Last update 07 Nov 2024

Riluzole

Last update 07 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

IT-Riluzole, Riluzole (JAN/USP/INN), BF-37

+ [9]

Target

SCNA

Mechanism

SCNA modulators(Sodium channel alpha subunit modulators), T lymphocytes inhibitors

Therapeutic Areas

Nervous System DiseasesEndocrinology and Metabolic DiseaseOther Diseases

Active Indication

Amyotrophic Lateral Sclerosis

Inactive Indication

Huntington DiseaseMultiple System AtrophySupranuclear Palsy, Progressive

Originator Organization

Sanofi

Active Organization

Aquestive Therapeutics, Inc.

Zentiva ks

Sanofi Winthrop Industrie SA

+ [4]

Inactive Organization

Covis Pharma Holdings BV

Sanofi

Aventis Pharmaceuticals, Inc.

Drug Highest PhaseApproved

First Approval Date

US (12 Dec 1995),

Amyotrophic Lateral Sclerosis

RegulationOrphan Drug (US), Orphan Drug (JP)

Structure

Molecular FormulaC8H5F3N2OS

InChIKeyFTALBRSUTCGOEG-UHFFFAOYSA-N

CAS Registry1744-22-5

Clinical Trials associated with Riluzole

IRCT20240722062515N1 / SuspendedPhase 3IIT

Evaluation the comparative effect of Riluzole and Methylprednisolone combined treatment and Methylprednisolone in function of acute spinal cord injury (a clinical trial)

Start Date22 Sep 2024

Sponsor / Collaborator-

CTR20242159 / CompletedNot Applicable

利鲁唑片（50mg）在中国健康受试者中空腹给药条件下随机、开放、单剂量、三序列、三周期、部分重复交叉生物等效性试验

[Translation] A randomized, open-label, single-dose, three-sequence, three-period, partially repeated crossover bioequivalence study of riluzole tablets (50 mg) in Chinese healthy subjects under fasting conditions

主要研究目的：按有关生物等效性试验的规定，选择Sanofi Mature IP为持证商的利鲁唑片（商品名：RILUTEK，规格：50mg）为参比制剂，对石家庄四药有限公司生产并提供的利鲁唑片（规格：50mg）进行空腹给药人体生物等效性试验，比较受试制剂中药物的吸收速度和吸收程度与参比制剂的差异是否在可接受的范围内，评估两种制剂在空腹给药条件下的生物等效性。次要研究目的：观察健康志愿受试者口服受试制剂利鲁唑片（规格：50mg）和参比制剂利鲁唑片（商品名：RILUTEK，规格：50mg）的安全性。

[Translation]

The main purpose of the study: According to the relevant provisions of the bioequivalence test, the riluzole tablets (trade name: RILUTEK, specification: 50 mg) of which Sanofi Mature IP is the licensee was selected as the reference preparation, and the human bioequivalence test of the riluzole tablets (specification: 50 mg) produced and provided by Shijiazhuang Siyao Co., Ltd. was conducted on the fasting administration, to compare whether the absorption rate and degree of the drug in the test preparation are within the acceptable range with the reference preparation, and to evaluate the bioequivalence of the two preparations under fasting administration conditions. Secondary purpose of the study: To observe the safety of oral administration of the test preparation riluzole tablets (specification: 50 mg) and the reference preparation riluzole tablets (trade name: RILUTEK, specification: 50 mg) by healthy volunteers.

Start Date15 Jul 2024

Sponsor / Collaborator

Shijiazhuang No. 4 Pharmaceutical Co., Ltd.

ChiCTR2300074440 / SuspendedPhase 3IIT

A multicenter,randomized controlled trial assessing the efficacy of Riluzole in facilitating the repairement of spinal cord

Start Date01 Jan 2024

Sponsor / Collaborator

Jiangsu Provincial People's Hospital

100 Clinical Results associated with Riluzole

100 Translational Medicine associated with Riluzole

100 Patents (Medical) associated with Riluzole

1,837

Literatures (Medical) associated with Riluzole

01 Apr 2025·Neural Regeneration Research

Treating amyotrophic lateral sclerosis with allogeneic Schwann cell–derived exosomal vesicles: a case report

Article

Author: Pearse, Damien D. ; Dietrich, W. Dalton ; Levi, Allan D. ; Guest, James D. ; Silvera, Risset ; Goldschmidt, Alexander J.P. ; Brooks, Adriana ; Graham, Patricia ; Goldschmidt-Clermont, Pascal J. ; Jimsheleishvili, George ; Khan, Aisha

