#USA #Cambridge #ALS #NeuroSense #PrimeC

NeuroSense Therapeutics Achieves Significant Milestone

NeuroSense Therapeutics Ltd., a clinical-stage biotechnology firm, has made substantial progress in its efforts to develop PrimeC, a groundbreaking treatment for amyotrophic lateral sclerosis (ALS). The latest update reveals that NeuroSense received encouraging feedback from the U.S. Food and Drug Administration (FDA) regarding its proposed Phase 3 clinical trial design. This positive development sets the stage for the company to take crucial steps toward addressing the urgent needs of those affected by this debilitating disease.

The pivotal meeting, known as the Type C meeting, was held between NeuroSense and the FDA to discuss the intricacies of the Phase 3 study. During the meeting, both parties engaged in productive dialogue about the trial's design, efficacy measures, safety assessments, and other critical factors. NeuroSense expressed satisfaction with the FDA's response, emphasizing that an understanding of the trial design is essential for meeting regulatory standards and striving for potential drug approval.

Recognizing the importance of this feedback, NeuroSense plans to finalize and submit the trial protocol in the first half of 2025, aiming to kick off patient enrollment around mid-2025. The study will encompass approximately 300 participants, utilizing a randomized approach and employing a 2:1 ratio of PrimeC to placebo. Characterized as a multi-center, multinational, prospective, double-blind, placebo-controlled study, it also features an open-label extension, allowing participants to transition to PrimeC following 12 months of treatment.

Alon Ben-Noon, the CEO of NeuroSense, highlighted the significance of this milestone in their drug development journey. He remarked on the FDA's feedback and expressed confidence that it reinforces the potential of PrimeC as a viable therapeutic option for ALS patients, whose therapeutic landscape currently remains limited, thus making this moment all the more pressing.

NeuroSense previously completed the PARADIGM study, a Phase 2b trial consisting of 68 participants across countries such as Canada, Italy, and Israel. This study evaluated the efficacy of PrimeC in slowing disease progression and improving survival rates for individuals diagnosed with ALS. The results of PARADIGM, combined with the FDA's positive reception of the forthcoming study design, lay a solid foundation for NeuroSense as it prepares for the next crucial phase of clinical trials.

Understanding ALS

Amyotrophic lateral sclerosis, commonly known as ALS, is a severe neurodegenerative disease that leads to total paralysis and ultimately death within 2 to 5 years following diagnosis. Currently, ALS affects over 5,000 new patients across the U.S. each year, contributing to a staggering annual disease burden exceeding $1 billion. Projections reveal an expected 24% increase in the number of individuals living with ALS by 2040 in both the U.S. and EU, highlighting the urgent need for innovative treatment solutions.

Key Features of PrimeC

PrimeC, NeuroSense’s flagship drug candidate, is an innovative oral formulation that combines two FDA-approved medications: ciprofloxacin and celecoxib. This unique combination has been designed to selectively target multiple pathological processes associated with ALS, including motor neuron degeneration, neuroinflammation, iron overload, and RNA dysregulation, with the goal of impeding ALS progression.

In earlier clinical trials, PrimeC demonstrated significant efficacy, including the reduction of functional and respiratory decline among participants. Due to its promise, it has been granted Orphan Drug Designation by both the FDA and the European Medicines Agency.

NeuroSense's Vision

NeuroSense is devoted to transforming the treatment landscape of debilitating neurodegenerative diseases such as ALS, Alzheimer's, and Parkinson’s. The company is grounded in a strategy that prioritizes the development of combination therapies capable of targeting various pathways relevant to these complex conditions. This multifaceted approach aims to meet the substantial unmet medical needs faced by patients worldwide.

As NeuroSense moves forward with the anticipated Phase 3 study, the incoming year holds great significance as they continue to seek effective solutions for ALS patients, underscoring a commitment to innovation in the field of biotechnology. By fostering relationships with regulatory entities like the FDA and focusing on research and development, NeuroSense is poised to pave the way for future advancements that can offer hope to those battling ALS.