Clene Inc. (NASDAQ:CLNN) saw its stock price climb 20% on Monday after the company announced plans to pursue an accelerated approval route for its ALS treatment candidate, CNM-Au8, following a recent Type C meeting with the U.S. Food and Drug Administration.
According to the final minutes from that FDA meeting, the agency stated that Clene’s "proposed data may be capable of supporting the submission and review of an [NDA] under the accelerated approval pathway" for CNM-Au8 based on neurofilament light, or NfL, biomarker data.
The FDA acknowledged that NfL has potential to serve as a reasonably likely surrogate endpoint to support accelerated approval under the Subpart H pathway. Clene said it intends to submit a New Drug Application for CNM-Au8 in the third quarter of 2026.
The agency asked Clene to include additional material in the NDA to establish a link between the magnitude of NfL reduction reported and an actual clinical benefit for patients. Clene stated that it has prepared the requested information and will include it in the filing.
The planned NDA will draw on both biomarker and clinical datasets. Specifically, Clene will include NfL biomarker and clinical data from the Phase 2 HEALEY ALS Platform Trial and its open-label extension, the Phase 2 RESCUE-ALS Trial, as well as data from the NIH-sponsored Expanded Access Protocol for CNM-Au8.
"We are encouraged by the FDA’s careful evaluation of the benefits and risks associated with Clene’s ALS drug candidate, CNM-Au8, including the biomarker data the Company provided," said Rob Etherington, President and CEO of Clene.
Looking ahead, Clene plans to initiate a Phase 3 confirmatory study for CNM-Au8 in the first quarter of 2027. The company also notes that CNM-Au8 previously received Orphan Drug Designation from the FDA for the treatment of ALS.
The FDA's response in the meeting minutes suggests a regulatory pathway that could allow CNM-Au8 to be reviewed under accelerated approval, contingent on the agency's assessment of the submitted biomarker and clinical evidence. The company’s near-term calendar includes compiling the requested linkage data for the NDA submission in Q3 2026, and preparing for a Phase 3 confirmatory trial beginning in early 2027.
Market reaction to the announcement was immediate, reflected in the 20% rise in CLNN shares on the Nasdaq following the disclosure.
Clene’s filing will rely on a combination of trial data and the company’s prepared analyses to address the FDA’s request tying NfL reductions to clinical outcomes, while the Phase 3 study is positioned to provide the confirmatory evidence the agency may seek.