Clene received final minutes from a Type C meeting with the U.S. Food and Drug Administration indicating the agency stated the company’s proposed data may be capable of supporting a New Drug Application under the accelerated approval pathway for the treatment of amyotrophic lateral sclerosis based on neurofilament light biomarker data.
The FDA noted NfL could potentially serve as a reasonably likely surrogate endpoint and reminded Clene that the submission must demonstrate an effect of CNM-Au8 on NfL and show that the magnitude of change is reasonably likely to predict clinical benefit. The submission would occur under Subpart H (21 CFR 314.510).
Clene expects to submit an NDA in the third quarter of 2026, and the agency requested additional information linking NfL reductions to clinical benefit, which the company has prepared and will include in the filing, according to the company.
The planned NDA will be supported by NfL biomarker and clinical data from the Phase 2 HEALEY ALS Platform Trial and its open-label extension, the Phase 2 RESCUE-ALS Trial, and an NIH-sponsored expanded access protocol. The company describes CNM-Au8 as an investigational first-in-class therapy targeting mitochondrial function and notes that CNM-Au8 has received FDA orphan drug designation for ALS.
Clene also intends to commence a Phase 3 confirmatory study in the first quarter of 2027.