Schwann cells are essential for the maintenance and function of motor neurons, axonal networks, and the neuromuscular junction. In amyotrophic lateral sclerosis, where motor neuron function is progressively lost, Schwann cell function may also be impaired. Recently, important signaling and potential trophic activities of Schwann cell-derived exosomal vesicles have been reported. This case report describes the treatment of a patient with advanced amyotrophic lateral sclerosis using serial intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles, marking, to our knowledge, the first instance of such treatment. An 81-year-old male patient presented with a 1.5-year history of rapidly progressive amyotrophic lateral sclerosis. After initial diagnosis, the patient underwent a combination of generic riluzole, sodium phenylbutyrate for the treatment of amyotrophic lateral sclerosis, and taurursodiol. The patient volunteered to participate in an FDA-approved single-patient expanded access treatment and received weekly intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles to potentially restore impaired Schwann cell and motor neuron function. We confirmed that cultured Schwann cells obtained from the amyotrophic lateral sclerosis patient via sural nerve biopsy appeared impaired (senescent) and that exposure of the patient’s Schwann cells to allogeneic Schwann cell-derived exosomal vesicles, cultured expanded from a cadaver donor improved their growth capacity in vitro. After a period of observation lasting 10 weeks, during which amyotrophic lateral sclerosis Functional Rating Scale-Revised and pulmonary function were regularly monitored, the patient received weekly consecutive infusions of 1.54 × 1012 (×2), and then consecutive infusions of 7.5 × 1012 (×6) allogeneic Schwann cell-derived exosomal vesicles diluted in 40 mL of Dulbecco’s phosphate-buffered saline. None of the infusions were associated with adverse events such as infusion reactions (allergic or otherwise) or changes in vital signs. Clinical lab serum neurofilament and cytokine levels measured prior to each infusion varied somewhat without a clear trend. A more sensitive in-house assay suggested possible inflammasome activation during the disease course. A trend for clinical stabilization was observed during the infusion period. Our study provides a novel approach to address impaired Schwann cells and possibly motor neuron function in patients with amyotrophic lateral sclerosis using allogeneic Schwann cell-derived exosomal vesicles. Initial findings suggest that this approach is safe.

01 Jan 2025·EXPERIMENTAL NEUROLOGY

Potential role of solid lipid curcumin particle (SLCP) as estrogen replacement therapy in mitigating TDP-43-related neuropathy in the mouse model of ALS disease

Article

Author: Lee, Yi-Chao ; Ashan, Asmar ; Hsieh, Yi-Chen ; Majumder, Pritha ; Huang, Chi-Chen ; Hu, Chaur-Joug ; Hsu, Tsung-I ; Huang, Jeffrey K ; Ahsan, Asmar ; Huang, Jeffrey K.

BACKGROUND:

Amyotrophic lateral sclerosis (ALS) was first identified in 1869, but it wasn't until the 2014 Ice Bucket Challenge that widespread attention was drawn to the disease. Since then, substantial research has been dedicated to developing treatments for ALS. Despite this, only three drugs - riluzole, edaravone and AMX0035, have been approved for clinical use, and they can only temporarily alleviate mild symptoms without significant disease modification or cure. Therefore, there remains a critical unmet need to identify disease modifying or curative therapies for ALS. The higher incidence and more severe progression of ALS and FTLD (frontotemporal lobar degeneration) observed in men and postmenopausal woman compared to young women suggests that sex hormones may significantly influence disease onset and progression. In both animal models and human clinical studies, 17β estradiol (E2) has been shown to delay and improve the outcomes of many neurodegenerative diseases. Here, we examined the role of TDP-43 in the regulation of estrogen-related enzymes, CYP19A1 and CYP3A4. In addition, we examined the impact of curcumin on the regulation of estrogen E2 levels and TDP-43-associated neuropathy as a potential therapeutic strategy for the treatment of FTLD and ALS.

METHODS:

Prp-TDP-43^A315T mice was used as a model of ALS/FTLD to examine the expression patterns of E2 and its biosynthesis and degradation enzymes, CYP19A1 and CYP3A4. Moreover, the molecular mechanisms and the potency of solid lipid curcumin particles (SLCP) as an E2 replacement therapy for TDP-43 associated neuropathy was analyzed. We further examined the survival rates and the pathological TDP43 patterns in female and male Prp-TDP-43^A315T mice administrated with or without SLCP. In addition, the changed expression levels of enzymes corresponding to E2 biosynthesis and degradation in the spinal cord of female and male Prp-TDP-43^A315T mice with or without SLCP were determined.

