Clene (CLNN) Emerging Growth Conference summary

Regulatory update and FDA engagement

• Ongoing discussions with the FDA focused on the Accelerated Approval pathway for ALS, with the agency requesting robust neurofilament light (NfL) data to demonstrate both correlation and causation with clinical benefit.

• Submission of a comprehensive New Drug Application (NDA) package is planned for the beginning of the fourth quarter, including clinical study reports and efficacy data.

• The FDA is expected to review the submission and potentially set a PDUFA date by year-end, with a commercialization decision possible by early summer 2027.

• The company has addressed all agency requests from the May meeting minutes and is finalizing the NDA submission.

Clinical data highlights

• Survival benefit is concentrated in patients with a neurofilament biomarker response, showing a 74% lower risk of death after 12 months compared to controls (p=0.1).

• Functional benefits, measured by ALSFRS and SVC, are significant in patients with neurofilament decline or stabilization (p=0.3 for ALSFRS, p=0.03 for SVC).

• Randomization-based causal analysis confirmed the survival benefit is linked to neurofilament responders, meeting FDA requirements.

• The drug has demonstrated long-term safety, with 1,250 collective patient years and some patients on therapy for up to 6.8 years.

Consistency across studies and programs

• A 10% reduction in neurofilament was observed in both the HEALEY ALS and NIH-sponsored expanded access programs.

• Similar neurofilament reductions were seen in the sicker EAP population and confirmed in the RESCUE-ALS study, supporting consistency of effect.

• The program remains unique in achieving a double-blind, statistically significant neurofilament reduction in phase II.