uniQure Shares Fall 33% After FDA Demands Sham-Controlled Phase III Trial
uniQure stock plunged 33% after the FDA required a randomized, double-blind, sham-controlled Phase III trial for its Huntington’s disease therapy AMT-130. Management said AMT-130’s three-year Phase I/II data showed a 75% disease progression slowdown versus external controls, but FDA requires a pivotal sham-controlled Phase III study.
1. FDA Requires Pivotal Phase III Trial
The FDA notified uniQure that external comparator Phase I/II data are insufficient for approval and strongly recommended a randomized, double-blind, sham surgery-controlled Phase III trial for AMT-130 in Huntington’s disease, triggering a 33% stock drop.
2. Phase I/II Findings
uniQure cited its three-year Phase I/II results showing a statistically significant 75% slowdown in disease progression on the Composite Unified Huntington’s Disease Rating Scale compared to matched external controls, alongside reductions in neurofilament light levels and supportive functional measures.
3. Patient Risk and Ethical Concerns
Management emphasized the ethical and logistical burdens of a sham surgery design—including invasive procedures, extended anesthesia, and patient risks—and highlighted community concerns over potential loss of benefit during blinded follow-up.
4. Upcoming Regulatory Meetings
uniQure plans to request a Type B meeting with the FDA in Q2 2026 to discuss Phase III study designs, and will update its Phase I/II analysis to include four-year follow-up, with timelines and endpoints to be finalized after the meeting.