Wave Life Sciences terminates WVE-004 program

24 May 2023

US RNA medicines company Wave Life Sciences (Nasdaq: WVE) saw its shares close up 2.7% at $3.81 yesterday, despite announcing that it is discontinuing an important program.

The Cambridge, Massachusetts-based company announced top-line results from the Phase Ib/IIa FOCUS-C9 study evaluating WVE-004 as an investigational treatment for C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) (C9-ALS/FTD).

Maximal mean reduction in poly(GP) of 48-51% across dose cohorts and increased CSF neurofilament light chain (NfL) did not translate to clinical benefit on Revised ALS Functional Rating Scale (ALSFRS-R) or Dementia Staging Instrument plus National Alzheimer's Coordinating Center (NACC) frontotemporal lobar degeneration (FTLD) Behavior and Language Domains (CDR plus NACC FTLD).

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