WVE-004’s Clinical Advantages for C9-ALS and C9-FTD: Michael Panzara, MD, MPH


The potential of a new investigational treatment for C9orf72-associated amyotrophic lateral sclerosis and frontotemporal could offer long-lasting effects for patients.

“The potential advantages of this particular compound are that…we would not have to administer it as frequently as some older generation compounds…there’s a lot of potential for patients with this compound, and the FOCUS-C9 study will help tell us if we’re on the right track.”

The limited treatment options for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) presents one of the biggest challenges for these patient populations; although, a new investigational treatment, WVE-004, may provide optimism for patients and their families. Michael Panzara, MD, MPH, chief medical officer, and head, therapeutics discovery and development, Wave Life Sciences, spoke with NeurologyLive about the molecule, which recently began dosing in the phase 1b/2a FOCUS-C9 study (NCT04931862). The treatment targets C9orf72-associated ALS (C9-ALS) and FTD (C9-FTD) and has shown a widespread distribution throughout the central nervous system, impacting the spinal cord, the cortex, and other areas of the brain. 

When studied in animal models during preclinical trials, 2 doses of WVE-004 were observed for up to 6 months, eliciting durable effects in reducing toxic dipeptide repeat proteins, namely polyGP. According to Panzara, the ability to replicate the effect in humans and administer infrequent, intrathecal doses can also aid in changing perspectives of both C9-ALS and C9-FTD, from “universally fatal” diseases to diseases that are manageable through the slowing of disease progression.

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