Positive Phase 2b Trial Outcomes for NeuroSense's ALS Drug PrimeC

NeuroSense Therapeutics, a biotechnology firm dedicated to developing treatments for severe neurodegenerative conditions, has announced promising new findings from its Phase 2b PARADIGM trial. This trial evaluates the efficacy of PrimeC, NeuroSense's leading drug candidate, in treating amyotrophic lateral sclerosis (ALS), an incurable disease characterized by rapid progression to paralysis and death post-diagnosis.

The six-month, double-blind study has shown that PrimeC not only has a significant impact on slowing the disease's progression, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), but also improves patients' quality of life and complication-free survival rates. Remarkably, PrimeC was found to reduce the risk of ALS-related complications or death by up to 53%, a substantial figure that underscores the drug's potential in managing this devastating illness.

The trial's secondary endpoints focused on life quality and survival, revealing that PrimeC positively influenced patients' mental and physical well-being. Survival analyses indicated that PrimeC could delay the onset of respiratory insufficiency and hospitalization due to ALS-related issues. Ferenc Tracik, M.D., NeuroSense's Chief Medical Officer, highlighted the importance of these findings, emphasizing the drug's potential to offer substantial benefits to the ALS patient community.

The positive results build upon previous top-line outcomes from December 2023, which confirmed PrimeC's safety and tolerability profile and demonstrated a notable slowing of disease progression. PrimeC outperformed the placebo group with a 37.4% difference in ALSFRS-R scores and a 17.2% difference in Slow Vital Capacity (SVC), reinforcing its status as a promising ALS treatment.

PrimeC is an innovative oral formulation combining two FDA-approved drugs, ciprofloxacin and celecoxib, aiming to address multiple ALS mechanisms, including motor neuron degeneration and inflammation. It has received Orphan Drug Designation from both the U.S. Food and Drug Administration and the European Medicines Agency, reflecting its potential as a much-needed therapy for ALS.

The PARADIGM trial, involving participants from Canada, Italy, and Israel, has shown high continuation rates, with 96% of those completing the double-blind phase choosing to continue PrimeC treatment in an open-label extension. The trial's intention-to-treat analysis also pointed towards PrimeC's efficacy, with a 29% difference in ALSFRS-R and a 13% difference in SVC, favoring PrimeC over the placebo.

NeuroSense's CEO, Alon Ben-Noon, expressed anticipation for forthcoming data on neurofilaments and other biomarkers, which could expedite PrimeC's development. The company is keen on advancing therapies that target multiple disease pathways, given the complexity of neurodegenerative diseases.

ALS remains a significant medical challenge, with over 5,000 new cases diagnosed annually in the U.S., incurring an annual cost of $1 billion. The patient population is expected to grow by 24% by 2040 in the U.S. and EU, highlighting the urgency for effective treatments like PrimeC.

NeuroSense Therapeutics is committed to addressing the unmet needs of patients with ALS and other neurodegenerative diseases, such as Alzheimer's and Parkinson's, by developing therapies that target multiple disease pathways based on robust scientific research and biomarker analysis. The company's strategy aligns with the current understanding that a multifaceted approach is necessary to combat these complex conditions effectively.