Clene (CLNN) investor relations material

Regulatory interactions and clinical data

• Multiple meetings with the agency culminated in March, with feedback that survival data alone is insufficient for accelerated approval; a biomarker is required.

• Concordant neurofilament light (NfL) reduction (~10%) was demonstrated in both HEALEY and NIH-funded EAP studies, addressing agency requests.

• Statistically significant benefits in ALS functional rating scale (ALSFRS) and breathing capacity were observed in patients with NfL stabilization or decline.

• The agency requested evidence that the modest 10% NfL reduction is reasonably likely to predict clinical benefit, which was supported by new data.

• Unique concurrent control data from the HEALEY trial allowed robust comparison, as other arms showed no benefit and were discontinued.

Product candidate and mechanism

• The asset is an oral suspension of gold nanocrystals, acting as a biocatalyst rather than a classical pharmaceutical.

• Nanoparticles cross the blood-brain barrier and drive bioenergetic changes, reducing reactive oxygen species and increasing NAD and ATP.

• The approach targets neuron survival and function in ALS, aiming to maintain cardiac and pulmonary function.

Clinical development and regulatory pathway

• Filing of a new drug application (NDA) is targeted for early Q4, with all clinical study reports nearly complete.

• The agency will review data from five phase II programs, preclinical studies, and in-house manufacturing details.

• A confirmatory phase III trial with survival as the primary endpoint is planned, enrolling 800 subjects over two years, and must begin before approval.

• The agency has been consistent in its review team, and the company is prepared for a potential advisory committee meeting, though none is currently scheduled.