uniQure Updates AMT-191 Clinical Data for Fabry Disease
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 17h ago
0mins
Should l Buy QURE?
Source: Benzinga
- Clinical Trial Progress: uniQure has updated preliminary safety and exploratory efficacy data from its Phase 1/2a trial of AMT-191 involving 11 patients, with all showing significant increases in α-galactosidase A activity across three dosing cohorts, indicating the gene therapy's potential in treating Fabry disease.
- Dose-Dependent Response: α-galactosidase A activity increased to 0.34-82.2 times, 1.6-312.52 times, and 27.7-223.7 times the normal level across the different dose groups, demonstrating a dose-dependent response, with these increases remaining stable over follow-up periods of up to more than a year, indicating the therapy's durability.
- Safety Analysis: No serious adverse events were observed at the 4×10^13 gc/kg and 2×10^13 gc/kg doses, although two patients at the 4×10^13 gc/kg dose experienced asymptomatic Grade 3 liver enzyme elevations, confirmed as dose-limiting toxicity, showcasing the manageable safety profile of the therapy.
- Market Reaction: Following the announcement, uniQure's shares rose by 6.10% to $26.01, reflecting positive market sentiment towards the clinical data of AMT-191 and potentially boosting investor confidence in the company's future prospects.
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Analyst Views on QURE
Wall Street analysts forecast QURE stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for QURE is 49.88 USD with a low forecast of 33.00 USD and a high forecast of 70.00 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.
10 Analyst Rating
8 Buy
2 Hold
0 Sell
Strong Buy
Current: 24.520
Low
33.00
Averages
49.88
High
70.00
Current: 24.520
Low
33.00
Averages
49.88
High
70.00
About QURE
Uniqure NV is a company based in the Netherlands specialized in gene therapy. It seeks to develop one-time administered treatments with potentially curative results for patients suffering from genetic and other devastating diseases. It develops, both internally and through partnerships, a pipeline of gene therapies. It produces adeno-associated virus based, or AAV-based, gene therapies in its own facilities with a proprietary, commercial-scale, current good manufacturing practices, compliant, manufacturing process. AMT-061, the Company’s lead product candidate for patients with hemophilia B, is going through a dosing phase of a pivotal study. AMT-130, the product candidate for patients with Huntington’s disease is in Phase I/II clinical study.
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.
uniQure Updates AMT-191 Clinical Data for Fabry Disease
- Clinical Trial Progress: uniQure has updated preliminary safety and exploratory efficacy data from its Phase 1/2a trial of AMT-191 involving 11 patients, with all showing significant increases in α-galactosidase A activity across three dosing cohorts, indicating the gene therapy's potential in treating Fabry disease.
- Dose-Dependent Response: α-galactosidase A activity increased to 0.34-82.2 times, 1.6-312.52 times, and 27.7-223.7 times the normal level across the different dose groups, demonstrating a dose-dependent response, with these increases remaining stable over follow-up periods of up to more than a year, indicating the therapy's durability.
- Safety Analysis: No serious adverse events were observed at the 4×10^13 gc/kg and 2×10^13 gc/kg doses, although two patients at the 4×10^13 gc/kg dose experienced asymptomatic Grade 3 liver enzyme elevations, confirmed as dose-limiting toxicity, showcasing the manageable safety profile of the therapy.
- Market Reaction: Following the announcement, uniQure's shares rose by 6.10% to $26.01, reflecting positive market sentiment towards the clinical data of AMT-191 and potentially boosting investor confidence in the company's future prospects.
See More
uniQure Updates AMT-191 Clinical Data for Fabry Disease
- Efficacy Data Update: As of January 8, 2026, all 11 patients in the three dosing cohorts of AMT-191 exhibited significant elevations in α-Gal A activity, with the lowest dose showing increases of 0.34 to 82.2-fold, indicating the therapy's potential in treating Fabry disease.
- Good Tolerability: No serious adverse events related to AMT-191 were observed at the 4x10^13 gc/kg and 2x10^13 gc/kg doses, demonstrating the safety profile of the therapy, although two patients at the 4x10^13 gc/kg dose experienced asymptomatic Grade 3 liver enzyme elevations.
- Patient Treatment Transition: Six out of 11 patients have discontinued enzyme replacement therapy by the data cutoff date, meeting pre-specified criteria, which highlights AMT-191's potential to replace traditional treatments and enhance patient options.
- Ongoing Clinical Trials: The Phase I/IIa clinical trial of AMT-191 is still underway, with uniQure planning to provide further updates in the future to support the therapy's potential as a one-time gene treatment for patients with Fabry disease.
