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New pharmacokinetic and cardiac biomarker data further demonstrate ifetroban's potential to protect the heart and reduce cardiac damage in DMD patients NASHVILLE, Tenn., June 23, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company committed to developing new products for rare diseases, shared the latest positive results from its Phase 2 FIGHT DMD trial evaluating ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD) heart disease, at the annual Parent Project Muscular Dystrophy (PPMD) conference in Las Vegas. These new results highlight multiple indicators of cardiac benefit with ifetroban treatment in DMD heart disease, the leading cause of death in DMD, including previously unreported pharmacokinetic findings and cardiac biomarker data that demonstrate the drug's potential to prevent ongoing heart damage. The trial results were presented this afternoon by FIGHT DMD Trial Principal Investigator Larry W. Markham, MD, Professor of Pediatrics and Medicine at Riley Children's Hospital and the Indiana University School of Medicine, as part of the "Therapies that Slow Progression" conference session. "PPMD has had a focus on heart disease and has been a part of this trial since its inception. We are proud to share the latest promising findings from the FIGHT DMD Trial with a group that has consistently supported us throughout our efforts to develop an effective treatment for DMD heart disease," said Dr. Markham. The complete slide presentation is now available on Cumberland's website. "Seeing these promising results validates our belief that targeted heart therapies can make a meaningful difference for our children," said Terry Marlin, President and Founder of FIGHTDMD, a community-based organization with the mission of advancing DMD research. "This trial represents what's possible when families refuse to accept the status quo and invest in advancing medical research." FIGHTDMD helped co-fund the preclinical studies at Vanderbilt's Monroe Carell Jr. Children's Hospital which formed the foundation for the FDA grant funding this trial. The trial was named The FIGHT DMD Trial in honor of their organization's early support. The Marlin family also provided input into the clinical trial design and contributed personal photos for the trial's patient brochure. The 12-month Phase 2 FIGHT DMD trial (NCT03340675) demonstrated that high-dose ifetroban treatment resulted in a significant 5.4% improvement in left ventricular ejection fraction (LVEF) compared to a control group composed of placebo-treated patients combined with propensity score-matched natural history patients. This represents a clinically meaningful difference in a progressive disease where heart function typically declines over time. Critically, high-dose ifetroban treatment was associated with reduced blood levels of cardiac damage markers (NT-proBNP and cardiac troponin I), while these markers of heart damage increased in placebo-treated patients, suggesting ifetroban could help prevent ongoing cardiac injury. This