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NeuroSense Therapeutics have announced that it partnered with PhaseV, a pioneer in causal machine learning for clinical trial analysis and optimisation, with respect to the planned Phase 3 trial of PrimeC as a treatment for amyotrophic lateral sclerosis.
As part of the collaboration, PhaseV conducted an independent analysis of NeuroSense's PARADIGM Phase 2b study using a causal ML and predicts a high probability of success in multiple subgroups for the planned Phase 3 trial of PrimeC as a treatment for ALS. The external results provide important insights that will significantly inform study design, patient enrolment and ensure cost-effectiveness.
"There remains a critical need for new innovative approaches to address this devastating neurodegenerative disease," said Alon Ben-Noon, CEO of NeuroSense. "Our recently announced subgroup analysis from the PARADIGM study is very encouraging and suggests the potential of PrimeC to change this reality. Through our initial collaboration with PhaseV, we gained an even greater understanding of the effect of PrimeC across multiple patient subgroups. We will apply these insights to optimise the design of our Phase 3 study with the aim of maximising meaningful clinical results that will differentiate PrimeC in the market. We plan to continue to collaborate with PhaseV as we develop our Phase 3 trial."
NeuroSense Therapeutics recently reported positive efficacy and safety data from its Phase 2b trial (PARADIGM) with its lead drug candidate for ALS, PrimeC, and is planning on embarking on a Phase 3 pivotal trial in the next few months. In addition to PrimeC demonstrating a statistically significant 37% slowing of disease progression, as measured by the ALSFRS-R score (p=0.03), in the per-protocol population from the PARADIGM trial, it also announced a statistically significant slowing of disease progression in high-risk ALS patients treated with PrimeC by 43% (p=0.02) as compared to placebo in the pre-specified per protocol (PP) population analysis after 6 months of treatment. The slowing of disease progression demonstrated by PrimeC versus placebo translates to a 5.04 points difference in the ALSFRS-R in favor of PrimeC (Confidence Interval: 0.862, 9.214; n=38).High-risk patients, defined by the European Network for the Cure of ALS (ENCALS) Risk Factor as those with a higher risk for rapid disease progression, comprise approximately 50% of the total ALS population.
NeuroSense soon plans to submit its End of Phase 2 package to the FDA and EMA, including the updated Phase 3 study protocol, which will be discussed in the meetings.
"Although there is an improved understanding of the underlying mechanisms of ALS, therapeutic options remain limited due to the complexity and heterogeneity of the disease," said Dr. Raviv Pryluk, CEO and Co-founder of PhaseV. "NeuroSense's ALS drug candidate PrimeC showed great promise in its Phase 2b study. Through a unique combination of causal-ML, real-world data, and advanced statistical methods, we confirmed the potential clinical benefit of PrimeC and provided actionable insights for the Phase 3 study. Our analysis predicted a high rate of success for PrimeC in the Phase 3 clinical trial for multiple recommended subgroups."
PhaseV's proprietary technology addresses the challenges of drug development by providing tools for the identification of subgroups and endpoints most likely to succeed in subsequent clinical trials. This has helped companies to better understand how their drug candidates will perform in Phase 3 clinical trials and to optimise the design of the Phase 3 trial accordingly in order to achieve meaningful and successful outcomes.