Edaravone

JERSEY CITY, N.J., March 12, 2025 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of eleven abstracts covering research in amyotrophic lateral sclerosis (ALS) at the 2025 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference being held in Dallas, Texas, March 16-19. "We are looking forward to presenting our latest ALS research, including an innovative study exploring how clinical algorithms may aid in the diagnosis of ALS," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs, MTPA. "We remain dedicated to advancing ALS research and fostering scientific dialogue at medical meetings and congresses on our mission to provide meaningful treatment options and help improve the lives of those affected by this disease." RADICAVA ORS® (edaravone) Studies: Final efficacy and safety results from two multi-center, Phase 3b, double-blind, parallel group studies will be presented, including a comparison of daily dosing of RADICAVA ORS® (edaravone) with the FDA-approved on/off regimen, assessed at 48 weeks (MT-1186-A02) and its extension study (MT-1186-A04). Safety data from a Phase 3 open-label safety extension study for up to 96 weeks (MT-1196-A03) will also be shared. An additional presentation will examine the long-term function and survival of patients treated with RADICAVA ORS using data from propensity score-matched patients in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database. Phase 3b Study MT-1186-A02 to Investigate the Superiority of Daily Dosing vs the FDA-approved On/Off Regimen of Oral Edaravone in Patients with ALS (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Phase 3, Open-Label, Safety Extension Study of Oral Edaravone Administered Over 96 Weeks in Patients with ALS (MT-1186-A03) (Stephen Apple, M.D.; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Phase 3b Study MT-1186-A04 Extension Study to Evaluate the Continued Efficacy and Safety of Radicava ORS® (Oral Edaravone) for up to an Additional 48-Weeks in Patients With Amyotrophic Lateral Sclerosis (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Analysis of Long-term Function and Survival of Radicava ORS® (Oral Edaravone)-Treated Patients With Amyotrophic Lateral Sclerosis vs Propensity Score–Matched PRO-ACT Historical Controls (Stephen Apple, M.D.; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Real World Data: Presentations will highlight a clinical algorithm designed to analyze electronic medical record (EMR) data of neurologists, which aims to reduce delays in ALS diagnosis and treatment. Findings from the ALS/Motor Neuron Disease National History Consortium database will provide insights into the survival, treatment patterns and clinical outcomes of patients treated for ALS. In addition, studies examining referral patterns for ALS diagnosis, as well as demographics and healthcare resource utilization (HCRU) trends across specialty and non-specialty treatment centers, will be shared. Further, two studies will explore point-of-care quality classifications and at-home telespirometry assessments, evaluating forced vital capacity as well as erect and supine slow vital capacity in individuals with ALS. Smartphone Application-Mediated Supervised At-Home Telespirometry Erect and Supine Slow Vital Capacity (eSVC/sSVC) Measurements in Subjects With Amyotrophic Lateral Sclerosis Identify Statistically Significant Differences in eSVC/sSVC Decline as Function of Non-Invasive Ventilation Treatment Status [NCT05106569] (Stephen Apple, M.D.; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Smartphone Application–Mediated, Supervised, At-Home Telespirometry Vital Capacity Measurements in Amyotrophic Lateral Sclerosis: Comparison of American Thoracic Society/European Respiratory Society Point-of-Care Quality Assessment Algorithms Implemented for Slow Vital Capacity and Forced Vital Capacity [NCT05106569] (Stephen Apple, M.D.; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Referral Patterns for Patients With Amyotrophic Lateral Sclerosis Enrolled in a US-Based Administrative Claims Database (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Investigating Application of a Clinical Algorithm on Real-World Electronic Medical Record Data for Earlier Detection of Amyotrophic Lateral Sclerosis (Dung Pham, Ph.D.; MTP-CA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Treatment of Amyotrophic Lateral Sclerosis in General and Specialist US Healthcare Settings: Results From a Real-World Survey (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Survival Benefits and Treatment Patterns of Edaravone–Treated People With Amyotrophic Lateral Sclerosis in the ALS/MND Natural History Consortium (Malgorzata Ciepielewska, M.S.; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 Preclinical Data: Data from a preclinical study examining the effects of edaravone on spinal motor neurons derived from an ALS patient carrying the A382T mutation in TDP-43, as analyzed through a comprehensive transcriptomic approach, will be presented. Transcriptomic Signature Induced by Edaravone in Motor Neurons From an ALS Patient With a TDP-43 Mutation (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA) Poster Session: 6:00 p.m. – 8:00 p.m. CT, March 16 – March 18, 2025 About RADICAVA® (edaravone) and RADICAVA ORS® (edaravone) The U.S. Food and Drug Administration (FDA) approved RADICAVA® (edaravone) on May 5, 2017, and the oral formulation RADICAVA ORS® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA recognized RADICAVA ORS with Orphan Drug Exclusivity based on the major contribution to patient care of the innovative oral formulation. RADICAVA is administered in 28-day cycles by intravenous (IV) infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily for 10 days within a 14-day period, followed by a two-week drug-free period. 