uniQure's gene therapy slows Huntington's progression

uniQure has released new data indicating that its experimental gene therapy, AMT-130, can slow the progression of Huntington's disease over a period of 24 months. Huntington's disease is a genetic neurodegenerative disorder that leads to the gradual decay of nerve cells in the brain, affecting around 70,000 individuals in Europe and the United States. Those suffering from the disease experience a range of symptoms including motor dysfunctions such as chorea, behavioral issues, and cognitive decline. Despite a well-understood causative basis, there are currently no approved treatments available to delay the onset or progression of Huntington's disease.

uniQure is currently conducting two phase 1/2 clinical trials of AMT-130, enrolling a total of 29 patients who were administered either a high or low dose of the gene therapy, and ten patients who underwent sham surgeries. Follow-up data from 21 patients revealed that those who received the high dose of AMT-130 exhibited a statistically significant slowing in disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), compared to a propensity score-weighted external control group.

Specifically, patients in the high-dose cohort showed an average 0.2 point reduction in cUHDRS over 24 months, while the external control group experienced a one-point reduction. This data indicates an 80% slowing in disease progression for the high-dose group. Meanwhile, the lower-dose group observed a 0.7 point reduction in cUHDRS, translating to a 30% slowdown in disease progression.

Additional measures of motor and cognitive function remained stable near baseline levels throughout the 24-month period for those who received the high dose of AMT-130. Furthermore, there was a significant decrease in a crucial biomarker of neurodegeneration found in the cerebrospinal fluid of patients treated with the gene therapy.

Walid Abi-Saab, the chief medical officer of uniQure, emphasized the groundbreaking nature of these findings. "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," he stated. Abi-Saab also highlighted the unique advantage offered by the one-time administration of AMT-130, which allows the company to continually gather long-term patient outcomes from the ongoing phase 1/2 studies, bolstering the therapeutic potential of the treatment.

Looking ahead, uniQure plans to engage with the US Food and Drug Administration (FDA) in the latter half of 2024 to present the updated data. The company aims to discuss potential expedited clinical development pathways and seek accelerated approval for AMT-130, hopeful that this gene therapy could become a pioneering treatment for Huntington’s disease.