Alnylam Pharmaceuticals reported on Friday that its experimental Huntington's disease treatment, brezosiran, successfully reduced levels of the mutant huntingtin protein in a small study. The company stated that the data suggest the drug can partially silence the specific gene responsible for the neurological disorder. In the highest dose group, patients experienced an average decline in mutant huntingtin within their spinal fluid of either 60% or 64%, a figure that varied based on the specific assay method used.
Pushkal Garg, head of research and development at Alnylam, presented these findings at the European Huntington's Disease Network Clinical Research Congress. Garg described the safety profile as encouraging, noting that the team achieved high levels of gene knockdown while maintaining good safety and tolerability. The company characterized the overall results as very encouraging given the observed efficacy and lack of significant safety issues.
Alnylam plans to initiate a placebo-controlled study next year. The objective of this subsequent trial is to determine whether brezosiran can actually slow the progression of the disease, moving beyond the biomarker reductions seen in the initial data.