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NeuroSense Therapeutics Ltd. (NeuroSense), a late-clinical stage biotechnology company developing novel treatments for severe neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has completed the review of the Investigational New Drug (IND) amendment application and authorized the company to initiate the pivotal phase 3 clinical trial for the evaluation of its lead drug candidate, PrimeC, for the treatment of amyotrophic lateral sclerosis (ALS).
With the FDA's clearance, NeuroSense is preparing for trial initiation and aims to have its first patient enrolled in the coming months upon securing the strategic resources needed to launch the trial.
The global pivotal phase 3 trial, PARAGON, is powered at over 95% to achieve its primary endpoint and to expand upon the results of NeuroSense's phase 2b PARADIGM trial, which demonstrated promising clinical and biomarker outcomes and a favourable safety and tolerability profile.
"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," stated Alon Ben-Noon, chief executive officer of NeuroSense. "We recognize the significant unmet need of people living with ALS and remain committed to delivering a meaningful therapy through our efforts."
Based on prior successful discussions with the FDA and in line with its recent comments and recommendations, PARAGON is expected to be conducted in the US and EU and include 300 people living with ALS randomized in a ratio of 2:1 (PrimeC : Placebo). The prospective, double-blind, 12-month placebo-controlled trial, has an open label extension to evaluate safety and efficacy of PrimeC. The trial will employ an adaptive design allowing for interim analyses to optimize sample size and assess early efficacy and futility boundaries.
Amyotrophic lateral sclerosis (ALS) is an incurable neurodegenerative disease that causes complete paralysis and death within 2-5 years from diagnosis. Every year, more than 5,000 people are diagnosed with ALS in the US alone, with an annual disease burden of $1 billion. The number of people living with ALS is expected to grow by 24% by 2040 in the US and EU.
PrimeC, NeuroSense's lead drug candidate, is a novel extended-release oral formulation composed of a unique fixed-dose combination of two FDA-approved drugs: ciprofloxacin and celecoxib. PrimeC is designed to synergistically target several key mechanisms of ALS and Alzheimer's disease (AD) that contribute to motor neuron degeneration, inflammation, iron accumulation and impaired ribonucleic acid (RNA) regulation to potentially inhibit the progression of ALS and AD.
NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on discovering and developing treatments for patients suffering from debilitating neurodegenerative diseases.
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