FDA authorizes new Phase 3 clinical trial of PrimeC for ALS
Study to test therapy in around 300 following promising Phase 2b results
- The FDA has authorized a Phase 3 trial of PrimeC, an experimental oral therapy for ALS.
- In a prior Phase 2b study, PrimeC slowed ALS progression by 37.4% and improved survival by 43% in key patient analyses.
- The new PARAGON trial will test PrimeC in about 300 people with ALS over a 12-month treatment period.
The U.S. Food and Drug Administration (FDA) has authorized Neurosense Therapeutics to begin a pivotal Phase 3 trial of its experimental oral therapy PrimeC for the treatment of amyotrophic lateral sclerosis (ALS).
The authorization follows the FDA’s positive feedback on the design of the PARAGON trial and its completed review of an amended investigational new drug application requesting clearance to test PrimeC. The company expects to enroll the first participant in the coming months, once the needed resources are in place.
“This FDA clearance marks a meaningful advancement for Neurosense and for people living with ALS. We believe this progress lays a strong foundation for additional achievements across several fronts in the near future,” Alon Ben-Noon, Neurosense’s CEO, said in a company press release.
ALS progression underscores the importance of developing new therapies
ALS affects how nerve cells communicate with muscles, causing progressive muscle weakness that makes everyday activities increasingly difficult.
While existing treatments can help manage symptoms and slow disease progression, their benefits are limited, and more effective therapies are still urgently needed.
“We recognize the significant unmet need of people living with ALS and remain committed to delivering a meaningful therapy through our efforts,” Ben-Noon said.
PrimeC is an extended-release tablet that contains a patented combination of the antibiotic ciprofloxacin and the anti-inflammatory medication celecoxib. It is designed to target inflammation and several other biological pathways thought to drive ALS. Both ciprofloxacin and celecoxib are approved in the U.S. for multiple indications.
The PARAGON trial builds on positive data from the completed Phase 2b PARADIGM (NCT05357950) study. In PARADIGM, 68 adults with ALS took either PrimeC or a placebo as two tablets twice a day for six months, followed by a year-long open-label extension in which all participants received PrimeC.
PrimeC showed encouraging effects on ALS progression in prior trial
Results showed that people who took PrimeC for six months experienced a slower disease progression on the ALS Functional Rating Scale-Revised (ALSFRS-R) compared with those on a placebo. The difference became significant among participants who strictly adhered to the study protocol — a group in which PrimeC slowed disease progression by 37.4%.
The benefits were sustained in the open-label extension. Compared with patients who started on a placebo and switched to PrimeC after six months, those who took PrimeC for a full year had a 43% higher survival rate. The benefits were more pronounced in those who adhered to the trial’s established protocol.
The pivotal Phase 3 clinical trial, PARAGON, is now expected to evaluate PrimeC’s efficacy in a larger patient population. The trial will run in the U.S. and European Union and is expected to include about 300 people with ALS.
Participants will be randomly assigned to receive either PrimeC or a placebo for 12 months. Afterward, all will have the option to enter an open-label extension to continue PrimeC treatment.
PARAGON will use an adaptive design, which allows for interim analyses that can help adjust the required sample size and evaluate early signs of benefit or lack of effect.
More information about PARAGON’s design and timelines — as well as about the status of PrimeC in Canada, where it is under regulatory review for potential early approval — will be shared during Neurosense’s investor webinar on Dec. 8.
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