Wave Life Sciences Jump As Low Doses of WVE-004 Lowers Disease Biomarker In Neurological Indications

Wave Life Sciences Ltd's WVE FOCUS-C9 study of WVE-004 shows reductions of poly(GP) dipeptide repeat proteins in cerebrospinal fluid (CSF) with low, single doses of WVE-004. 

• The Phase 1b/2a trial is assessing WVE-004 in C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD). 
• Poly(GP) is a key C9-ALS/C9-FTD disease biomarker that, when reduced in CSF, indicates WVE-004's engagement of target in the brain and spinal cord.
• Reductions in poly(GP) were observed across all active treatment groups (10 mg, n=2 patients; 30 mg, n=4 patients; 60 mg, n=3 patients), reaching statistical significance versus placebo (n=3 patients) after single 30 mg doses, with a 34% reduction in poly(GP) at day 85. 
• Related: Wave Life Sciences, Takeda Discontinue Research On Preclinical CNS Programs.
• At the analysis time, none of the patients dosed with 60 mg had reached day 85.
• The poly(GP) reduction in the 30 mg single dose cohort does not appear to have plateaued.
• Thus Wave will extend the observation period to around six months to identify the maximum reduction of poly(GP) and duration of effect of low single doses. 
• The FOCUS-C9 trial has been adapted to include additional patients receiving 20 mg and 30 mg single doses of WVE-004.
• Adverse events (AEs) were balanced across treatment groups, including placebo, and mostly mild to moderate intensity. 
• Dosing in a multidose cohort at 10 mg monthly is also well underway, and additional single and multidose data are expected throughout 2022.
• Price Action: WVE shares are up 21.2% at $2.46 during the market session on the last check Monday.