Interim data presented in July 2024 indicated a significant dose-dependent effect on slowing progression, measured by the cUHDRS, when compared to a natural history cohort.

Looking ahead, uniQure plans to submit additional regulatory updates and guidance regarding the Biologics License Application for AMT-130 in the second quarter of 2025.

uniQure is dedicated to advancing gene therapies for severe conditions, including Huntington's disease, building on its history of success with treatments like hemophilia B.

Huntington's disease affects approximately 70,000 people in the U.S. and Europe, caused by a genetic mutation linked to a CAG repeat expansion in the huntingtin gene, leading to severe physical and mental decline.

uniQure Inc. announced on April 17, 2025, that the FDA has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease, a serious neurodegenerative disorder with no current disease-modifying therapies.

The Breakthrough Therapy designation is based on interim data from ongoing Phase I/II trials, which demonstrated a dose-dependent slowing of disease progression over 24 months.

This Breakthrough Therapy designation adds to AMT-130's existing accolades, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations from the FDA.

The Breakthrough Therapy designation aims to expedite the development and review of therapies for serious conditions, providing enhanced FDA support and intensive guidance on drug development.

Dr. Walid Abi-Saab, the Chief Medical Officer of uniQure, emphasized the urgent need for effective Huntington's disease treatments and highlighted the promising interim data suggesting that AMT-130 can slow disease progression.

Currently, 45 patients have received AMT-130 in these trials, showcasing the treatment's potential.

The designation is supported by significant trial data showing a marked slowing of disease progression in treated patients, reinforcing the therapy's potential impact.

Abi-Saab reiterated the importance of the Breakthrough Therapy designation and the company's commitment to advancing innovative gene therapies.