ALS therapy moves closer to Health Canada filing planned for December
Neurosense says regulator raised no concerns about its PrimeC filing timeline
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- ALS is a progressive neurodegenerative disease that causes worsening muscle weakness and can lead to paralysis
- Symptoms can include increasing difficulty with movement, speaking, swallowing, and breathing.
- Neurosense plans to seek Canadian approval of PrimeC, its experimental oral therapy for ALS.
Neurosense Therapeutics plans to seek Health Canada approval by the end of the year for PrimeC, its experimental oral therapy for amyotrophic lateral sclerosis (ALS).
The planned new drug submission (NDS) filing follows completion of a pre-NDS process and finalization of the related meeting minutes. The process is designed to clarify regulatory requirements before a formal submission. Neurosense and Health Canada agreed on what information should be included in the NDS and how the application should be organized.
Health Canada filing targeted for December
According to Neurosense, Health Canada raised no concerns about the proposed timeline and advised the company to target early December for the filing.
“We now have an alignment with the Canadian regulator and a clear operational target,” Alon Ben-Noon, CEO of Neurosense, said in a company press release. “Our team is advancing the remaining clinical, biomarker, manufacturing and regulatory components of the NDS, with the objective of filing in December 2026.”
Neurosense is seeking to pursue the filing through Canada’s Notice of Compliance with Conditions pathway, which can allow earlier access to promising therapies for serious diseases with unmet medical needs based on promising clinical evidence.
Once the NDS is submitted, Health Canada will first screen it to determine whether it contains enough information for a full review. If the application is accepted for review, the regulator will conduct a comprehensive scientific evaluation of PrimeC’s data before deciding whether to approve the therapy in Canada.
“Canada represents an important potential market for PrimeC and, more importantly, an opportunity to bring a promising therapy to people living with ALS,” Ben-Noon said.
Conditional approval pathway could require more evidence
If PrimeC is authorized through the Notice of Compliance with Conditions pathway, Neurosense would be required to provide additional evidence to confirm its clinical benefits.
ALS is a progressive neurodegenerative disease in which the nerve cells that control movement become damaged and die. This causes worsening muscle weakness that can lead to paralysis, along with difficulties speaking, swallowing, and breathing.
Although there are a few treatment options available, they have modest clinical benefits, and there remains a substantial need for new ALS therapies.
PrimeC is an extended-release oral tablet containing fixed doses of the antibiotic ciprofloxacin and the anti-inflammatory celecoxib, both of which are approved for use in other indications. The combination is designed to slow disease progression by targeting inflammation and other biological processes thought to contribute to nerve cell damage in ALS.
The planned NDS filing is supported by evidence from the now-completed Phase 2b PARADIGM trial (NCT05357950) and its open-label extension, which evaluated PrimeC in adults with ALS. The trial randomized 69 adults, but one was later found to have been misdiagnosed, leaving 68 participants with ALS in the analysis.
Participants were randomly assigned to receive PrimeC or a placebo, taken as two tablets twice daily for six months. They could continue standard ALS treatments during the trial. Of those who completed the six-month dosing period, 96% entered a 12-month open-label extension in which everyone received PrimeC.
The PARADIGM study met its main efficacy goal of lowering levels of TDP-43, a protein that abnormally accumulates in nerve cells in nearly all ALS cases. PrimeC significantly lowered TDP-43 levels compared with placebo after six months, with the reductions sustained and becoming more pronounced over 18 months. Participants who received PrimeC for the full 18 months maintained lower TDP-43 levels than those who received placebo for six months before switching to PrimeC.
PrimeC data suggest slower ALS progression
Data spanning PARADIGM and its extension also suggested that PrimeC could significantly slow disease progression, as assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standardized measure of a person’s ability to perform daily living tasks. At 18 months, ALSFRS-R scores were an average of 7.92 points higher in participants who initially received PrimeC than in those who initially received a placebo.
Longer-term findings also suggested a survival benefit with earlier PrimeC treatment. After 18 months of follow-up, estimated median survival was 36.3 months among participants who started PrimeC at the beginning of the study, compared with 21.4 months among those who started on placebo and switched to PrimeC after six months — a difference of 14.9 months. In an analysis that accounted for differences between the groups at the start of the study, earlier PrimeC treatment was associated with a 65% lower risk of death.
In parallel with the NDS filing to Health Canada, Neurosense is preparing to launch PARAGON, a pivotal Phase 3 clinical trial that will evaluate PrimeC’s efficacy in a larger group of people with ALS. Late last year, the U.S. Food and Drug Administration (FDA) authorized the trial. PARAGON is expected to enroll about 300 people with ALS across the U.S. and Europe.
If Health Canada grants PrimeC authorization under the Notice of Compliance with Conditions pathway, PARAGON could provide the additional evidence needed to confirm the therapy’s clinical benefits. Positive PARAGON results could also support an application seeking FDA approval of PrimeC.
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