PrimeC Safe, Well-tolerated; Earns US Patent Allowance

PrimeC is safe and well-tolerated for the treatment of amyotrophic lateral sclerosis (ALS) and showed signs of slowing disease progression in patients, findings from a recently-completed Phase 2a study show.

All 15 participants in the NST002 clinical trial (NCT04165850) have chosen to continue receiving the treatment in an extension study, NeuroSense Therapeutics announced in a press release.

As part of the company’s plans to make this treatment available to ALS patients within the next four years, NeuroSense is now outlining a placebo-controlled trial with an upgraded formulation to confirm the benefits of PrimeC in ALS.

“This study outcome provides important validation of our pharmaceutical pipeline and particularly PrimeC,” said Alon Ben-Noon, NeuroSense’s CEO.

“We are very satisfied with the study’s results and are currently planning a robust pivotal trial to confirm the efficacy of PrimeC in a double-blind placebo-controlled study with fine-tuned doses and a unique upgraded formulation, which maximizes the synergistic effect between the compounds of our combination drug PrimeC,” added Ben-Noon.

Along with completing the study, NeuroSense received a patent allowance for PrimeC in the U.S., meaning that the U.S. Patent and Trademark Office believes the treatment qualifies for a patent.

The company has submitted one additional patent for the new PrimeC formulation and expects to submit more, based on the results of upcoming trials.

PrimeC is a combination treatment composed of the medications ciproflaxacin and celecoxib. Ciprofloxacin is an antibiotic often used to treat bacterial infections, and celecoxib is a prescription non-steroidal anti-inflammatory agent, similar to aspirin and ibuprofen.

Used together, these medicines target two key features of ALS that contribute to motor neuron degeneration — inflammation of the nervous system and impaired RNA regulation.

PrimeC has won orphan drug designation in both Europe and the United States.

After successful studies of PrimeC in fish models of ALS, NeuroSense launched two clinical trials to investigate PrimeC in humans, including the recently completed NST002 trial in Israel and a similar Phase 1 trial in the U.S.

The Israeli trial enrolled 15 adults with ALS, who received a capsule of PrimeC three times a day for 12 months.

The main objective was to investigate PrimeC’s safety and tolerability, which were determined by the occurrence of adverse side effects or the decision to stop treatment at any point prior to the study’s end.

Investigators also measured PrimeC’s efficacy, using both the ALS Functional Rating Scale – Revised, which is an established measure of functional status, and vital capacity, a standard measure of lung function.

Interim results reported last year showed that, starting three months after their first dose, patients began to experience slower declines in functional status and lung function than what usually was seen in historical controls. These trends continued to be observed following nine months of treatment, and now were confirmed with 12 months of data.

“We are excited by the potential efficacy of the drug, despite the limited size of the trial population, and are hopeful and enthusiastic for the initiation of a larger trial,” said Vivian Drory, MD, principal investigator of the NST002 study.