RESULTS:

We found that in addition to E2, the expression patterns of CYP19A1 and CYP3A4 proteins differed between Prp-TDP-43^A315T mice compared to wild-type control, suggesting that toxic phosphorylated TDP43 oligomers may disrupt the balance between CYP19A1 and CYP3A4 expression, leading to reduced estrogen biosynthesis and accelerated degradation. In addition, we found that oral administration of SLCP prolonged the survival rates in female Prp-TDP-43^A315T mice and significantly reduced the pathological insoluble phosphorylated TDP-43 species. Furthermore, SLCP attenuated disease progression associated with TDP-43-related neuropathies through modulating estrogen biosynthesis and the activity of CYP450 enzymes.

CONCLUSIONS:

Our results showed that Prp-TDP-43^A315T mice exhibit altered estradiol levels. Moreover, we demonstrated the efficacy of SLCP as an estrogen replacement therapy in mitigating TDP-43-associated disease progression and pathogenesis. These findings suggest that SLCP could be a promising strategy to induce E2 expression for the treatment of ALS and FTLD.

01 Dec 2024·EPILEPSY & BEHAVIOR

Drug repurposing in status epilepticus

Review

Author: Walker, Matthew C

The treatment of status epilepticus (SE) has changed little in the last 20 years, largely because of the high risks and costs of new drug development for SE. Moreover, SE poses specific challenges to drug development, such as patient diversity, logistical hurdles, and the need for acute treatment strategies that differ from chronic seizure prevention. This has reduced the appetite of industry to develop new drugs in this area. Drug repurposing is an attractive approach to address this unmet need. It offers significant advantages, including reduced development time, lower costs, and higher success rates, compared to novel drug development. Here I demonstrate how novel methods integrating biological knowledge and computational methods can be applied to drug repurposing in status epilepticus. Biological approaches focus on addressing mechanisms underlying drug resistance in SE (using for example ketamine, tacrolimus and safinamide) and longer-term consequences (using for example omaveloxolone, celecoxib and losartan). Additionally, artificial intelligence platforms, such as ChatGPT, can rapidly generate promising drug lists, while in silico methods can analyze gene expression changes to predict molecular targets. Combining AI and in silico approaches has identified several candidate drugs, including metformin, sirolimus and riluzole, for SE treatment. Despite the promise of repurposing, challenges remain, such as intellectual property issues and regulatory barriers. Nonetheless, drug repurposing presents a viable solution to the high costs and slow progress of traditional drug development for SE. This paper is based on a presentation made at the 9th London-Innsbruck Colloquium on Status Epilepticus and Acute Seizures, in April 2024.

115

News (Medical) associated with Riluzole

28 Oct 2024

·www.prnewswire.com

NeuroSense Secures Key FDA Meeting to Advance its Phase 3 ALS Trial and NDA Submission

