Alnylam Announces Positive Initial Phase 1b Data for Brezosiran (ALN-HTT02) Demonstrating Deep and Durable Huntingtin Lowering in Huntington’s Disease

Blair R. Leavitt, MDCM, FRCPC, neurologist, Director of Research at the Centre for Huntington Disease at the University of British Columbia Hospital, and principal investigator

“The evidence suggesting that brezosiran has the potential to achieve robust and durable HTT-lowering, while maintaining an encouraging safety profile, is a very promising advance for this program at a time when there are still no approved disease-modifying therapies for Huntington disease.”

Blair R. Leavitt, MDCM, FRCPC, neurologist, Director of Research at the Centre for Huntington Disease at the University of British Columbia Hospital, and principal investigator

“Brezosiran is designed to lower all forms of HTT, including full-length mutant HTT and the shorter mutant HTT1a, which a growing body of evidence suggests may play a key role in the progression of Huntington disease.”

Pushkal Garg, M.D., Chief Research and Development Officer at Alnylam

“The deep and durable mutant huntingtin lowering observed after a single dose, together with the encouraging safety profile seen to date, give us confidence to advance brezosiran into a pivotal study.”

Pushkal Garg, M.D., Chief Research and Development Officer at Alnylam

“We plan to evaluate our differentiated huntingtin-lowering approach in a rigorous placebo-controlled study to determine whether it can alter the course of Huntington’s disease.”

Pushkal Garg, M.D., Chief Research and Development Officer at Alnylam

“We are deeply grateful to the people living with Huntington’s disease, their families and caregivers, advocacy organizations, investigators and clinical trial staff whose partnership makes this work possible.”