RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (Australia), Overseas New Drugs Urgently Needed in Clinical Settings (China), Orphan Drug (Japan)

Structure/Sequence

Molecular FormulaC10H10N2O

InChIKeyQELUYTUMUWHWMC-UHFFFAOYSA-N

CAS Registry89-25-8

Clinical Trials associated with Edaravone

ChiCTR2500099139

Phase 4IIT

Effects of concentrated Edaravone and Dexborneol Concentrated Solution for Injection on inflammatory factors in patients with amyotrophic lateral sclerosis

Start Date20 Mar 2025

Sponsor / Collaborator

Yichang Central People's Hospital

[+1]

NCT06757504

/ RecruitingPhase 2

A Multi-Center, Randomized, Double-Blind, Parallel Group, Placebo-controlled Phase II Trial of TTYP01 Tablets in Adolescents and Children With Autism Spectrum Disorders

This is a multi-center, randomized, double-blind, multiple dose levels, parallal group, placebo-controlled study, to evaluate the safety, PK profiles and preliminary efficacy of TTYP01 tablets in adolescents and children with ASD.

Start Date13 Jan 2025

Sponsor / Collaborator

Shanghai Auzone Biological Technology Co., Ltd.

ChiCTR2400094236

/ SuspendedPhase 3IIT

A clinical study on the treatment of cerebral small vessel disease-related cognitive dysfunction with edaravone sublingual tablets

Start Date30 Dec 2024

Sponsor / Collaborator-

100 Clinical Results associated with Edaravone

100 Translational Medicine associated with Edaravone

100 Patents (Medical) associated with Edaravone

3,934

Literatures (Medical) associated with Edaravone

01 Dec 2025·MOLECULAR BIOLOGY REPORTS

Edaravone reduces brain injury in hepatic encephalopathy by upregulation of Nrf2/HO-1 and inhibition of NF-κB, iNOS/NO and inflammatory cytokines

Article

Author: Amirshahrokhi, Keyvan ; Imani, Mahsa

BACKGROUND:

Brain damage is the most important complication in patients with hepatic encephalopathy (HE). Oxidative stress and inflammation are essential factors in the progression of brain injury caused by HE. The aim of this study was to investigate the potential therapeutic effect of edaravone and its underlying mechanisms against brain injury associated with HE in mice.

METHODS AND RESULTS:

HE was induced by the injection of thioacetamide (200 mg/kg) for 2 days and then mice treated with edaravone (10 or 20 mg/kg/day, ip) for four consecutive days. The brain tissues were dissected for histopathological, biochemical, ELISA, RT-qPCR and immunofluorescence analysis. The results showed that edaravone improved the locomotor function and ameliorated brain histopathological changes in mice with HE. Edaravone inhibited oxidative stress markers by increasing the levels of glutathione, catalase, superoxide dismutase, glutathione reductase and the upregulation of nuclear erythroid 2-related factor (Nrf2)/HO-1 pathway in the brain tissue. Administration of edaravone significantly decreased the expression of p-NF-κB and iNOS. Edaravone treatment reduced the levels of NO, MPO and MMP-9 in the brain of mice. Additionally, the brain levels and expressions of inflammatory cytokines IL-1β, IL-6, TNF-α and IFN-γ were downregulated in mice treated with edaravone.

CONCLUSIONS:

These results suggest that edaravone exerts significant neuroprotection by modulating of inflammatory and oxidative responses in HE and may serve as a promising agent for the treatment of brain injury associated with HE.

01 Nov 2025·Neural Regeneration Research

Biochemical dissection of STAT3 signaling in amyotrophic lateral sclerosis

Article

Author: Apolloni, Savina ; D’Ambrosi, Nadia

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by the loss of upper and lower motor neurons, clin. marked by muscle atrophy and weakness.Although the clin. course is highly variable, the average time from the onset of symptoms to the need for respiratory support or death is 3-5 years.ALS is the most prevalent motor neuron disease in adults, occurring at a rate of 2 per 100,000 individuals and affecting 5.4 per 100,000 individuals overall.

01 Nov 2025·JOURNAL OF PHOTOCHEMISTRY AND PHOTOBIOLOGY A-CHEMISTRY

Molecularly engineered tandem dye-sensitized solar cells achieving 11.02% efficiency through complementary sensitizer integration

Author: Hashem, A. H. ; El-Sadek, Mustafa H. ; Elmorsy, Mohamed R. ; Abdel-Latif, Ehab ; Moawad, Beshoy M. ; Hafiz, Youssef Y. ; Kamel, Abdullah I. ; Shenouda, Thomas T. F. ; Badawy, Safa A.

