UniQure's Huntington's Gene Therapy Shows Promising Results, Setting Stage for FDA Submission
UniQure has announced groundbreaking results from its phase 1/2 trial of AMT-130, a gene therapy for Huntington's disease, potentially paving the way for the first genetic treatment for this devastating neurodegenerative disorder. The therapy demonstrated a significant 75% slowing of disease progression after three years, exceeding expectations and positioning the company for a biologics license application (BLA) submission to the FDA in early 2026.
Clinical Trial Success and Disease-Modifying Potential
The pivotal phase 1/2 trial evaluated 29 patients, with data from 12 high-dose recipients showing remarkable efficacy at the 36-month mark. Patients receiving high-dose AMT-130 experienced a mean change from baseline in the composite Unified Huntington's Disease Rating Scale (cUHDRS) of -0.38, compared to -1.52 in the control group. This 75% reduction in disease progression surpassed analysts' expectations and previous interim results.
Additionally, the therapy demonstrated a 60% slowing of disease progression as measured by Total Functional Capacity (TFC), a key secondary endpoint. Sarah Tabrizi, director of the University College London Huntington's Disease Center, emphasized the significance of these results, stating, "That means for one year of disease progression, they will have four years longer in terms of disease-free lives."
Regulatory Path and Market Potential
UniQure plans to meet with the FDA by the end of the year to discuss the data before filing a BLA in the first quarter of 2026. The company aims for a potential U.S. launch in late 2026, pending approval. The FDA has granted AMT-130 two designations that could expedite its review process.
The success of AMT-130 represents a potential breakthrough in a field that has seen numerous setbacks. Recent years have witnessed companies like Roche, Wave Life Sciences, and Sage Therapeutics shelving their Huntington's disease assets. UniQure's positive results have sparked renewed optimism, with the company's stock price more than tripling following the announcement.
Technical Insights and Safety Profile
AMT-130 is designed to silence a gene that produces a mutant form of the huntingtin protein, which is responsible for damaging nerve cells. The therapy's effects were not only observed in clinical measures but also in biomarkers. Levels of neurofilament light (NfL), a key marker of neurodegeneration, were "well below baseline" in treated patients.
The treatment was reported to be "well-tolerated" with a "manageable safety profile," addressing previous concerns about potential adverse events. This safety data is particularly crucial given the therapy's one-time administration approach and the FDA's recent scrutiny of gene therapies.
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 to seek approval of Huntington’s gene therapy after trial win
New study data showed treatment slowed signs of disease progression by 75% after three years, a finding that could have “massive effects on patients’ lives,” an investigator said.
- 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 key factors that differentiate AMT-130 from previous Huntington's disease treatments developed by Roche, Wave Life Sciences, and Sage Therapeutics?
What are the specific FDA designations granted to AMT-130, and how might they expedite its review process?
What is the estimated market size and unmet medical need for Huntington's disease therapies in the U.S. and globally?
How does the reduction in neurofilament light (NfL) levels in treated patients correlate with long-term clinical outcomes for AMT-130?
What potential challenges could UniQure face in scaling up manufacturing and distribution for AMT-130 ahead of its anticipated U.S. launch in late 2026?