See More
uniQure Faces Investigation Over FDA Data Issues
- Insufficient FDA Data: On November 3, 2025, uniQure revealed that the FDA informed the company that data for its AMT-130 Biologics License Application (BLA) lacked sufficient evidence, leading to uncertainty regarding the timing of the BLA submission, which directly impacts future product launch plans.
- Stock Price Plunge: Following this announcement, uniQure's stock price plummeted over 50%, from $67.69 on October 31, 2025, to $34.29 on November 3, indicating extreme market pessimism regarding the company's prospects.
- Legal Investigation Initiated: Kessler Topaz Meltzer & Check LLP is investigating potential violations of federal securities laws by uniQure, aiming to protect investor rights and pursue possible legal remedies, highlighting concerns over corporate governance and compliance.
- Investor Impact: This incident not only undermines shareholder confidence in uniQure but may also lead to difficulties in future financing, thereby affecting its research and development capabilities and market competitiveness, especially in the capital-intensive gene therapy sector.
See More
uniQure Faces Investigation Over FDA Data Issues
- FDA Notification Impact: On November 3, 2025, uniQure announced that the FDA deemed the data for its AMT-130 gene therapy insufficient to support its Biologics License Application (BLA), leading to uncertainty regarding the timing of the application.
- Stock Price Plunge: Following this announcement, uniQure's stock price plummeted over 50%, closing at $34.29 on November 3, 2025, down from $67.69 on October 31, reflecting extreme market pessimism about the company's prospects.
- Legal Investigation Initiated: Kessler Topaz Meltzer & Check LLP is investigating potential violations of federal securities laws by uniQure, aiming to protect investor rights and seek possible legal remedies.
- Investor Attention: The investigation has garnered significant attention from uniQure investors, who are encouraged to fill out an online form for more information, indicating heightened concern and unease regarding the company's future developments.
See More
Analysis of QURE's 52-Week High and Low Points
- Price Range Analysis: QURE's 52-week low is $7.76 per share, while the high is $71.50, with the last trade at $23.88, indicating significant volatility in its stock price over the past year, reflecting varying market expectations for its future performance.
- Technical Indicator Focus: The current stock price is below the 200-day moving average, which may suggest increased selling pressure in the short term, prompting investors to monitor subsequent trends to assess potential investment risks.
- Market Sentiment Insight: Although the current stock price is significantly lower than the 52-week high, it remains above the low, indicating that the market still holds some confidence in QURE's fundamentals, potentially attracting investors looking for entry points at lower prices.
- Investor Strategy Adjustment: Investors should consider QURE's price volatility and technical indicators, along with market sentiment, to adjust their strategies in response to potential market changes.
See More
uniQure (QURE) Stock Plummets 50% After FDA Rejects BLA Submission for AMT-130
- FDA Notification Impact: On November 3, 2025, uniQure disclosed that the FDA indicated its clinical data for AMT-130, a gene therapy for Huntington's disease, was insufficient to support the Biologics License Application (BLA), leading to significant regulatory challenges for the company.
- Stock Price Volatility: Following this announcement, uniQure's stock plummeted over 50%, from $67.69 on October 31, 2025, to $34.29, reflecting extreme market pessimism regarding the company's future prospects.
- Investor Scrutiny: Kessler Topaz Meltzer & Check LLP is investigating potential violations of federal securities laws by uniQure, indicating heightened investor concerns about the company's compliance and transparency.
- Future Uncertainty: uniQure stated that the timing for the BLA submission for AMT-130 is now unclear, which could impact the company's fundraising and R&D efforts, posing a threat to its long-term strategic objectives.
See More
uniQure Updates AMT-191 Clinical Data for Fabry Disease
- Clinical Trial Progress: uniQure has updated preliminary safety and exploratory efficacy data from its Phase 1/2a trial of AMT-191 involving 11 patients, with all showing significant increases in α-galactosidase A activity across three dosing cohorts, indicating the gene therapy's potential in treating Fabry disease.
- Dose-Dependent Response: α-galactosidase A activity increased to 0.34-82.2 times, 1.6-312.52 times, and 27.7-223.7 times the normal level across the different dose groups, demonstrating a dose-dependent response, with these increases remaining stable over follow-up periods of up to more than a year, indicating the therapy's durability.
- Safety Analysis: No serious adverse events were observed at the 4×10^13 gc/kg and 2×10^13 gc/kg doses, although two patients at the 4×10^13 gc/kg dose experienced asymptomatic Grade 3 liver enzyme elevations, confirmed as dose-limiting toxicity, showcasing the manageable safety profile of the therapy.
- Market Reaction: Following the announcement, uniQure's shares rose by 6.10% to $26.01, reflecting positive market sentiment towards the clinical data of AMT-191 and potentially boosting investor confidence in the company's future prospects.
