Clinical Studies to Evaluate Orphan Drug for Amyotrophic Lateral Sclerosis

In addition to safety and tolerability, a pair of clinical studies will evaluate the drug’s efficacy and ability to slow disease progression and to improve quality of life in patients with amyotrophic lateral sclerosis.

The initiation of 2 clinical studies have been announced to evaluate the benefit of a combination drug known as PrimeC, which aims to slow or halt disease progression in patients with amyotrophic lateral sclerosis (ALS).

The studies plan to enroll 45 patients with ALS, 15 participants at each site. In addition to safety and tolerability, the study will evaluate the drug’s efficacy and ability to slow disease progression and improve quality of life.

PrimeC targets 2 fundamental mechanisms underlying the pathogenesis of ALS. The 2 clinical studies were initiated following exceptional results achieved in pre-clinical models of ALS, according to the company press release. Preclinical data analysis of motor neurons, neuromuscular junctions, and aspects of the immune system demonstrate that PrimeC is neuroprotective, according to the release.

“PrimeC was shown to be very successful in pre-clinical studies and we are glad to be testing it in our clinic now, hopeful to see how it will affect the patients,” said Vivian Drory, professor and expert at Tel Aviv Sourasky Medical Center, in the release.

The FDA has granted an orphan drug designation to NeuroSense for the use of PrimeC as treatment for patients with ALS. The designation grants PrimeC 7 years of market exclusivity in the United States.

Reference

NeuroSense Therapeutics Receives Orphan Drug Designation for Amyotrophic Lateral Sclerosis (ALS) Drug, and Announces Initiation of Two Clinical Studies in ALS [news release]. Herzliya, Israel. Published February 6, 2020. https://www.biospace.com/article/releases/neurosense-therapeutics-receives-orphan-drug-designation-for-amyotrophic-lateral-sclerosis-als-drug-and-announces-initiation-of-two-clinical-studies-in-als/. Accessed February 6, 2020.