Wave Life Sciences Touts Encouraging Data From Duchenne Muscular Dystrophy Study

Wave Life Sciences Ltd WVE announced an update from the initial cohort of the Phase 1b/2a proof-of-concept study of WVE-N531 in three boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.
High muscle concentrations of WVE-N531 and exon skipping were observed six weeks after initiating biweekly multi-dosing at 10 mg/kg, achieving proof-of-concept in the study.
Related: Wave Life Sciences Shares Move Higher On Therapeutic Pact With GSK Targeting Genetic Disorders.
WVE-N531 also appeared safe and well-tolerated.
WVE-N531 resulted in a mean tissue concentration of 42 micrograms/gram (6.1 micromolar).
Plasma concentrations and other pharmacokinetic parameters following a single dose of 10 mg/kg demonstrated a half-life of 25 days, which may support monthly dosing.
Adverse events were mild. There were no serious adverse events, no trends in labs, and no oligonucleotide class-related safety events.
Price Action: WVE shares are down 3.47% at $5.01 on the last check Monday.