Meeting discussion to focus on finalizing Phase 3 study design and NDA readiness, ensuring a clear regulatory path for submission based on trial success Parallel preparations in Canada: NeuroSense plans to submit its dossier in Q2 2025, with a regulatory decision on commercialization expected by Q1 2026 CAMBRIDGE, Mass., Oct. 28, 2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a late-clinical stage biotechnology company developing novel treatments for severe neurodegenerative diseases, today announced it is scheduled for a Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss the design of its upcoming Phase 3 clinical trial for PrimeC, a potential treatment for Amyotrophic Lateral Sclerosis (ALS). The FDA Type C meeting, scheduled for November 6, 2024, will focus on finalizing the design of the Phase 3 trial, a pivotal step toward securing approval for the drug. NeuroSense will also review its readiness for a future New Drug Application (NDA) submission, ensuring its regulatory strategy aligns with the FDA's requirements. NeuroSense plans to give an update on the results of the meeting following receipt of the meeting minutes which are expected approximately one month after the meeting. PrimeC, NeuroSense's lead product, has shown promising results in clinical trials, including a statistically significant reduction in disease progression clinical and biological markers. With an urgent unmet need in ALS, NeuroSense is positioning PrimeC as a potential breakthrough therapy. In parallel, NeuroSense plans to submit its regulatory dossier to Health Canada in Q2 2025, with a regulatory decision anticipated by Q1 2026. The Company estimates the potential market opportunity for PrimeC in Canada alone to be approximately $100 million to $150 million in annual revenue. This decision follows the recommendations of Canadian regulatory experts and recent clinical findings, which demonstrated that PrimeC significantly reduced disease progression (p=0.009) and improved survival rates by 43% compared to placebo, highlighting its potential as a breakthrough therapy for ALS. About ALS Amyotrophic lateral sclerosis ("ALS") is an incurable neurodegenerative disease that causes complete paralysis and death within 2-5 years from diagnosis. Every year, more than 5,000 people are diagnosed with ALS in the U.S. alone, with an annual disease burden of $1 billion. The number of people living with ALS is expected to grow by 24% by 2040 in the U.S. and EU. About ALSFRS-R Disease progression is measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), which is the most widely used ALS tracking tool accepted by the FDA, utilized by neurologists treating ALS patients, in clinical trials, and by other regulators to determine disease progression. It tracks 12 changes in a person's physical abilities over time including functions such as: speech, walking, climbing stairs, dressing/hygiene, handwriting, turning in bed, cutting food, salivation, swallowing, and breathing. A single point change on the ALSFRS-R has a significant impact on ALS patients, such as the transition from independent feeding to requiring assistance or independent breathing to needing to use a machine ventilator. About PARADIGM PARADIGM is a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b (NCT05357950) clinical trial of PrimeC in ALS. The trial included 68 participants living with ALS in Canada, Italy, and Israel. 96% of the trial participants who completed the 6-month double-blind portion of the trial chose to receive treatment with PrimeC through a 12-month open label extension. Furthermore, to date (June 2024) all participants that completed the 18-month trial treatment duration, requested to continue PrimeC, which is provided to them in an Investigator Initiated Trial, not limited with time. As previously reported, in the 6-month double-blind segment of the trial, the data showed clinically meaningful signs of efficacy with a 29% difference in favor of PrimeC vs placebo in analysis of the intent to treat (ITT) population. In the PP top-line analysis from PARADIGM, a statistically significant slowing of disease progression was observed with a 37.4% (p=0.03) difference in ALSFRS-R in favor of PrimeC vs placebo. Most patients enrolled in both the active and placebo arms of the trial were concurrently treated with Riluzole, the ALS standard of care medication, indicating PrimeC slowed disease progression well beyond the level afforded by the FDA approved ALS drug. About PrimeC PrimeC, NeuroSense's lead drug candidate, is a novel extended-release oral formulation composed of a unique fixed-dose combination of two FDA-approved drugs: ciprofloxacin and celecoxib. PrimeC is designed to synergistically target several key mechanisms of ALS that contribute to motor neuron degeneration, inflammation, iron accumulation and impaired ribonucleic acid ("RNA") regulation to potentially inhibit the progression of ALS. NeuroSense completed a Phase 2a clinical trial which met its safety and efficacy endpoints including reducing functional and respiratory deterioration and statistically significant changes in ALS-related biological markers indicating PrimeC's biological activity. PrimeC was granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency. About NeuroSense NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on discovering and developing 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 most significant unmet medical needs of our time, with limited effective therapeutic options available for patients to date. Due to the complexity of neurodegenerative diseases and based on strong scientific research on a large panel of related biomarkers, NeuroSense's strategy is to develop combined therapies targeting multiple pathways associated with these diseases. For additional information, we invite you to visit our website and follow us on LinkedIn, YouTube and X. Information that may be important to investors may be routinely posted on our website and these social media channels. Forward-Looking Statements 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 contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "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 inherent uncertainties, risks and assumptions that are difficult to predict and include statements regarding the timing of regulatory filings and regulatory decisions, the market opportunity in Canada and securing regulatory approval in global markets. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. The future events and trends may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward looking statements. These risks include the risk of a delay in submission by the Company of its regulatory dossier, that regulatory approvals for PrimeC will be delayed or not obtained in Canada or elsewhere; that the market opportunity in Canada will not be as currently estimated; unexpected R&D costs or operating expenses, insufficient capital to complete development of PrimeC, a delay in the reporting of additional results from PARADIGM clinical trial, the timing of expected regulatory and business milestones, risks associated with meeting with the FDA and Health Canada to determine the best path forward following the results from PARADIGM clinical trial, including a delay in any such meeting; the potential for PrimeC to safely and effectively target ALS; preclinical and clinical data for PrimeC; the uncertainty regarding outcomes and the timing of current and future clinical trials; timing for reporting data; the development and commercial potential of any product candidates of Neurosense; the ability of NeuroSense to remain listed on Nasdaq; and other risks and uncertainties set forth in NeuroSense's filings with the Securities and Exchange Commission (SEC). You should not rely on these statements as representing our views in the future. More information about the risks and uncertainties affecting NeuroSense is contained under the heading "Risk Factors" in the Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2024 and NeuroSense's subsequent filings with the SEC. Forward-looking statements contained in this announcement are made as of this date, and NeuroSense undertakes no duty to update such information except as required under applicable law. Logo - SOURCE NeuroSense WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Clinical ResultPhase 3Orphan DrugNDA

