uniQure's Stock Plummets 50% After FDA Rejects BLA Submission for AMT-130

Stifel lowered the firm's price target on uniQure to $40 from $50 and keeps a Buy rating on the shares. The firm "refreshed diligence" on many companies within its biotechnology coverage universe and made a number of model, estimate and price target changes in a 2026 look ahead note for the group.

Mizuho analyst Uy Ear lowered the firm's price target on uniQure to $33 from $60 and keeps an Outperform rating on the shares after lowering the firm's view of the probability of success and pushing out its expected launch year for AMT-130 by one year following the company announcing that the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 for Huntington's disease are currently unlikely to provide the primary evidence to support a Biologics License Application.

Leerink lowered the firm's price target on uniQure to $60 from $68 and keeps an Outperform rating on the shares following quarterly results and earnings call, which was "unsurprisingly" focused on the regulatory path for AMT-130 in Huntington's disease. Leerink acknowledges the "surprise" last week when uniQure sounded the alarm and disclosed that the FDA no longer agreed that the data from the Phase 1/2 studies and prespecified analyses vs. an external control may be adequate to provide primary evidence in support of a BLA submission. On the call today, uniQure emphasized that this FDA reversal does not change their data for AMT-130 in HD that support real and serious disease modification. While this news has made timing of a BLA submission uncertain, the company plans to urgently engage with the FDA to discuss next steps, beginning with the formal meeting minutes.

William Blair analyst Myles Minter downgraded Biohaven (BHVN) to Market Perform from Outperform. The announcement of a CRL for troriluzole in spinocerebellar ataxia halts not only a near-term source of revenue but also the ability of the company to access $150M in capital through its note purchase agreement with Oberland Capital, the analyst tells investors. Even with a 60% reduction in annual R&D spending, the firm believes Biohaven will need additional capital to continue to fund its pipeline or seek strategic alternatives for its deprioritized pipeline assets, notes the analyst, who is moving to the sidelines to await regulatory clarity given that the FDA has now been restrictive to both uniQure's (QURE) and Biohaven's comparisons to natural history as a regulatory path forward in rare neurological disorders.