WVE-004 Treatment for C9orf72 ALS and FTD: Merit Cudkowicz, MD

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The director of the Sean M. Healey & AMG Center for ALS outlined the trial, which will evaluate ideal dose level and frequency of the investigational treatment for patients with C9-ALS and C9-FTD.

“I think the potential for this drug, in this phase 1 trial, is that we’re going to identify the dose that hits target and is safe so that we can move forward to the pivotal efficacy trial.”

Dosing recently began for patients with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) in the phase 1b/2a FOCUS-C9 trial (NCT04931862). The study will evaluate a new investigational treatment, WVE-004, which was developed by Wave Life Sciences to focus specifically on patients with C9orf72-associated ALS (C9-ALS) and FTD (C9-FTD).

The chair of the clinical advisory board for the trial, Merit Cudkowicz, MD, spoke with NeurologyLive on the design of the trial, what makes it unique, and the potential for clinical outcomes. Cudkowicz, who is also director of the Sean M. Healey & AMG Center for ALS and the chief of neurology at Massachusetts General Hospital, commented on the trial’s adaptive, basket design, as it includes patients with C9-ALS, as well as patients with C9-FTD.

The “innovate design,” according to Cudkowicz, looks into polyGP dipeptide repeat proteins in the cerebrospinal fluid and the effectiveness of WVE-004 in reducing that marker. Clinical data from the study are expected to be generated through 2022, with dose escalation and dosing frequency dependent on analysis from an independent data safety monitoring board.