Clene (CLNN) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company overview and lead asset

• Clinical-stage biotech focused on nanotherapeutics for neurodegenerative diseases, with 13 years of development experience.

• Lead asset is an orally administered nanotherapeutic targeting ALS, with over 1,250 patient-years of exposure and hundreds currently on compassionate use.

• Asset supports neuronal bioenergetics and reduces oxidative stress, aiming to improve neural function.

Clinical data and regulatory strategy

• Two phase II ALS studies completed; HEALEY study missed primary endpoint but achieved secondary (survival) and exploratory (clinical worsening, neurofilament light reduction) endpoints.

• Neurofilament light (NfL) reduction emerged as a surrogate endpoint, with consistent ~10% reduction observed in both phase II and expanded access programs.

• FDA requested demonstration that NfL reduction is reasonably likely to predict clinical benefit; recent data show statistically significant correlation between NfL reduction, survival, and functional outcomes.

Safety, tolerability, and manufacturing

• Asset has shown a strong safety profile with no serious drug-related adverse events over nearly seven years of use.

• Mild side effects include headache and GI symptoms; no known drug-drug interactions.

• Manufacturing is in-house, with preparations underway for commercialization.