Tandem dye-sensitized solar cells (DSSCs) represent a promising avenue for advancing solar energy conversion through the mol.-level design of light-harvesting systems.In this study, we report a tandem DSSC architecture that integrates two chem. distinct sensitizers, triphenylamine-based organic dye MRSF-3 and benchmark ruthenium dye N719, selected for their complementary absorption and photophys. characteristics.The device employed a double-sided Pt electrode, with N719 in the top cell and MRSF-3 in the bottom cell, achieving a notable power conversion efficiency (η_cell) of 11.02%.Detailed electrochem. impedance spectroscopy (EIS) and Bode frequency analyses revealed enhanced charge separation, reduced recombination, and prolonged electron lifetimes compared to single-sensitizer systems, highlighting the synergy between the dyes at the mol. level.Thermogravimetric anal. (TGA) confirmed the high thermal stability of both sensitizers, supporting their potential for long-term application.This study provides mol.-level insights into light-induced charge dynamics in hybrid systems and demonstrates how rational photochem. design can optimize light absorption and electron transfer in photoactive mol. assemblies.Our findings contribute to the fundamental understanding of photochem. processes in tandem architectures and underscore their potential in efficient solar energy conversion.

209

News (Medical) associated with Edaravone

19 Aug 2025

·www.prnewswire.com

Mitsubishi Tanabe Pharma America Announces Publication of Long-Term Function and Survival Analysis of RADICAVA ORS® (edaravone)-Treated Patients with ALS

