ALS Treatment Breakthrough: Clinical Trial Data Shows Promise for NeuroSense Therapeutics' Dual-Drug Approach

The Case For A New ALS Therapy

Amyotrophic lateral sclerosis remains one of medicine’s most urgent challenges. Diagnosed in roughly 5,000 Americans each year, this progressive neurological disease attacks the nerve cells controlling voluntary muscle movement, inevitably leading to complete paralysis and death within two to five years of diagnosis. Current therapeutic options on the market provide limited benefit, offering insufficient disease slowing or meaningful improvement in patient quality of life. This unmet medical need has created significant pressure within the pharmaceutical industry to develop more effective interventions.

NeuroSense Therapeutics Ltd. (NASDAQ:NRSN) appears to be moving in a promising direction with its lead candidate, PrimeC—a combination therapy designed to address ALS through a fundamentally different mechanism than existing treatments.

How PrimeC Works Differently

Rather than relying on a single active ingredient, PrimeC employs a synergistic dual-drug formulation combining two FDA-approved medications at specific dosing levels. Celecoxib addresses neuroinflammation, glutamate excitotoxicity, and oxidative stress, while Ciprofloxacin targets microRNA synthesis and iron accumulation—two pathways implicated in ALS progression. This multi-target approach reflects growing recognition that ALS pathology is sufficiently complex to warrant combination therapy strategies.

PARADIGM Trial Delivers Compelling Efficacy Signals

NeuroSense’s Phase 2b trial, known as PARADIGM, enrolled participants from Canada, Italy, and Israel in a randomized, double-blind, placebo-controlled design. After six months of treatment, participants were randomized 2:1 to receive PrimeC or placebo. The results exceeded expectations. The combination therapy achieved a 29.2% decline in disease progression and a 13.3% slowing of respiratory function loss compared to placebo—outcomes that become even more striking when examining the per-protocol population, which demonstrated a 37.4% decline in disease progression and 17.2% slowing of respiratory decline.

Beyond efficacy, PrimeC met its primary safety endpoint, with a tolerability profile comparable to placebo. This solid safety foundation addresses a critical regulatory requirement as the program advances toward commercialization.

Patient Enthusiasm Suggests Strong Market Potential

Perhaps the most telling metric comes from patient behavior. Among trial participants who completed the initial six-month phase, 96% voluntarily elected to continue PrimeC through a 12-month open-label extension. By the end of the full 18-month treatment period, all completing participants requested to continue therapy via an Investigator Initiated Trial—an unusually high retention rate that reflects both efficacy perception and tolerability.

Independent validation comes from recent research conducted at the Ichida Stem Cell Lab at the University of Southern California, which demonstrated that PrimeC ranked among the most effective compounds tested for improving motor neuron survival when benchmarked against two FDA-approved ALS medications and several other developmental candidates. The ALS treatment market represents approximately $3 billion in annual opportunity, and the scale of improvement evident in PARADIGM positions this approach as potentially game-changing.

Critical Milestones Ahead In 2024

The near-term catalyst pipeline appears robust. Strategic collaboration results with Biogen Inc. (NASDAQ:BIIB) are expected imminently, with the partnership evaluating PrimeC’s impact on neurofilament levels—a biomarker directly correlated with disease progression. Biogen has retained right of first refusal for co-development and commercialization rights, suggesting confidence in the therapeutic potential.

In the first half of 2024, NeuroSense Therapeutics plans to report primary endpoints for ALS biomarkers TDP-43 and ProstaglandinJ2, providing additional biological evidence of target engagement and PrimeC’s mechanism of action.

Regulatory engagement is accelerating as well. The company expects End of Phase 2 meetings with both the FDA and European Medicines Agency by Q2 2024—meetings typically required before advancing to Phase 3 evaluation. NeuroSense is simultaneously engaging with major pharmaceutical companies specializing in CNS therapeutics regarding potential strategic partnerships and commercialization opportunities.

What This Means For The Field

Merit Cudkowicz, M.D., Chair of Neurology at Massachusetts General Hospital and member of NeuroSense’s Scientific Advisory Board, characterized the clinical data as compelling, emphasizing that any therapy capable of slowing ALS progression while preserving quality of life addresses an enormous unmet medical need. Jeffrey Rosenfeld, MD, PhD, from Loma Linda University, highlighted that the magnitude of improvement is particularly noteworthy given ALS’s natural disease trajectory, and that the therapeutic benefit emerged consistently from the earliest timepoint throughout the entire study period.

With Phase 3 trials anticipated and regulatory pathways potentially accelerated through continued collaboration, NeuroSense Therapeutics appears positioned to advance PrimeC toward market authorization—representing meaningful progress in an area where patient options remain desperately limited.