Ultragenyx Pharmaceutical Accused of Misleading Investors
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1h ago
0mins
Should l Buy RARE?
Source: Businesswire
- Class Action Filed: Robbins LLP has initiated a class action against Ultragenyx Pharmaceutical (NASDAQ: RARE) on behalf of all investors who purchased shares between August 3, 2023, and December 26, 2025, indicating strong investor concern over potential misleading actions by the company.
- Study Results Failure: The complaint alleges that Ultragenyx misrepresented the efficacy of setrusumab in its Phase III Orbit and Cosmic studies, claiming it would significantly reduce annualized fracture rates in Osteogenesis Imperfecta patients, but ultimately failed to achieve statistical significance, leading to a sharp decline in investor confidence.
- Stock Price Plunge: Following the announcement of study failures on December 29, 2025, Ultragenyx's stock price plummeted from $34.19 per share on December 26 to $19.72 on December 29, marking a staggering 42.32% drop in just one day, reflecting the market's pessimistic outlook on the company's future.
- Investor Rights Protection: Robbins LLP encourages investors interested in serving as lead plaintiffs in the class action to reach out, demonstrating the firm's commitment to protecting shareholder rights and holding companies accountable for their actions.
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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: 24.800
Low
35.00
Averages
61.65
High
120.00
Current: 24.800
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.
Ultragenyx Reports Long-Term Data on UX111 for Sanfilippo Syndrome
- Clinical Trial Results: Ultragenyx announced long-term data for UX111, showing a significant 23.2-point improvement (p<0.0001) in Bayley-III cognitive raw scores for 17 patients under two years old compared to natural history data during 24-60 months, indicating substantial efficacy of the gene therapy in enhancing cognitive function.
- Functional Improvements: In addition to cognitive gains, caregiver-reported outcomes using Vineland 3 demonstrated comparable improvements in communication, motor, and personal subdomains, further validating the clinical benefits of UX111.
- Biomarker Enhancements: As of the September 2025 cutoff, the median reduction in cerebrospinal fluid heparan sulfate (CSF-HS) was 63.98% (p<0.001), with 88.2% of treated children achieving a 50% or greater reduction, underscoring the treatment's effectiveness.
- FDA Application Progress: These long-term data have been included in the resubmitted Biologics License Application (BLA), with the company anticipating an accelerated review by the FDA, expected to conclude in the third quarter of 2026, which could significantly enhance UX111's market prospects.
See More
Ultragenyx Pharmaceutical Accused of Misleading Investors
- Class Action Filed: Robbins LLP has initiated a class action against Ultragenyx Pharmaceutical (NASDAQ: RARE) on behalf of all investors who purchased shares between August 3, 2023, and December 26, 2025, indicating strong investor concern over potential misleading actions by the company.
- Study Results Failure: The complaint alleges that Ultragenyx misrepresented the efficacy of setrusumab in its Phase III Orbit and Cosmic studies, claiming it would significantly reduce annualized fracture rates in Osteogenesis Imperfecta patients, but ultimately failed to achieve statistical significance, leading to a sharp decline in investor confidence.
- Stock Price Plunge: Following the announcement of study failures on December 29, 2025, Ultragenyx's stock price plummeted from $34.19 per share on December 26 to $19.72 on December 29, marking a staggering 42.32% drop in just one day, reflecting the market's pessimistic outlook on the company's future.
- Investor Rights Protection: Robbins LLP encourages investors interested in serving as lead plaintiffs in the class action to reach out, demonstrating the firm's commitment to protecting shareholder rights and holding companies accountable for their actions.
See More
Ultragenyx Faces Class Action Lawsuit Allegations
- Lawsuit Background: Robbins Geller Rudman & Dowd LLP announces a class action lawsuit against Ultragenyx Pharmaceutical Inc., representing purchasers of its common stock from August 3, 2023, to December 26, 2025, alleging violations of the Securities Exchange Act of 1934 by its executives.
- False Statement Allegations: The lawsuit claims that Ultragenyx misled investors regarding the statistical significance of fracture rates in its Phase III Orbit study, resulting in stock price declines of over 25% and 42% in July and December 2025, respectively, as the market reacted to the news.
- Impact of Study Failures: On December 29, 2025, Ultragenyx revealed that both its Orbit and Cosmic studies failed to achieve statistical significance against primary endpoints, raising further doubts about its product development capabilities and potentially affecting future financing and investor confidence.
- Investor Rights Protection: Under the Private Securities Litigation Reform Act of 1995, any investor who purchased Ultragenyx stock during the class period can seek to be appointed as lead plaintiff, representing other shareholders in pursuit of potential recovery, highlighting the importance of protecting investor rights.