Results from this analysis were published in Muscle and Nerve JERSEY CITY, N.J., Aug. 19, 2025 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the publication of a retrospective analysis of patients living with amyotrophic lateral sclerosis (ALS) in Muscle and Nerve. The analysis evaluated patients taking RADICAVA ORS® (edaravone) compared with Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) historical placebo controls (patients who did not receive active investigational treatment, but may have received riluzole in their respective clinical trials), with evidence suggesting treatment with RADICAVA ORS decreased physical functional decline and offering insights into survival outcomes. "These findings contribute to the ongoing effort to address unmet needs in ALS treatment by providing insight into the benefits observed with RADICAVA ORS," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "By publishing this analysis, we aim to deliver meaningful insights for healthcare providers, patients and caregivers." The primary analysis, based on data from clinical studies MT-1186-A02/A04, evaluated long-term survival and functional outcomes of 78 propensity score-matched patients treated with RADICAVA ORS compared to 78 matched historical placebo controls from the PRO-ACT database. The main statistical comparison was made between the Combined (FDA approved On/Off + investigational Once Daily) RADICAVA ORS versus PRO-ACT placebo group. The post-hoc analysis, based on data from clinical studies MT-1186-A01/A02/A03/A04, expanded the evaluation to a broader ALS population (n=210 RADICAVA ORS patients vs. 210 PRO-ACT placebo patients) to confirm robustness of the initial results. Propensity score matching on 10 baseline variables was used to assess the impact of RADICAVA ORS suspension on function and survival. The analysis showed evidence for a slowing of functional decline and improved survival outcomes with long-term RADICAVA ORS treatment versus PRO-ACT placebo group patients with ALS. The analysis also showed a smaller ALS Functional Rating Scale-Revised (ALSFRS-R) change from baseline, demonstrating a slower rate of functional decline observed over 48 weeks. Key findings from the analysis include: An association was observed between treatment with RADICAVA ORS and improved survival outcomes compared to matched historical placebo patients in the PRO-ACT database. In the primary analysis cohort, the combined RADICAVA ORS group demonstrated a survival benefit vs the PRO-ACT placebo group over approximately 22 months (P=0.005). Three out of the 78 patients (3.8%) died in the RADICAVA ORS group vs 14/78 (17.9%) in the PRO-ACT placebo group. The baseline risk-adjusted hazard ratio showed an 84% decreased risk of death, indicating a longer survival time, in the combined RADICAVA ORS vs PRO-ACT placebo groups (P=0.005). Additionally, positive trends favoring both the Once Daily (n=65) and On/Off RADICAVA ORS (n=65) groups vs PRO-ACT placebo group (n=65) were observed for number of deaths and time to death, but neither one reached statistical significance. In the post hoc broader ALS cohort, patients from studies MT-1186-A01/A02/A03/A04 showed a difference in time to death over approximately 35 months (P<0.001) Additionally, a post hoc covariate-adjusted restricted mean survival time (RMST) analysis to confirm the average survival time over approximately 34 months showed a mean prolongation of 7.3 months in survival (On/Off and Once Daily RADICAVA ORS: 29.6 months; PRO-ACT placebo group: 22.2 months; P<0.001). The proportion of events in the RADICAVA ORS suspension group (29/210 patients, 13.8%) was significantly smaller than the PRO-ACT placebo group (69/210 patients, 32.9%). RMST is a metric that estimates the average time a group survives relative to a comparator. Over 48 weeks, treatment with RADICAVA ORS was associated with a slower rate of decline in ALSFRS-R scores, suggesting potential benefit in maintaining physical function compared to matched PRO-ACT placebo group. In the primary analysis cohort, patients in both groups had a mean baseline ALSFRS-R score of 41.0 points. The least squares (LS) mean change from baseline to week 48 was −8.4 ± 1.0 points for RADICAVA ORS compared to −14.1 ± 1.0 points for PRO-ACT placebo patients, a difference of 5.6 points (95% CI, 2.8–8.4; P<0.001). In the broader post-hoc cohort, the LS mean change was −10.5 ± 0.7 points for RADICAVA ORS compared to −12.9 ± 0.6 points for PRO-ACT placebo patients, a difference of 2.4 points (95% CI, 0.6–4.2; P=0.008). Lastly, it is important to note that results from this analysis are not generalizable and cannot be used to determine definitive conclusions about the effects of treatment. The p-values are nominal as no multiplicity adjustments were made for these analyzed data. Additionally, this was a non-randomized analysis, and results should be interpreted with caution. Please see publication for additional limitations. "There remains no cure for ALS, underscoring the importance of treatments that can slow functional loss for those living with this fatal neurodegenerative condition," said Fumihiro Takahashi, Senior Biostatistician, Mitsubishi Tanabe Pharma Corporation and lead author of the publication. "This analysis provides additional data to help characterize functional outcomes and potential impact of RADICAVA ORS on disease progression in ALS." This analysis was funded and conducted by MTPA. The full publication is titled, "Analysis of Long-term Function and Survival of Edaravone Oral Suspension-Treated Patients With Amyotrophic Lateral Sclerosis Using PRO-ACT Data as Historical Placebo Controls." About the PRO-ACT Database PRO-ACT Dataset is the world's largest ALS