Clene (CLNN) said it plans to file a New Drug Application for its investigational ALS therapy CNM-Au8 at the start of the fourth quarter, seeking accelerated approval from the FDA. The announcement came during a fireside chat at the 12th Annual Cantor Fitzgerald Global Healthcare Conference on September 9, 2026.
CNM-Au8, an oral nanotherapeutic suspension, targets amyotrophic lateral sclerosis, a uniformly fatal disease affecting an estimated 35,000 patients in the U.S. The drug delivers bioenergetic metabolite nutrients to failing neurons to reduce reactive oxygen species and support mitochondrial function, according to CEO Rob Etherington.
In the Phase 2 HEALEY ALS Platform Trial, Clene’s arm enrolled 160 subjects—120 on the active drug and 40 on placebo—as part of 480 total across eight regimens in the 6-month double-blind study. While the trial missed its primary ALSFRS functional-response endpoint, CNM-Au8 was the only program among eight to achieve the secondary endpoint of survival, an exploratory endpoint for clinical worsening, and a statistically significant reduction in neurofilament light protein (NfL). The 10% NfL reduction was later reproduced in an NIH-funded expanded-access program involving more than 300 patients who were further along in disease progression.
Etherington noted that responders showed a 7-point improvement in ALSFRS scores, which he described as roughly three times the 2.3-point change observed with a previously approved competitor.
Safety data across programs total more than 1,250 collective patient-years and over 900 exposed patients through clinical trials and four FDA-approved expanded-access protocols. Some patients have remained on the drug since late 2019. Clene reported zero serious adverse events linked to the therapy, with side effects limited to occasional headaches and mild gastrointestinal symptoms, and no hepatic or renal toxicity. The drug is not hepatically metabolized and showed no interactions with standard ALS medications.
The company expects the FDA to take 60 to 73 days to determine a Prescription Drug User Fee Act date, potentially in late Q4 2024. That would initiate a 6-month review window pointing to a decision around June or July 2025. A confirmatory Phase 3 trial, RESTORE-ALS, is designed with survival as the primary endpoint and a 2-year follow-up for approximately 700 patients, expected to begin in the first half of 2025.
Clene received $45 million—nearly 10% of a $500 million congressional appropriation through the ACT for ALS act—to expand its compassionate-use program.
On the commercial front, Clene plans an independent launch in ALS, citing the concentrated nature of the patient population across roughly 50 U.S. centers of excellence. The company envisions a field force of high single digits to low teens of key account managers and is working with patient networks including I AM ALS and Synapticure. Manufacturing is handled in-house across four clean rooms being prepared for commercial supply. Etherington referenced Amylyx’s approach of bringing 4,000 individuals onto therapy in its first year using a targeted model.
Cash runway extends to the end of 2024, and Clene indicated that warrant tranches tied to PDUFA and regulatory approval milestones could generate additional capital if triggered. Etherington said, “The market cap that Clene is today will be very different tomorrow.”
