UniQure has set its sights on submitting its Huntington’s disease gene therapy for approval next year after the asset was shown to slow progression of the neurodegenerative condition by 75% in a phase 1/2 trial.
The trial saw 29 patients with the disease receive either a low or a high dose of the gene therapy, dubbed AMT-130. Today’s data come from 12 patients who received the high dose and were evaluated at the 36-month mark, with the outcomes compared to a natural history data set.
The 36-month analysis showed a “statistically significant” 75% slowing of disease progression among patients who received the higher dose of AMT-130. Specifically, these patients saw a mean reduction on the Unified Huntington's Disease Rating Scale of 0.38, compared to a 1.52 reduction among patients in the historical data set.
The readout thrilled investors, who tripled uniQure's stock price when markets opened this morning. The company's shares began trading at around $40 on Wednesday, compared to a Tuesday closing price of $13.66.
As well as hitting this primary endpoint, uniQure also pointed to a key secondary endpoint win as measured by total functional capacity (TFC), which measures an individual’s ability to maintain functional independence in their day-to-day life. Here, patients on high-dose AMT-130 saw their TFC score drop by 0.36 compared to a 0.88 drop for the historical data set—equivalent to a 60% slowing of disease progression for patients on the gene therapy.
The company acknowledged “favorable trends” in various measures of motor and cognitive function as well as highlighted an 8.2% reduction in cerebrospinal neurofilament light protein, a biomarker of neurodegeneration.
Armed with the phase 1/2 data, uniQure Chief Medical Officer Walid Abi-Saab, M.D., said the biotech is “eager to discuss the data” in a meeting expected later this year, aligning with “the goal of submitting a BLA in the first quarter of 2026.” The aim is to take AMT-130 to market next year.
“We are incredibly excited about these topline results and what they may represent for individuals and families affected by Huntington’s disease,” Abi-Saab said. “These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease, while also providing important evidence supporting one-time, precision-delivered gene therapies for the treatment of neurological disorders.”
Huntington’s is a rare inherited disease that causes the progressive degeneration of nerve cells in the brain, leading to impairment of physical abilities, movement, thinking and psychiatric disorders. Treatments are available to manage symptoms, but there is currently no disease-altering therapy available.
AMT-130’s clinical journey got off to a bumpy start back in 2022 when the therapy was scrutinized after safety signals prompted a pause in dosing for two groups in an open-label, European phase 1/2 trial. The pause, which was recommended by a safety monitoring board after three patients were hospitalized with various symptoms, was lifted in November 2022.
The earliest disease data were later deemed “noisy and difficult to interpret,” but interim findings in December 2023 received a warmer reception. By the time interim 24-month results read out from the phase 1/2 study last year, Abi-Saab was confident enough to tout the study as the “first clinical trial of any investigational medicine for Huntington’s disease to show evidence of a potential long-term clinical benefit.”
The past year has been a reminder of what a tricky indication Huntington’s can be. What appeared to be a phase 2 win for PTC Therapeutics’ Novartis-partnered candidate in May still knocked 20% of the biotech’s stock amid doubts about the path to accelerated approval. Back in November 2024, Bayer abandoned work on its own clinical-stage gene therapy for the disease.