clinical trial data repository, compiling placebo and treatment-arm data from 36 phase II/III clinical trials and over 12,500 fully anonymized longitudinal Subject records funded by The ALS Therapy Alliance, Prize4Life, Inc., Northeast ALS Consortium (NEALS), Neurological Clinical Research Institute of Mass. General Hospital, ALS Finding A Cure, and The ALS Association. Neurological Clinical Research Institute of Mass. General Hospital created and maintained the PRO-ACT Dataset and serves as the coordinating center and data distributor of the PRO-ACT Dataset. Find out more at . About Studies MT-1186-A01/A02/A03/A04 Study MT-1186-A01 (NCT04165824) was a phase 3, global, multicenter, open-label study that evaluated the long-term safety and tolerability of the approved edaravone oral suspension 105 mg On/Off dosing regimen over 48 weeks, and Study MT-1186-A03 (NCT04577404) was its extension study, which provided up to 96 weeks of additional treatment. Study MT-1186-A02 (NCT04569084), a post-marketing commitment following the FDA approval of RADICAVA® (edaravone), was a phase 3b, multicenter, double-blind, parallel-group, randomized study that evaluated whether investigational Once Daily edaravone oral suspension dosing was superior to the approved On/Off dosing based on Combined Assessment of Function and Survival, and assessed safety and tolerability over 48 weeks in patients with ALS; and Study MT-1186-A04 (NCT05151471) was its extension study, which provided up to 48 weeks of additional treatment. In Study MT-1186-A02, a pre-planned futility analysis was conducted after 50% of the planned study population (N=190) reached 48 weeks, which assessed the study's primary endpoint and the probability of the study results changing if all participants completed the 48-week study period. Through that interim analysis, the Independent Data Monitoring Committee (IDMC) concluded that there was a low statistical probability for the investigational once-daily dosing regimen to show superiority to the current on/off dosing regimen as measured by the ALS Functional Rating Scale Revised (ALSFRS-R) score at study completion; therefore, study discontinuation was recommended by the IDMC. About RADICAVA ORS® (edaravone) The U.S. Food and Drug Administration (FDA) approved RADICAVA ORS® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA granted RADICAVA ORS Orphan Drug Exclusivity based on its major contribution to patient care by providing an oral suspension route of administration that avoids the burdens of IV administration. RADICAVA ORS is taken daily for 14 consecutive days followed by a 14-day drug-free period for the initial treatment cycle. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. Each 105 mg (5mL) dose of RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.1 Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as RADICUT® for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), Malaysia (December 2021), Australia (February 2023) and Brazil (February 2024). Marketing authorization for RADICAVA® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022. To date, in the U.S., RADICAVA ORS, along with the previously available IV RADICAVA® (edaravone), have been used to treat over 19,000 people with ALS, with over 2.5-million days of therapy, and have been prescribed by over 2,600 HCPs.2-4 INDICATION RADICAVA ORS® (edaravone) is indicated for the treatment of amyotrophic lateral sclerosis (ALS). IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions RADICAVA ORS® (edaravone) is contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals, and erythema multiforme) and cases of anaphylaxis (urticaria, decreased blood pressure, and dyspnea) have occurred. Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA ORS, treat per standard of care, and monitor until the condition resolves. Sulfite Allergic Reactions RADICAVA ORS contain sodium bisulfite, a sulfite that may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible people. The overall prevalence of sulfite sensitivity in the general population is unknown but occurs more frequently in asthmatic people. Adverse Reactions The most common adverse reactions (≥10%) reported in RADICAVA® (edaravone)-treated patients and at least 2% more frequently than placebo were contusion (15% vs 9%), gait disturbance (13% vs 9%), and headache (10% vs 6%), respectively. In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS. Pregnancy Based on animal data, RADICAVA ORS may cause fetal harm. To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You may also report suspected adverse reactions to the FDA at 1-800-FDA-1088 or . Please see the full Prescribing Information, also available at . About Mitsubishi Tanabe Pharma America, Inc. Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit or follow us on X (formerly Twitter), Facebook and LinkedIn. About Mitsubishi Tanabe Pharma Corporation Mitsubishi Tanabe Pharma Corporation (MTPC) is one of the oldest pharmaceutical companies in the world, founded in 1678. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MTPC sets the MISSION of "Creating hope for all facing illness." To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on "precision medicine" to provide drugs with high treatment satisfaction and additionally working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to . Media inquiries: [email protected] 1 RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022 2 Data on file. Mitsubishi Tanabe Pharma America, Inc. 3 Data on file. Mitsubishi Tanabe Pharma America, Inc. 4 Data on file. Mitsubishi Tanabe Pharma America, Inc. SOURCE Mitsubishi Tanabe Pharma America WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultDrug ApprovalPhase 3Orphan Drug

