NeuroSense Therapeutics (NRSN) Status Update summary

Clinical development and trial updates

• Planning to initiate the pivotal PARAGON phase III ALS trial in mid-2026, with enrollment across the US, Europe, and Israel, targeting 300 participants and using a Bayesian adaptive design with interim analyses.

• FDA has cleared the PARAGON protocol, and operational preparations are underway; pre-NDS meeting with Health Canada is scheduled for April 2026 to align on regulatory strategy.

• The primary endpoint for PARAGON is ALSFRS adjusted for survival at 12 months, which is accepted by the FDA.

• Phase II PARADIGM study showed over 30% functional advantage and 58% reduction in risk of death for PrimeC, supporting phase III design.

• Patients can continue standard of care (Riluzole or Radicava) during phase III, balanced between arms to reflect real-world practice.

Regulatory and data strategy

• Health Canada submission will include new long-term survival data and additional biomarker data to support conditional approval.

• Accelerated approval pathways, such as breakthrough or fast track, are under consideration depending on data robustness.

• Biomarkers are integral to both clinical and regulatory strategy, demonstrating biological activity and target engagement; will be incorporated into phase III.

Alzheimer's disease program

• Proof of concept phase II RoAD study in Alzheimer's has completed database lock; top-line results expected in Q1 2026.

• The study enrolled eight participants and generated a diverse biological dataset, now ready for analysis.