UniQure’s New Gene Therapy Shows Promise in Slowing Huntington’s Disease Progression

In the high-dose group, patients experienced a mean 0.2-point reduction on the composite Unified Huntington’s Disease Rating Scale (cUHDRS), compared to a 1-point reduction in a control group, translating to an 80% slowing of disease progression. The low-dose group saw a 0.7-point reduction, indicating a 30% slowing. Additionally, levels of neurofilament light protein, a biomarker for neurodegeneration, decreased by 11% across all patients who received AMT-130, compared to a 26% increase in a natural history study.

CMO Walid Abi-Saab highlighted the importance of these findings, noting that the majority of patients had reached the two-year milestone, allowing for robust statistical analysis. This data is particularly promising given the mixed results from previous trials, which had cast doubt on the therapy’s efficacy and led to a significant drop in UniQure’s stock price.

AMT-130, an AAV5 gene therapy delivered directly to the brain, targets exon 1 of the huntingtin gene. Its potential for long-term effects from a one-time administration sets it apart from other treatments. The therapy’s success so far has led to a significant increase in UniQure’s stock, which rose by over 50% following the announcement.

Looking ahead, UniQure is in discussions with the FDA about future trial plans and the possibility of accelerated approval. However, CEO Matt Kapusta acknowledged the potential need for a large-scale Phase 3 trial, which could require a partnership with a larger company due to the substantial financial and time commitments involved. The company plans to meet with the FDA before the end of the year and expects to release 36-month data by mid-2025.

Overall, the latest results from UniQure’s AMT-130 gene therapy trials offer renewed hope for a treatment that can significantly slow the progression of Huntington’s disease, a debilitating neurodegenerative disorder.

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