25 Jul 2025

·www.globenewswire.com

AB Science has received approval from several European countries to initiate the confirmatory phase 3 study of masitinib in ALS

AB SCIENCE HAS RECEIVED APPROVAL FROM SEVERAL EUROPEAN COUNTRIES TO INITIATE THE CONFIRMATORY PHASE 3 STUDY OF MASITINIB IN AMYOTROPHIC LATERAL SCLEROSIS THIS APPROVAL FOLLOWS PROTOCOL VALIDATION BY THE EMA AND FDA AUTHORIZATION July 24, 2025, 8am CET AB Science SA (Euronext - FR0010557264 - AB) today announced that the confirmatory Phase 3 study with masitinib in amyotrophic lateral sclerosis (ALS), study AB23005, has been authorized by the first set of European countries (Spain, Greece, Slovenia) in Step 2 of the Clinical Trials Information System (CTIS) procedure. This authorization follows EMA's validation of the harmonized protocol, approved at the end of Step 1 of the CTIS procedure, and followed the authorization from the FDA. Consequently, AB Science can now initiate this registration study in Europe and the United States. Professor Albert Ludolph, MD, PhD (University of Ulm, Germany), principal investigator of the study, said: "Masitinib ALS study AB23005 has great potential because it’s design is based on strong clinical and preclinical data. That is to say, results from the first 48-week Phase 2B/3 study, AB10015, which generated a strong hypothesis with significant survival of several months in the patient population being targeted in this confirmatory study, and also numerous preclinical studies that provide compelling insight into masitinib’s mechanism of action in ALS and its effect on biomarkers such as neurofilaments (NfL) by targeting mast cells and microglia." Alignment with the FDA and EMA on the Phase 3 design and, in particular, the study population focused on patients who respond best to masitinib. Study AB23005 is a prospective, multicenter, randomized, double-blind, placebo-controlled, two-arm study in patients with amyotrophic lateral sclerosis (ALS), to confirm the efficacy and safety of masitinib (at a dose of 4.5 mg/kg/day in combination with riluzole) as compared against riluzole in combination with placebo after 48 weeks of treatment. The study will include 408 patients (randomized 1:1) with ALS, with normal disease progression (i.e., functional decline of less than 1.1 points per month) and no total loss of function (i.e., a score of at least 1 on each of the 12 items of the ALSFRS-R score). US patients receiving edaravone will also be eligible to participate in the study, with the use of this drug being a stratification factor. This design has been validated in discussions with European health authorities, particularly with regard to the criteria for the optimal population selected for the confirmatory study: Patients without rapid progression: Experts of the EMA's Scientific Advisory Group on Neurology (SAG-N) considered the categorization of the study population with normal progressors, defined as an average rate of change in the ALSFRS-R of less than 1.1 points per month, as clinically relevant and consistent with the expected progression of the disease, and therefore acceptable when predefined, which is the case for this study. Patients without complete loss of function: The SAG-N experts considered that the ALSFRS-R scale is widely used in clinical practice and that administration criteria are available for healthcare professionals. Therefore, the subgroup of patients with very severe ALS (who have a score of zero on at least one of the 12 individual items of the ALSFRS-R) can be easily identified in clinical practice. In this subgroup, defined as patients before complete loss of function and with normal disease progression, which corresponds to the optimal population of best responders to masitinib that is targeted in study AB23005, the AB10015 study generated extremely robust results, with a median survival increase of +12 months. This optimal population represents approximately 75% of the total patient population. The optimal population included approximately 90 patients per treatment group in the AB10015 study. The effect of masitinib was statistically significant (p=0.0290) on the CAFS endpoint, which is the endpoint recognized by the FDA. The AB23005 study will enroll approximately 200 patients per treatment group, more than double the number in the AB10015 study, in order to achieve high statistical power for this test and maximize the chances of statistical success. Masitinib, thanks to its well-demonstrated mechanism of action, particularly its immunomodulatory properties targeting mast cells and microglia, preserves neuromuscular function. The immunomodulatory properties of masitinib in ALS have been well demonstrated in a preclinical setting using a relevant model that replicates the complexity of a multicomponent immune response with concomitant evidence of neurodegeneration [1-4]. The mechanism of action of masitinib in ALS has been confirmed by independent research. Harrison and colleagues showed that treatment of SOD1G93A mice with masitinib significantly reduced macrophage infiltration, prevented the loss of terminal Schwann cells and , and improved reinnervation of partially denervated plantar muscles [5]. Recent pharmacological data have shown that mast cells infiltrate the degenerating spinal cord in both mouse models of ALS and in patients with ALS [6]. The results indicate a protective effect of masitinib. Masitinib has demonstrated its ability to reduce blood levels of neurofilament light (NfL) in a model of neurodegenerative disease (EAE model) [7]. Masitinib has demonstrated its ability to restore motor performance in zebrafish expressing the TDP-43 mutant. Overall, these preclinical results establish the biological plausibility of masitinib for use in ALS and support the hypothesis that masitinib may offer clinical benefit if administered early in the disease course (i.e., before the point of permanent loss of function). Based on the results of the AB10015 study, AB Science has filed a patent application for methods of treating ALS (i.e., a secondary medical use patent) with its lead molecule, masitinib, and this patent has been granted in all jurisdictions where it has been filed. This patent provides strong protection for masitinib in the treatment of ALS until 2037 and in all geographical areas where masitinib could be marketed, including Europe (patent EP 3240538), the United States (US 10092564), Canada (CA 3018635), China (ZL201780019760. 9), South Korea (KR 10-2293847), Japan (JP 7250312B2), Singapore (SG 11201808106Y), Hong Kong (HK 1261581), Israel (IL 261856), Australia (AU M53001274), Eurasia (EA 201800499), Mexico (MX 390495), New Zealand (NZ 745778) and South Africa (ZA 2018/05810). In addition, masitinib has been designated as an orphan drug for ALS by both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). This orphan drug designation confers 10 and 7 years of marketing exclusivity in Europe and the United States, respectively, from the date of product registration. Such status may also be sought in Japan. If granted, the period of commercial exclusivity is extended to 10 years (compared with 4 to 6 years for conventional drugs). References: [1] Trias E, Kovacs M, King PH, et al. Glia. 2020;68(6):1165-1181. [2] Trias E, King PH, Si Y, et al. JCI Insight. 2018;3(19):e123249. [3] Trias E, Ibarburu S, Barreto-Núñez R, et al. JCI Insight. 2017;2(20):e95934. [4] Trias E, Ibarburu S, Barreto-Núñez R, et al. J Neuroinflammation. 2016;13(1):177. [5] Harrison JM, Rafuse VF. Neurobiol Dis. 2020;145:105052. [6] Kovacs M, Alamón C, Maciel C, et al. Acta Neuropathol Commun. 2021;9(1):136. [7] Hermine O, Gros L, Tran T-A, et al. (2025) PLoS ONE 20(4):e0322199. Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment. AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultOrphan Drug