biochemical evidence of reduced cardiac damage, combined with functional heart improvement, provides compelling evidence of ifetroban's cardioprotective effects. The study also revealed pharmacokinetic insights, showing that DMD patients receiving higher doses than typical adults achieved similar plasma levels, with no evidence of drug accumulation, supporting the 300 mg daily dosing used in the high-dose group. Despite the higher dosing requirements, ifetroban was well-tolerated with an acceptable pharmacokinetic profile in patients with DMD. The observed improvement in cardiac function together with the reduction in cardiac damage biomarkers suggest a clinically significant impact for ifetroban in both heart disease and potential to impact overall survival in DMD. Demonstrating confidence in the treatment, all patients who completed the 12-month study opted to continue with the open-label extension. "These new results reinforce our conviction that ifetroban has the potential to address the leading cause of death in DMD patients," said A.J. Kazimi, Cumberland CEO. "The improvement in heart function observed, combined with the reduction in cardiac damage markers, suggests we may be able to meaningfully impact the progressive heart disease that affects virtually all DMD patients. What's particularly encouraging is that there is both functional improvement and biochemical evidence of reduced cardiac injury - exactly what you'd want to see in a therapy designed to protect the heart. We're deeply grateful to both Terry Marlin and FIGHT DMD for their early vision and funding and to PPMD for their longstanding support and partnership throughout this journey – as both organizations have been instrumental in making this breakthrough possible. We're optimistic about taking the next steps toward bringing this much-needed treatment to DMD patients and their families." Ifetroban is a once-daily oral medication that works by blocking the thromboxane receptor, which plays a key role in inflammation and fibrosis. The drug has received both Orphan Drug Designation Rare Pediatric Disease Designation, from the FDA for the indication of DMD heart disease. There is currently no approved treatment specifically targeting DMD heart disease, highlighting the critical unmet medical need in this patient population where cardiac complications are universal and represent the leading cause of death. Cumberland has secured a growing portfolio of patents protecting the product for this DMD heart disease indication. Next steps include the analysis of long-term treatment results and discussions with the FDA to determine the regulatory pathway forward based on these encouraging results. More information regarding The FIGHT DMD Trial can be found here: www.fightdmdtrial.com About Duchenne Muscular Dystrophy (DMD) DMD is a rare and incurable pediatric disease caused by mutations in the gene encoding dystrophin, a protein critical for muscle function, including the heart. Patients with DMD slowly lose muscle function, resulting in the inability to