New miRNA findings unveiled at the AAN, reveal key biological mechanisms behind PrimeC's clinical benefit in ALS.CAMBRIDGE, Mass., April 9, 2025 /PRNewswire/ -- NeuroSense Therapeutics, Ltd. (NASDAQ: NRSN), a leading clinical-stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, today announced promising new findings from its Phase 2b PARADIGM clinical trial. The data highlights the significant impact of PrimeC, the company's investigational therapy for the trea ...

Core Insights - NeuroSense Therapeutics reported significant clinical progress in 2024, particularly with its lead drug candidate PrimeC, which demonstrated transformative results in a Phase 2b study for ALS [3][8] - The company plans to advance PrimeC towards a Phase 3 study, with positive FDA feedback on the proposed study design [3][9] Business Highlights - PrimeC achieved primary and secondary clinical endpoints in the PARADIGM Phase 2b study, showing a 33% reduction in disease progression after 18 months and a 58% improvement in survival rates [2][8] - Ongoing partnership discussions are aimed at advancing PrimeC to Phase 3 study and commercialization, with plans to file for early commercialization in Canada, targeting a market opportunity of $100 million to $150 million in peak annual revenues [9][12] Financial Results - Research and development expenses decreased by 21.9% from $7.3 million in 2023 to $5.7 million in 2024, attributed to reduced subcontractor and consultant expenses [4] - General and administrative expenses also decreased by 12.5% from $4.8 million in 2023 to $4.2 million in 2024, mainly due to lower share-based compensation and insurance costs [5] - As of December 31, 2024, NeuroSense had cash of approximately $3.4 million, with total assets increasing to $4.575 million from $3.185 million in 2023 [10][11] Company Overview - NeuroSense is focused on developing treatments for severe neurodegenerative diseases, including ALS, Alzheimer's, and Parkinson's disease, addressing significant unmet medical needs [15] - PrimeC is a novel extended-release oral formulation combining two FDA-approved drugs, designed to target multiple mechanisms contributing to ALS progression [13][14]

Financial Performance - The company reported net losses of $10.2 million and $10.1 million for the years ended December 31, 2024 and 2023, respectively[56]. - The company anticipates continuing to incur significant losses and negative cash flows for the foreseeable future[56]. - The company has a limited operating history and has incurred significant losses since its inception in February 2017[56]. - The company may need to seek additional funds sooner than planned due to changes in its operating plan[52]. - The company will require substantial additional financing to achieve its goals, and failure to obtain this capital could delay or terminate product development[50]. Cash and Financing - As of December 31, 2024, the company had cash and cash equivalents of $3.4 million[50]. - The company expects to finance its cash needs through equity, convertible debt, or debt financings until it can generate substantial product revenues[61]. Product Development and Regulatory Approval - The company has invested almost all of its resources in the research and development of its lead product candidate, PrimeC[63]. - The company has not yet obtained any regulatory approvals for its product candidates, which are in various stages of development[60]. - The FDA has accepted the IND submission for PrimeC, allowing the company to initiate pivotal clinical trials in the United States[76]. - The company plans to initiate a pivotal clinical trial for PrimeC in the second half of 2025, following positive results from previous studies[75]. - The company has completed a Phase 2b trial for PrimeC, demonstrating a clear safety profile and efficacy signals[75]. - The FDA's approval process may require additional post-approval clinical trials, which could delay commercialization[70]. - The company has not yet obtained regulatory approval for any product candidates in the United States or other countries[69]. - The company faces risks associated with patient enrollment in clinical trials, which can impact trial timing and success[83]. - The COVID-19 pandemic has caused delays in regulatory approvals and trial enrollment, affecting the timeline for top-line results[85]. Clinical Trials and Manufacturing Risks - The company has limited experience in conducting clinical trials, which may lead to increased costs and extended timelines[73]. - The company relies on third-party contract manufacturers and suppliers for all raw materials and finished products, which poses risks of supply disruption[111]. - Manufacturing pharmaceutical products is complex and may encounter difficulties, potentially delaying the development and commercialization of product candidates[115]. - Compliance with Good Clinical Practice (GCP) regulations is critical; failure to comply may result in unreliable clinical data and delayed marketing applications[119]. Intellectual Property and Competition - The company has a patent portfolio that includes U.S. Patent 10,980,780, expiring in 2038, related to methods for treating ALS using ciprofloxacin and celecoxib[144]. - The company may face challenges in enforcing its patents, which could limit its ability to prevent competitors from using similar technologies[149]. - Limitations on the scope of intellectual property rights may allow competitors to design around the company's patents and compete effectively[146]. - The company may encounter difficulties in protecting its intellectual property in foreign jurisdictions, particularly in developing countries[167]. - Generic drug manufacturers may challenge the company's patents, leading to complex and lengthy litigation[172]. Market and Regulatory Environment - The biopharmaceutical industry is highly competitive, with numerous companies developing similar therapeutics for conditions like ALS and Alzheimer's disease[100]. - The company faces ongoing regulatory obligations that may result in significant expenses and affect profitability if compliance is not maintained[199]. - Current and future healthcare legislation may impact the company's operations and profitability, particularly regarding reimbursement and coverage of its products[189]. - The U.S. healthcare industry is focused on cost containment, with third-party payors challenging drug prices and limiting reimbursement levels[213]. Management and Operational Challenges - The company currently has 17 full and part-time employees and will need to expand its resources to manage operations and clinical trials effectively[124]. - The company may face challenges in managing growth effectively, which could disrupt operations and delay strategic objectives[125]. - The company relies on attracting and retaining qualified management and scientific personnel for successful product development and commercialization[130]. - Competition for qualified personnel in the pharmaceutical field is intense, which may hinder the company's ability to hire necessary staff as it expands clinical and commercial activities[131]. Financial and Stock Market Considerations - The company received a notice from Nasdaq on December 21, 2023, indicating non-compliance with the Minimum Equity Rule, requiring at least $2.5 million in shareholders' equity[221]. - On January 6, 2025, the company announced it had regained compliance with the Minimum Equity Rule after submitting a plan to Nasdaq[221]. - The company has never paid cash dividends and does not anticipate doing so in the foreseeable future, focusing instead on funding business development[232]. - The market price of the company's ordinary shares and Warrants may fluctuate significantly due to various factors, including market conditions and operational results[225].

