CNM-Au8

Clene granted in-person meeting with FDA to discuss potential accelerated approval of CNM-Au8 for ALS, based on clinical data showing significant reduction in neurofilament light chain levels and improved survival. CNM-Au8, a gold nanoparticle suspension, has been tested in HEALEY ALS Platform Trial and RESCUE-ALS Phase 2 trial, with over 700 ALS patients treated and no significant safety concerns reported.

Clene Inc. secured an in-person meeting with senior FDA leadership to discuss CNM-Au8 for ALS, presenting biomarker, clinical endpoint, and survival data. The company has over 700 patient-years of safety data for CNM-Au8 with no significant concerns.

Despite challenges, the HEALEY ALS Platform Trial continues with optimism, as Clene Nanomedicine and Prilenia Therapeutics progress their ALS treatments based on positive secondary endpoint data. The trial, initiated by a $40 million donation from Sean Healey and AMG, aims to efficiently test multiple therapies simultaneously. While primary endpoints have not been met, the trial's design allows for quick identification of non-viable treatments, and positive outcomes in subgroups have supported further development for some candidates.

The ALS drug development pipeline is rich with promising therapies targeting TDP-43 pathology, including Biogen’s BIIB-105, AbbVie/Calico’s ABBV-CLS-7262, Denali’s DNL-343, and QurAlis’ QRL-201. Trials range from Phase 1 to Phase 3, with completion dates extending to 2026. These therapies aim to slow ALS progression by addressing TDP-43 mislocalization and aggregation, a hallmark of ALS pathology. The article also highlights the importance of clinical trials in advancing ALS treatment and the potential for FDA approval of these therapies.