From Setback To Surge: UniQure's Huntington's Hope Is Poised For Upside

2025-11-10 12:44:37 ET

UniQure ( QURE )

• Rating: Buy
• Price target: $66 (137% upside)

Executive Summary

• What happened: In a pre-BLA meeting, the FDA did not agree to accepting UniQure's (QURE) AMT-130's (the company's Huntington's disease gene therapy) Phase 1/2 data for accelerated approval as the trial used external controls (which was done under FDA agreement per the company). The timing for the BLA filing is unclear. The company expects FDA minutes within 30 days and will work with the FDA for the best path forward for the drug's approval. The stock has fallen >50% from its 52-week high. Management requested an urgent Type A meeting. EU filing through EMA pathways is still on the dock. Patient advocacy groups (HDSA, EURORDIS) have been mobilized for immediate FDA engagement).

• AMT-130 is the first disease-modifying therapy for this debilitating disease: AMT-130 showed 75% slowing of composite Unified Huntington’s Disease Rating Scale, cUHDRS decline at 36 months in the high dose cohort compared to propensity score-matched external controls in the Phase 1/2 Enroll-HD trial (p=0.003), with acceptable tolerability. The trial also met a key secondary endpoint by showing significant slowing of the disease progression as measured by Total Functional Capacity compared to external controls at 36 months.
• Regulatory context: The FDA has permitted external controls in the past in selected rare diseases with no approved therapies. With the recent strictness in FDA stance toward accelerated drug approvals after the management change earlier this year, accelerated approvals have seen more scrutiny, as shown by the FDA complete response letter, CRL, for Biohaven's ( BHVN ) BLA in spinocerebellar ataxia.
• Regulatory model: We model ~78% probability of eventual approval (accelerated approval or Phase 3 required) based on Monte Carlo regulatory scenarios (details given below). We model ~35% near-term probability of accelerated approval.
• Our call, Buy: We model base case risk-adjusted NPV of $66/share based on discounted cash flow valuation, implying 137% upside. Near-term catalysts include FDA meeting minutes by the first week of December and any company discussions with the FDA about alternative regulatory path.

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