UniQure Stock Plummets 52% on Potential FDA Decision. Here’s Why.

The company believes the Food and Drug Administration may deal a setback to one of its investigational therapies. (Dreamstime)

Key Points

• UniQure shares fall sharply after the FDA indicates Phase I/II study data for gene therapy AMT-130 may be insufficient for a BLA.
• AMT-130, an investigational gene therapy for Huntington’s disease, previously received Breakthrough Therapy designation.
• Coming into Monday, uniQure stock has risen 283% this year.

Shares of uniQure tumbled Monday after the biotech company reported a likely regulatory setback for one of its gene therapies.

Created with Highcharts 9.0.1uniQure’s stock priceSource: FactSetAs of Nov. 3

Created with Highcharts 9.0.1Feb. 2025Oct.010203040506070$80

The Food and Drug Administration no longer agrees that the data from Phase I/II studies of uniQure’s therapy AMT-130 is adequate to support a Biologics License Application, the company said it believes. AMT-130 is an investigational gene therapy for Huntington’s disease, which the FDA granted a Breakthrough Therapy designation earlier this year.

A Biologics License Application, or BLA, is a formal request to the FDA to market a distribute a biological product, such as a gene therapy.

UniQure stock plummeted 52% on Monday. Shares closed Friday up 283% on the year and more than 1,100% over the past 12 months.

“We remain fully committed to working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S,” said CEO Matt Kaputsa, who added that the FDA’s feedback was a “drastic change” from its previous guidance.

UniQure stock soared in December, when the company announced an agreement with the FDA to submit a BLA for AMT-130. Shares again skyrocketed in September when the company reported that AMT-130 effectively slowed the progression of Huntington’s disease, an inherited condition that causes nerve cells in the brain to decay. A high dose of the gene therapy slowed disease progression by 75% at 36 months, the company reported.

Less than a week after that announcement, uniQure closed a $345 million public equity offering, saying it would use the proceeds in part to fund “the potential commercial launch of AMT-130.”

Monday’s update therefore came as a surprise, said Cantor Fitzgerald analysts Kristen Kluska and Richard Miller.

“We now believe there will be challenges ahead with a BLA submission at this time,” wrote Kluska and Miller in a research note Monday.

Cantor Fitzgerald reiterated an Overweight rating for uniQure stock but lowered its price target to $55 from $80 following a call with the company’s executives.

Write to Nate Wolf at nate.wolf@barrons.com