See More
Ultragenyx Faces Class Action Lawsuit Impacting Stock Price
- Class Action Initiated: Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit for allegedly providing misleading information to investors regarding its stock transactions between August 3, 2023, and December 26, 2025, which could lead to significant legal liabilities for the company.
- Stock Price Plummets: Following the announcement on December 29, 2025, that its Phase III Orbit and Cosmic studies failed to meet primary endpoints, Ultragenyx's stock price dropped dramatically from $34.19 to $19.72, a staggering 42.32% decline in a single day, reflecting a pessimistic market sentiment towards the company's future.
- Study Results Disappoint: The company's studies failed to demonstrate that setrusumab effectively reduces fracture rates despite increasing bone density, contrasting sharply with management's previous optimistic claims, which may undermine future investor confidence.
- Investor Advocacy Call: Levi & Korsinsky LLP is urging RARE shareholders to step forward before the April 6, 2026 deadline to seek lead plaintiff status, emphasizing the importance of investor rights and the challenges to corporate governance.
See More
Ultragenyx Under Investigation After 87.64% Stock Drop Following Phase 3 Study Results
- Stock Price Plunge: Ultragenyx's stock plummeted by $2.02, or 87.64%, to close at $0.28 per share on December 29, 2025, following the announcement of two Phase 3 study results that failed to meet primary endpoints, severely impacting investor confidence.
- Study Results Analysis: While both studies achieved strong statistical significance in improving bone mineral density, they did not demonstrate statistical significance in reducing clinical fracture rates compared to placebo or bisphosphonates, raising concerns about the company's future product viability.
- Legal Investigation Initiated: Pomerantz LLP is investigating whether Ultragenyx and its executives engaged in securities fraud or other unlawful business practices, which could lead to further legal liabilities and financial repercussions.
- Company Background: Pomerantz LLP, a premier firm in securities class litigation with over 85 years of experience, emphasizes its commitment to fighting for victims of securities fraud, indicating that this investigation could have long-term implications for Ultragenyx's reputation and operations.
See More
Ultragenyx Reports Significant Improvements in Gene Therapy for Sanfilippo Syndrome
- Efficacy Data Update: Ultragenyx's new data for the UX111 gene therapy indicates a significant 23.2-point improvement in the Bayley-III cognitive raw score for children under two years old during a 24-60 month follow-up, suggesting promising outcomes for early intervention in Sanfilippo syndrome Type A.
- Multiple Improvement Metrics: In addition to cognitive score enhancements, improvements were also observed in receptive communication, expressive communication, fine motor, and gross motor skills, indicating UX111's functional benefits across various domains, potentially enhancing patients' quality of life.
- Long-Term Follow-Up Data: The study's data includes up to 8.5 years of follow-up, providing a comprehensive understanding of UX111's efficacy, which may bolster its clinical application potential and future market acceptance.
- Regulatory Progress: Ultragenyx received an FDA Complete Response Letter for UX111 in July 2025 and subsequently resubmitted its Biologics License Application (BLA) in late January, demonstrating the company's ongoing commitment to advancing UX111 towards U.S. approval.
See More
Ultragenyx Reports Long-Term Data on UX111 for Sanfilippo Syndrome
- Clinical Trial Results: Ultragenyx announced long-term data for UX111, showing a significant 23.2-point improvement (p<0.0001) in Bayley-III cognitive raw scores for 17 patients under two years old compared to natural history data during 24-60 months, indicating substantial efficacy of the gene therapy in enhancing cognitive function.
- Functional Improvements: In addition to cognitive gains, caregiver-reported outcomes using Vineland 3 demonstrated comparable improvements in communication, motor, and personal subdomains, further validating the clinical benefits of UX111.
- Biomarker Enhancements: As of the September 2025 cutoff, the median reduction in cerebrospinal fluid heparan sulfate (CSF-HS) was 63.98% (p<0.001), with 88.2% of treated children achieving a 50% or greater reduction, underscoring the treatment's effectiveness.
- FDA Application Progress: These long-term data have been included in the resubmitted Biologics License Application (BLA), with the company anticipating an accelerated review by the FDA, expected to conclude in the third quarter of 2026, which could significantly enhance UX111's market prospects.
See More
Ultragenyx Pharmaceutical Accused of Misleading Investors
- Class Action Filed: Robbins LLP has initiated a class action against Ultragenyx Pharmaceutical (NASDAQ: RARE) on behalf of all investors who purchased shares between August 3, 2023, and December 26, 2025, indicating strong investor concern over potential misleading actions by the company.
- Study Results Failure: The complaint alleges that Ultragenyx misrepresented the efficacy of setrusumab in its Phase III Orbit and Cosmic studies, claiming it would significantly reduce annualized fracture rates in Osteogenesis Imperfecta patients, but ultimately failed to achieve statistical significance, leading to a sharp decline in investor confidence.