Core Insights - NeuroSense Therapeutics is presenting new clinical data from its Phase 2b trial of PrimeC, a novel therapy for amyotrophic lateral sclerosis (ALS), at the 77th Annual American Academy of Neurology Meeting [1][2][3] Company Overview - NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, including ALS, Alzheimer's disease, and Parkinson's disease [7][8] - The company aims to address significant unmet medical needs in neurodegenerative diseases, which currently have limited effective therapeutic options [8] Product Information - PrimeC is NeuroSense's lead drug candidate, an extended-release oral formulation combining two FDA-approved drugs: ciprofloxacin and celecoxib [6] - The therapy targets multiple mechanisms contributing to ALS, including motor neuron degeneration, inflammation, iron accumulation, and impaired RNA regulation [6] - PrimeC has shown safety and efficacy in a Phase 2a clinical trial, with significant changes in ALS-related biological markers indicating its biological activity [6] Clinical Trial Insights - The Phase 2b trial data presented by Jeremy Shefner, M.D., Ph.D., indicates PrimeC's potential to redefine ALS treatment by demonstrating safety, tolerability, and a marked reduction in ALS progression [2][3] - Jeffrey Rosenfeld, M.D., Ph.D., will discuss the role of microRNA modulation in ALS treatment, highlighting PrimeC's multi-targeted approach [3][4] - The anticipation for the forthcoming Phase 3 trial of PrimeC is heightened due to significant changes in relevant biomarker endpoints reflecting target engagement [4] ALS Disease Context - ALS is an incurable neurodegenerative disease that leads to complete paralysis and death within 2-5 years from diagnosis, with over 5,000 new cases diagnosed annually in the U.S. [5] - The annual disease burden of ALS in the U.S. is approximately $1 billion, and the number of people living with ALS is expected to grow by 24% by 2040 in the U.S. and EU [5]

