Results positive in study of antisense oligonucleotide for Huntington’s disease
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Wave Life Sciences has announced positive results from its phase 1b/2a clinical trial of WVE-003, a potential disease-modifying therapeutic for Huntington’s disease.
WVE-003 is a first-in-class, allele-selective antisense oligonucleotide designed to lower mutant huntingtin (mHTT) protein and preserve healthy, wild-type huntingtin (wtHTT) protein, according to a company press release.
The novel therapeutic is being examined in the SELECT-HD study, a global, multicenter, randomized, double-blind, placebo-controlled undertaking to assess safety and tolerability of single and multiple ascending doses.
In the multidose portion of SELECT-HD, participants were randomized 2:1 to receive either intrathecal doses of 30 mg WVE-003 (n=16) once every 8 weeks, or placebo (n = 7), with 12 weeks of follow up (total study period of 28 weeks).
Data showed that WVE-003 was generally safe and well-tolerated, with no serious adverse events reported; significant mHTT protein lowering was observed throughout the 28-week assessment period; at 24 weeks (8 weeks after last dose), mean mHTT lowering in cerebrospinal fluid (CSF) was 46% compared with placebo and at 28 weeks (12 weeks after last dose), mean mHTT lowering in CSF was 44% vs. placebo, supporting quarterly or less frequent dosing.
Researchers additionally found that most participants in the treatment group had neurofilament light protein (NfL) levels that were in the range of placebo or had NfL levels that increased and returned to the range of placebo.
“With these results, we have delivered the first-ever clinical demonstration of allele-selective silencing in any disease target,” Paul Bolno, MD, MBA, president and CEO at Wave Life Sciences, said in the release. “The translation of genetic insights and preclinical data in the clinic is also highly encouraging and reinforces the broader value of our pipeline.”
The company also said in the release that it would release data from studies regarding Duchenne muscular dystrophy and Alpha-1 antitrypsin deficiency later in 2024.