NeuroSense Therapeutics Ltd. secured U.S. Food and Drug Administration clearance to begin its pivotal Phase 3 study, PARAGON, for its lead candidate PrimeC in amyotrophic lateral sclerosis (ALS). The approval removes a critical regulatory hurdle and positions the company to advance PrimeC toward potential market approval.
The PARAGON trial will enroll 300 patients across the United States and the European Union, randomizing participants 2:1 to PrimeC versus placebo in a double‑blind, 12‑month, placebo‑controlled design that includes an open‑label extension. The adaptive design allows interim analyses to optimize sample size and assess early efficacy and futility boundaries, giving the company flexibility to adjust the study as data accrue.
PrimeC is a combination therapy of ciprofloxacin and celecoxib, both FDA‑approved drugs, and has received orphan drug status from the FDA and EMA. The combination targets multiple ALS mechanisms—inflammation, iron accumulation, and microRNA regulation—providing a novel therapeutic approach in a disease with limited treatment options.
Phase 2b PARADIGM data, which underpinned the Phase 3 launch, showed a 33% reduction in disease progression and a 58% improvement in survival. These results, achieved in a global, double‑blind, 12‑month study, give the company a strong foundation for the larger trial and support the hypothesis that PrimeC can alter disease trajectory.
Management emphasized the significance of the clearance. CEO Alon Ben‑Noon said, “This FDA clearance marks a meaningful advancement for NeuroSense and for people living with ALS. We believe this progress lays a strong foundation for additional achievements across several fronts in the near future.” The statement underscores confidence in the trial design and the company’s ability to conduct a multinational study.
The ALS market is projected to reach between USD 510.9 million and USD 754.2 million by 2035, growing at a CAGR of about 3.6%. PrimeC’s potential approval would address a substantial unmet need and could capture a meaningful share of this expanding market.
Investor sentiment reflected the regulatory milestone. In early trading on the announcement day, analysts maintained a ‘Buy’ stance for the company, citing the trial’s design and the company’s track record. Some analysts noted the company’s ongoing financial challenges, but the regulatory approval was viewed as a positive step toward future revenue generation.
The company will host an investor webinar on December 8, 2025, to provide further details on the PARAGON trial and its strategic implications.
The FDA clearance also signals confidence in NeuroSense’s operational capabilities, as the agency approved the company’s IND amendment and the multinational study plan. This approval is a key milestone that could accelerate the company’s path to commercialization and potential revenue generation in the ALS market.
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