Core Insights - NeuroSense Therapeutics has entered into a binding term sheet with a leading global pharmaceutical company to advance the development and commercialization of PrimeC, a treatment for amyotrophic lateral sclerosis (ALS) [13][14] - The agreement includes a substantial upfront payment, funding for a Phase 3 clinical trial, milestone payments, and a tiered royalty structure reaching double-digit percentages on annual net sales [14][15] - PrimeC is a proprietary fixed-dose combination of two FDA-approved drugs, designed to target multiple ALS disease pathways, and has shown positive results in a Phase 2b clinical trial [4][7] Company Overview - NeuroSense Therapeutics is a clinical-stage biotechnology company focused on developing treatments for neurodegenerative diseases, including ALS, Alzheimer's, and Parkinson's [9] - The company aims to address significant unmet medical needs through combined therapies targeting multiple disease pathways [9] Industry Context - ALS is an incurable neurodegenerative disease with a high annual disease burden of $1 billion in the U.S., affecting over 5,000 new patients each year [5] - The prevalence of ALS is expected to grow by 24% by 2040 in the U.S. and EU, highlighting the urgent need for effective treatments [5]

Core Insights - NeuroSense Therapeutics is advancing its lead drug candidate PrimeC for the treatment of ALS, with significant milestones achieved in clinical trials and regulatory discussions [2][5][6]. Clinical Development - The completion of the 18-month Phase 2b PARADIGM study marks a major milestone, showing PrimeC's potential to slow disease progression by 33% and improve survival rates by 58% [6]. - Positive feedback from the FDA on the Phase 3 study design positions the company to commence this pivotal study in mid-2025 [5]. - The company plans to submit a dossier for early commercialization in Canada, with a potential market opportunity estimated between $100 million to $150 million in peak annual revenue [7]. Financial Updates - NeuroSense secured a $5 million private placement in December 2024, enhancing its financial position to support ongoing clinical development [3][16]. - Research and development expenses decreased by 14% year-over-year, totaling $4.61 million for the nine months ended September 30, 2024 [13]. - As of September 30, 2024, the company reported cash of $0.34 million, not including the recent financing, and believes it has regained compliance with Nasdaq's equity requirements [14]. Regulatory and Market Strategy - The company is preparing for a meeting with EMEA and aims to submit a dossier to Health Canada in Q2 2025, with a regulatory decision expected by Q1 2026 [3][7]. - The company is also participating in various symposiums to present data from the PARADIGM study, enhancing visibility and credibility within the ALS research community [4][9]. Intellectual Property - A key U.S. patent was granted for PrimeC's formulation, extending its protection until 2042, which is crucial for maintaining competitive advantage in the market [10]. Biomarker and Mechanism Insights - PrimeC has shown promising results in regulating iron metabolism and improving key microRNAs, indicating its potential to engage critical genetic targets involved in ALS progression [11][12].

Core Viewpoint - NeuroSense Therapeutics is advancing its lead drug candidate PrimeC for the treatment of amyotrophic lateral sclerosis (ALS) following a productive Type C meeting with the FDA, which is a significant step towards commencing a Phase 3 clinical study in mid-2025 [1][2][4]. Company Developments - The FDA provided positive feedback on the trial design for the planned Phase 3 study, which is crucial for meeting regulatory expectations and potentially securing drug approval [2][4]. - NeuroSense plans to submit a final protocol to the FDA in the first half of 2025, aiming to enroll approximately 300 patients in a randomized, double-blind, placebo-controlled Phase 3 study [3][4]. - The Phase 3 study will evaluate the efficacy and safety of PrimeC, with participants transitioning to an open-label extension after 12 months of treatment [3][4]. Clinical Trial Insights - The completed Phase 2b PARADIGM study demonstrated PrimeC's significant impact on slowing disease progression and increasing survival rates in ALS patients [1][5]. - The PARADIGM trial involved 68 participants across multiple countries, with a design that included a 12-month open-label extension [7][8]. - Most participants in the trial were concurrently treated with Riluzole, indicating that PrimeC may offer benefits beyond the current standard of care [9]. Product Information - PrimeC is an extended-release oral formulation combining two FDA-approved drugs, ciprofloxacin and celecoxib, targeting multiple mechanisms of ALS [10][11]. - The drug has received Orphan Drug Designation from both the FDA and the European Medicines Agency, highlighting its potential in addressing an urgent medical need [11]. Industry Context - ALS is an incurable neurodegenerative disease with a significant annual disease burden of $1 billion in the U.S., and the number of diagnosed cases is expected to grow by 24% by 2040 [6].

