UniQure's Huntington's Gene Therapy Shows Promising Results, Poised for FDA Submission
UniQure has reported groundbreaking results from a pivotal Phase I/II trial of its gene therapy AMT-130 for Huntington's disease, demonstrating a 75% slowing of disease progression after three years. The company is now gearing up for a potential first-in-class approval and launch, with plans to submit a Biologics License Application (BLA) to the FDA in early 2026.
Landmark Clinical Data
The trial evaluated 24 patients, with 12 receiving a high dose of AMT-130 and 12 receiving a low dose. After 36 months, high-dose patients showed a mean change from baseline in the composite Unified Huntington's Disease Rating Scale (cUHDRS) of -0.38, compared to -1.52 in the external control group. This represents a statistically significant 75% slowing of disease progression.
Additionally, AMT-130 met a key secondary endpoint, significantly slowing disease progression as measured by the Total Functional Capacity (TFC). High-dose patients experienced a TFC score reduction of 0.36, compared to 0.88 in the control group, equating to a 60% slowing of functional decline.
Dr. Walid Abi-Saab, UniQure's Chief Medical Officer, stated, "These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington's disease."
Market Impact and Future Plans
The positive results have had a dramatic effect on UniQure's stock price, which surged by 156% in pre-market trading following the announcement. The company plans to submit a BLA for AMT-130 in the first quarter of 2026, with an anticipated U.S. launch later that year, pending approval.
If approved, AMT-130 would become the first genetic treatment for Huntington's disease, addressing a significant unmet need in the treatment of this rare, inherited neurodegenerative disorder. The therapy's success is particularly noteworthy given the recent setbacks in Huntington's disease research, including abandoned projects by Roche, Wave Life Sciences, and Sage Therapeutics.
UniQure CEO Matt Kapusta expressed optimism about the therapy's potential for conditional approval, highlighting the consistency between the three-year data and previous two-year results. The company has already aligned with the FDA on key components of the statistical analysis plan and chemistry, manufacturing, and controls information to support the BLA application through the accelerated pathway.
References
- UniQure ‘Exceeds Expectations’ in Much-Anticipated 3-Year Huntington’s Readout
If approved, uniQure’s gene therapy AMT-130—which slowed disease progression by 75%—would be the first genetic treatment for Huntington’s disease. A BLA submission is planned for the first quarter of 2026.
- uniQure hopes to launch 1st Huntington's gene therapy next year following phase 1/2 success
UniQure has set its sights on submitting its Huntington’s disease gene therapy for approval next year after the asset was shown to slow progression of the neurodegenerative condition by 75% in a phase 1/2 trial
Explore Further
What are the potential safety concerns or observed adverse events reported during the Phase I/II trial of AMT-130 for Huntington's disease?
How does AMT-130 compare in efficacy to other gene therapy or experimental treatments previously pursued by competitors like Roche, Wave Life Sciences, or Sage Therapeutics?
What is the estimated market size and patient population for Huntington's disease in the United States, and how could these factors impact AMT-130's market potential?
What are UniQure's plans for manufacturing and scaling production of AMT-130 to meet anticipated demand following FDA approval?
What are the anticipated challenges or regulatory hurdles in the submission and approval process for AMT-130 through the FDA's accelerated pathway?