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. 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) 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 and RADICAVA ORS have been used to treat over 16,000 people with ALS, with over 2.0-million days of therapy, and have been prescribed by over 2,400 HCPs.2-4 IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions RADICAVA (edaravone) and RADICAVA ORS (edaravone) are 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 with RADICAVA. Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA or RADICAVA ORS, treat per standard of care, and monitor until the condition resolves. Sulfite Allergic Reactions RADICAVA and 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-treated patients were contusion (15%), gait disturbance (13%), and headache (10%). In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS. Pregnancy Based on animal data, RADICAVA and 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 . INDICATION RADICAVA and RADICAVA ORS are indicated for the treatment of amyotrophic lateral sclerosis (ALS). For more information, including full Prescribing Information, please visit . 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), the pharma arm of Mitsubishi Chemical Group (MCG), is one of the oldest pharmaceutical companies in the world, founded in 1678, and focusing on ethical pharmaceuticals. 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 by identifying patient populations with high potential for efficacy and safety. In addition, MTPC is 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

SALT LAKE CITY, March 12, 2025 (GLOBE NEWSWIRE) -- Clene, Inc. (Nasdaq: CLNN) and its subsidiary, Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today announced new evidence from a cross-regimen, post hoc analysis of long-term survival in HEALEY ALS Platform Trial participants. The analyses further substantiate that treatment with CNM-Au8® 30 mg delivers a significant survival benefit for people living with ALS. New Survival Analyses The analyses compared survival in participants who received CNM-Au8 30 mg (Regimen C) to those of Regimen A in the HEALEY ALS Platform Trial. Regimen A provided a large concurrent control group vs. CNM-Au8 treatment using the same randomization criteria established within the HEALEY master protocol. Long-term survival status, determined through public records and site reporting, was evaluated over a follow-up period of up to 48 months. 78% of participants across both groups received standard ALS background therapy (riluzole, edaravone, or both) at baseline. Overall Survival Improvement (All-Cause Mortality) Across the Full Analysis Set: Median Survival: CNM-Au8 30 mg group (Regimen C, n=59) achieved 951 days versus 753 days in the Regimen A comparator group (n=162) – a gain of 198 days (6.5 months) Restricted Mean Survival Time (RMST) Benefit: covariate-adjusted RMST improvement of 124 days (4.1 months) was observed (95% CI: 3 to 245 days, p=0.045). RMST is a metric that estimates the average time a group survives relative to a comparator. The estimate incorporated the prespecified covariates for survival analyses from the HEALEY ALS Platform Trial (i.e., months from symptom onset, pre-treatment ALSFRS-R slope, age, background riluzole treatment, background edaravone treatment) Sensitivity analyses, which included additional covariate models such as baseline serum neurofilament light (NfL) levels, use of ALS background therapy, and the TRICALS risk score, confirmed the robustness and statistical significance of the findings Enhanced Benefit in Moderate to Severe ALS: In participants with baseline serum NfL > 33 pg/mL and TRICALS risk score range between –6.5 and –2.5 (i.e., filtering slow progressors where there was an imbalance between groups), median survival improved from 589 days (Regimen A, n=120) to 951 days (CNM-Au8 30 mg, n=51), representing an 11.9 month gain Mortality risk in this group decreased by 44% (Cox HR: 0.556, 95% CI: 0.367–0.842, p=0.006) with an RMST improvement of 197 days (6.5 month gain; 95% CI: 65 to 329 days, p=0.004), when using the prespecified covariates for survival analyses from the HEALEY ALS Platform Trial Median Survival: CNM-Au8 30 mg group (Regimen C, n=59) achieved 951 days versus 753 days in the Regimen A comparator group (n=162) – a gain of 198 days (6.5 months) Restricted Mean Survival Time (RMST) Benefit: covariate-adjusted RMST improvement of 124 days (4.1 months) was observed (95% CI: 3 to 245 days, p=0.045). RMST is a metric that estimates the average time a group survives relative to a comparator. The estimate incorporated the prespecified covariates for survival analyses from the HEALEY ALS Platform Trial (i.e., months from symptom onset, pre-treatment ALSFRS-R slope, age, background riluzole treatment, background edaravone treatment) Sensitivity analyses, which included additional covariate models such as baseline serum neurofilament light (NfL) levels, use of ALS background therapy, and the TRICALS risk score, confirmed the robustness and statistical significance of the findings In participants with baseline serum NfL > 33 pg/mL and TRICALS risk score range between –6.5 and –2.5 (i.e., filtering slow progressors where there was an imbalance between groups), median survival improved from 589 days (Regimen A, n=120) to 951 days (CNM-Au8 30 