CLNN News & Events

Clene announced receipt of final meeting minutes following its recent Type C meeting with the FDA. During the meeting and confirmed in the final meeting minutes, the FDA stated that Clene's "proposed data may be capable of supporting the submission and review of an [New Drug Application (NDA)] under the accelerated approval pathway for the treatment of ALS." The FDA reminded the company that the submission should demonstrate the effectiveness of an effect of CNM-Au8 on NfL and show that the magnitude of change in NfL is reasonably likely to predict clinical benefits in patients with ALS. Clene intends to submit its NDA in the third quarter of 2026, which will remain a matter of FDA review. The agency also noted that "NfL could potentially serve as a reasonably likely surrogate endpoint to support (an) accelerated approval." This submission would occur under the Subpart H accelerated approval pathway in ALS. The Agency has also requested that the company provide additional information in its NDA, including to support a connection between the reported magnitude of reduction in NfL and clinical benefit, which Clene has prepared and will include in the submission.

Reports FY25 revenue $200k, consensus $152k. "Our next key milestone is the upcoming Type C face-to-face meeting with the U.S. Food and Drug Administration on Clene's biomarker and survival data, which if positive, will facilitate filing of an new drug application under an accelerated approval pathway for ALS by the end of the second quarter of 2026," said Rob Etherington, President and CEO of Clene. "In parallel, we continue to advance plans to initiate our confirmatory Phase 3 trial in ALS, which is required to be "underway" by CNM-Au8 approval in ALS, as well as to continue working with the FDA on the initiation of a Phase 3 clinical trial using cognition as an endpoint in MS. Based on our combined findings to date, we continue to believe that patients across multiple neurodegenerative conditions may benefit from the improved mitochondrial function plus energy metabolism associated with CNM-Au8 treatment."

Clene and its wholly owned subsidiary Clene Nanomedicine issued a letter to stockholders outlining key anticipated regulatory and clinical milestones for CNM-Au8 in 2026. "AFollowing the completion of multiple Phase 2 clinical trials and continued support of open label expanded access programs, we have now treated over 800 patients with CNM-Au8, our orally administered nanocrystal suspension, encompassing one of the largest sets of clinical experience in ALS. The learnings and data that we have discovered place us at an exciting and critical juncture... Sufficient cash to fund operations into the fourth quarter of 2026... Based on the significance of our clinical data and our engagement with the FDA, we believe that CNM-Au8 is well suited for the accelerated approval pathway as described below: 1. Prolonged Survival and Associated Declines in Biomarkers of Neurodegeneration: We have generated extensive clinical data demonstrating prolonged survival in ALS associated with CNM-Au8 30mg treatment... 2. Favorable Safety and Benefit/Risk Profile: CNM-Au8's groundbreaking clean-surfaced nanotherapeutic suspension, an oral liquid that patients drink daily and tastes like water, has demonstrated a consistent safety and tolerability profile... 3. Confirmatory Phase 3 RESTORE-ALS Trial Planned to Begin Later in 2026: To confirm the survival benefit observed with CNM-Au8 30 mg treatment across several Phase 2 trials and to meet FDA requirements for the accelerated approval pathway, we are planning to dose the first patient in our confirmatory Phase 3 RESTORE-ALS trial later this year."

Clene announced that the U.S. Food and Drug Administration, FDA, has granted Clene an in-person Type C Meeting later this quarter. Key highlights: The FDA has granted an in-person Type C meeting during the first quarter of 2026; New independent analyses across large observational ALS cohorts demonstrate that modest NfL reductions are significantly associated with lower mortality risk, supporting NfL reduction as a candidate surrogate endpoint for accelerated approval; New exploratory findings demonstrate that in responders with IGFBP7 biomarker decline, CNM-Au8 30mg was strongly associated with 78% reduced mortality risk in the HEALEY ALS Platform Trial, consistent with emerging genetic evidence linking lower IGFBP7 to ALS reversals