uniQure’s investigational gene therapy has been shown to slow the progression of Huntington’s disease, according to new 24-month data shared by the company.
Affecting approximately 70,000 people in Europe and the US, Huntington’s is an inherited neurodegenerative disorder that causes nerve cells in the brain to decay over time.
Patients can experience motor symptoms such as chorea, as well as behavioural abnormalities and cognitive decline, and despite the “clear aetiology” of the disease, uniQure outlined that there are currently no approved therapies to delay its onset or slow progression.
The gene therapy specialist is conducting two phase 1/2 clinical trials of its candidate, AMT-130, with a total of 29 patients receiving one of two doses of the therapy and ten receiving imitation surgery.
According to follow-up data from 21 patients, a statistically significant slowing in disease progression measured by cUHDRS, a Huntington’s disease rating scale, was observed through 24 months in those receiving the high dose of AMT-130.
A mean 0.2 reduction in cUHDRS was seen in patients in the high-dose AMT-130 cohort, compared to a one point reduction for those in a propensity score-weighted external control, representing an 80% slowing of disease progression.
The lower-dose AMT-130 group saw a 0.7 reduction in cUHDRS, equating to a 30% slowing of disease progression.
Trends in measurements of motor and cognitive function showed near-baseline stability throughout the 24 months of follow-up in patients receiving the high dose of AMT-130, and a statistically significant reduction of a key biomarker of neurodegeneration in cerebrospinal fluid was shown in patients treated with uniQure’s therapy.
Walid Abi-Saab, uniQure’s chief medical officer, said: “We believe this is the first clinical trial of any investigational medicine for Huntington’s disease to show evidence of a potential long-term clinical benefit and reduction of a key marker of neurodegeneration.
“Moreover, given the one-time administration of AMT-130, we are in a unique position to continue accumulating longer-term patient outcomes from the phase 1/2 studies to support the emerging therapeutic benefit.”
uniQure said it expects to meet with the US Food and Drug Administration in the second half of 2024 to present the updated data and to discuss potential expedited clinical development pathways and accelerated approval of AMT-130.