QURE - uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for Huntington's Disease | Benzinga
~ Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control ~
~ Achieved statistically significant lowering of CSF neurofilament light protein (NfL) compared to baseline at 24 months in patients treated with AMT-130; Mean CSF NfL levels for both doses were below baseline at 24 months ~
~ Granted first-ever Regenerative Medicine Advanced Therapy (RMAT) designation in Huntington's disease; uniQure expects to meet with the FDA in the second half of 2024 to discuss potential for expedited clinical development ~
~ Investor conference call and webcast today at 8:30 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM, July 09, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 24 months of follow-up data from 29 treated patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington's disease.
"We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington's disease and lowering of NfL in the CSF at 24 months," stated Walid Abi-Saab, M.D., chief medical officer of uniQure. "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 I/II studies to support the emerging therapeutic benefit. We look forward to holding an initial, multi-disciplinary RMAT meeting with the FDA later this year to discuss the potential for expedited clinical development of AMT-130."
"These updated results are exciting and provide compelling evidence of potential therapeutic benefit," stated Victor Sung, M.D., professor of neurology at the University of Alabama at Birmingham (UAB), director of the UAB Huntington's Disease Clinic, co-director of the UAB School of Medicine Neuroscience Module, and trustee on the National Board of the Huntington's Disease Society of America. "The preservation of motor and cognitive function observed through two years, combined with reduced NfL levels below baseline, defy expectations about the natural progression of Huntington's disease. cUHDRS, in particular, has been shown to be a robust and sensitive measure of disease progression, and offers an opportunity for enhanced clinical trial efficiency relative to individual measurements. These long-term data provide encouraging support of durable disease-modification and offer much needed hope for a community that is in desperate need of therapeutic options."
Exploratory Efficacy and Safety Data1
uniQure is conducting two multi-center Phase I/II clinical trials of AMT-130 in the U.S. (n=26) and Europe/UK (n=13). A total of 29 patients received one of two doses of AMT-130 (n=12 low dose; n=17 high dose) and 10 control patients received imitation surgery. Across both studies, 24-month follow-up data from a total of 21 patients (n=12 low dose; n=9 high dose) were available for analysis as of a March 31, 2024 cut-off date.
For the first time, uniQure conducted a statistical analysis of clinical outcomes for the 21 treated patients at 24 months compared to an expanded, propensity-weighted external control (n=154) developed in collaboration with the Cure Huntington's Disease Initiative (CHDI) using data from the TRACK-HD, TRACK-ON and PREDICT-HD natural history studies. The external control includes patients that met the Phase I/II clinical trial eligibility criteria, and whose data contributions were statistically weighted using propensity scoring to closely match the baseline characteristics of patients treated with AMT-130. Disease-related outcomes for these well-balanced cohorts were then compared after 24 months follow-up.
- A statistically significant, dose-dependent, slowing in disease progression measured by cUHDRS was observed through 24 months in patients receiving the high dose of AMT-130.
- At 24 months, the mean change in cUHDRS for patients receiving the high-dose of AMT-130 was -0.2 compared to -1.0 for patients in the propensity score-weighted external control, representing an 80% slowing of disease progression (p=0.007).
- At 24 months, the mean change in cUDHRS for patients receiving the low-dose of AMT-130 was -0.7 compared to -1.0 for patients in the propensity ...
- At 24 months, the mean change in cUHDRS for patients receiving the high-dose of AMT-130 was -0.2 compared to -1.0 for patients in the propensity score-weighted external control, representing an 80% slowing of disease progression (p=0.007).