- Stock Price Plunge: Following the announcement of study failures on December 29, 2025, Ultragenyx's stock price plummeted from $34.19 per share on December 26 to $19.72 on December 29, marking a staggering 42.32% drop in just one day, reflecting the market's pessimistic outlook on the company's future.
- Investor Rights Protection: Robbins LLP encourages investors interested in serving as lead plaintiffs in the class action to reach out, demonstrating the firm's commitment to protecting shareholder rights and holding companies accountable for their actions.
See More
Ultragenyx Faces Class Action Lawsuit Allegations
- Lawsuit Background: Robbins Geller Rudman & Dowd LLP announces a class action lawsuit against Ultragenyx Pharmaceutical Inc., representing purchasers of its common stock from August 3, 2023, to December 26, 2025, alleging violations of the Securities Exchange Act of 1934 by its executives.
- False Statement Allegations: The lawsuit claims that Ultragenyx misled investors regarding the statistical significance of fracture rates in its Phase III Orbit study, resulting in stock price declines of over 25% and 42% in July and December 2025, respectively, as the market reacted to the news.
- Impact of Study Failures: On December 29, 2025, Ultragenyx revealed that both its Orbit and Cosmic studies failed to achieve statistical significance against primary endpoints, raising further doubts about its product development capabilities and potentially affecting future financing and investor confidence.
- Investor Rights Protection: Under the Private Securities Litigation Reform Act of 1995, any investor who purchased Ultragenyx stock during the class period can seek to be appointed as lead plaintiff, representing other shareholders in pursuit of potential recovery, highlighting the importance of protecting investor rights.
See More
Ultragenyx Faces Class Action Lawsuit Impacting Stock Price
- Class Action Initiated: Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit for allegedly providing misleading information to investors regarding its stock transactions between August 3, 2023, and December 26, 2025, which could lead to significant legal liabilities for the company.
- Stock Price Plummets: Following the announcement on December 29, 2025, that its Phase III Orbit and Cosmic studies failed to meet primary endpoints, Ultragenyx's stock price dropped dramatically from $34.19 to $19.72, a staggering 42.32% decline in a single day, reflecting a pessimistic market sentiment towards the company's future.
- Study Results Disappoint: The company's studies failed to demonstrate that setrusumab effectively reduces fracture rates despite increasing bone density, contrasting sharply with management's previous optimistic claims, which may undermine future investor confidence.
- Investor Advocacy Call: Levi & Korsinsky LLP is urging RARE shareholders to step forward before the April 6, 2026 deadline to seek lead plaintiff status, emphasizing the importance of investor rights and the challenges to corporate governance.
See More
Ultragenyx Under Investigation After 87.64% Stock Drop Following Phase 3 Study Results
- Stock Price Plunge: Ultragenyx's stock plummeted by $2.02, or 87.64%, to close at $0.28 per share on December 29, 2025, following the announcement of two Phase 3 study results that failed to meet primary endpoints, severely impacting investor confidence.
- Study Results Analysis: While both studies achieved strong statistical significance in improving bone mineral density, they did not demonstrate statistical significance in reducing clinical fracture rates compared to placebo or bisphosphonates, raising concerns about the company's future product viability.
- Legal Investigation Initiated: Pomerantz LLP is investigating whether Ultragenyx and its executives engaged in securities fraud or other unlawful business practices, which could lead to further legal liabilities and financial repercussions.
- Company Background: Pomerantz LLP, a premier firm in securities class litigation with over 85 years of experience, emphasizes its commitment to fighting for victims of securities fraud, indicating that this investigation could have long-term implications for Ultragenyx's reputation and operations.
See More
Ultragenyx Reports Significant Improvements in Gene Therapy for Sanfilippo Syndrome
- Efficacy Data Update: Ultragenyx's new data for the UX111 gene therapy indicates a significant 23.2-point improvement in the Bayley-III cognitive raw score for children under two years old during a 24-60 month follow-up, suggesting promising outcomes for early intervention in Sanfilippo syndrome Type A.
- Multiple Improvement Metrics: In addition to cognitive score enhancements, improvements were also observed in receptive communication, expressive communication, fine motor, and gross motor skills, indicating UX111's functional benefits across various domains, potentially enhancing patients' quality of life.
- Long-Term Follow-Up Data: The study's data includes up to 8.5 years of follow-up, providing a comprehensive understanding of UX111's efficacy, which may bolster its clinical application potential and future market acceptance.
- Regulatory Progress: Ultragenyx received an FDA Complete Response Letter for UX111 in July 2025 and subsequently resubmitted its Biologics License Application (BLA) in late January, demonstrating the company's ongoing commitment to advancing UX111 towards U.S. approval.
See More
