uniQure Shares Plunge 40% as FDA Demands New AMT-130 Trial
FDA advised uniQure that AMT-130 Phase 1/2 data are insufficient as primary evidence for a BLA submission, recommending a prospective randomized, double-blind, sham-controlled trial. uniQure will request a Type B meeting in Q2 2026 and reported $622.5 million in cash and investments funding operations into H2 2029.
1. FDA Rejects Phase 1/2 Data for AMT-130
FDA advised uniQure that data from its Phase 1/2 study of AMT-130 cannot serve as primary evidence for a Biologics License Application, and strongly recommended conducting a prospective, randomized, double-blind, sham surgery-controlled trial to support approval.
2. Next Steps and Regulatory Strategy
uniQure plans to request a Type B meeting in the second quarter of 2026 to clarify an efficient development pathway; CEO Matt Kapusta emphasized the need for continued dialogue to apply regulatory flexibility and determine a scientifically grounded path forward.
3. Pipeline Progress and Financial Position
As of December 31, 2025, uniQure held approximately $622.5 million in cash and investments, supporting operations into the second half of 2029; the company is also advancing AMT-191 in a Phase 1/2a Fabry disease trial and completing enrollment for AMT-260 in refractory temporal lobe epilepsy.
4. Stock Market Reaction
Following the FDA guidance, uniQure shares tumbled over 40% in premarket trading, trading near a 52-week low as investors weigh the impact of additional trial requirements on the lead Huntington’s disease therapy timeline.