02 Jul 2025

·www.einpresswire.com

Amyotrophic Lateral Sclerosis (ALS): Competitive Intelligence Insight into Next-Gen Therapies | DataM Intelligence

ALS treatment is evolving from symptom management to disease-modifying strategies gene therapies, antisense drugs, & neuroprotective agents promise real impact ALS, once incurable, is seeing remarkable progress. With gene interventions and neuroprotective molecules entering late-stage trials, the vision of slowing or halting progression is closer than ever.” — DataM Intelligence AUSTIN, TX, UNITED STATES, July 2, 2025 / EINPresswire.com / -- Amyotrophic Lateral Sclerosis (ALS) , often known as Lou Gehrig’s disease, is a fatal neurodegenerative disorder that progressively destroys motor neurons, leading to paralysis and death. It remains one of the most complex and devastating neurological diseases, with a prognosis of just 2 to 5 years post-diagnosis for most patients. However, the landscape in 2025 reflects significant progress. With deeper genetic insights and a diversified therapeutic pipeline, ALS is transitioning from symptom management toward disease modification. Download CI Sample Report: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🧬 Disease Overview ALS causes the degeneration of both upper and lower motor neurons, disrupting voluntary muscle activity. This leads to severe muscle wasting, difficulties in swallowing and breathing, and ultimately, respiratory failure. While about 90% of ALS cases are sporadic, the remaining 10% are familial, frequently linked to mutations in genes such as SOD1, C9orf72, TARDBP, and FUS. Diagnosis is typically clinical, supported by electromyography (EMG), nerve conduction studies, and exclusion of mimicking conditions. Biomarker development is ongoing, aiming to enable earlier diagnosis and patient stratification for clinical trials. 🌍 Epidemiology & Market Landscape Globally, ALS affects an estimated 2–5 people per 100,000, with an annual incidence of 1–2 per 100,000. The disease is more common in individuals aged 55–75 and is slightly more prevalent in men. Due to its high mortality and lack of curative therapies, ALS qualifies for orphan drug status in major markets. This regulatory incentive, combined with strong advocacy and rising R&D investment, is accelerating the therapeutic pipeline. The ALS treatment market, valued at around USD 2 billion in 2024, is projected to grow steadily with the entry of novel disease-modifying therapies over the next five years. 💊 Current Treatment Paradigm The current standard of care is limited to supportive and symptomatic therapies. These include: - Riluzole: Approved in 1995, it modestly extends survival by a few months by reducing glutamate-mediated excitotoxicity. - Edaravone: Approved in 2017, it acts as a free radical scavenger, slightly delaying functional decline in early-stage ALS. - Nuedexta: Although not approved specifically for ALS, it is commonly used to manage pseudobulbar affect (PBA), improving emotional stability. While helpful, these therapies do not halt or reverse the disease, highlighting a clear unmet need for disease-modifying approaches. Book Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🔬 Pipeline Innovation and Competitive Momentum The ALS pipeline in 2025 is driven by a multi-pronged approach, including gene silencing, neuroprotection, immunomodulation, and cellular therapies. Key areas of innovation include: Gene-Targeted Therapies - Biogen’s Tofersen, an antisense oligonucleotide (ASO) targeting SOD1 mutations, has shown promise in reducing SOD1 protein levels and slowing disease progression in familial ALS. It represents the first real attempt at targeting a genetic driver in ALS with precision. - Another gene-targeted agent, BIIB100, is in early-phase trials to treat C9orf72-associated ALS, a common cause of familial and sporadic cases. These therapies are designed to address root-cause mutations and could revolutionize ALS management in genetically stratified patients. Neuroprotective and Combination Therapies - AMX0035, a fixed-dose combination of sodium phenylbutyrate and taurursodiol, showed significant survival benefit and slower functional decline in late-phase trials. With a favorable safety profile and oral administration, it’s emerging as a strong candidate for broad use across ALS populations. - Other agents in this class aim to support mitochondrial function, reduce ER stress, or modulate excitotoxicity-core mechanisms implicated in ALS pathogenesis. Immuno-inflammatory Modulators - Masitinib, a tyrosine kinase inhibitor, targets neuroinflammation and microglial activation. Now in Phase III, it could become the first oral immune-modulating agent with broad applicability in ALS if efficacy is confirmed. - Avonex (interferon-beta) and other repurposed immune therapies are being evaluated to address inflammatory aspects of ALS, particularly in patients showing elevated biomarkers of immune activation. Stem Cell and Cellular Approaches Stem cell infusions, particularly mesenchymal stem cells (MSCs), are under evaluation for their potential to protect or repair damaged motor neurons. These approaches remain experimental but are gaining attention for their regenerative promise. 🔍 Competitive Intelligence – Descriptive Landscape Tofersen is leading the field in genetically targeted therapy for SOD1-mutated ALS. Its ability to slow functional decline and reduce neurofilament biomarkers puts it ahead in terms of precision and biological targeting. In the broader ALS population, AMX0035 offers a disease-modifying option with strong survival data and ease of use. It stands out as the first combination therapy likely to gain global traction for sporadic ALS. Masitinib, still under evaluation, represents a promising oral option for patients with inflammatory phenotypes. Its oral route and broad immunologic targeting could position it as a foundational therapy if efficacy and tolerability are confirmed. Meanwhile, stem cell therapies and early-stage gene-editing solutions such as BIIB100 are advancing with hopes of establishing novel, durable interventions. These will likely serve as adjuncts or niche solutions depending on patient genotype and disease stage. 🎯 Target Opportunity Profile (TOP): What Success Looks Like For any new therapy to thrive in the ALS market, it must deliver across a range of clinical and strategic attributes: - Clinical Efficacy: Demonstrated improvement in survival and/or functional endpoints such as ALSFRS-R or time to tracheostomy. - Safety and Tolerability: Minimal systemic side effects, especially critical in a fragile patient population with reduced physiological reserve. - Mechanism Differentiation: Gene-targeted therapies should address known mutations; other candidates must tackle neuroinflammation, oxidative stress, or neuronal energy failure. - Ease of Administration: Oral or infrequent dosing regimens are preferred. Intrathecal administration may be acceptable for precision therapies like ASOs. - Payer & Access Considerations: Orphan status, cost-effectiveness, and robust real-world data will be vital for widespread adoption and reimbursement. - Patient Stratification: Success depends on biomarker-driven selection, especially for genetic or inflammatory subtype-specific therapies. Ask for Customized CI Service as per Your Business Requirement: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🔮 Market Outlook: 2025 and Beyond The years ahead will witness critical inflection points: - Tofersen is expected to reshape treatment in genetically confirmed SOD1-ALS, catalyzing greater adoption of genetic testing. - AMX0035 will likely establish itself as the first broadly used disease-modifying therapy. - Masitinib may expand treatment choices for sporadic ALS if it confirms efficacy in ongoing trials. - Gene therapies and cell-based innovations will push the envelope toward true disease reversal or stabilization. ✅ Conclusion After decades of frustration, the ALS field is entering a breakthrough era. New therapies are moving beyond symptomatic support to targeting the underlying biology of the disease. From gene silencing to neuroprotection and immunomodulation, the coming years hold unprecedented potential to extend and improve lives. For patients, caregivers, and industry stakeholders, ALS is no longer a closed chapter-it’s a rapidly unfolding story of innovation. Read Related CI Reports: 1. Focal Segmental Glomerulosclerosis | Competitive Intelligence 2. Vitiligo | Competitive Intelligence Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 sai.k@datamintelligence.com Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Phase 3Drug ApprovalOrphan DrugOligonucleotideImmunotherapy

