UniQure restates FDA’s negative feedback on Huntington’s trial; Neurimmune expands AstraZeneca alliance

UniQure has reiterated that the U.S. Food and Drug Administration no longer considers data from its Phase I/II AMT-130 Huntington’s disease studies, which rely on an external natural-history control group, sufficient as primary evidence to support a planned biologics license application. 1 3 4

Despite earlier positive three-year data showing a substantial slowing of disease progression with high-dose AMT-130, the FDA has indicated that more robust controlled data are needed, creating a longer and more uncertain regulatory path. 1 2 4

UniQure is evaluating the FDA’s feedback and intends to seek additional meetings to explore options such as new or expanded studies, longer follow-up, or other pathways that could still enable an eventual U.S. filing for AMT-130. 1 2 3

The FDA’s shift is a regulatory alignment issue rather than a newly identified safety or efficacy problem; AMT-130 continues to appear generally safe, well tolerated, and potentially disease-modifying for Huntington’s disease. 1 3 4

Neurimmune and AstraZeneca have expanded their existing collaboration focused on antibody and neurodegeneration programs, building on prior work in areas such as cardiovascular and neurological disease. 5

The expanded Neurimmune–AstraZeneca alliance broadens AstraZeneca’s access to Neurimmune’s antibody discovery and human-derived immunology platforms, with AstraZeneca typically funding R&D in exchange for development, commercialization, and profit-sharing or royalty rights. 5

The new agreement is structured as a long-term strategic partnership that can generate milestone payments and royalties for Neurimmune if candidate molecules advance successfully through clinical development and reach the market. 5