Alnylam is reporting encouraging early safety and biomarker results for brezosiran, its huntingtin-lowering therapy for Huntington’s disease, and plans to move forward with larger efficacy studies in 2027.
Alnylam has shared an update on its Huntington's disease research programme following the presentation of new data at the European Huntington's Disease Network (EHDN) conference in Krakow.
The company's investigational treatment, brezosiran (previously known as ALN-HTT02), is designed to lower levels of the huntingtin protein associated with Huntington's disease. The treatment is still being studied and has not been approved for use by any regulatory authority.
According to Alnylam, results from the ongoing Phase 1b clinical trial showed that brezosiran reduced levels of mutant huntingtin protein in participants. The company reported that the treatment was generally well tolerated, with no treatment-related signs of brain inflammation or nerve cell damage observed during the study.
Participants already enrolled in the trial will be able to continue into an extension study, receiving brezosiran every six months for up to three years. This will allow researchers to continue monitoring the treatment's safety and effects over a longer period.
Alnylam has also announced plans for a larger placebo-controlled study, expected to begin in 2027 subject to regulatory agreement, to investigate whether brezosiran can slow the progression of Huntington's disease.
The company said it remains committed to working with the Huntington's disease community and keeping people informed as the research programme progresses.
Cath Stanley, BEM, Chief Executive, Huntington's Disease Association, said,
It is important to note that brezosiran remains an experimental treatment and further studies are needed to understand whether it can slow or prevent the progression of Huntington's disease, however we are pleased that there will be a new larger study.