Zinger Key Points
- Ultragenyx's Q1 2025 revenue rose 28% to $139.29 million; full-year guidance was set at $640 million–$670 million.
- Setrusumab is projected to generate $2.4 billion in peak sales by 2037, with $1.4 billion to Ultragenyx.
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On Wednesday, William Blair initiated coverage on Ultragenyx Pharmaceutical Inc. RARE, a biopharmaceutical company focused on developing and commercializing therapies for rare and ultra-rare genetic diseases.
Since 2017, the company has commercialized four products, which generated $560 million in revenue in 2024, up 29% year-over-year. In the first quarter of 2025, sales increased 28% to $139.29 million.
Ultragenyx expects fiscal 2025 sales of $640 million-$670 million versus consensus of $655.02 million.
“We anticipate Ultragenyx’s legacy products will continue to provide a strong revenue foundation for the company through the early 2030s,” writes William Blair analyst Sami Corwin.
Also Read: Ultragenyx Stays On JPMorgan’s Focus List As Analyst Highlights Strong Long-Term Positioning
William Blair initiated coverage with an Outperform rating, writing that the company is at an inflection point. Potentially, six additional therapeutics will be approved by the end of 2028, including three approvals in the next 18 months.
Corwin notes that these additional assets synergize with Ultragenyx’s existing commercial portfolio, aiding in strong commercial launches. She projects the company’s expanding revenue stream, supplemented by the potential receipt of three priority review vouchers, could enable the company to achieve GAAP profitability in 2027 and beyond.
William Blair noted that regulatory decisions and clinical data could be catalysts for the stock in the near term. Specifically, positive results from the Phase 3 trial of setrusumab in osteogenesis imperfecta (OI) could lead to significant upside.
Corwin projects the products could begin contributing to the top line as early as the fourth quarter of 2025 and could lead to a major revenue inflection in 2026 with the potential approval of pertuzumab in OI.
William Blair views setrusumab as a blockbuster product and estimates peak revenue of $2.4 billion in 2037, $1.4 billion of which will go to Ultragenyx.
Price Action: RARE stock is down 1.65% at $34.50 at the last check Wednesday.
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