Clene granted in-person FDA meeting on CNM-Au8 for ALS
Discussions to focus on data submitted in bid for accelerated approval
Clene has been granted an in-person meeting with the U.S. Food and Drug Administration (FDA) — to be held before the end of November, according to the company — to discuss a potential accelerated approval of CNM-Au8 as a treatment for amyotrophic lateral sclerosis (ALS).
Under such a pathway, the FDA may authorize a new therapy based on early clinical data suggesting it may be effective — though the developer would need to conduct additional testing to prove the treatment’s benefit.
The announcement comes a few days after the FDA informed Clene that the information the company provided in its briefing package did not support a submission for approval under the accelerated approval pathway. However, after further discussions, the regulatory agency agreed to a meeting in which experts will present data and answer questions related to CNM-Au8, Clene said in a company press release.
The in-person meeting will also involve senior leadership from the FDA, including the directors of the Office on New Drugs and the Office of Neuroscience, as well as a division of neurology review team.
“Clene looks forward to the opportunity to have experts present their views to the FDA and address questions on ALS biomarkers, related clinical endpoints, and survival data, all of which Clene believes are essential for the understanding of its CNM-Au8” therapy, the company stated.
CNM-Au8 not yet tested in usual larger Phase 3 clinical trials
CNM-Au8 is a gold nanoparticle suspension designed to support the energy needs of nerve cells — which have a high energy demand — and improve their general health and survival.
The therapy was examined in the HEALEY ALS Platform Trial (NCT04297683), a large study that’s testing several experimental ALS treatments simultaneously. It also was evaluated in the company-sponsored RESCUE-ALS Phase 2 trial (NCT04098406), which wrapped up in 2021.
Data from these studies have supported the idea that CNM-Au8 may slow the progression of ALS symptoms and extend survival, but larger Phase 3 trials would generally be required before the therapy could be considered for approval by the FDA.
Clene, however, is seeking an accelerated approval for CNM-Au8 based on clinical trial data demonstrating that the therapy candidate significantly lowers levels of neurofilament light chain (NfL), an established biomarker of nerve cell damage. NfL levels correlate with disease progression.
Specifically, the findings suggest that about half of ALS patients experienced notable decreases in NfL levels. Among these patients, the risk of death was reduced by approximately 65% when compared with patients who did not experience decreased NfL levels with CNM-Au8.
To date, more than 700 ALS patients have received CNM-Au8, and no significant safety concerns or serious adverse events related to treatment have been identified, according to Clene.
In the release, Clene noted that it is “appreciative of the FDA’s use of process and regulatory flexibility” in discussing a potential approval pathway for CNM-AU8.
About the Author