100 Deals associated with Edaravone

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KEGG	Wiki	ATC	Drug Bank
D01552	Edaravone	N07XX14	DB12243

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	Japan	Mitsubishi Tanabe Pharma Corp.	26 Jun 2015
Brain Infarction	Japan	Mitsubishi Tanabe Pharma Corp.	26 Jun 2015
Acute Ischemic Stroke	China	Nanjing Simcere Dongyuan Pharmaceutical Co. Ltd.	31 Dec 2003

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis 2, Juvenile	Phase 3	Japan	Mitsubishi Tanabe Pharma Corp.	01 Dec 2011
Alzheimer Disease	Phase 3	Netherlands	Treeway BV	-
Autism Spectrum Disorder	Phase 2	China	Shanghai Auzone Biological Technology Co., Ltd.	13 Jan 2025
Androgen-Insensitivity Syndrome	Phase 2	China	Shanghai Auzone Biological Technology Co., Ltd.	19 Sep 2023
Autistic Disorder	Phase 2	China	Shanghai Auzone Biological Technology Co., Ltd.	-
Liver Injury	Phase 1	Japan	Mitsubishi Tanabe Pharma Corp.	16 Nov 2016
Chronic Kidney Diseases	Phase 1	Japan	Mitsubishi Tanabe Pharma Corp.	27 Oct 2016
Ischemic stroke	Preclinical	China	Tongji University	01 Aug 2025
Cardiovascular Diseases	Preclinical	China	Peking University	18 Mar 2025
Cardiovascular Diseases	Preclinical	China	Peking University Third Hospital	18 Mar 2025

