FDA Mandates Sham-Controlled Trial, uniQure Targets Slashed to $12 and $11
FDA will require a sham-controlled Phase 3 trial for uniQure’s AMT-130 Huntington’s therapy, derailing plans for historical controls and prompting Mizuho to slash its price target from $33 to $12 and delay launch to 2030 with success odds cut to 15%. BofA also downgraded to Sector Perform with $11 target.
1. FDA Requires Sham-Controlled Phase 3 Trial
Regulators signaled that uniQure must conduct a prospective sham-controlled Phase 3 study for its AMT-130 Huntington’s therapy, reversing expectations of using historical controls and adding complexity to the approval path.
2. Mizuho Downgrade and Forecast Revision
Mizuho cut its uniQure rating from Outperform to Neutral, slashed its price target from $33 to $12, reduced the probability of success for AMT-130 from 65% to 15%, and pushed the projected launch from 2028 to 2030 due to increased trial requirements.
3. BofA Lowers Rating and Notes Pipeline Risks
Bank of America downgraded uniQure to Sector Perform with an $11 target, citing the FDA’s insistence on a randomized, sham-controlled study and highlighting pauses in its Fabry disease program and holds in SOD1-ALS and epilepsy trials.