walk, difficulty breathing, and heart failure. While current treatments can help manage some DMD symptoms, there are no approved therapies specifically targeting DMD-related heart disease, highlighting a critical unmet medical need. About Parent Project Muscular Dystrophy (PPMD) Parent Project Muscular Dystrophy is a grassroots, parent-led advocacy group with the mission to end DMD. Since its founding in 1994, PPMD has helped to accelerate treatments through research funding, to provide access to optimal DMD care for families, and to affect legislation through advocacy to improve the lives of children with DMD and their families. PPMD also hosts an annual conference which is the largest, most comprehensive, annual international conference focused entirely on DMD. This conference connects families across the world to share their stories and serves as a forum to highlight the progress in ending DMD. About FIGHT DMD FIGHT DMD is a community-based organization founded by Terry and Sonya Marlin with the mission of advancing research into DMD heart disease. The organization has provided crucial early-stage funding for multiple research projects focused on understanding and treating cardiac complications in DMD patients. Their support has been instrumental in advancing preclinical research that led to FDA grant funding for clinical trials, demonstrating the power of family-driven advocacy in catalyzing medical innovation. About Cumberland Pharmaceuticals Cumberland Pharmaceuticals Inc. is the largest biopharmaceutical company founded and headquartered in Tennessee and is focused on providing unique products that improve the quality of patient care. The company develops, acquires, and commercializes products for the hospital acute care, gastroenterology and oncology market segments. The company's portfolio of FDA-approved brands includes: Acetadote® (acetylcysteine) injection, for the treatment of acetaminophen poisoning; Caldolor® (ibuprofen) injection, for the treatment of pain and fever; Kristalose® (lactulose) oral, a prescription laxative, for the treatment of constipation; Sancuso® (granisetron) transdermal, for the prevention of nausea and vomiting in patients receiving certain types of chemotherapy treatment; Vaprisol® (conivaptan) injection, to raise serum sodium levels in hospitalized patients with euvolemic and hypervolemic hyponatremia; and Vibativ® (telavancin) injection, for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections. The company also has a series of Phase II clinical programs underway evaluating its ifetroban product candidate in patients with cardiomyopathy associated with Duchenne Muscular Dystrophy, Systemic Sclerosis and Idiopathic Pulmonary Fibrosis. For more information on Cumberland's approved products, including full prescribing information, please visit the individual product websites, which can be found on the company's website www.cumberlandpharma.com. Forward-Looking Statements This press release contains forward-looking statements, which are subject to certain risks and reflect Cumberland's