Core Insights - NeuroSense Therapeutics announced statistically significant positive results from the 18-month PARADIGM study, showing that PrimeC slowed disease progression in ALS patients by 33% and improved survival rates by 58% compared to placebo [1][8]. Company Overview - NeuroSense Therapeutics is a late-stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, including ALS, Alzheimer's, and Parkinson's [11]. - The company aims to address significant unmet medical needs in neurodegenerative diseases through combined therapies targeting multiple pathways [11]. Product Information - PrimeC is NeuroSense's lead drug candidate, an extended-release oral formulation combining ciprofloxacin and celecoxib, designed to target mechanisms contributing to ALS progression [9][10]. - The drug has shown efficacy in reducing functional and respiratory deterioration and has received Orphan Drug Designation from the FDA and EMA [10]. Study Details - The PARADIGM study is a Phase 2b clinical trial involving 68 participants across Canada, Italy, and Israel, designed to evaluate the efficacy of PrimeC in ALS [7]. - The study demonstrated a 36% improvement in ALS Functional Rating Scale-Revised (ALSFRS-R) scores at the 12-month readout, marking the best results in ALS clinical research to date [8]. Future Developments - Results from the PARADIGM study will be presented at the 2024 International Symposium on ALS/MND, with insights from Prof. Merit Cudkowicz, a leading figure in ALS research [2]. - The company plans to provide updates on its meeting with the FDA in the coming days [4].

Core Viewpoint - NeuroSense Therapeutics Ltd. has entered into a definitive agreement for a private placement to raise $5,000,000 through the sale of ordinary shares and warrants, aimed at supporting its development of treatments for severe neurodegenerative diseases [1][2]. Group 1: Private Placement Details - The company will sell a total of 4,000,000 ordinary shares and 8,000,000 warrants, with a combined purchase price of $1.25 per share, which is 25% above the closing price on November 29, 2024 [2]. - The warrants will have a term of five years and an exercise price of $1.25 per share [2]. - The proceeds from the private placement will be delivered in two tranches and are expected to be used for general corporate and working capital purposes [3]. Group 2: Company Overview - NeuroSense Therapeutics is focused on developing treatments for debilitating neurodegenerative diseases such as ALS, Alzheimer's, and Parkinson's disease, which represent significant unmet medical needs [6]. - The company aims to develop combined therapies targeting multiple pathways associated with these diseases, based on strong scientific research and a large panel of related biomarkers [6].

Core Insights - NeuroSense Therapeutics is participating in the 2024 International Symposium on ALS/MND, showcasing its commitment to ALS research and treatment development [1][5] - The company will present findings from its Phase 2b PARADIGM trial of PrimeC, a potential treatment for Amyotrophic Lateral Sclerosis (ALS) [2][3] Company Overview - NeuroSense Therapeutics is a late-clinical stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, including ALS [1][11] - The company aims to address significant unmet medical needs in neurodegenerative diseases through innovative therapies and biomarker research [11] Clinical Trial Details - The PARADIGM trial is a Phase 2b clinical trial involving 68 participants across Canada, Italy, and Israel, designed to evaluate the efficacy of PrimeC in ALS [8] - The trial demonstrated a 29% efficacy difference in favor of PrimeC compared to placebo in the intent-to-treat population, with a statistically significant 37.4% slowing of disease progression observed in the ALS Functional Rating Scale-Revised (ALSFRS-R) [9] Treatment Insights - PrimeC is an extended-release oral formulation combining two FDA-approved drugs, ciprofloxacin and celecoxib, targeting multiple mechanisms of ALS [10] - The drug has shown promising results in previous trials, including significant changes in ALS-related biological markers and a reduction in functional and respiratory deterioration [10] Symposium Participation - Key presentations at the symposium will include insights from leading experts in ALS research, such as Prof. Merit Cudkowicz and Dr. Cristian Lunetta, focusing on clinical outcomes and biomarker analysis from the PARADIGM trial [3][4] - The presence of NeuroSense's management team at the symposium highlights the company's leadership role in advancing ALS research [4]