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05151471 \| NCT04165824 \| NCT04569084 \| NCT04577404 (MT-1186-A04, Pubmed) Manual	Not Applicable	Amyotrophic Lateral Sclerosis	420	Edaravone oral suspension	crohhiwcux(eawqzspyuy) = 78 edaravone oral suspension-treated patients from Studies MT-1186-A02/A04 demonstrated a survival benefit versus 78 matched PRO-ACT placebo patients (p = 0.005) lfvgfuazog (xpvwdtgvuu ) View more	Positive	01 Jul 2025
NCT05151471 (MT-1186-A04, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	202	Edaravone (Edaravone 105 mg (Once Daily))	vknjneeubr(tdimvqmksp) = pixcqdxrvj kabrqamvtk (pfzxhwfcoa, tastxcjync - wrkazgfold) View more	-	27 Mar 2025
				Edaravone (Edaravone 105 mg (on/Off))	vknjneeubr(tdimvqmksp) = gnicjsjouc kabrqamvtk (pfzxhwfcoa, igxmwkcqak - rwvohibapo) View more
NCT04569084 (MT-1186-A02, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	384	Edaravone (Edaravone 105 mg (Once Daily))	djtzocarrh(pgxvfnlpxd) = xdsnapbswk feikjfsfzc (ryefusmoej, uzmzuyexpi - hskzlwnfiu) View more	-	27 Mar 2025
				Edaravone (Edaravone 105mg (2 Weeks On/Off ))	djtzocarrh(pgxvfnlpxd) = flzspzsisl feikjfsfzc (ryefusmoej, nbmqlkautr - lgvjlknmaj) View more
NCT05178810 (ADOREXT \| ADORE, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	313	FAB122 (FAB122)	rvxzhxzzwq(dssdsamgxc) = qgtoadsvtz auspcubqjo (pugatlskzb, 7.9) View more	-	18 Mar 2025
				Placebo (Placebo)	rvxzhxzzwq(dssdsamgxc) = wanfdkaxfn auspcubqjo (pugatlskzb, 8.4) View more
NCT05866926 (ADOREXT, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	201	FAB122 (FAB122)	wmrlmmgtba = rpymgqwcja lsxlzvdchz (vogaslwcqw, hjrvlsfxjd - fhucemftxu) View more	-	04 Mar 2025
				FAB122 (Placebo)	wmrlmmgtba = sgotmarums lsxlzvdchz (vogaslwcqw, fwktvoxoks - kzjdoicyzh) View more
NCT05568615 (CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	15	MT-1186	gtybuuiuga = zsidptymgn wgbfjqoyfx (sbkjagxxiq, feopbnighh - upatwgxyfv) View more	-	03 Feb 2025
NCT03346538 (CTgov)	Phase 2	Acute Ischemic Stroke	17	Approved dosing regimen placebo+Continuous infusion high-dose MCI-186 (Continuous Infusion High-dose Group (Group H))	aatwcknfki = abjopaqoxe sifwxgcqqc (slhzufvify, qqrywvyyoz - mgysadlokc) View more	-	09 Jan 2025
				Approved dosing regimen placebo+Continuous infusion low-dose MCI-186 (Continuous Infusion Low-dose Group (Group L))	aatwcknfki = djzrhdigad sifwxgcqqc (slhzufvify, exasglzcda - huisjaioiv) View more
NCT04577404 (MT-1186-A03, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	124	MT-1186	rcopdwbhkp = qbasajzalu nbqzqypfwi (udxdzafcqi, lmaasddkns - wpukcidbap) View more	-	28 Aug 2024
NCT04577404 (MT-1186-A03, Biospace) Manual	Phase 3	Amyotrophic Lateral Sclerosis	-	RADICAVA ORS	iruodhktuj(wwfbivafwc) = fall, muscular weakness, dyspnea, constipation, and dysphagia. wfzqbcwitm (adsckdtzqx )	Positive	17 Jun 2024
AAN2024	Not Applicable	Amyotrophic Lateral Sclerosis	-	Edaravone-naive patients	fytzzdhxdr(iiwwgcxyjg) = hhnvnvkcxo xllpqggait (dsjpbtahka )	-	09 Apr 2024
				Edaravone-experienced patients	fytzzdhxdr(iiwwgcxyjg) = lofnbccomr xllpqggait (dsjpbtahka )

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