current views on future events based on what it believes are reasonable assumptions. No assurance can be given that these events will occur. Forward-looking statements include, among other things, statements regarding the company's intent, belief or expectations, and can be identified by the use of terminology such as "may," "will," "expect," "believe," "intend," "plan," "estimate," "should," "seek," "anticipate," "look forward" and other comparable terms or the negative thereof. As with any business, all phases of Cumberland's operations are subject to factors outside of its control, and any one or combination of these factors could materially affect Cumberland's operation results. These factors include macroeconomic conditions, including rising interest rates and inflation, competition, an inability of manufacturers to produce Cumberland's products on a timely basis, failure of manufacturers to comply with regulations applicable to pharmaceutical manufacturers, natural disasters, public health epidemics, maintaining an effective sales and marketing infrastructure, and other events beyond the company's control as more fully discussed in its most recent annual report on Form 10-K as filed with the U.S. Securities and Exchange Commission ("SEC"), as well as the company's other filings with the SEC from time to time. There can be no assurance that results anticipated by the company will be realized or that they will have the expected effects. Readers are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date hereof. The company does not undertake any obligation to publicly revise these statements to reflect events after the date hereof. View original content to download multimedia:https://www.prnewswire.com/news-releases/cumberland-pharmaceuticals-shares-phase-2-fight-dmd-trial-results-at-the-parent-project-muscular-dystrophy-annual-conference-302487504.html SOURCE Cumberland Pharmaceuticals Inc. Investor Contact: Shayla Simpson, Cumberland Pharmaceuticals, (615) 255-0068; Media Contact: Emily Kent, Dalton Agency, (540) 621-5448

6月13日至14日，2025年第二届中欧脑心联盟俱乐部学术会议在上海圆满落幕。本次会议由中国神经科学学会精神病基础与临床分会、意大利地中海自由大学、意大利帕多瓦大学主办。会议汇聚了来自中欧的顶尖医学专家，围绕心血管医学、精神医学及皮肤医学等多个领域展开深入交流，为推动跨国医学创新发展搭建了重要平台。6月13日下午，进行了两个专题的会议。上半场聚焦“肺动脉高压规范化诊疗”，由意大利地中海自由大学的Sabino Iliceto教授、复旦大学附属华山医院的李圣青教授以及李氏大药厂控股有限公司（简称“李氏大药厂”或“集团”，股份编号：00950）董事总经理李烨妮女士担任共同会议主席，与中欧肺动脉高压领域的专家共同探讨了该领域的最新诊疗规范和研究进展。下半场则围绕“心血管病与皮肤医学”展开，由Sabino Iliceto教授、海军军医大学附属长征医院的温海教授和李烨妮女士担任主席，中欧皮肤领域的专家们就心血管疾病与皮肤医学之间的交叉关系进行了深入探讨，旨在促进学科间的融合与合作。6月14日上午，会议重点聚焦双心医学的前沿进展。东南大学附属中大医院的袁贵勇教授、意大利地中海自由大学的Sabino Iliceto教授以及李氏大药厂董事总经理李烨妮女士担任共同主席。来自意大利及国内精神科、心血管科、皮肤科等领域的顶尖专家齐聚一堂，围绕“双心医学的前沿进展”这一交叉领域的研究进行了深入交流。在开场致辞中，三位主席均强调了本次学术会议对双心医学发展的重要推动作用。袁永贵教授特别指出，通过心脏科与精神科的协作，中国心身医学在疑难病症诊疗、医疗资源利用、患者经济负担及医患沟通等方面取得了显著突破。意大利Sabino Iliceto教授则呼吁现代医学应突破单纯的器官诊疗模式，建立“身-心-脑轴”的整体观。李烨妮女士在致辞中回顾了李氏大药厂自2015年与意大利Iliceto教授团队开展双心医学学术会议中国巡讲以来的成果，已成功举办43场跨国论坛，覆盖3000余名临床医生。她表示，本次会议聚焦“双心医学的前沿进展”以及“精神医学与皮肤医学的交叉领域”，将进一步提高医学界对这些治疗领域的关注，推动医学的进步。上午的会议由上海市精神卫生中心的陈俊教授担任学术报告环节主持，中意专家进行了四场精彩纷呈的学术报告。