The biotech company plans to roll out 2 clinical trials for patients with amyotrophic lateral sclerosis following the orphan drug designation for PrimeC.
NeuroSense Therapeutics announced it has received an orphan drug designation from the FDA for PrimeC, a combination drug aimed to slow disease progression in amyotrophic lateral sclerosis (ALS), and thus will proceed with the initiation of 2 clinical studies.
This announcement comes less than a month after the FDA cleared the HEALEY-ALS platform trial. That trial will test zilucoplan, verdiperstat, and nanocrystalline gold, marking the first ALS trial to examine the effects of multiple drugs.
"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," Vivian Drory, MD, director, Neuromuscular Diseases Unit, Department of Neurology, Tel Aviv Sourasky Medical Center, said in a statement. “PrimeC has intriguing preclinical data and we expect a good safety profile. It is gratifying to be able to offer this novel experimental therapy to patients."
The 2 studies will not only test the tolerability and safety of the drug, but assess the efficacy and ability to slow the disease’s progression as well as improve patient quality of life. In total, the company plans to enroll 45 patients, 15 at each of 3 sites.
The first phase 2b study will be conducted by Drory and colleagues at TASMC in Israel, and the other will be conducted at 2 different sites: Barrow Neurological Institute (BNI) in Phoenix, Arizona, by Jeremy Shefner, MD, PhD, and Columbia University in New York City, by Jinsy Andrews, MD, MSc.
PrimeC received the orphan drug designation following its successful results in 2 different zebra fish models. In those preliminary trials, the ALS-mutated fish demonstrated a significant increase in swimming abilities when treated with PrimeC compared to when left untreated. The drug’s main mechanism of action targets 2 fundamental pathways that underlie the pathogenesis of ALS.
“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,” Alon Ben-Noon, CEO of NeuroSence Therapeutics, said in a statement.
The drug orphan designation gives PrimeC a periof of 7 years of market exclusivity in the US. Ben-Noon added that the company expects to see positive interim results reported within several months.
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: NeuroSense Therapeutics; Published February 6, 2020. finance.yahoo.com/news/neurosense-therapeutics-receives-orphan-drug-140000174.html. Accessed February 6, 2020.