Clene Inc. shares advanced 2.44% on Wednesday after the company outlined a clearer regulatory pathway for its lead amyotrophic lateral sclerosis drug CNM-Au8, following feedback from the U.S. Food and Drug Administration.
The FDA indicated that data from Clene’s clinical programs may support a New Drug Application under the accelerated approval pathway for ALS, the company said at the Emerging Growth Conference. Management noted that agency feedback received in early May emphasized the need for robust neurofilament light chain data demonstrating a connection to clinical benefit.
Rob Etherington, Clene’s president and CEO, said the FDA’s written guidance confirmed the agency’s view that the proposed dataset could underpin a submission. “They actually said, ‘We need you to demonstrate the evidence of effectiveness of CNM-Au8 on reducing neurofilament and show us that the magnitude of change in neurofilament is connected to clinical benefit,’” he told conference attendees.
Clene plans to submit its NDA for CNM-Au8 in the fourth quarter, with a potential PDUFA review target by the end of 2024. If the timeline holds, the drug could reach the market by early summer 2027.
The company highlighted survival and functional benefits observed in clinical data. In the HEALEY ALS trial, patients randomized to CNM-Au8 showed a 74% lower risk of death after 12 months compared to concurrent controls, with the benefit persisting over a four-year follow-up period. Neurofilament light chain reductions of 10% were replicated across the HEALEY trial, RESCUE-ALS, and an NIH-sponsored expanded access program, even among sicker patients.
Functional improvements were also noted in neurofilament responders, including statistically significant gains on the ALS Functional Rating Scale and slow vital capacity, a measure of breathing function. Safety data accumulated 1,250 patient-years, with some patients remaining on continuous treatment for up to 6.8 years since 2019.
Clene’s stock closed at $4.19, up $0.10, following the update. The shares have traded between $3.88 and $13.50 over the past 52 weeks, with a market capitalization of approximately $54.6 million.
