- uniQure added that the FDA has recommended that the company conduct a prospective, randomized, double-blind, sham-surgery-controlled study.
- The company stated that it intends to continue engaging with the FDA regarding Phase III trial development.
- uniQure added that it plans to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches.
uniQure NV (QURE) shares plunged by nearly 45% in Monday’s pre-market trade after the company issued an update about its investigational gene therapy for Huntington’s disease (HD).
uniQure said the FDA does not agree that Phase I/II data, compared with an external control, are sufficient to serve as primary evidence of effectiveness for a marketing application for its Huntington’s disease gene therapy, AMT-130.
“While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said uniQure CEO Matt Kapusta.
Retail sentiment on Stocktwits around uniQure trended in the ‘extremely bullish’ territory, with message volumes at ‘extremely high’ levels.
FDA Urges Sham-Controlled Trial
uniQure added that the FDA has recommended that the company conduct a prospective, randomized, double-blind, sham-surgery-controlled study.
The company stated that it intends to continue engaging with the FDA regarding Phase III trial development. uniQure added that it plans to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches.
“We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130,” Kapusta added.
uniQure’s Gene Therapy Pipeline
uniQure currently has six gene therapies in its pipeline, each at different stages of progress and approvals.
The company’s Hemophilia B gene therapy, AMT-061, has already been approved, and the AMT-130 therapy for Huntington’s disease is currently at the most advanced stage of trials.
uniQure’s other gene therapies include treatments for Fabry disease, Amyotrophic Lateral Sclerosis, and Temporal Lobe Epilepsy, apart from other undisclosed programs.
QURE stock is down 35% year-to-date, but up 29% over the past 12 months.
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