HERZLIYA, ISRAEL / ACCESSWIRE / January 30, 2024 / Amyotrophic lateral sclerosis (ALS) is a progressive neurological disease that kills the nerve cells controlling the voluntary movement of muscles. It is incurable and results in complete paralysis and ultimately death within two to five years from diagnosis. Each year, about 5,000 people in the U.S. are diagnosed with this devastating disease, amounting to 15 new cases daily, according to the ALS Association.
There are treatments on the market, but they don't do much to slow the progression of the disease or improve quality of life meaningfully enough. NeuroSense Therapeutics Ltd. (NASDAQ:NRSN), may have a better approach with its lead therapeutic candidate, PrimeC.
Dual-Action To Target ALS
Unlike current treatments available to ALS patients, PrimeC consists of specific doses of two FDA-approved drugs, which aim to work synergistically on several ALS targets. Celecoxib is used to reduce neuroinflammation, glutamate excitotoxicity, and oxidative stress, while Ciprofloxacin regulates microRNA synthesis and iron accumulation.
The top-line results of these two drugs combined showed a 29.2% decline in disease progression and a 13.3% slowing of the loss of respiratory function in NeuroSense's phase 2b ALS trial of PrimeC (PARADIGM). In the Per Protocol Population, an even more impressive outcome of a statistically significant 37.4% decline in disease progression and a 17.2% slowing of the loss of respiratory function was witnessed. The trial met its primary endpoint of safety and tolerability with results comparable to the placebo. That establishes a solid safety profile for PrimeC as it forges ahead in hopefully bringing the treatment to commercialization. "I am excited by the top-line clinical data from PARADIGM. The clinical advancement of a new therapy that helps slow down the progression of ALS, with the potential to preserve quality of life, has the capacity to significantly positively impact people living with ALS and their families," said Merit Cudkowicz, M.D., Chair of Neurology at Massachusetts General Hospital, the Director of the Healey & AMG Center for ALS, Julieanne Dorn Professor of Neurology at Harvard Medical School, and member of NeuroSense's Scientific Advisory Board.
NeuroSense: ALS Patients Want PrimeC
PrimeC seems to be proving to be popular with trial participants. NeuroSense said 96% of people in the trial who completed the 6-month portion chose to receive PrimeC through a 12-month open-label extension. What's more, all trial participants who completed the 18-month trial treatment requested to continue taking PrimeC via an Investigator Initiated Trial.
PARADIGM is a prospective, multinational, randomized, double-blind, placebo-controlled trial. Participants from Canada, Italy, and Israel were enrolled and were dosed for six months after being randomized 2:1 to receive PrimeC or placebo, respectively.
But it's not only NeuroSense that's optimistic about PrimeC to address what is a $3 billion a year market opportunity. An independent study performed at the Ichida Stem Cell Lab at the University of Southern California last year showed PrimeC performed among the best in improving motor neuron survival when compared to two FDA-approved ALS drugs as well as several other ALS drugs in development.
Major Milestones Expected In 2024
And that was all in 2023. This year, the company is gearing up for even more developments and milestones including results from a strategic collaboration with Biogen Inc. (NASDAQ: BIIB). With results expected soon, Biogen and NeuroSense are evaluating the impact PrimeC has on neurofilament levels in trial participants. Neurofilament is related to ALS disease progression. Once results are in, Biogen has the right of first refusal to co-develop and commercialize PrimeC for the treatment of ALS for a limited time. That's something investors may want to keep on their radar.
The first half of 2024 is also expected to see NeuroSense report primary endpoints of ALS hallmark biomarkers, TDP-43 and ProstaglandinJ2, to evaluate PrimeC's biological activity and target engagement. "The magnitude of this apparent improvement is especially noteworthy considering the natural history of ongoing disease progression. Furthermore, the most exciting aspects of these data include not only the statistical differences in ALSFRS-R score at the specified timepoints, but also that the improved trajectory of the ALSFRS-R decline was evident from the earliest timepoint, throughout the entire study period," said Jeffrey Rosenfeld, MD, PhD, Professor of Neurology at Loma Linda University and member of NeuroSense's Scientific Advisory Board. "The complexity of ALS pathology warrants a multi-drug therapeutic strategy and it is especially gratifying to see this combination therapy advance."
Up next for PrimeC is a phase 3 trial, and if things work out, marketing approval. To see that along, NeuroSense expects to have End of Phase 2 meetings with the FDA and the European Medicines Agency in the second quarter of 2024. It is also in talks with several big pharma companies focused on CNS therapeutics to explore potential strategic partnerships and business opportunities.
"The clinical efficacy seen in the topline PARADIGM trial results demonstrate PrimeC's potential to render a significant and meaningful clinical benefit to people living with ALS, as any slowing of progression of this incurable disease is meaningful from both a clinical and patient perspective," said Alon Ben-Noon, CEO of NeuroSense. "This is perhaps one of the most significant outcomes seen to date. We believe that these data in conjunction with hopefully correlative neurofilament readouts this month could lead to a strategic partnership and an expedited regulatory pathway for PrimeC towards the market."
Featured photo by Milad Fakurian on Unsplash.
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SOURCE: NeuroSense Therapeutics Ltd
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