Goldman Sachs Downgrades Ultragenyx to Neutral
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1 hour ago
0mins
Should l Buy RARE?
Source: seekingalpha
- Rating Downgrade: Goldman Sachs has downgraded Ultragenyx Pharmaceutical (RARE) from buy to neutral due to disappointing phase 3 trial results for seralutinib in treating osteogenesis imperfecta, which failed to meet the primary endpoint of statistical significance.
- Price Target: The bank has set a price target of $25, reflecting a 23% upside based on the March 23 close, indicating a cautious outlook on Ultragenyx's future growth potential amid trial setbacks.
- Shift in Key Drivers: Analyst Salveen Richter noted that while the secondary endpoint of bone mineral density was statistically significant, the limited regulatory and commercial path for setrusumab has shifted focus to the Angelman syndrome asset GTX-102 as the new key value driver.
- Profitability Outlook: Achieving GAAP profitability by 2027 is deemed possible, contingent upon the approvals and launches of gene therapies DTX401 and UX111 in the second half, which could unlock priority review vouchers valued at approximately $150 million each in their model.
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Analyst Views on RARE
Wall Street analysts forecast RARE stock price to rise
18 Analyst Rating
17 Buy
1 Hold
0 Sell
Strong Buy
Current: 20.270
Low
35.00
Averages
61.65
High
120.00
Current: 20.270
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.
- Legal Investigation: Faruq & Faruq LLP is investigating potential claims against Ultragenyx Pharmaceutical for investors who purchased securities between August 3, 2023, and December 26, 2025, urging them to apply for lead plaintiff status in the federal securities class action by the April 6, 2026 deadline.
- False Statements Allegations: The lawsuit alleges that Ultragenyx and its executives violated federal securities laws by misleading investors about the reliability of information regarding setrusumab's effects on Osteogenesis Imperfecta patients, failing to disclose that the Phase III Orbit study did not achieve statistical significance, thereby misleading the investing public.
- Stock Price Plunge: Following the July 9, 2025 announcement that the Phase III Orbit study failed to achieve statistical significance, Ultragenyx's stock price fell over 25%; on December 29, 2025, the stock plummeted more than 42% after the company revealed that both the Phase III Orbit and Cosmic studies did not meet primary endpoint statistical significance, indicating severe investor confidence erosion.
- Investor Rights Protection: Faruq & Faruq LLP encourages anyone with information regarding Ultragenyx's conduct, including whistleblowers and former employees, to contact the firm to ensure investor rights are protected and to support potential class action litigation.
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- Rating Downgrade: Goldman Sachs has downgraded Ultragenyx Pharmaceutical (RARE) from buy to neutral due to disappointing phase 3 trial results for seralutinib in treating osteogenesis imperfecta, which failed to meet the primary endpoint of statistical significance.
- Price Target: The bank has set a price target of $25, reflecting a 23% upside based on the March 23 close, indicating a cautious outlook on Ultragenyx's future growth potential amid trial setbacks.
- Shift in Key Drivers: Analyst Salveen Richter noted that while the secondary endpoint of bone mineral density was statistically significant, the limited regulatory and commercial path for setrusumab has shifted focus to the Angelman syndrome asset GTX-102 as the new key value driver.
- Profitability Outlook: Achieving GAAP profitability by 2027 is deemed possible, contingent upon the approvals and launches of gene therapies DTX401 and UX111 in the second half, which could unlock priority review vouchers valued at approximately $150 million each in their model.
See More
- Class Action Initiation: Robbins Geller law firm announces that purchasers of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) common stock between August 3, 2023, and December 26, 2025, can seek lead plaintiff status in a class action lawsuit, indicating significant investor distrust in the company's management.
- False Statement Allegations: The lawsuit alleges that Ultragenyx and its executives made false and misleading statements throughout the class period, failing to disclose risks associated with the Phase III Orbit study, which misled investors about the company's prospects and negatively impacted stock prices.
- Stock Price Plunge: Following the July 9, 2025 announcement that the Phase III Orbit study failed to achieve statistical significance, Ultragenyx's stock price fell over 25%; the subsequent announcement on December 29, 2025, of further study failures led to an additional drop of over 42%, reflecting extreme market pessimism regarding the company's future.
- Investor Rights Protection: Under the Private Securities Litigation Reform Act of 1995, any investor who purchased Ultragenyx stock during the class period can seek lead plaintiff status, emphasizing the importance of protecting investor rights and ensuring accountability from corporate management.
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- Class Action Notification: The Schall Law Firm reminds investors of a class action lawsuit against Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) for violations of securities laws during the period from August 3, 2023, to December 26, 2025, with a deadline to contact the firm by April 6, 2026.
- False Statements Exposed: The complaint alleges that Ultragenyx made false and misleading statements regarding its drug candidate's effects on Osteogenesis Imperfecta patients, leading to significant investor losses once the truth was revealed.
- Clinical Trial Failure: The Phase III ORBIT study failed to achieve a statistically significant reduction in annualized fracture rate, indicating that the company's public statements were false and materially misleading throughout the class period, exacerbating investor losses.
- Legal Consultation Opportunity: The Schall Law Firm offers legal consultation for affected shareholders, encouraging them to seek compensation through the class action lawsuit, while noting that investors are not represented until the class is certified.
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- Lawsuit Background: Bronstein, Gewirtz & Grossman, LLC, a nationally recognized investor-rights law firm, has filed a class action lawsuit against Ultragenyx Pharmaceutical Inc. and certain officers, seeking damages for investors who purchased securities between August 3, 2023, and December 26, 2025.
- Allegations of Misrepresentation: The complaint alleges that defendants made false and/or misleading statements and failed to disclose risks associated with the Phase III Orbit study of setrusumab for Osteogenesis Imperfecta, misleading investors about the reliability of the study's results.
- Investor Actions: Affected investors are encouraged to apply to be lead plaintiffs by April 6, 2026, allowing them to share in any recovery without needing to serve as lead plaintiffs, thus broadening participation in the lawsuit.
- Law Firm's Credentials: Bronstein, Gewirtz & Grossman, LLC has a strong reputation in securities fraud class actions, having recovered hundreds of millions for investors, underscoring their commitment to restoring investor capital and ensuring corporate accountability.
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- Investigation Launched: Faruq & Faruqi, LLP is investigating potential claims against Ultragenyx Pharmaceutical Inc, particularly for investors who purchased or acquired securities between August 3, 2023, and December 26, 2025, aiming to protect investor rights.
- Legal Consultation Opportunity: Investors who suffered losses during the specified period can contact Faruq & Faruqi partner Josh Wilson directly at 877-247-4292 or 212-983-9330 (Ext. 1310) to discuss their legal options and ensure timely action.
- Class Action Deadline: The firm reminds investors that the deadline to seek the role of lead plaintiff in a federal securities class action against Ultragenyx is set for April 6, 2026, necessitating prompt action from investors to safeguard their rights.
- Investor Protection Emphasis: As a leading national securities law firm, Faruq & Faruqi emphasizes the importance of investor rights, aiming to provide necessary support and legal protection for affected investors through investigations and legal actions.
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