Ultragenyx Pharmaceutical's 2025 Conference Presentation: A Rare Disease Company's Path to Full Year GAAP Profitability

Ultragenyx Pharmaceutical Inc. (RARE) is a rare disease company focused on transformative treatments. The company is on a pathway to full year GAAP profitability in 2027 and has 4 current commercial programs with significant and growing revenue. Ultragenyx has 3 near-term launches, including 2 gene therapies and an OI program, as well as the Angelman program. The company is expected to reach full year profitability in 2027.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a leading rare disease company, is poised to achieve full-year GAAP profitability by 2027. The company, which focuses on transformative treatments, has 4 current commercial programs with significant and growing revenue. These programs include 2 gene therapies and an OI program, as well as the Angelman program.

Ultragenyx is expected to reach full-year profitability in 2027, with revenue projected to reach $640 million to $670 million this year. The company has 3 near-term launches planned, including the 401 and 111 gene therapies and the OI program (143). Additionally, the Angelman program is in Phase III, with data expected in the back half of next year.

The company’s Chief Medical Officer, Dr. Eric Crombez, and Executive VP of Corporate Strategy and CFO, Howard Horn, recently participated in a conference call hosted by Cantor Fitzgerald & Co. They discussed the company's progress and upcoming developments. Horn noted that the company is on a pathway to profitability in 2027, with several exciting programs in the pipeline.

The OI program, which includes more severe patients (types 3s and 4s), is of particular interest. Crombez explained that setrusumab, the antibody used in the program, works by increasing bone formation. The Phase II data exceeded expectations, showing significant bone mineral density increases and reductions in annualized fracture rates for more severe patients. This positive outcome led to modifications in the Phase III trial design.

The company is also working with the FDA to resubmit the BLA for the 111 program and conclude the submission for the 401 BLA. Additionally, the Angelman Phase III trial is fully enrolled, with data expected in the back half of next year.

The FDA has recently announced the Rare Disease Evidence Principles (RDEP) program, which aims to expedite the development and review of rare disease medicines. This program allows for the acceptance of evidence from single-arm clinical trials and other supportive evidence sources, potentially speeding up the approval process for rare disease treatments.

While the RDEP program is promising, it is unclear what tangible impact it will have on the development process and approval timeline. Companies like Neurogene, Rocket Pharmaceuticals, Ultragenyx, and UniQure are already planning to use single-arm studies to support their approval applications for gene therapies.

Overall, Ultragenyx Pharmaceutical Inc. (RARE) is well-positioned to achieve its financial goals, with a strong pipeline of transformative treatments and a focus on rare diseases.

References:
[1] https://www.biopharmadive.com/news/fda-rare-disease-evidence-principles-drug-reviews/759237/
[2] https://seekingalpha.com/article/4819641-ultragenyx-pharmaceutical-inc-rare-presents-at-cantor-global-healthcare-conference-2025