If approved, uniQure’s gene therapy AMT-130—which slowed disease progression by 75%—would be the first genetic treatment for Huntington’s disease. A BLA submission is planned for the first quarter of 2026.
UniQure is gearing up to launch the first-ever genetic treatment for Huntington’s disease after three-year data from a pivotal Phase I/II trial of its gene therapy showed 75% slowing of the intractable neurodegenerative disease.
In a note Wednesday morning, Stifel analysts said the data “clearly exceed expectations,” bolstering the company’s case with the FDA. Shares of uniQure were up 156% in pre-market trading Wednesday.
A single high dose of AMT-130 slowed disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) compared to an external control, hitting statistical significance and meeting the study’s primary endpoint, uniQure reported Wednesday. It also aced a key secondary endpoint, significantly slowing disease progression as measured by the Total Functional Capacity (TFC) compared to the control group.
The trial evaluated 12 patients receiving a high dose of the gene therapy and 12 patients receiving a low dose after 36 months. Patients given high-dose AMT-130 had a mean change from baseline in cUHDRS—which measures Huntington’s disease progression—of -0.38, in comparison to the control group, which saw a –1.52 change.
This 75% slowing of disease surpassed Stifel’s hopes for at least around 60%, the analysts wrote, while the secondary win on TFC was an “upside surprise.”
Additionally, levels of neurofilament light (NfL)—a key marker of neurodegeneration—were “well below baseline,” according to uniQure. Elevation in NfL in the cerebrospinal fluid has been shown to be “strongly associated” with greater clinical severity of Huntington’s, according to uniQure. Finally, AMT-130 was “well-tolerated,” with what the biotech called a “manageable safety profile.”
uniQure plans to submit a biologics license application for AMT-130 in the first quarter of 2026, with an anticipated U.S. launch later next year, “pending approval.”
This past June, uniQure announced it had “reached alignment with the FDA on several key components of the statistical analysis plan and chemistry, manufacturing and controls information that will support” the BLA application through the accelerated pathway.
Stifel noted that 75% was “largely consistent” with the 80% disease slowing shown by AMT-130 at 2 years in the same trial. In an interview with BioSpace in June, uniQure CEO Matt Kapusta said the company would be seeking “consistency” in this three-year readout with the two-year data.
“If we’re able to demonstrate in treated patients at three years after a one-time administration of AMT 130 that there’s meaningful slowing of disease progression compared to this very closely matched external control cohort, I think that would be immensely powerful, and I think potentially supportive of a conditional approval,” he said at the time.
uniQure’s readout is welcome news for the Huntington’s space, which has been mired in a desert of disappointment the past five years. Roche, Wave Life Sciences and Sage Therapeutics have all shelved assets—though Wave is back with a next-gen antisense oligonucleotide that showed positive results last year in a mid-stage study. PTC Therapeutics, meanwhile, claimed a mid-stage win for its small molecule, PTC518, in May, though it was met with mixed analyst reaction.
