Key item data includes:
- The study met its primary safety endpoint in a 12-month clinical trial with 15 participants living with ALS.
Efficacy and biomarkers:
- Exploratory efficacy was assessed by ALSFRS-R and FVC, and blood samples were collected before and during the study for biomarker analysis.
- PrimeC showed statistically significant changes in ALS-related biomarkers of serum neuron-derived exosomes (NDEs) such as TDP-43 and LC3, as measured by ExoSORT™, indicating positive biological activity.
- The trial was open-label, comparing generated data to the PROACT database using propensity matching.
- This study confirms the safety and tolerability of PrimeC in ALS.
- Biomarker analyzes suggest early evidence of a biological effect.
“This clinical study is a very important achievement and milestone for the ALS community. It is very encouraging to see that there was both biological activity and clinical signals of a treatment effect,” said Viviane Drory, MD, director of the Neuromuscular Diseases Unit at Tel Aviv Sourasky Medical Center and principal investigator of the study. “I am grateful to all participants, their families and staff who took part in this study, and proud and excited to participate in the PARADIGM study, which uses an improved formulation of PrimeC.”
Based on the results of this study, NeuroSense initiated PARADIGM, a Phase IIb clinical trial in May 2022. PARADIGM is randomizing 69 people living with ALS in a 2:1 ratio to receive PrimeC or placebo, respectively. The study’s primary endpoints include assessment of ALS biomarkers, assessment of clinical efficacy, improvement in quality of life, and safety and tolerability.
“This study, along with previously published preclinical work, strongly encourages the continued development of Prime C. We need more treatments for ALS, and PrimeC is an exciting candidate,” said Jeremy M. ShefnerMD, PhD, FAAN, Professor of Neurology, Chief Medical Officer for Clinical Research at the Barrow Neurological Institute, and co-author of the article.
dr. Erez Eitan, study co-author and NeuroDex Scientific Director, said, “We are proud to be working with the NeuroSense team on the implementation of ExoSORT™, our proprietary method for isolating neuron-derived exosomes at from blood samples for the identification and measurement of biomarkers When we discovered that TDP43, a major biomarker of ALS, and LC3, a biomarker of autophagy, can be measured in NDE and are different in patients ALS, we were hoping this would help advance therapeutic development. We are grateful to NeuroSense for providing us with the opportunity to test the biomarkers in their clinical trials.” NeuroDex is advancing brain diagnostics with its proprietary cell-specific exosome diagnostics platform, which provides a non-invasive diagnostic tool , inexpensive and robust.
“We thank the trial participants and their families and caregivers for choosing to participate in this study. We would also like to thank all of our valued collaborators for their teamwork and tireless efforts in the field and in this study,” said the head of NeuroSense. Scientific Program, Dr. Shiran Zimri.
“We are very pleased to have our PrimeC Phase IIa findings published in a leading ALS journal, as interest in PrimeC continues to grow in the ALS scientific community. Our clinical research teams and scientists actively present at conferences as we engage in patient advocacy to bring much-needed effective treatment to those suffering from this debilitating disease,” said NeuroSense CEO Alon Ben Noon.
PrimeC, NeuroSense’s lead drug candidate, is a combination therapy that has received Orphan Drug Designation from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). NeuroSense has completed a Phase IIa clinical study which successfully met its safety and efficacy criteria, including reduction in functional and respiratory deterioration and statistically significant changes in ALS-related biomarkers indicating activity organic from PrimeC. The improved formulation of PrimeC, which is a unique sustained-release tablet developed to maximize synergy between compounds, is currently being evaluated in a Phase IIb clinical trial, PARADIGM, for the treatment of ALS. The study’s primary endpoints include assessment of ALS biomarkers, assessment of clinical efficacy, improvement in quality of life, and safety and tolerability. Through a collaboration with Massachusetts General Hospital in Boston on novel neuron-derived exosomes (NDEs), NeuroSense is working to better determine biological changes in ALS-related pathologies and the effect of PrimeC on relevant targets.
Amyotrophic lateral sclerosis (ALS) is an incurable neurodegenerative disease that causes complete paralysis and death within 2 to 5 years of diagnosis. Each year, more than 5,000 patients are diagnosed with ALS in the United States alone, with an annual disease burden of $1 billion. The number of ALS patients is expected to increase by 24% by 2040 in the US and EU.
NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on the discovery and development of treatments for patients suffering from debilitating neurodegenerative diseases. NeuroSense believes that these diseases, which include amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and Parkinson’s disease, among others, represent one of the greatest unmet medical needs of our time, with treatment options limited efficacy for patients to date. Due to the complexity of neurodegenerative diseases and based on sound scientific research on a broad panel of related biomarkers, NeuroSense’s strategy is to develop combination therapies targeting multiple pathways associated with these diseases.
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This press release contains “forward-looking statements” that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements in this press release can be identified by the use of words such as “anticipate”, “believe”, “intend”, “could”, “estimate”, “expect”, “have the ‘intent to’, ‘seek,’ ‘may’, ‘could’, ‘plan’, ‘potential’, ‘predict’, ‘project’, ‘target’, ‘aim’, ‘should’, ‘will’, ‘ would” or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on NeuroSense Therapeutics’ current expectations and are subject to uncertainties, risks and inherent assumptions that are difficult to predict and include statements regarding patent applications; the company’s PrimeC development program; the potential for PrimeC to safely and effectively target ALS; preclinical and clinical data for PrimeC; clinical trial schedule current and future; the nature, strategy and corporate guidance and other updates thereto; and the development and commercial potential of any of the Company’s product candidates. In addition, certain forward-looking statements are based on assumptions about future events which may not prove to be accurate. The forward-looking statements contained in this announcement are made as of this date, and NeuroSense Therapeutics Ltd. assumes no obligation to update this information, except as required by applicable law.
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SOURCE NeuroSense Therapeutics