FDA Hears Directly From Huntington's Disease Community in Externally-Led Meeting
• The FDA met with Huntington's disease (HD) patients and caregivers to hear their experiences and challenges, informing future drug development and regulatory decisions.
• Discussions focused on the impact of HD at pre-symptomatic, early, and mid-stages, including cognitive, psychiatric, and physical symptoms, and their effects on daily life.
• Participants emphasized the need for treatments addressing cognitive changes, delaying disease progression, and improving quality of life, while highlighting challenges in clinical trial participation.
• The meeting underscored the importance of patient perspectives in shaping HD treatment strategies and regulatory pathways, with ongoing efforts to gather further community input.
Sage Therapeutics Halts Dalzanemdor Development After Phase II Huntington's Disease Trial Fails
• Sage Therapeutics discontinues dalzanemdor (SAGE-718) development after its Phase II DIMENSION trial failed to meet primary and secondary endpoints in Huntington's disease patients.
• The 12-week, double-blind, placebo-controlled study involving 189 patients showed no statistically significant difference in cognitive improvement compared to placebo.
• This decision follows a recent restructuring at Sage, including R&D team reductions, and earlier setbacks with their depression drug Zurzuvae.
• The company expressed disappointment, acknowledging the unmet need for new Huntington's disease treatments and thanking study participants.
Sage Therapeutics Halts Dalzanemdor Development After Phase II Huntington's Disease Trial Fails
• Sage Therapeutics discontinues dalzanemdor (SAGE-718) development after its Phase II DIMENSION trial failed to meet primary and secondary endpoints in Huntington's disease patients.
• The DIMENSION study, a 12-week, double-blind, placebo-controlled trial, included 189 patients but showed no statistically significant difference in cognitive improvement.
• Dalzanemdor was generally well-tolerated, with most adverse events being mild to moderate, but the lack of efficacy led to the program's termination.
• This decision follows a recent restructuring at Sage, including halving its R&D team, after its depression drug Zurzuvae's limited approval.
Huntington's Disease Research Sees Renewed Hope with Promising Trial Results
• A phase 1/2 gene therapy trial demonstrated an 80% slowing of disease progression in Huntington's disease (HD) patients, as measured by the composite Unified Huntington's Disease Rating Scale.
• Wave Life Sciences' ASO, WVE-003, showed a statistically significant 46% reduction in mutant huntingtin protein, prompting plans for accelerated FDA approval.
• Numerous companies are actively developing HD therapeutics, employing diverse approaches based on an expanding understanding of the disease, signaling a dynamic future for HD treatment.
• Researchers are exploring innovative strategies like CRISPR-based genome editing and neural progenitor transplantation to counteract HD pathology, expanding therapeutic horizons.
© Copyright 2025. All Rights Reserved by MedPath