NeuroSense Therapeutics Ltd. (Nasdaq: NRSN) is set to advance its amyotrophic lateral sclerosis (ALS) treatment, PrimeC, through a strategic partnership and positive regulatory feedback. The company has entered a binding term sheet with a major global pharmaceutical firm, securing substantial financial support for the Phase 3 clinical trial. Simultaneously, the U.S. Food and Drug Administration (FDA) has provided encouraging feedback on the design of the planned Phase 3 study for PrimeC, paving the way for a pivotal trial in mid-2025. These developments mark significant milestones in the quest to address the urgent unmet medical need in ALS, a devastating neurodegenerative disease.
Financial Agreement with Global Pharma
The binding term sheet with the global pharmaceutical company includes a substantial upfront payment to NeuroSense upon signing a definitive agreement, along with funding for the Phase 3 clinical trial. The agreement also encompasses regulatory and net sales milestone payments, and a tiered royalty structure reaching double-digit percentages on annual net sales. Under the agreement, the pharmaceutical company would have exclusive rights to distribute, market, promote, sell, and develop PrimeC for ALS in certain key markets, while NeuroSense retains full rights in other territories. The finalization of a definitive agreement is anticipated in the first quarter of 2025.
FDA's Positive Feedback on Phase 3 Design
NeuroSense has concluded a Type C meeting with the FDA, focusing on the design of the proposed Phase 3 clinical study for PrimeC. The FDA's positive feedback on the trial design is a critical step, indicating alignment with regulatory expectations and the potential to generate sufficient data for drug approval. The company plans to submit the final protocol to the FDA in the first half of 2025, with the aim of commencing enrollment of approximately 300 patients in mid-2025. The Phase 3 study is expected to be a randomized, multi-center, multinational, prospective, double-blind, placebo-controlled study, with an open-label extension.
PrimeC: A Novel Therapeutic Approach for ALS
PrimeC is a proprietary fixed-dose combination of two FDA-approved drugs, ciprofloxacin and celecoxib, uniquely formulated to enhance bioavailability and provide synergistic effects that target multiple ALS disease pathways. The drug is designed to address key mechanisms of ALS, including motor neuron degeneration, inflammation, iron accumulation, and impaired ribonucleic acid (RNA) regulation. PrimeC has already demonstrated positive safety and efficacy in NeuroSense's Phase 2b PARADIGM clinical trial, strengthening confidence in its potential to address the urgent unmet medical need in ALS.
PARADIGM Trial: Promising Results
The PARADIGM trial was a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b clinical trial of PrimeC in ALS, involving 68 participants across Canada, Italy, and Israel. During the first six months, 45 participants received PrimeC, and 23 received a placebo. This was followed by a 12-month open-label extension where all participants received PrimeC in a blinded manner. Notably, most patients were concurrently treated with Riluzole, the standard of care medication for ALS, suggesting that PrimeC slowed disease progression beyond the level afforded by the existing FDA-approved drug. Data from the 18-month readout showed that, compared with patients who were initially assigned to a placebo before switching to PrimeC, disease progression was slowed by 33% in those who received PrimeC from the start, with an increase of 58% in the survival rate.
Addressing a Critical Unmet Need
ALS is an incurable neurodegenerative disease characterized by complete paralysis and death within 2-5 years of diagnosis. Each year, over 5,000 people are diagnosed with ALS in the U.S. alone, contributing to an annual disease burden of $1 billion. With the number of people living with ALS expected to grow by 24% by 2040 in the U.S. and EU, the development of effective treatments is of paramount importance. PrimeC has been granted Orphan Drug Designation by both the U.S. FDA and the European Medicines Agency, underscoring its potential to address this critical unmet need.
Early Commercialization in Canada
NeuroSense is also pursuing early commercialization approval for PrimeC in Canada, with a dossier submission to Health Canada expected in Q2 2025 and a regulatory decision anticipated by Q1 2026. The company estimates a potential market opportunity of $100M to $150M in peak annual revenue in Canada.
