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Clene Reports Full Year 2023 Financial Results and Recent Operating Highlights
Initiated discussions with FDA regarding accelerated approval pathway for CNM-Au8® for the treatment of ALS with the objective of submitting an NDA in
About this update from Clene Inc.
[{"type":"text","content":"Initiated discussions with FDA regarding accelerated approval pathway for CNM-Au8® for the treatment of ALS with the objective of submitting an NDA in 2024Released long-term data from the open-label extension of the HEALEY ALS Platform Trial in which CNM-Au8 demonstrated statistically significant reductions of plasma neurofilament light chain (NfL) levels at 76 weeks relative to placebo (18 months from randomization, p=0.023) as well as a 60% decreased risk of long-term all-cause mortality using rank-preserving structural failure time model analysis (>18 months, p=0.0167)Demonstrated a statistically significant survival benefit (p=0.0001) in Expanded Access Program (EAP) participants (n=220) treated with CNM-Au8 compared to historical ALS disease progression controls with decreased risk of all-cause mortality ranging from 57%-68% Reported additional long-term data from the Phase 2 VISIONARY-MS trial in which CNM-Au8 treatment was associated with vision improvement as measured by low contrast letter acuity (LCLA) across both eyes vs. baseline through 35 months from randomization, p18 months, p=0.0167) in participants originally randomized to CNM-Au8 30 mg compared to those originally randomized to placebo using the rank-preserving structural failure time model (RPSFTM). Additionally, CNM-Au8 30 mg treatment had greater overall treatment effect in delaying the time to morbidity events in the highest risk participants based on baseline NfL levels. These biomarker and survival data from the long-term open label extension together reinforce evidence of a treatment effect consistent with delayed time to event results observed in the original double-blind Phase 2 period analyses. In December 2023, Clene announced that it met with the FDA to discuss the regulatory pathway for CNM-Au8 for the treatment of ALS and to explore the potential for an accelerated NDA filing under an accelerated approval pathway. The FDA determined that the initial findings on biomarker NfL reduction from the Phase 2 programs submitted earlier in 2023 were insufficient to support accelerated approval at that time. Clene is currently preparing supplemental data on NfL biomarkers and improved clinical outcomes in people living with ALS to address the FDA’s questions, including increased survival time. Clene plans to submit additional data to the FDA to advance the...