首都医科大学附属北京安贞医院的何东方教授以“双心医学”为核心，强调了其在临床实践中的重要性；帕多瓦大学的Francesco Tona教授从神经影像学角度分享了“Takotsubo心肌病的脑源性起源”的研究成果；Giovanni Civieri博士则围绕“忧脑・伤心：抑郁症与心血管疾病的关联”展开，阐述了抑郁症与心血管疾病之间的紧密联系，并强调了抑郁症治疗对降低心血管疾病风险的重要性；复旦大学附属中山医院的戴能博士通过实际病例和影像学分析，探讨了“心脑交互与冠状动脉斑块”的预后价值。在随后的讨论环节中，专家们围绕上午的讲题进行了热烈讨论，分享了各自在抑郁症和心血管疾病相关研究与临床实践中的见解，现场气氛活跃，中意双方专家就双心诊疗领域的前沿研究和合作进行了深入探讨。下午会议开展前，首先进行了Matrix Breaking News 2025第二期内容的录制。地中海自由大学的Sabino Iliceto教授首先介绍了Matrix Breaking News 2025项目，随后复旦大学附属中山医院的王继纳教授联合帕多瓦大学的Giovanni Civieri博士现场就肾移植和抑郁症的关联进行讨论交流。下午场的会议聚焦精神医学与皮肤医学领域，由上海市精神卫生中心的彭代辉教授与意大利地中海自由大学的Sabino Iliceto教授担任共同主席。来自意大利及国内精神科、皮肤科、脑科学等领域的顶尖专家再次汇聚，就“精神医学与皮肤医学”这一主题进行了深入探讨和交流。彭代辉教授在开场致辞中强调了精神医学在皮肤科相关疾病诊治中的重要性，并指出了精神科医生在治疗过程中可能面临的多学科交叉问题，呼吁加强跨学科交流与合作。Sabino Iliceto教授则指出抑郁与皮肤病之间存在很强的相关性，抑郁情绪会对身体的各个系统和器官产生影响，包括皮肤系统。同济大学附属同济医院的黄欣教授担任主持，开启了下半场的学术盛宴。上海市精神卫生中心的洪波教授以“精神类药物在心身性皮肤病中的应用”为题，详细介绍了心身性皮肤病的诊疗情况、精神类药物的应用以及对未来加强精神皮肤医学相关培训的展望；帕多瓦大学的Stefano Piaseric教授则以“脑-皮轴：综合视角”为题，介绍了银屑病的免疫发病机制，并深入讲解了抑郁症和皮肤疾病之间形成恶性循环的因素；复旦大学脑科学研究院的韩清见研究员以“躯体感觉神经与皮肤免疫交互作用”为题，通过详细的图解展示了皮肤免疫与躯体感觉神经的联系和作用机制。在讨论环节中，六位专家围绕下午的讲题进行了深入探讨，分享了各自对精神医学与皮肤医学前沿研究与临床实践的洞见，现场专家积极参与提问和讨论，进一步推动了中意双方在精神医学与皮肤医学交叉领域的前沿研究和合作。此次会议还专门举行了“跨越山海・心身同治”为主题中欧脑心俱乐部媒体发布会，吸引了《国际循环》《人民日报》《神经时讯》《门诊》《医学界》等多家媒体到场。在媒体发布会上，媒体记者就心身医学的现状和未来、多学科合作、企业责任和创新研发等关键问题进行了提问，专家们一一耐心解答，现场气氛热烈。此次接受访谈的共有五位专家学者，面对媒体提问，意大利地中海自由大学的Sabino Iliceto教授指出，现代医学正从“器官中心”向“整体观”转变，大脑是身心交互枢纽，心理因素在慢性病发展中作用关键，中国双心医学发展需建立“大脑优先”诊疗范式，人工智能将成重要工具。东南大学附属中大医院袁永贵教授称，心身医学近年来在科普、诊疗理念、规范体系、脑科学研究等方面取得了实质性进展，未来应建立“三维协同”模式，强调媒体传播对公众心理觉察能力培养的重要性。上海市精神卫生中心的陈俊教授表示，心身医学是精神医学重要部分，应予以高度重视，精神科医生要推动多学科形成“大医学”思维。帕多瓦大学的Francesco Tona教授指出，心脏病学医生需明确心理与心血管疾病双向影响机制，与精神科医生合作实现“心身同治”。李氏大药厂创始人李小羿博士表示，心身医学作为现代医学的重要组成部分，强调精神心理因素与生理因素的相互作用，对疾病的预防、治疗及康复具有深远意义。2025-2027年是我国卫生健康领域重要的“儿科和精神卫生服务年”，这充分体现了国家对精神卫生领域的高度重视。李氏大药厂作为医药企业，始终致力于推动精神卫生领域的发展，积极响应政府“心身同治”的号召。结合自身产品管线和优势，李氏大药厂制定了明确的行动规划，今年的工作重点和主题确定为“抑郁是一种全身性疾病的影响因素”。在产品研发方面，李氏大药厂计划于今年推出芮畅舒®洛沙平吸入剂，该药物专门用于成人精神分裂症或双相Ⅰ型障碍相关激越的急性治疗，能够更好地满足精神疾病患者的急性治疗需求。在未来的关键时期，李氏大药厂将继续秉承创新和以患者为中心的理念，通过实际行动支持国家政策的落地，为推动我国精神卫生事业的发展贡献力量。最后，在共同主席的总结下，2025年第二届中欧脑心联盟俱乐部学术会议圆满落幕。此次会议的成功举办，为精神医学、心血管医学和皮肤医学之间，以及国内与国外医学专家、科学研究与临床实践之间搭建了一座宝贵的桥梁。正如会议所倡导的，科学无国界，期待中欧脑心联盟俱乐部未来能够搭建更多的前沿学术交流平台，持续推动跨国医学领域的创新发展，为全球医学事业的进步注入新的活力。往期回顾◆ 中外交流越山海，学术领航促未来——2025中欧脑心联盟俱乐部：心血管病与皮肤医学专场成功举办◆ 中欧脑心联盟俱乐部-肺动脉高压专场◆ 砺行致远，双证铸基——广州兆科联发有限公司成功通过ISO9001及ISO13485质量管理体系认证◆ 【2025 ASCO速递】原研“格拉司琼透皮贴片”善可舒®-口服抗肿瘤药物的“止吐新防线”！关于李氏大药厂- 亞太地區最具創新力的上市企業 -李氏大药厂控股有限公司（简称“李氏大药厂”或“集团”, 股份编号: 00950）成立于1994年，是一家结合了研发驱动和市场导向的生物医药集团公司，总部位于香港。李氏大药厂于2002年7月在香港联合交易所（简称：“港交所”）创业板上市，并于2010年5月成功转至港交所主板上市。李氏大药厂在中国大陆拥有两大综合性生产研发基地，即：兆科合肥基地（位于安徽省合肥市高新区）和兆科广州基地（位于广东省广州市南沙区），目前员工总数超过1100人。李氏大药厂立足中国大陆，放眼国际，通过坚实和稳固的投入建设，全面覆盖整合了药物研发、临床研究、药政注册、生产制造、销售与市场推广等各个环节。集团集中于心血管科、妇科与产科、儿科、罕见病、肿瘤科、皮肤科、精神科等多个重要疾病领域开发不同阶段的专利新药和高端仿制药。李氏大药厂与20余家国际公司建立了广泛的合作关系，目前在中国大陆、香港、澳门及台湾地区推广及销售25种包含专利新药、仿制药物及授权引进的国外已上市进口药品。李氏大药厂现有超过40个处于不同开发阶段的产品，既有自主研发产品，也有通过来自美国、欧洲及日本等公司通过授权引进进行研发、生产以及商业化的产品。李氏大药厂的使命是致力于研发新药，治疗顽疾，改善生活质量，为患者带来福音，目标成为亚洲成功的生物制药集团，提供治疗疾病和改善生活质量的创新产品。下滑查看更多免责声明：本公众号发布或转载企业宣传资讯，仅供读者参考之用，并不构成收购、购买或认购依据，并不构成任何个人化的投资劝诱或建议。因本公布全部或任何部分内容而产生或因倚赖该等内容而引致的任何损失不承担任何责任。企业文化公共关系部出品图片来源：会议现场撰稿：张慧玲摄像：廖宁排版：樊扬波校对：丁雪森审核：方玲与“李”同行招商热线020-39062882