uniQure Stock Hits 18-Month High After FDA Agreement Clears Pathway For Huntington’s Treatment: Retail Gets More Optimistic

uniQure’s AMT-130 previously received a regenerative medicine advanced therapy designation from the FDA in May.

Shares of gene therapy company uniQure more than doubled on Tuesday before trimming some gains, reaching their highest levels since mid-June 2023.

The surge came after uniQure announced it had reached an agreement with the U.S. Food and Drug Administration (FDA) on key aspects of an Accelerated Approval pathway for its AMT-130 treatment, a potential therapy for Huntington’s disease. 

This agreement means data from the ongoing Phase I/II studies, compared to an external control, could serve as the primary basis for a biologics license application (BLA) submission without requiring an additional pre-submission study.

uniQure’s AMT-130 previously received a regenerative medicine advanced therapy (RMAT) designation from the FDA in May, underscoring its potential to address unmet medical needs in Huntington’s disease. 

Analysts have viewed this development as a major milestone.

Cantor Fitzgerald analyst Kristen Kluska raised her price target for uniQure to $58 from $28 and maintained an ‘Overweight’ rating, noting that the FDA’s approval signals potential pharma interest given the clearer path toward market entry.

Stifel also weighed in, describing the development as “a best-case scenario” and noting that uniQure can use external controls and data from ongoing studies. 

Stifel has a ‘Buy’ rating and predicts the FDA agreement will bolster confidence in uniQure’s pipeline, despite the low initial expectations for this accelerated pathway.

Retail sentiment on Stocktwits turned ‘extremely bullish’, with QURE trending among the top 10 tickers late Tuesday morning.

uniQure’s stock has more than doubled in value this year.

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