UniQure shares skyrocket as gene therapy slows Huntington’s progression in trial

(This Sep 24 story has been repeated with no changes to text)

By Kamal Choudhury and Mrinalika Roy

(Reuters) -UniQure’s experimental gene therapy for Huntington’s disease slowed progression of the brain disorder by 75% in a key study, marking potentially breakthrough results that nearly tripled the value of its U.S.-listed shares on Wednesday.

Shares of the Dutch gene therapy maker were up 196% at $40.49 in morning trading, on track to add about $1.47 billion in market capitalization, if gains hold, as investors responded to the upbeat trial data for a disease that currently has no approved treatments to slow its progression.

Patients who received a high dose of the company’s therapy, called AMT-130, in an early-to-mid-stage study showed a 75% reduction in disease progression in a three-year analysis, based on a widely used clinical scale.

The therapy also slowed the decline of functional abilities by 60%, a key secondary goal.

A GAME CHANGER

Sarah Tabrizi, a Huntington’s disease researcher with nearly 30 years of experience, said the data was “game changing,” offering the first clear evidence of a therapy that may modify the course of the rare disease.

“75% slowing of disease progression is greater than what we even anticipated … and that means for one year of disease progression it slowed by, they will have four years longer in terms of disease-free life.”

Existing medications for the inherited brain disorder only manage symptoms such as involuntary movements but do not address the underlying cause.

“These data look pretty definitive to us and actually exceed what we thought was reasonable to expect,” Leerink Partners analyst Joseph Schwartz said in a note.

LAYING OUT REGULATORY PATHWAY

UniQure plans to submit a marketing application to the U.S. Food and Drug Administration in early 2026, with hopes of launching the therapy later that year if approved.

The data seemed sufficient to support an approval with minimal need for regulatory flexibility, which reduces investor concerns about FDA hurdles, Schwartz said.

CEO Matt Kapusta said the company’s licensed facility, which already produces a commercial gene therapy, has begun key tests to ensure it can reliably produce AMT-130.

The company estimates about 6,000 initially treatable U.S. patients at launch, from a total of 40,000 symptomatic patients nationwide.

“With gene therapy pricing, even a small percent penetration can lead to meaningful revenues,” Cantor analysts said in a note.

Meanwhile, UniQure separately said it had secured a $175 million loan from Hercules Capital to support the potential U.S. launch of AMT-130.

The company had cash and cash equivalents and investment securities of $378.5 million, at the end of June, which it said would fund operations into the second half of 2027.

(Reporting by Kamal Choudhury and Mrinalika Roy in Bengaluru; Editing by Sahal Muhammed and Sriraj Kalluvila)