09 Oct 2024

·www.prnewswire.com

NeuroSense Therapeutics Plans to File for Early Commercialization of ALS Treatment in Canada

The preparations for the regulatory submission for early commercialization in Canada follows the positive Phase 2b trial results which show PrimeC reduces ALS disease progression by 36% and improves survival rates by 43% Company plans to seek approval in additional countries CAMBRIDGE, Mass., Oct. 9, 2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ('NeuroSense'), a late-clinical stage biotechnology company developing novel treatments for severe neurodegenerative diseases, today announced it has initiated the regulatory process to seek early commercialization approval for PrimeC under Health Canada's Notice of Compliance with Conditions (NOC/c) policy. This submission is based on the promising results from the company's Phase 2b ALS PARADIGM clinical trial, alongside supporting clinical and preclinical data. This decision aligns with the recommendations of Canadian regulatory experts and follows recent clinical findings, where PrimeC demonstrated a 36% reduction in disease progression (p=0.009) and a 43% improvement in survival rates compared to placebo, underscoring its potential as a breakthrough therapy for ALS. Alon Ben-Noon, NeuroSense CEO expressed optimism about the company's prospects: "We are excited to take this important step toward bringing PrimeC to ALS patients in Canada. The compelling results from our clinical trials give us confidence that PrimeC can address the urgent unmet needs in ALS treatment. Our goal is to expedite access to this promising therapy, and we are committed to advancing regulatory approvals in Canada and beyond." About ALS Amyotrophic lateral sclerosis ("ALS") is an incurable neurodegenerative disease that causes complete paralysis and death within 2-5 years from diagnosis. Every year, more than 5,000 people are diagnosed with ALS in the U.S. alone, with an annual disease burden of $1 billion. The number of people living with ALS is expected to grow by 24% by 2040 in the U.S. and EU. About ALSFRS-R Disease progression is measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), which is the most widely used ALS tracking tool accepted by the FDA, utilized by neurologists treating ALS patients, in clinical trials, and by other regulators to determine disease progression. It tracks 12 changes in a person's physical abilities over time including functions such as: speech, walking, climbing stairs, dressing/hygiene, handwriting, turning in bed, cutting food, salivation, swallowing, and breathing. A single point change on the ALSFRS-R has a significant impact on ALS patients, such as the transition from independent feeding to requiring assistance or independent breathing to needing to use a machine ventilator. About PARADIGM PARADIGM is a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b (NCT05357950) clinical trial of PrimeC in ALS. The trial included 68 participants living with ALS in Canada, Italy, and Israel. 96% of the trial participants who completed the 6-month double-blind portion of the trial chose to receive treatment with PrimeC through a 12-month open label extension. Furthermore, to date (June 2024) all participants that completed the 18-month trial treatment duration, requested to continue PrimeC, which is provided to them in an Investigator Initiated Trial, not limited with time. As previously reported, in the 6-month double-blind segment of the trial, the data showed clinically meaningful signs of efficacy with a 29% difference in favor of PrimeC vs placebo in analysis of the intent to treat (ITT) population. In the PP top-line analysis from PARADIGM, a statistically significant slowing of disease progression was observed with a 37.4% (p=0.03) difference in ALSFRS-R in favor of PrimeC vs placebo. Most patients enrolled in both the active and placebo arms of the trial were concurrently treated with Riluzole, the ALS standard of care medication, indicating PrimeC slowed disease progression well beyond the level afforded by the FDA approved ALS drug. About PrimeC PrimeC, NeuroSense's lead drug candidate, is a novel extended-release oral formulation composed of a unique fixed-dose combination of two FDA-approved drugs: ciprofloxacin and celecoxib. PrimeC is designed to synergistically target several key mechanisms of ALS that contribute to motor neuron degeneration, inflammation, iron accumulation and impaired ribonucleic acid ("RNA") regulation to potentially inhibit the progression of ALS. NeuroSense completed a Phase 2a clinical trial which met its safety and efficacy endpoints including reducing functional and respiratory deterioration and statistically significant changes in ALS-related biological markers indicating PrimeC's biological activity. PrimeC was granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency. About NeuroSense NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on discovering and developing 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 most significant unmet medical needs of our time, with limited effective therapeutic options available for patients to date. Due to the complexity of neurodegenerative diseases and based on strong scientific research on a large panel of related biomarkers, NeuroSense's strategy is to develop combined therapies targeting multiple pathways associated with these diseases. For additional information, we invite you to visit our website and follow us on LinkedIn, YouTube and X. Information that may be important to investors may be routinely posted on our website and these social media channels. Forward-Looking Statements 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 contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "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 inherent uncertainties, risks and assumptions that are difficult to predict and include statements regarding PrimeC as a potential treatment for people with ALS. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. The future events and trends may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward looking statements. These risks include the risk that regulatory approvals for PrimeC will not be obtained in Canada or elsewhere; unexpected R&D costs or operating expenses, insufficient capital to complete development of PrimeC, a delay in the reporting of additional results from PARADIGM clinical trial, the timing of expected regulatory and business milestones, risks associated with meeting with the FDA and Health Canada to determine the best path forward following the results from PARADIGM clinical trial, including a delay in any such meeting; the potential for PrimeC to safely and effectively target ALS; preclinical and clinical data for PrimeC; the uncertainty regarding outcomes and the timing of current and future clinical trials; timing for reporting data; the development and commercial potential of any product candidates of Neurosense; the ability of NeuroSense to remain listed on Nasdaq; and other risks and uncertainties set forth in NeuroSense's filings with the Securities and Exchange Commission (SEC). You should not rely on these statements as representing our views in the future. More information about the risks and uncertainties affecting NeuroSense is contained under the heading "Risk Factors" in the Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2024 and NeuroSense's subsequent filings with the SEC. Forward-looking statements contained in this announcement are made as of this date, and NeuroSense undertakes no duty to update such information except as required under applicable law. Logo: SOURCE NeuroSense WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Clinical ResultOrphan Drug

