WVE - Wave Life gains most in more than a year after early-stage data for ALS drug

The shares of clinical-stage genetic medicine company, Wave Life Sciences (NASDAQ:WVE +29.6%) have recorded the best intraday gain in more than a year on Monday in reaction to favorable early-stage data for WVE-004, an experimental therapy for amyotrophic lateral sclerosis and frontotemporal dementia. The adaptive Phase 1b/2a FOCUS-C9 trial was designed to find the optimum dose level and frequency of WVE-004, an antisense oligonucleotide targeting C9orf72-associated ALS and FTD. The early findings have indicated that low, single doses of WVE-004 were linked to a durable decline of a key C9-ALS/C9-FTD biomarker, poly (GP) dipeptide repeat proteins in cerebrospinal fluid (CSF). A decline of GP in CSF implies the activity of WVE-004 in the brain and spinal cord, Wave Life Sciences (WVE) said. Following single 30 mg doses, all active treatment groups (10 mg, n=2 patients; 30 mg, n=4 patients; 60 mg, n=3 patients) demonstrated statistical significant reductions of poly(GP) versus

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Wave Life gains most in more than a year after early-stage data for ALS drug