#United States #Cambridge #ALS #NeuroSense #PrimeC

NeuroSense Secures FDA Approval for Pivotal ALS Trial

NeuroSense Therapeutics Ltd., a biotechnology company focused on developing innovative therapies for severe neurodegenerative diseases, has gained significant momentum with the recent clearance from the U.S. Food and Drug Administration (FDA). This authorization allows NeuroSense to move forward with its pivotal Phase 3 clinical trial for its lead drug candidate, PrimeC, aimed at treating amyotrophic lateral sclerosis (ALS).

FDA Clearance Highlights

The FDA's clearance was granted after a thorough review of the Investigational New Drug (IND) amendment application, which signifies that NeuroSense is now ready to initiate the next critical phase of its clinical testing. The company is working diligently to enroll its first patient in the trial soon after ensuring that necessary resources are in place. This ambitious trial, known as PARAGON, is designed to evaluate PrimeC's effectiveness and safety for individuals living with ALS.

Expanding Upon Previous Successes

The pivotal Phase 3 trial will be a continuation of NeuroSense's promising results from the earlier Phase 2b PARADIGM trial. Preliminary findings from PARADIGM showcased encouraging clinical outcomes as well as biomarker improvements, along with an acceptable safety and tolerability profile. The Phase 3 study has been structured to have a robust statistical power of over 95% to meet its primary endpoint, reinforcing the foundation established in prior trials.

“It’s an exciting time for NeuroSense and for all those affected by ALS,” said Alon Ben-Noon, the CEO of NeuroSense. “This FDA clearance is a significant advancement towards fulfilling our commitment to provide meaningful therapies for ALS patients. We recognize the pressing needs of these individuals and are determined to make strides in offering effective solutions.”

Trial Design and Logistics

The PARAGON clinical trial will be conducted in both the U.S. and the European Union. It aims to recruit 300 participants diagnosed with ALS, with a random allocation ratio of 2:1 between PrimeC and a placebo. This double-blind, 12-month study will control for bias and features an open-label extension to further assess PrimeC's safety and efficacy. The trial is also designed with an adaptive framework to allow for interim analyses to fine-tune sample sizes and evaluate early indications of efficacy and futility.

NeuroSense will provide further insights into the specifics of the PARAGON trial's design and timelines at an investor webinar scheduled for December 8, 2025. Interested parties can register for the webinar through NeuroSense’s official channels.

Understanding ALS

Amyotrophic lateral sclerosis, commonly known as ALS, is an aggressive neurodegenerative disease characterized by rapid loss of muscle control, leading to complete paralysis and, ultimately, death within 2 to 5 years post-diagnosis. Each year, around 5,000 new ALS cases are reported in the U.S., contributing to a staggering annual burden of about $1 billion. Projections indicate that this number will escalate significantly, with an expected 24% increase in ALS cases by 2040 in the U.S. and European markets.

PrimeC: A Unique Approach

PrimeC represents a pioneering treatment, being a novel extended-release oral formulation that combines two FDA-approved drugs—ciprofloxacin and celecoxib. This fixed-dose combination is designed to target multiple pathogenic mechanisms implicated in ALS and Alzheimer's disease (AD). By addressing inflammation, iron accumulation, and RNA regulation, PrimeC aims to interrupt the disease's progression effectively.

About NeuroSense

NeuroSense Therapeutics is a clinical-stage biotechnology firm dedicated to identifying and developing therapeutic options for patients impacted by debilitating neurodegenerative disorders. The company is committed to addressing one of the most pressing medical challenges of our time, as effective treatments for diseases like ALS, Alzheimer's, and Parkinson's remain largely elusive. Through strategic research and innovative therapeutic combinations, NeuroSense aspires to pioneer new pathways forward in neurodegeneration therapies.

In conclusion, NeuroSense’s progress with PrimeC and its upcoming clinical trial presents a beacon of hope for ALS patients and signifies an essential step in advancing treatments within the neurodegenerative disease arena.