Ultragenyx Under Investigation After 87.64% Stock Drop Following Phase 3 Study Results
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1h ago
0mins
Should l Buy RARE?
Source: Globenewswire
- 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.
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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: 24.760
Low
35.00
Averages
61.65
High
120.00
Current: 24.760
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 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 Gene Therapy FDA Review Period Extended
- FDA Review Expectation: Ultragenyx anticipates a 6-month review period for its UX111 gene therapy, with a potential FDA decision expected by Q3 2026, which could significantly impact the company's future market strategy and cash flow.
- Resubmission Details: The company has resubmitted its application for UX111, including substantial long-term data on neurologic benefits and responses to FDA observations from July, demonstrating its ongoing commitment to regulatory compliance and drug development.
- Market Reaction: Following the announcement of the resubmission, Ultragenyx shares fell by 3%, reflecting investor concerns over FDA review uncertainties and the company's future profitability, with a 46% decline in stock price over the past 12 months.
- Disease Context: Sanfilippo syndrome type A is a severe genetic disorder causing global developmental delays and early death in children; if approved, UX111 would be the first therapy for this condition, representing significant market potential and societal value.
See More
Ultragenyx Pharmaceutical Inc. Faces Setback as Phase III Studies Fail to Meet Primary Endpoint
- 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.
See More
Ultragenyx Pharmaceutical's Phase III Study Fails to Meet Primary Endpoint, Analysts Cut Price Targets
- Disappointing Study Results: Ultragenyx's Phase III Orbit and Cosmic studies, announced on December 29, 2025, failed to meet their primary endpoints, leading to diminished investor confidence and analysts rapidly cutting price targets.
- Bone Density Improvement Not Translated: Although the studies showed improvements in patients' bone density, they failed to establish a causal link to reduced annualized fracture rates, increasing regulatory and market pressures on the company.
- Analyst Sentiment Shift: Barclays analysts noted limited opportunities for drug approval despite some trends of clinical benefit, reflecting a pessimistic outlook on Ultragenyx's future prospects.
- Increased Investor Scrutiny: Levi & Korsinsky LLP is investigating Ultragenyx's business operations, indicating heightened investor concern over the company's future and potential legal risks.
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 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 Gene Therapy FDA Review Period Extended
- FDA Review Expectation: Ultragenyx anticipates a 6-month review period for its UX111 gene therapy, with a potential FDA decision expected by Q3 2026, which could significantly impact the company's future market strategy and cash flow.
- Resubmission Details: The company has resubmitted its application for UX111, including substantial long-term data on neurologic benefits and responses to FDA observations from July, demonstrating its ongoing commitment to regulatory compliance and drug development.
- Market Reaction: Following the announcement of the resubmission, Ultragenyx shares fell by 3%, reflecting investor concerns over FDA review uncertainties and the company's future profitability, with a 46% decline in stock price over the past 12 months.
- Disease Context: Sanfilippo syndrome type A is a severe genetic disorder causing global developmental delays and early death in children; if approved, UX111 would be the first therapy for this condition, representing significant market potential and societal value.
See More
Ultragenyx Pharmaceutical Inc. Faces Setback as Phase III Studies Fail to Meet Primary Endpoint
- 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.
See More
Ultragenyx Pharmaceutical's Phase III Study Fails to Meet Primary Endpoint, Analysts Cut Price Targets
- Disappointing Study Results: Ultragenyx's Phase III Orbit and Cosmic studies, announced on December 29, 2025, failed to meet their primary endpoints, leading to diminished investor confidence and analysts rapidly cutting price targets.
- Bone Density Improvement Not Translated: Although the studies showed improvements in patients' bone density, they failed to establish a causal link to reduced annualized fracture rates, increasing regulatory and market pressures on the company.
- Analyst Sentiment Shift: Barclays analysts noted limited opportunities for drug approval despite some trends of clinical benefit, reflecting a pessimistic outlook on Ultragenyx's future prospects.
- Increased Investor Scrutiny: Levi & Korsinsky LLP is investigating Ultragenyx's business operations, indicating heightened investor concern over the company's future and potential legal risks.
See More
