Top-line results of PARADIGM trial into PrimeC expected by year’s end

Safety and efficacy findings will cover 69 patients in Phase 2b study

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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NeuroSense Therapeutics is planning to soon announce top-line safety and efficacy results from its PARADIGM trial, which is testing PrimeC in people with amyotrophic lateral sclerosis (ALS).

News of trial findings being expected in December follows the dosing of a final patient in the randomized portion of the Phase 2b trial (NCT05357950). A total of 69 people have received the experimental therapy or a placebo for six months, and most (96%) have chosen to enter an open-label extension where all are being treated with PrimeC for up to one year.

The company also plans to report findings across a number of PARADIGM biomarker analyses in the first half of 2024.

“We are excited to soon begin sharing the results of the double-blind segment of our Phase 2b trial,” Alon Ben Noon, NeuroSense’s CEO, said in a company press release.

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PARADIGM trial assessing a longer-acting formulation of PrimeC

“We would like to thank the trial participants, their caregivers and families, as well as the sites’ Principal Investigators and study coordinators for their tremendous contribution to PARADIGM,” Ben Noon added.

PrimeC is an oral, fixed-dose combination of two medications approved in the U.S. for other indications, with established safety profiles. One is ciprofloxacin, an antibiotic, and the other is celecoxib, an anti-inflammatory.

Together, these compounds are designed to target several key mechanisms that contribute to the nerve cell degeneration that causes ALS. Specifically, it aims to block inflammation, iron accumulation, and impaired RNA regulation to slow disease progression.

In a previous Phase 2a clinical trial (NCT04165850) in Israel, an initial formulation of PrimeC was found to be safe and well tolerated in 15 ALS patients. The therapy also slowed functional and respiratory decline compared with an historical group of untreated patients, and lowered ALS biomarkers, particularly TDP-43.

PARADIGM is testing a longer-acting formulation of PrimeC that releases the medications over an extended period of time, requiring less frequent dosing.

Participants, whose symptoms of muscle weakness began less than 30 months before screening, were randomly assigned to either PrimeC or a placebo for six months. The active medication was given at a total daily dose of 1,496 mg, taken as two tablets twice daily, and patients were allowed to continue their standard ALS therapies.

Efficacy measured by changes in disability, lung health with treatment

The study’s primary goals are to determine PrimeC’s safety and its effects on key ALS blood biomarkers, TDP-43 and prostaglandin J2. Secondary goals include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), a measure of functional disability, changes in lung health and quality of life, and survival outcomes.

Data covering these secondary efficacy measures, as well as safety results, are expected to be announced in December, and the biomarker findings by June. NeuroSense also expects to report, by the close of March, results of a blood biomarker analysis being conducted under a collaboration with Biogen.

This analysis is looking at changes in plasma levels of neurofilaments, established markers of nerve damage, among people treated with PrimeC. Biogen is conducting the analysis, and under the agreement has a right of first refusal to co-develop and co-commercialize PrimeC depending on the results.

PrimeC has been designated an orphan drug in the U.S. and the European Union, a status meant to accelerate the development and regulatory review of therapies for rare diseases.