Key Takeaways and Future Opportunities for WVE-004: Michael Panzara, MD, MPH

“Approaching these diseases as a spectrum…[and] approaching the treatment like you would a spectrum of phenotypes of a single underlying disorder—that is something that when we've worked with the ALS and FTD communities, they have appreciated us bringing that to the surface.”

The phase 1b/2a FOCUS-C9 trial, which will evaluate the safety and efficacy of WVE-004, an investigational treatment for C9orf72-related frontotemporal dementia (C9-FTD) and amyotrophic lateral sclerosis (C9-ALS), was designed with both patients and physicians in mind. According to Michael Panzara, MD, MPH, the trial aims to bring physicians from both treatment areas together, encouraging them to approach ALS and FTD as a spectrum of a single underlying disease. The study was designed “with both groups in the room,” ensuring that necessary aspects were included in the clinical trial, Panzara told NeurologyLive.

If successful, WVE-004 would broaden limited treatment options for patients with both C9-ALS and C9-FTD, and according to Panzara, who is also chief medical officer, head of therapeutics discovery and development at Wave Life Sciences, growing interest in both fields generates opportunities for patients to be involved in trials.

Clinical data from the FOCUS-C9 trial is anticipated to be generated through 2022. At multiple points throughout the course of the trial, data will be evaluated by an independent safety monitoring board, to further inform dosing frequency and escalation.