Clene Inc. announced that it has finished the FDA‑recommended biomarker analyses for its lead ALS candidate, CNM‑Au8, using data from the NIH‑sponsored Expanded Access Program. The analyses, completed on December 3 2025, show statistically significant reductions in neurofilament light (NfL) and glial fibrillary acidic protein (GFAP) across 183 patients enrolled at eight U.S. sites.
The decline in NfL and GFAP is clinically meaningful because both proteins are established surrogate markers of neuronal injury and glial activation. In the EAP cohort, the average NfL reduction was 35 % and GFAP fell 28 %, with p‑values below 0.01 for both metrics. These results satisfy the FDA’s 2024 guidance that required demonstration of biomarker change as a surrogate for survival benefit.
Clene’s data also confirm a positive correlation between biomarker decline and overall survival. Patients whose NfL levels dropped by more than 30 % lived an average of 12 % longer than those with smaller changes, reinforcing the drug’s disease‑modifying potential and providing a robust surrogate endpoint for accelerated approval. The company’s analysis aligns with the FDA’s request to evaluate NfL trajectories in placebo switchers and to include additional biomarkers in the submission.
Clene has scheduled a Type C meeting with the FDA for the first quarter of 2026 to present the full dataset and discuss the accelerated‑approval pathway. The company also plans to file a New Drug Application in the same quarter, positioning CNM‑Au8 for potential market entry once the FDA accepts the biomarker evidence. The timing of the meeting and filing underscores the company’s intent to move quickly through the regulatory process.
Financially, Clene reported a net loss of $8.8 million in Q3 2025, a 9 % increase from the $8.0 million loss in Q3 2024, and revenue of $15,000, down sharply from $87,000 in the prior year. Cash and cash equivalents stood at $7.9 million as of September 30 2025, giving the company a runway into the second quarter of 2026. The modest revenue decline reflects the company’s focus on clinical development rather than commercial sales, while the loss highlights the need for additional financing to support the upcoming NDA and Phase 3 confirmatory trial.
Rob Etherington, President and CEO, said the biomarker data “make a strong case for accelerated approval” and that the company is “following the FDA’s roadmap” to bring a disease‑modifying therapy to patients. The announcement is expected to reinforce investor confidence in Clene’s scientific strategy, even as the company’s cash position remains tight and it seeks further capital to fund the next development milestones.
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