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End of Phase 2 meeting with the FDA and EMA expected in Q2 2024 and several biomarker study results anticipated as early as Q1 2024, as the Company advances partnership discussions
Statistically Significant Reduction in ALS Disease Progression in Phase 2b PARADIGM Trial
Top-line results in the Phase 2b PARADIGM trial of PrimeC revealed a statistically significant slowing of disease progression in ALS patients, compared to placebo. The study demonstrated a 37.4% (p=0.03) difference in the FDA-approved ALS functional rating scale revised (ALSFRS-R), compared to placebo, and a strong safety profile. These data highlight a novel approach to treating ALS, with potential implications in other neurodegenerative conditions. PrimeC is a unique fixed-dose combination of ciprofloxacin and celecoxib, which may utilize complementary and synergistic mechanisms to preserve motor neuron health in ALS.
Jeffrey Rosenfeld, MD, PhD, Professor of Neurology at Loma Linda University and member of NeuroSense's Scientific Advisory Board, stated that the magnitude of this apparent improvement is especially noteworthy considering the natural history of ongoing disease progression. Furthermore, Prof. Rosenfeld commented that 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. "These data provide an exciting justification and indication for a pivotal Phase 3 trial. The complexity of ALS pathology warrants a multi-drug therapeutic strategy and it is especially gratifying to see this combination therapy advance. The pending data on biomarkers of neurodegeneration will also be of great interest, as we continue to better understand the benefits of PrimeC and promote this approach."
"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. 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," stated Alon Ben-Noon, NeuroSense's CEO.
In line with the Phase 2b findings, an independent study performed at the Ichida Stem Cell Lab at University of Southern California in 2023, 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 in an in vitro study utilizing induced pluripotent stem cells (iPSCs).
Primary Biomarker Endpoints Anticipated in 2024
NeuroSense expects to report results from a strategic collaboration with Biogen in the second half of January, evaluating the impact of PrimeC on neurofilament levels in participants enrolled in PARADIGM. Upon receipt of results, Biogen has the right of first refusal to co-develop/commercialize PrimeC for the treatment of ALS for a limited time. The Company also expects to report primary endpoints of ALS hallmark biomarkers, TDP-43 and ProstaglandinJ2, to evaluate PrimeC's biological activity and target engagement, in the first half of 2024.
Next Steps in PrimeC Development Pathway
The FDA has confirmed NeuroSense's chemistry, manufacturing, and controls (CMC) development plans, in advance of an expected Phase 3 pivotal trial and potential subsequent marketing approval. The Company expects End of Phase 2 meetings to be scheduled with the FDA and the European Medicines Agency for Q2 2024.
Further advancing its platform combination drug therapy platform, in 2023 NeuroSense also reported positive biomarker results in Parkinson's and Alzheimer's Disease (AD). The Company recently commenced enrollment in RoAD, its Phase 2, randomized, prospective double-blind, single-center, placebo-controlled study to evaluate safety, tolerability, target engagement, and efficacy of PrimeC in patients with mild to moderate AD.
Finally, driven by the significant milestones achieved during the year, NeuroSense has entered into advanced discussions with fundamental biotech VCs and several big pharma companies focused on CNS therapeutics, to explore potential strategic partnerships and business opportunities.
"I thank our devoted team, invaluable collaborators, and the inspiring ALS community for their dedication and support in reaching this crucial milestone in 2023 and positioning the Company for success in 2024. We remain, as always, committed to the development of improved approaches to treat serious neurological disorders. These efforts are not just about advancing medical science; they are about improving patient's lives. Our focus continues to be on delivering optimal value to our shareholders and stakeholders and making a meaningful difference in the healthcare landscape," added Ben-Noon.
Posted In: NRSN
