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Clene Announces Biomarker Analyses To Be Included in NDA Submission Supporting ALS Accelerated Approval

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  • Clene Inc. announced new post hoc analyses of CNM-Au8® data showing survival & functional benefits concentrated in ALS patients whose neurofilament light chain (NfL) biomarker levels declined or stabilized.
  • These findings address FDA questions from a March 2026 meeting & will be included in Clene's planned New Drug Application (NDA) for accelerated approval.
  • In the HEALEY ALS Platform Trial, CNM-Au8 30 mg-treated participants with declining or stable NfL had a 74% lower risk of death at 12 months & a 38% lower risk of death over the full follow-up period compared to controls.
  • Participants with NfL decline or stabilization in HEALEY demonstrated a survival advantage reaching nearly six months at four years of follow-up.
  • CNM-Au8-treated patients with NfL decline or stabilization scored significantly better on combined measures of survival & function, including the ALS Functional Rating Scale-Revised (ALSFRS-R) & Slow Vital Capacity (SVC).
  • A causal analysis using pre-treatment characteristics alone identified a 41% lower risk of death for predicted NfL responders on CNM-Au8 30 mg versus controls, with no significant benefit for predicted non-responders.
  • The RESCUE-ALS trial confirmed these trends, showing a 76% lower adjusted risk of death for NfL responders within the treated group & a 69% lower risk compared to real-world controls.
  • Independent observational cohorts (APST & ANSWER ALS) involving over 2,000 patients who never received CNM-Au8 established that a 10% NfL decline correlates with a 6% to 10% reduction in the hazard of death.
  • Modeling suggests even modest NfL reductions of 5% to 10% are associated with improved ALSFRS-R scores & reduced mortality hazard.
  • Negative controls (sex, BMI, GFAP, Tau) showed no association with clinical outcomes, confirming the benefit is specific to NfL response.
  • CNM-Au8 has a consistent safety profile across 1,280 participant-years of exposure, with no serious adverse events assessed as related to the drug as of August 10, 2026.
  • Clene plans to initiate the Phase 3 confirmatory trial RESTORE-ALS to prospectively evaluate the NfL biomarker relationship using baseline NfL levels for enrollment stratification.
  • The company intends to file its NDA in early Q4, asserting the totality of evidence meets the accelerated approval standard regarding NfL as a reasonably likely surrogate endpoint.