NeuroSense’s CMC earn FDA OK ahead of PrimeC Phase 3 trial
Results from Phase 2b PARADIGM trial expected next month
The U.S. Food and Drug Administration (FDA) has signed off on the chemistry, manufacturing, and controls (CMC) clinical development plan for NeuroSense Therapeutics‘ lead candidate for amyotrophic lateral sclerosis (ALS) ahead of a planned Phase 3 study.
The Phase 3 trial of PrimeC is due to start in 2024 pending positive results from the company’s ongoing PARADIGM Phase 2b clinical trial (NCT05357950), whose results are expected in December. CMC refers to the manufacturing practices and product specifications that must be followed and met to ensure product safety and consistency between batches.
“This confirmation from the FDA of our CMC strategy for a future pivotal Phase 3 clinical trial and commercial launch of PrimeC in the treatment of ALS marks a critical milestone in our drug development program,” Alon Ben-Noon, CEO of NeuroSense, said in a company press release. “It sets the stage for a smooth transition, particularly as we anticipate clinical efficacy top-line results (secondary endpoints) from our Phase 2b trial very soon.”
PrimeC is an oral, fixed-dose combination of ciprofloxacin, an antibiotic, and celecoxib, an anti-inflammatory. Both are approved in the U.S. for other indications. The compound’s combined action would block key mechanisms in ALS to slow disease progression.
What is PARADIGM trial assessing?
An initial formulation of PrimeC was deemed safe and well tolerated, in a Phase 2a clinical trial (NCT04165850) with 15 ALS patients. After treatment, patients saw a slower decline in their functional and respiratory abilities over a historical group of untreated patients. Key ALS blood biomarkers, particularly TDP-43, also were reduced.
PARADIGM is assessing the safety and effectiveness of a longer-acting formulation of PrimeC, which would release the medications over time, requiring less frequent dosing.
A total of 69 ALS patients from Canada, Italy, and Israel were randomly assigned to a placebo or PrimeC for six months, while continuing with their standard ALS treatments. PrimeC was given at a total daily dose of 1,496 mg, taken as two tablets twice daily.
The trial will also measure PrimeC’s impact on key ALS blood biomarkers, TDP-43 and prostaglandin J2. Changes in functional disability, assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), as well as lung health, quality of life, and survival will also be assessed.
After completing six months of treatment in the trial’s randomized part, most patients (96%) joined an open label extension, where they are receiving PrimeC for a year.
The company plans to announce safety results and data from the trial’s secondary measures in December. Biomarker analyses, including those conducted in collaboration with Roche, are due in the first half of 2024.
PrimeC has received orphan drug status in the U.S. and the European Union. The designation is meant to accelerate the development and regulatory review of rare disease therapies.
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