Clene (CLNN) Emerging Growth Virtual Conference summary

Company overview and mission

• Focused on developing therapies to improve mitochondrial health and protect neuronal function for neurodegenerative diseases, with a primary emphasis on ALS and ongoing interest in MS.

• Lead asset CNM-Au8 is a nanotherapeutic suspension designed to cross the blood-brain barrier and support neuronal housekeeping.

• Over 800 patients have been treated, including more than 500 through expanded access protocols.

Clinical data and regulatory engagement

• CNM-Au8 demonstrated statistically significant reductions in neurofilament and GFAP biomarkers, both linked to improved survival in ALS.

• Survival benefit observed in multiple studies, including a 94% risk reduction in the HEALEY study and durable effects out to 36 months.

• FDA provided three options for further evidence, leading to extensive biomarker and survival data submission.

• The agency is considering whether biomarker changes are reasonably likely to predict clinical benefit and may serve as confirmatory evidence for accelerated approval.

Upcoming milestones and timelines

• Type C FDA meeting expected by end of Q1, with meeting minutes anticipated early Q2.

• NDA filing targeted for end of Q2, with potential PDUFA date in Q3 and possible commercialization in early 2027.

• Confirmatory Phase III trial is planned, with the goal of providing patient access to CNM-Au8 as early as next year.