07 Oct 2024

·www.prnewswire.com

NeuroSense Therapeutics Announces Acceptance of Two PrimeC Abstracts at the 2024 NEALS Annual Meeting

CAMBRIDGE, Mass., Oct. 7, 2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (Nasdaq: NRSN) ("NeuroSense"), a late-clinical stage biotechnology company developing novel treatments for severe neurodegenerative diseases, today announced the acceptance of two abstracts focusing on the company's lead drug candidate, PrimeC, for the treatment of Amyotrophic Lateral Sclerosis (ALS). These abstracts will be presented at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis (NEALS) Consortium Meeting, taking place October 21 to October 24 in Clearwater, Florida. The abstracts will highlight groundbreaking data from NeuroSense's Phase 2b PARADIGM study. The first abstract, presenting clinical outcomes, will be delivered by renowned clinician Dr. Merit Cudkowicz, Chief of Neurology at Massachusetts General Hospital (MGH) and Director of the Sean M. Healey & AMG Center for ALS, USA. The second abstract, focused on biomarker analysis, will be presented by Dr. Cristian Lunetta, a leading neurologist and ALS specialist from the NEuroMuscular Omnicentre (NEMO) in Milan, Italy. These presentations underscore NeuroSense's commitment to driving forward ALS research and the development of innovative treatments for this devastating disease. The company's recent findings demonstrated that PrimeC reduced disease progression by 36% reduction in disease progression (p=0.009) and a 43% improvement in survival rates compared to placebo, further solidifying PrimeC's potential as a breakthrough therapy for ALS. About ALS Amyotrophic lateral sclerosis ("ALS") is an incurable neurodegenerative disease that causes complete paralysis and death within 2-5 years from diagnosis. Every year, more than 5,000 people are diagnosed with ALS in the U.S. alone, with an annual disease burden of $1 billion. The number of people living with ALS is expected to grow by 24% by 2040 in the U.S. and EU. About ALSFRS-R Disease progression is measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), which is the most widely used ALS tracking tool accepted by the FDA, utilized by neurologists treating ALS patients, in clinical trials, and by other regulators to determine disease progression. It tracks 12 changes in a person's physical abilities over time including functions such as: speech, walking, climbing stairs, dressing/hygiene, handwriting, turning in bed, cutting food, salivation, swallowing, and breathing. A single point change on the ALSFRS-R has a significant impact on ALS patients, such as the transition from independent feeding to requiring assistance or independent breathing to needing to use a machine ventilator. About PARADIGM PARADIGM is a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b (NCT05357950) clinical trial of PrimeC in ALS. The trial included 68 participants living with ALS in Canada, Italy, and Israel. 96% of the trial participants who completed the 6-month double-blind portion of the trial chose to receive treatment with PrimeC through a 12-month open label extension. Furthermore, to date (June 2024) all participants that completed the 18-month trial treatment duration, requested to continue PrimeC, which is provided to them in an Investigator Initiated Trial, not limited with time. As previously reported, in the 6-month double-blind segment of the trial, the data showed clinically meaningful signs of efficacy with a 29% difference in favor of PrimeC vs placebo in analysis of the intent to treat (ITT) population. In the PP top-line analysis from PARADIGM, a statistically significant slowing of disease progression was observed with a 37.4% (p=0.03) difference in ALSFRS-R in favor of PrimeC vs placebo. Most patients enrolled in both the active and placebo arms of the trial were concurrently treated with Riluzole, the ALS standard of care medication, indicating PrimeC slowed disease progression well beyond the level afforded by the FDA approved