Clene (CLNN) Emerging Growth Conference 89 summary

Regulatory progress and clinical data

• Granted an in-person Type C FDA meeting this quarter to discuss biomarker and survival data for ALS treatment.

• Biomarker analyses showed significant reductions in neurofilament and GFAP, both linked to longer survival in ALS patients.

• Survival benefit consistently demonstrated across multiple data sets, despite missing the ALSFRS functional endpoint in the HEALEY study.

• Data package includes three key biomarkers: neurofilament, GFAP, and IGFBP, all showing concordant responses and association with survival.

• Plan to file a New Drug Application for accelerated approval if FDA concurs, targeting submission in Q2 and potential commercialization early next year.

Accelerated approval pathway and next steps

• Accelerated approval pathway pursued using biomarker as surrogate endpoint, following precedent set by Biogen's Tofersen.

• Confirmatory phase III study planned, focusing on survival as the primary endpoint.

• FDA meeting scheduled for later this quarter, with outcome determining NDA filing timeline.

• If NDA is accepted, potential FDA decision and commercialization could occur by early next year.

Financial update and investor participation

• Recent tranche financing raised $6 million at $6.50 per share, extending cash runway by an additional quarter.

• Additional tranches could raise $7 million at $7 and $15 million at $12.50, triggered by regulatory milestones.

• Three major healthcare investors participated: Vivo, Castellan, and Boxer Capital.

• Funding now supports operations through potential approval in a positive scenario.