza® (evinacumab) to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first medicine in the EU approved for children as young as 6-months old with this ultrarare inherited condition that causes dangerously high cholesterol levels.

The approval is based on model-based extrapolation analysis predicting similar or higher LDL-C reduction in young patients compared to adults when receiving a 15 mg/kg dose every 4 weeks. Supportive data from five patients aged 1-4 years showed clinically meaningful LDL-C reduction consistent with older patients. The safety profile is expected to be similar to that in older pediatric patients.

Evkeeza is currently reimbursed and commercially available in eight countries including the UK, U.S., Canada, and Japan, with early access schemes in 13 additional countries.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of 15,175 restricted stock units to 15 newly hired non-executive officers. The awards were approved by the company's compensation committee and granted under the Ultragenyx Employment Inducement Plan on December 16, 2024. The stock units will vest over four years, with 25% vesting annually on each grant date anniversary, contingent on continuous employment. This grant was made in accordance with Nasdaq Listing Rule 5635(c)(4) as an employment inducement.

Ultragenyx Pharmaceutical (NASDAQ: RARE) has initiated patient dosing in its pivotal Phase 3 Aspire study evaluating GTX-102 for Angelman syndrome. The global study will enroll approximately 120 children aged 4-17 with genetically confirmed maternal UBE3A gene deletion. Participants will be randomized 1:1 to receive GTX-102 or sham treatment over 48 weeks.

The treatment group will receive three monthly 8mg loading doses, followed by maintenance doses up to 14mg quarterly. The study's primary endpoint focuses on cognitive improvement measured by Bayley-4 cognitive raw score, with a key secondary endpoint using the Multi-domain Responder Index across cognition, communication, behavior, motor function, and sleep. The company plans to initiate the Aurora study in 2025 to evaluate GTX-102 in other Angelman syndrome genotypes and age groups.