ALS drug. About PrimeC PrimeC, NeuroSense's lead drug candidate, is a novel extended-release oral formulation composed of a unique fixed-dose combination of two FDA-approved drugs: ciprofloxacin and celecoxib. PrimeC is designed to synergistically target several key mechanisms of ALS that contribute to motor neuron degeneration, inflammation, iron accumulation and impaired ribonucleic acid ("RNA") regulation to potentially inhibit the progression of ALS. NeuroSense completed a Phase 2a clinical trial which met its safety and efficacy endpoints including reducing functional and respiratory deterioration and statistically significant changes in ALS-related biological markers indicating PrimeC's biological activity. PrimeC was granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency. About NeuroSense NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on discovering and developing 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 most significant unmet medical needs of our time, with limited effective therapeutic options available for patients to date. Due to the complexity of neurodegenerative diseases and based on strong scientific research on a large panel of related biomarkers, NeuroSense's strategy is to develop combined therapies targeting multiple pathways associated with these diseases. For additional information, we invite you to visit our website and follow us on LinkedIn, YouTube and X. Information that may be important to investors may be routinely posted on our website and these social media channels. Forward-Looking Statements 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 contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "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 inherent uncertainties, risks and assumptions that are difficult to predict and include statements regarding PrimeC as a potential treatment for people with ALS. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. The future events and trends may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward looking statements. These risks include unexpected R&D costs or operating expenses, insufficient capital to complete development of PrimeC, a delay in the reporting of additional results from PARADIGM clinical trial, the timing of expected regulatory and business milestones, risks associated with meeting with the FDA to determine the best path forward following the results from PARADIGM clinical trial, including a delay in any such meeting; the potential for PrimeC to safely and effectively target ALS; preclinical and clinical data for PrimeC; the uncertainty regarding outcomes and the timing of current and future clinical trials, timing for reporting data and the development and commercial potential of any product candidates of Neurosense; the ability of NeuroSense to remain listed on Nasdaq; and other risks and uncertainties set forth in NeuroSense's filings with the Securities and Exchange Commission (SEC). You should not rely on these statements as representing our views in the future. More information about the risks and uncertainties affecting NeuroSense is contained under the heading "Risk Factors" in the Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2024 and NeuroSense's subsequent filings with the SEC. Forward-looking statements contained in this announcement are made as of this date, and NeuroSense undertakes no duty to update such information except as required under applicable law. SOURCE NeuroSense WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Clinical ResultOrphan Drug

100 Deals associated with Riluzole

External Link

KEGG	Wiki	ATC	Drug Bank
D00775	Riluzole	N07XX02	DB00740

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	US	Covis Pharma Holdings BV	12 Dec 1995

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Multiple System Atrophy	Phase 3	GB	Aventis Pharmaceuticals, Inc.	01 Apr 2000
Supranuclear Palsy, Progressive	Phase 3	GB	Aventis Pharmaceuticals, Inc.	01 Apr 2000
Huntington Disease	Phase 3	-	Sanofi	01 Nov 1999

