ADDF and AFTD Partner to Support Wave Life Sciences’ FTD and ALS Clinical Program

The Alzheimer’s Drug Discovery Foundation (ADDF) and The Association for Frontotemporal Degeneration (AFTD) announced today that they have partnered to support Wave Life Sciences’ FOCUS-C9 Phase 1b/2a clinical trial investigating WVE-004 as a potential treatment for C9orf72-associated frontotemporal degeneration (C9-FTD), as well as amyotrophic lateral sclerosis (C9-ALS). The partnership provides an investment from ADDF and AFTD that will support the evaluation of fluid biomarkers, functional assessments, and digital biomarkers in FOCUS-C9, potentially leading to clinically meaningful endpoints to inform development of treatments for FTD.

ADDF and AFTD made the decision to support the FOCUS-C9 trial following a review of Wave’s clinical research application for the Treat FTD Fund, which supports the development of new medicines to treat FTD. Specifically, members of the Treat FTD Fund Joint Steering Committee, a panel of experts convened by ADDF in collaboration with AFTD, and ADDF’s Scientific Review Board reviewed and commented on the Phase 1b/2a study plan, preclinical data supporting the program, and credentials of the study team.

“This investment exemplifies many of our priorities—collaboration, innovative science, and developing more rigorous ways to conduct clinical trials,” said Howard Fillit, M.D., Co-Founder and Chief Scientific Officer at the ADDF. “We need to work together—as ADDF and AFTD have done for years—to expand our scientific knowledge about all neurodegenerative diseases so we can help deliver meaningful treatments for people with FTD, Alzheimer’s and other related dementias.”

“AFTD is proud to support, through the Treat FTD Fund, this innovative and potentially significant clinical trial,” said AFTD CEO Susan L-J Dickinson. “For so many people living with FTD, this trial represents hope for effective treatments and for making the journey easier for the next family facing this disease. Our ongoing collaborations with both ADDF and Wave Life Sciences point to a future free of this disease, and we are grateful to all the clinical investigators and persons diagnosed with FTD who will participate in this important research.”

The FOCUS-C9 trial is novel in that it is a “basket” type study designed to assess the effects of a genetically targeted treatment in patients with different disease phenotypes (FTD, FTD with ALS, or ALS) who share a common molecular etiology, as has been used in oncology trials but has yet to be applied in neurology and to the C9orf72 population specifically. Wave’s focus on C9-FTD makes this a unique program in the C9orf72 clinical research landscape. It is also unique in the use of novel oligonucleotide chemistry which has shown improved cellular and nuclear uptake.

“We are grateful to the panel of experts from ADDF and AFTD for their support and acknowledgement of the innovative approach we’ve taken to rapidly progress our clinical program,” said Michael Panzara, M.D., M.P.H., Chief Medical Officer and Head of Therapeutics Discovery and Development at Wave Life Sciences. “In addition to advancing WVE-004 as a novel, genetically-targeted treatment for FTD and ALS, we are looking forward to sharing the many learnings that will emerge from this trial with the broader medical and scientific communities.”

WVE-004 is a stereopure antisense oligonucleotide designed to selectively target transcriptional variants containing a hexanucleotide repeat expansion (G₄C₂) associated with the C9orf72 gene, thereby sparing C9orf72 protein. G₄C₂ expansions in C9orf72 are one of the most common genetic causes of the sporadic and inherited forms of ALS and FTD.