Clene Nanomedicine has secured an in-person meeting with the U.S. Food and Drug Administration (FDA) to discuss the potential accelerated approval of CNM-Au8 for the treatment of amyotrophic lateral sclerosis (ALS). The meeting, scheduled before the end of November, will involve senior FDA officials and neurology experts to review data on ALS biomarkers, clinical endpoints, and survival data associated with CNM-Au8.
CNM-Au8 and Accelerated Approval Pathway
Clene is pursuing an accelerated approval pathway for CNM-Au8, which allows the FDA to approve therapies based on early clinical data suggesting potential effectiveness. However, further studies are required to confirm the treatment's benefits. This approach follows initial feedback from the FDA indicating that Clene's briefing package did not initially support accelerated approval, leading to further discussions and the agreement for an in-person meeting.
Mechanism of Action and Clinical Trials
CNM-Au8 is a gold nanoparticle suspension designed to support the energy demands of nerve cells and enhance their overall health and survival. The therapy has been evaluated in the HEALEY ALS Platform Trial and the RESCUE-ALS Phase 2 trial. While these studies suggest CNM-Au8 may slow the progression of ALS and extend survival, larger Phase 3 trials are typically required for full approval.
Biomarker Data and Survival Benefits
Clene is advocating for accelerated approval based on clinical trial data demonstrating that CNM-Au8 significantly reduces levels of neurofilament light chain (NfL), a recognized biomarker for nerve cell damage that correlates with disease progression. Data indicates that approximately half of ALS patients treated with CNM-Au8 experienced notable decreases in NfL levels. Among these patients, the risk of death was reduced by approximately 65% compared to those without decreased NfL levels.
Safety Profile and Patient Experience
To date, over 700 ALS patients have been treated with CNM-Au8, with no significant safety concerns or serious adverse events reported. Clene has expressed appreciation for the FDA's flexibility in considering a potential approval pathway for CNM-Au8, emphasizing the importance of biomarker data and clinical endpoints in understanding the therapy's impact.