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04761614 (ASCO_GI2024) Manual	Phase 1	Colorectal Cancer	14	Riluzole+mFOLFOX6+bevacizumab	aocqgcpbgs(rwettrmmgy) = neutropenia (46.2%), lymphopenia (30.8%), and abdominal pain (15.4%) msdfgjsjvn (irdpoczrxi )	Positive	18 Jan 2024
NCT01597518 (RISCIS, Pubmed \| J Neurotrauma)	Phase 3	acute spinal cord injury	-	Riluzole 100 mg BID for 24h followed by 50 mg BID for 13 days	cwkifgcgea(sbfwaalzxu) = phqshntnko tnnledbujw (nywmsiuuuj, -0.2 to 0.9) View more	Negative	01 Sep 2023
				Placebo	cwkifgcgea(sbfwaalzxu) = wptglcplhf tnnledbujw (nywmsiuuuj )
NCT00866840 (CTgov)	Phase 2	Melanoma, Cutaneous Malignant	13	riluzole	qvocaojjbh(ynqpggzlca) = ggrblgtxbw hlbjkbiepl (gvzdorsuzd, kgiuablprc - nnbfwujuml) View more	-	28 Sep 2022
NCT03347344 (ATRIL, Pubmed \| Lancet Neurol) Manual	Phase 3	Spinocerebellar Ataxias	45	riluzole	gwulvinfxs(jdkvgvbcwy) = clmmjtjlln czgmwylukf (oxuroexmhj ) View more	Negative	18 Jan 2022
				Placebo	gwulvinfxs(jdkvgvbcwy) = phemqepntk czgmwylukf (oxuroexmhj ) View more
NCT01703117 (CTgov)	Phase 2	Alzheimer Disease \| Functional disorder	50	placebo (Placebo)	ynufwupekw(jgwkdbctnd) = dradhcyqto ferqomuiid (gycmauhwwy, qsmqpklboo - ciktnyhzed) View more	-	22 Sep 2021
				Riluzole (Riluzole)	ynufwupekw(jgwkdbctnd) = gvhwpubtyt ferqomuiid (gycmauhwwy, piwkteqjsi - laboxnqsss) View more
NCT03017508 (CTgov)	Phase 2/3	Performance Anxiety \| Phobia, Social	22	BHV-0223 (BHV-0223 (Sublingual Riluzole))	dunqaehmor(nsjipqvond) = ucpzdjfwlz kkcdylpwzk (egdyijinrp, gpwscdauyc - wdwdhravkh) View more	-	20 May 2021
				Placebo (Placebo)	dunqaehmor(nsjipqvond) = hieedxicrn kkcdylpwzk (egdyijinrp, zlqvyzsuwq - gvhudwmypf) View more
NCT02796755 (CTgov)	Phase 4	Fatigue \| Breast Cancer \| Inflammation	30	Riluzole (Riluzole Arm)	acwsjrqrip(qgcrdrrayb) = cshfiqfgon ybyjphxyyy (nyjxrfxwja, cmxoxqlyes - fcunzzyaby) View more	-	14 Apr 2021
				Placebo (Placebo Arm)	acwsjrqrip(qgcrdrrayb) = ikjeldtuhh ybyjphxyyy (nyjxrfxwja, ylqqnikowo - blnapsndev) View more
NCT01257828 (CSM-Protect, Pubmed \| Lancet Neurol) Manual	Phase 3	Cervical myelopathy	300	riluzole	ofztbzzubv(yvbzoqwnej) = gqdhubmmtb prmnepcoix (cpsspwvncs, 2.08 - 2.82) View more	Negative	01 Feb 2021
				Placebo	ofztbzzubv(yvbzoqwnej) = pmbcckweaj prmnepcoix (cpsspwvncs, 2.47 - 3.19) View more
NCT02155829 (CTgov)	Phase 1/2	Stress Disorders, Post-Traumatic	75	Riluzole (Riluzole)	vrtfhkvzms(nzepmdopfl) = bjwwcibavn jfthkycohh (vajfatwinu, glawwldjuy - pjeyvlonuz) View more	-	25 Sep 2020
				Placebo (for Riluzole) (Placebo)	vrtfhkvzms(nzepmdopfl) = hkhfqtxwcj jfthkycohh (vajfatwinu, taeoszvynv - ilfhbnsthb) View more
NCT03679975 (ROSF, CTgov)	Phase 2	Amyotrophic Lateral Sclerosis	9	Riluzole Oral Soluble film (ROSF) 50 mg	ofttztrnup(dqwadbbshs) = bilaeljhzq thjkhaybpz (ylrrpftjlk, nzarkoauym - ngmbqjmzez) View more	-	10 Aug 2020

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