HERZLIYA, Israel—NeuroSense Therapeutics has announced the initiation of two clinical studies to evaluate the benefit of PrimeC, the company’s combination drug which aims to slow or halt amyotrophic lateral sclerosis (ALS) progression for ALS patients. NeuroSense recently received Orphan Drug Designation from the U.S. Food and Drug Administration for the use of PrimeC to treat ALS. The designation grants PrimeC seven years of market exclusivity in the U.S.
“PrimeC has intriguing preclinical data and we expect a good safety profile,” noted Jeremy Shefner, chair of Neurology at the Barrow Neurological Institute (BNI). “It is gratifying to be able to offer this novel experimental therapy to patients.”
Prof. Vivian Drory is conducting a Phase 2a study in Tel-Aviv Sourasky Medical Center (TASMC). A similar study is being conducted in the U.S. at two sites: BNI in Phoenix, Arizona, led by Prof. Jeremy Shefner; and Columbia University in New York City, led by Dr. Jinsy Andrews. The studies plan to enroll 45 patients, 15 at each site. In addition to safety and tolerability, NeuroSense will evaluate the drug’s efficacy and ability to slow disease progression and improve quality of life.
“PrimeC, developed by NeuroSense, was shown to be very successful in preclinical studies and we are glad to be testing it in our clinic now, hopeful to see how it will affect the patients. Patients are excited to participate in the trial and the recruitment rate is very high,” said Drory, an ALS expert from TASMC.
PrimeC targets two fundamental mechanisms underlying the pathogenesis of ALS. The two clinical studies were initiated following results that PrimeC achieved in preclinical models of ALS. In these studies, conducted on two different zebra-fish models — each with a different ALS-causing mutation — the transgenic fish were treated either with PrimeC or left untreated.
The swimming abilities of the PrimeC-treated ALS fish increased drastically compared to the controls, and significantly more than with any other compound previously tested in these models. And the analysis of motor neurons, neuromuscular junctions and aspects of the immune system in the treated ALS fish indicated that PrimeC is neuroprotective.
“We are excited by the potential PrimeC holds to benefit ALS patients. We are pleased to be evaluating it in these patients, providing them hope while testing the drug’s efficacy. The special connection the company has with patients and their families contributes to the way we progress in our development, and we are thankful for this opportunity,” added Alon Ben-Noon, CEO of NeuroSense. “We hope and expect to see positive interim results within several months.”
