| Valuation method | Value, $ | Upside, % |
|---|---|---|
| Artificial intelligence (AI) | 25.15 | 4 |
| Intrinsic value (DCF) | 15.45 | -36 |
| Graham-Dodd Method | n/a | |
| Graham Formula | n/a |
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is a pioneering biopharmaceutical company specializing in rare and ultra-rare genetic diseases. Headquartered in Novato, California, Ultragenyx focuses on identifying, developing, and commercializing novel therapies for underserved patient populations. The company’s product portfolio includes Crysvita (burosumab) for X-linked hypophosphatemia, Mepsevii for Mucopolysaccharidosis VII, Dojolvi for long-chain fatty acid oxidation disorders, and Evkeeza (evinacumab) for homozygous familial hypercholesterolemia. Additionally, Ultragenyx has a robust pipeline of gene therapies and monoclonal antibodies targeting conditions like glycogen storage disease, osteogenesis imperfecta, and Angelman syndrome. With strategic collaborations with Kyowa Kirin, REGENXBIO, and Bayer, Ultragenyx leverages cutting-edge science to address unmet medical needs in the rare disease space. Operating in North America, Europe, and internationally, the company is a key player in the $200B+ global rare disease market, positioning itself for long-term growth through innovation and strategic partnerships.
Ultragenyx presents a high-risk, high-reward investment opportunity due to its focus on rare diseases, which often command premium pricing and limited competition. The company’s revenue growth is supported by its commercial products, but profitability remains elusive, with a net loss of $569M in the latest fiscal year. Its strong cash position ($174M) and manageable debt ($40M) provide financial flexibility, but reliance on pipeline success and regulatory approvals introduces volatility. The stock’s low beta (0.34) suggests relative stability, but clinical trial outcomes and reimbursement challenges in ex-U.S. markets remain key risks. Investors bullish on gene therapy and rare diseases may find Ultragenyx compelling, but the lack of profitability and cash burn (-$414M operating cash flow) warrant caution.
Ultragenyx competes in the niche but rapidly growing rare disease therapeutics market, where differentiation hinges on scientific innovation and commercialization capabilities. Its competitive advantage lies in its focused pipeline targeting ultra-rare conditions with high unmet need, reducing direct competition. Crysvita, developed with Kyowa Kirin, dominates the XLH market, while Evkeeza (licensed from Regeneron) is the only FDA-approved treatment for homozygous familial hypercholesterolemia. The company’s gene therapy candidates (e.g., DTX401, DTX301) leverage AAV8 technology, competing with BioMarin and Pfizer in gene therapy. However, Ultragenyx faces scalability challenges due to the small patient populations it serves, requiring high pricing and efficient global commercialization. Its partnerships with REGENXBIO (for AAV vectors) and Arcturus (for mRNA) enhance R&D capabilities but expose it to collaboration risks. Competitors like BioMarin and Alexion (now part of AstraZeneca) have deeper commercial infrastructures, while smaller biotechs like Sarepta threaten in gene therapy. Ultragenyx’s ability to secure orphan drug exclusivity and expand label indications will be critical to maintaining its edge.