mg, n=51), representing an 11.9 month gain Mortality risk in this group decreased by 44% (Cox HR: 0.556, 95% CI: 0.367–0.842, p=0.006) with an RMST improvement of 197 days (6.5 month gain; 95% CI: 65 to 329 days, p=0.004), when using the prespecified covariates for survival analyses from the HEALEY ALS Platform Trial Strongest Survival Benefit Observed in Subset Who Met Planned Phase 3 RESTORE-ALS Trial Enrollment Criteria: Among participants who met the core RESTORE-ALS Trial criteria (e.g., baseline serum NfL > 33 pg/mL, TRICALS risk score range between –6.5 and –2.5, baseline slow vital capacity > 60%, and symptom onset < 36 months), median survival improved from 628 days (Regimen A, n=94) to 1079 days (CNM-Au8 30 mg, n=40), an increase of 451 days (14.8 months) This subset experienced a 49% reduction in mortality risk (Cox HR: 0.514, 95% CI: 0.319–0.830, p=0.006) and an RMST improvement of 215 days (7.1 months; 95% CI: 70 to 360 days, p=0.004), when using the planned covariates for the RESTORE-ALS trial Among participants who met the core RESTORE-ALS Trial criteria (e.g., baseline serum NfL > 33 pg/mL, TRICALS risk score range between –6.5 and –2.5, baseline slow vital capacity > 60%, and symptom onset < 36 months), median survival improved from 628 days (Regimen A, n=94) to 1079 days (CNM-Au8 30 mg, n=40), an increase of 451 days (14.8 months) This subset experienced a 49% reduction in mortality risk (Cox HR: 0.514, 95% CI: 0.319–0.830, p=0.006) and an RMST improvement of 215 days (7.1 months; 95% CI: 70 to 360 days, p=0.004), when using the planned covariates for the RESTORE-ALS trial These results are consistent with previous survival benefits observed in the HEALEY ALS Platform Trial’s 24-week double-blind period, the open-label extension of the Phase 2 RESCUE-ALS trial, and analyses of Expanded Access Programs compared to ALS natural history controls. “We are highly encouraged by these results, as the significant survival advantage demonstrated by CNM-Au8 not only reinforces its potential to extend life for people living with ALS but also validates our strategic direction as we prepare for the launch of our confirmatory Phase 3 RESTORE-ALS study in mid-2025,” stated Rob Etherington, President and CEO of Clene. “We look forward to discussing these findings with the FDA as we advance toward commercialization.” Merit Cudkowicz, M.D., M.S.c., Principal Investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, and executive director of the Mass General Brigham Neuroscience Institute, said, “The innovative design of the HEALEY ALS Platform Trial has enabled us to extract clear and meaningful survival data that helps make decisions about CNM-Au8 drug development.” About Regimen A Regimen A was one of the first three regimens investigated in the HEALEY ALS Platform Trial. Eligible participants were randomized in a 3:1 ratio to receive active treatment or matching placebo for a planned duration of 24 weeks. Participants assigned to Regimen A had to receive both quadrivalent and serotype B meningococcal vaccinations at least 14 days prior to the first dose of study drug, and participants were excluded from Regimen A if they had a history of meningococcal disease or prior treatment with a complement inhibitor. Regimen A was stopped prematurely for futility after all participants had been randomized, and approximately 70% had completed the Week 24 visit. Participants were instructed to discontinue study dosing, and a final early termination study visit was conducted. Long-term survival status of Regimen A participants was tracked from public records and site reporting independently of the early termination. There was no difference in long-term survival in participants randomized to Regimen A active compared to Regimen A placebo, supporting the combined analyses of the entire Regimen A population for comparisons of long-term survival to CNM-Au8 30 mg (Regimen C) participants. About Clene Clene Inc., (Nasdaq: CLNN) (along with its subsidiaries, “Clene” and its wholly owned subsidiary Clene Nanomedicine, Inc.), is a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, including amyotrophic lateral sclerosis, Parkinson’s disease, and multiple sclerosis. CNM-Au8® is an investigational first-in-class therapy that improves central nervous system cells’ survival and function via a mechanism that targets mitochondrial function and the NAD pathway while reducing oxidative stress. CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc. The company is based in Salt Lake City, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit www.clene.com or follow us on X (formerly Twitter) and LinkedIn. About CNM-Au8® CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc. About RESTORE-ALS RESTORE-ALS is a Phase 3 confirmatory global, multi-center, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of CNM-Au8 in participants diagnosed with ALS on stable background therapy. The study is designed to investigate the effects of CNM-Au8 on improved survival (primary endpoint) and delayed time to ALS clinical worsening events (secondary efficacy endpoint). Participants will be randomized in a 2:1 ratio to receive either active treatment with CNM-Au8 30 mg or matched placebo daily during the 108-week double-blind treatment period. The Phase 3 RESTORE-ALS clinical trial, due to launch in mid-2025, is planned to serve as the confirmatory clinical trial required to meet the FDA’s guidance for an “underway” clinical trial when a New Drug Application requesting Accelerated Approval is submitted. 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