Future Plans for WVE-004 in C9-FTD and C9-ALS: Michael Panzara, MD, MPH

“Should we see a change in the biomarker here, where the only explanation for that reduction is that we have done it with our drug and reduced an actual component of the pathophysiology of the disease, that would tell us that we are doing exactly what we set out to do, we are altering the natural course of this toxin, which will tell us that this is very likely to predict clinical effects in larger studies and longer studies.”

As treatment options are few for those with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), investigators are working to identify viable treatment routes and generate a meaningful impact for this patient population. Michael Panzara, MD, MPH, chief medical officer, and head, therapeutics discovery and development, Wave Life Sciences, spoke with NeurologyLive on the key outcomes to be observed for WVE-004, an investigational treatment that recently began dosing as part of the phase 1b/2a FOCUS-C9 trial (NCT04931862).

Investigators will observe the efficacy of WVE-004 in influencing underlying pathologies for C9orf72-associated ALS (C9-ALS) and FTD (C9-FTD) and measure biomarker outcomes, namely the effect on polyGP, a toxic dipeptide repeat protein. Panzara discussed the need to advance drug development for both C9-ALS and C9-FTD, with WVE-004 hopefully seeing success in slowing down disease progression and expanding approaches for developing additional treatments.