Antisense Drug Trial Provides Key Insights About Huntington’s Disease
The halted GENERATION HD1 (GENHD1) study of the investigational antisense drug tominersen provided insights about Huntington’s disease staging and informed future clinical trials, researchers said. The phase III study involved 791 manifest Huntington’s disease patients who received intrathecal tominersen 120 mg either every 8 weeks or every 16 weeks, or placebo. Based on an overall […]
Investigational ALS Drug May Have Clinical Benefit, FDA Staff Says
Tofersen, an investigational antisense drug developed to treat amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene (SOD1-ALS), may have clinical benefit, FDA reviewers indicated in briefing documents ahead of an advisory committee meeting. On Wednesday, the Peripheral and Central Nervous System Drugs Advisory Committee will vote on whether evidence supporting a […]
Investigational ALS Drug Betters Biomarkers, but Can It Improve Motor Functions?
Tofersen, an investigational antisense oligonucleotide developed to treat an inherited form of amyotrophic lateral sclerosis (ALS), changed biomarkers and curbed neurodegeneration but did not improve motor control and muscle strength at 6 months, the phase III VALOR trial showed. In a subgroup of patients with fast-progressing SOD1 ALS, the 28-week change in scores on the […]