See More
uniQure Updates AMT-191 Clinical Data for Fabry Disease
- Efficacy Data Update: As of January 8, 2026, all 11 patients in the three dosing cohorts of AMT-191 exhibited significant elevations in α-Gal A activity, with the lowest dose showing increases of 0.34 to 82.2-fold, indicating the therapy's potential in treating Fabry disease.
- Good Tolerability: No serious adverse events related to AMT-191 were observed at the 4x10^13 gc/kg and 2x10^13 gc/kg doses, demonstrating the safety profile of the therapy, although two patients at the 4x10^13 gc/kg dose experienced asymptomatic Grade 3 liver enzyme elevations.
- Patient Treatment Transition: Six out of 11 patients have discontinued enzyme replacement therapy by the data cutoff date, meeting pre-specified criteria, which highlights AMT-191's potential to replace traditional treatments and enhance patient options.
- Ongoing Clinical Trials: The Phase I/IIa clinical trial of AMT-191 is still underway, with uniQure planning to provide further updates in the future to support the therapy's potential as a one-time gene treatment for patients with Fabry disease.
See More
uniQure Faces Investigation Over FDA Data Issues
- Insufficient FDA Data: On November 3, 2025, uniQure revealed that the FDA informed the company that data for its AMT-130 Biologics License Application (BLA) lacked sufficient evidence, leading to uncertainty regarding the timing of the BLA submission, which directly impacts future product launch plans.
- Stock Price Plunge: Following this announcement, uniQure's stock price plummeted over 50%, from $67.69 on October 31, 2025, to $34.29 on November 3, indicating extreme market pessimism regarding the company's prospects.
- Legal Investigation Initiated: Kessler Topaz Meltzer & Check LLP is investigating potential violations of federal securities laws by uniQure, aiming to protect investor rights and pursue possible legal remedies, highlighting concerns over corporate governance and compliance.
- Investor Impact: This incident not only undermines shareholder confidence in uniQure but may also lead to difficulties in future financing, thereby affecting its research and development capabilities and market competitiveness, especially in the capital-intensive gene therapy sector.
See More
uniQure Faces Investigation Over FDA Data Issues
- FDA Notification Impact: On November 3, 2025, uniQure announced that the FDA deemed the data for its AMT-130 gene therapy insufficient to support its Biologics License Application (BLA), leading to uncertainty regarding the timing of the application.
- Stock Price Plunge: Following this announcement, uniQure's stock price plummeted over 50%, closing at $34.29 on November 3, 2025, down from $67.69 on October 31, reflecting extreme market pessimism about the company's prospects.
- Legal Investigation Initiated: Kessler Topaz Meltzer & Check LLP is investigating potential violations of federal securities laws by uniQure, aiming to protect investor rights and seek possible legal remedies.
- Investor Attention: The investigation has garnered significant attention from uniQure investors, who are encouraged to fill out an online form for more information, indicating heightened concern and unease regarding the company's future developments.
See More
Analysis of QURE's 52-Week High and Low Points
- Price Range Analysis: QURE's 52-week low is $7.76 per share, while the high is $71.50, with the last trade at $23.88, indicating significant volatility in its stock price over the past year, reflecting varying market expectations for its future performance.
- Technical Indicator Focus: The current stock price is below the 200-day moving average, which may suggest increased selling pressure in the short term, prompting investors to monitor subsequent trends to assess potential investment risks.
- Market Sentiment Insight: Although the current stock price is significantly lower than the 52-week high, it remains above the low, indicating that the market still holds some confidence in QURE's fundamentals, potentially attracting investors looking for entry points at lower prices.
- Investor Strategy Adjustment: Investors should consider QURE's price volatility and technical indicators, along with market sentiment, to adjust their strategies in response to potential market changes.
See More
uniQure (QURE) Stock Plummets 50% After FDA Rejects BLA Submission for AMT-130
- FDA Notification Impact: On November 3, 2025, uniQure disclosed that the FDA indicated its clinical data for AMT-130, a gene therapy for Huntington's disease, was insufficient to support the Biologics License Application (BLA), leading to significant regulatory challenges for the company.
- Stock Price Volatility: Following this announcement, uniQure's stock plummeted over 50%, from $67.69 on October 31, 2025, to $34.29, reflecting extreme market pessimism regarding the company's future prospects.
- Investor Scrutiny: Kessler Topaz Meltzer & Check LLP is investigating potential violations of federal securities laws by uniQure, indicating heightened investor concerns about the company's compliance and transparency.
- Future Uncertainty: uniQure stated that the timing for the BLA submission for AMT-130 is now unclear, which could impact the company's fundraising and R&D efforts, posing a threat to its long-term strategic objectives.
See More
