Ultragenyx Pharmaceutical Inc. Faces Setback as Phase III Studies Fail to Meet Primary Endpoint
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1h ago
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Source: PRnewswire
- Study Results Fail: Ultragenyx's Phase III Orbit and Cosmic studies, announced on December 29, 2025, failed to meet their primary endpoint of reducing clinical fracture rates compared to placebo, casting doubt on the efficacy of its drug setrusumab and potentially impacting future drug approvals.
- Bone Density Improvement Ineffective: Although the studies showed improvements in patients' bone density, they failed to establish a causal link to reduced fracture rates, undermining the credibility of Ultragenyx's previous reliance on Phase II results and likely leading to decreased investor confidence.
- Analyst Expectations Turn Cautious: Following the study results, analysts quickly slashed Ultragenyx's price targets, with Barclays noting limited opportunities for drug approval despite some trends of clinical benefit, reflecting a pessimistic sentiment towards the company's future.
- Legal Investigation Initiated: Levi & Korsinsky LLP is investigating Ultragenyx's business operations, indicating investor concerns regarding the company's transparency and governance, which may lead to further legal and financial risks.
Analyst Views on RARE
Wall Street analysts forecast RARE stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for RARE is 61.65 USD with a low forecast of 35.00 USD and a high forecast of 120.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
18 Analyst Rating
17 Buy
1 Hold
0 Sell
Strong Buy
Current: 23.400
Low
35.00
Averages
61.65
High
120.00
Current: 23.400
Low
35.00
Averages
61.65
High
120.00
About RARE
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. Its four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, UX701, UX143, UX111, and GTX-102. UX143 for the treatment of Osteogenesis Imperfecta.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
