NeuroSense Therapeutics is set to meet with the U.S. Food and Drug Administration (FDA) to discuss the Phase 3 clinical trial design for PrimeC, their investigational drug for Amyotrophic Lateral Sclerosis (ALS). The Type C meeting, scheduled for November 6, 2024, is a crucial step in securing potential approval for PrimeC, addressing a significant unmet need in ALS treatment. The company will also review its readiness for a future New Drug Application (NDA) submission, ensuring its regulatory strategy aligns with the FDA's requirements.
PrimeC's Promising Clinical Data
PrimeC, a novel extended-release oral formulation combining ciprofloxacin and celecoxib, has shown encouraging results in clinical trials. Data from the PARADIGM study, a Phase 2b trial, demonstrated a statistically significant slowing of disease progression (p=0.009) and a 43% improvement in survival rates compared to placebo. These findings suggest PrimeC's potential as a disease-modifying therapy for ALS.
Regulatory Strategy and Market Opportunity
In addition to the FDA meeting, NeuroSense is preparing to submit its regulatory dossier to Health Canada in Q2 2025, with a regulatory decision anticipated by Q1 2026. The company estimates the potential market opportunity for PrimeC in Canada alone to be approximately $100 million to $150 million in annual revenue.
ALS: An Unmet Medical Need
ALS is a progressive neurodegenerative disease characterized by the degeneration of motor neurons, leading to paralysis and death within 2-5 years of diagnosis. Approximately 5,000 people are diagnosed with ALS each year in the U.S., resulting in an annual disease burden of $1 billion. The ALS Functional Rating Scale-Revised (ALSFRS-R) is the most widely used tool to track disease progression. A single point change on the ALSFRS-R has a significant impact on ALS patients, such as the transition from independent feeding to requiring assistance or independent breathing to needing to use a machine ventilator.
PARADIGM Trial Details
The PARADIGM trial (NCT05357950) was a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b clinical trial of PrimeC in ALS. The trial included 68 participants living with ALS in Canada, Italy, and Israel. In the 6-month double-blind segment of the trial, the data showed clinically meaningful signs of efficacy with a 29% difference in favor of PrimeC vs placebo in analysis of the intent to treat (ITT) population. In the PP top-line analysis from PARADIGM, a statistically significant slowing of disease progression was observed with a 37.4% (p=0.03) difference in ALSFRS-R in favor of PrimeC vs placebo. Most patients enrolled in both the active and placebo arms of the trial were concurrently treated with Riluzole, the ALS standard of care medication, indicating PrimeC slowed disease progression well beyond the level afforded by the FDA approved ALS drug.
PrimeC's Mechanism of Action
PrimeC is designed to synergistically target several key mechanisms of ALS, including motor neuron degeneration, inflammation, iron accumulation, and impaired ribonucleic acid (RNA) regulation. It is composed of a fixed-dose combination of ciprofloxacin and celecoxib. PrimeC has been granted Orphan Drug Designation by both the FDA and the European Medicines Agency.
