Edaravone

来源：药讯随说 3月12日（当地时间），Clene Inc及其子公司Clene Nanomedicine宣布其纳米药物CNM-Au8在肌萎缩侧索硬化（ALS，渐冻症）临床研究（HEALEY ALS平台研究）患者的交叉方案长期生存事后分析结果积极。 HEALEY ALS平台研究（主研究：NCT04297683）为一项评估多种方案在稳定背景治疗（利鲁唑和/或依达拉奉）ALS患者中有效性、安全性、PK和PD的全球多中心、随机、双盲、安慰剂对照的确证性III期临床研究。研究主要终点为生存改善，次要有效性终点为ALS临床恶化事件延迟时间。 平台试验包括7个方案，分别为：Zilucoplan（泽勒普肽，方案A：NCT04436497）、Verdiperstat（方案B：NCT04436510）、CNM-Au8（方案C：NCT04414345）、Pridopidine（普利多匹定，方案D：NCT04615923）、Trehalose（海藻糖，方案E：NCT05136885）、ABBV-CLS -7262(方案F：NCT05740813）、DNL343（方案G：NCT05842941）。 方案A：患者3：1随机接受24周活性治疗（每天一次皮下注射Zilucoplan，0.3mg/kg）或匹配安慰剂治疗。 方案C：患者2：1随机接受108周活性治疗（30mg）或匹配安慰剂治疗. 本次公布结果如下： 1、全分析集（FAS）： 中位生存：CNM-Au8 30mg组 (方案C,n=59)和方案A对照组（n=162）中位生存天数分别为951天、753天，增加198天（6.5月）； 限制平均生存时间（RMST）：协变量校正RMST增加124天（4.1月）（95%CI：3-245；p=0.045）； 2、中-重度ALS患者增强获益： 基线血清NfL（神经丝轻链蛋白）≥33pg/ml和TRICALS风险得分-6.5至-2.5间人群中，方案C（n=51）较方案A（n=120）中位生存时间从589天提高至951天，增加11.9月； 死亡风险降低44%（Cox HR=0.556;95%CI:0.367-0.842;p=0.006),RMST增加197天（6.5月，95%CI：65-329；p=0.004）； 3、符合入选标准集【血清NfL（神经丝轻链蛋白）≥33pg/ml，TRICALS风险得分-6.5至-2.5间，基线慢肺活量≥60%，发病时间≤36月】 方案C（n=40）较方案A（n=94）中位生存时间从628天提高至1079天，增加14.8月（451天）； 死亡风险降低49%（Cox HR=0.514;95%CI:0.319-0.83;p=0.006),RMST提高215天（7.1月，95%CI：70-360；p=0.004） CNM-Au8是一种具有催化活性、表面清洁、浓缩的金元素多面纳米晶体的水悬浮液。通过提供和/或接收电子驱动大脑中关键的细胞能量生成反应，通过增加神经元和胶质细胞对疾病相关应激的耐受性，使神经修复和髓鞘再生成为可能，并减少有害的反应性分子。 免责声明： 文章内容仅供参考，不构成投资建议。投资者据此操作，风险自担, 关于对文中陈述、观点判断保持中立，不对所包含内容的准确性、可靠性或完整性提供任何明示或暗示的保证。请读者仅作参考，并请自行承担全部。 联系我们 SIT 2025 展位火热预定中！ 扫码立即咨询 电话：13816031174 （同微信） 赞助形式包括但不仅限于演讲席位、会场展位、会刊彩页、晚宴赞助、会议用品宣传等。 点击此处“阅读全文”咨询更多精彩！

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