FDA Refuses Neurology-Focused Clene's Accelerated Approval For Amyotrophic Lateral Sclerosis Treatment, Shares Fall

Clene Inc CLNN provided an amyotrophic lateral sclerosis (ALS) regulatory update from its recent meeting with the FDA to discuss CNM-Au8 for ALS, presenting initial clinical and Neurofilament Light Chain (NfL) biomarker results from the completed Phase 2 ALS studies. 

The FDA determined that the initial findings on biomarker NfL reduction from the Phase 2 programs were insufficient to support accelerated approval. 

Clene looks forward to providing supplemental data for further engagement with the FDA in the first half of 2024, including additional long-term clinical evidence and biomarker results of CNM-Au8's treatment benefit in people with ALS. 

Clene plans to demonstrate how CNM-Au8's mechanism of action is linked to the reduction in NfL and the association between observed NfL reductions and improved clinical outcomes in ALS patients, including increased survival time.

Concurrently, Clene reported new data from the 12-month long-term open-label extension (OLE) of the CNM-Au8 treatment arm in the HEALEY ALS Platform Trial.

Plasma neurofilament light chain (NfL), a blood-based biomarker associated with neurodegeneration, declined by 16% from baseline to 76 weeks of treatment. 

CNM-Au8 was associated with a 10% relative reduction in plasma NfL over the 24-week double-blind treatment period of the HEALEY ALS Platform Trial.

Under an assumption of a constant common treatment effect from CNM-Au8, treatment with CNM-Au8 demonstrated a 60% decreased risk of long-term all-cause mortality in participants initially randomized to treatment with CNM-Au8 compared to those originally randomized to placebo after adjusting for the estimated benefit received after switching to CNM-Au8.

Price Action: CLNN shares are down 41.80% at $0.30 on the last check Thursday.