Developer to seek approval in Canada for ALS treatment aiding survival
Trial data show participants on combo therapy living more than 3 years
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- Neurosense Therapeutics announced it will seek approval in Canada for PrimeC, its oral treatment candidate for ALS.
- New trial data submitted to Health Canada showed a 14.9-month survival benefit with the combination therapy.
- The developer is now also planning a pivotal trial in the U.S. to further test its patented treatment.
Neurosense Therapeutics announced that it’s preparing a new drug submission to Health Canada — following a meeting with the regulatory agency — to seek approval of PrimeC, its combination therapy for amyotrophic lateral sclerosis (ALS), in the North American nation.
During that meeting, the U.S. biotech company presented additional findings from the Phase 2b PARADIGM trial (NCT05357950) — including data showing a median survival benefit of more than one year. The new data also provided evidence that PrimeC significantly reduced blood levels of TDP-43, a protein whose abnormal accumulation is a hallmark in most ALS cases.
The meeting also included perspectives from a Canadian ALS specialist and additional analyses, which Neurosense believes will further support its application for approval in Canada, according to a company press release.
“The additional evidence generated through the PARADIGM program … has significantly strengthened our planned submission package and reinforces our confidence in PrimeC’s potential to address the substantial unmet need faced by people living with ALS,” said Alon Ben-Noon, Neurosense’s CEO, specifically pointing out a “meaningful long-term survival outcome.” Long-term data show treated participants living longer than three years.
Ben-Noon said the meeting marked “an important milestone” in the company’s regulatory strategy and thanked Health Canada for its constructive engagement throughout the presubmission process.
The company also announced plans to launch a pivotal Phase 3 trial, dubbed PARAGON, following a green light from the U.S. Food and Drug Administration. That trial is expected to enroll about 300 people with ALS, mainly in the U.S., per Neurosense.
Median survival of over 1 year seen with ALS treatment
ALS is caused by the loss of motor neurons, the nerve cells that control voluntary muscle movement, which leads to muscle weakness that gradually worsens as the disease progresses.
PrimeC is an extended-release tablet containing a patented combination of the antibiotic ciprofloxacin and the anti-inflammatory medication celecoxib, both approved in the U.S. for other indications. The combo therapy is designed to target inflammation and other biological processes that drive ALS symptoms.
The PARADIGM study enrolled 68 adults with ALS, randomly assigned to receive PrimeC or a placebo, once daily for six months, as an add-on to standard ALS treatments. After completing the main trial, patients could enter an open-label extension in which all received PrimeC.
In the placebo-controlled portion of the trial, participants who received PrimeC tended to experience slower disease progression, though this difference was not statistically significant. However, among participants who closely adhered to the study protocol, disease progression was significantly slower — by 37.4% — for those treated with PrimeC.
Long-term follow-up also showed that participants treated continuously with PrimeC had an estimated median survival of 36.3 months, compared with 21.4 months for those who started the treatment after receiving a placebo for six months. Per the investigators, that corresponds to a 65% lower risk of death.
The company has now updated Health Canada with the new evidence, which includes the achievement of one of the study’s prespecified primary endpoints, or goals: the average difference in blood levels of TDP-43 between the PrimeC and the placebo groups after six months. The new data also showed a median survival benefit of 14.9 months, according to the company. Neurosense also submitted external natural history analyses and additional data supporting both PrimeC’s mechanism of action and clinical translation.
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