Elamipretide Hydrochloride

Program underscores Stealth's commitment to patient access and partnership with the Barth syndrome community NEEDHAM, Mass., Oct. 9, 2025 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a commercial-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced the launch of its Mito Assist™ Patient Support Program for access to FORZINITY™ (elamipretide) injection. FORZINITY was granted accelerated approval by the U.S. Food and Drug Administration (FDA) on September 19, 2025 as the first treatment for Barth syndrome, to improve muscle strength in adult and pediatric patients weighing at least 30 kilograms (kg). As part of this initiative, Stealth has selected AnovoRx Specialty Pharmacy ("AnovoRx") as its exclusive commercial distribution partner for FORZINITY in the United States. Through this partnership, AnovoRx will play a key role in helping patients, caregivers, and healthcare providers navigate the insurance coverage and access process. "We have been deeply committed to the Barth syndrome community for over a decade, and access to FORZINITY for eligible patients remains our highest priority," said Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics. "We are investing significantly in our access infrastructure to make sure every eligible patient who may benefit from FORZINITY can receive it. Partnering with AnovoRx allows us to deliver a high-touch experience for families as they navigate access and coverage." Mito Assist™ will offer personalized patient support services to help affected individuals and caregivers understand coverage options, manage insurance submissions, and access financial assistance resources. This includes: Co-pay assistance for eligible commercially insured patients. Patient assistance programs for uninsured or underinsured individuals. Support for at-home subcutaneous injection training. Dedicated case management through AnovoRx to provide ongoing communication with patients, caregivers, and healthcare providers. Patients currently receiving elamipretide through Stealth's Expanded Access Program who meet the weight requirement of 30kg will be contacted directly by their healthcare providers and AnovoRx case managers to facilitate a seamless transition to commercial therapy once FORZINITY™ becomes available. Stealth anticipates that commercial product will be available through AnovoRx by December 2025, pending final distribution readiness and payer coverage activation. Patients and healthcare providers can learn more by calling 1-833-458-9099. About FORZINITY™ (elamipretide) injection INDICATION FORZINITY™ is a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). CONTRAINDICATIONS Serious hypersensitivity to elamipretide or any of the ingredients. WARNINGS AND PRECAUTIONS FORZINITY is not approved in neonates. FORZINITY contains Benzyl Alcohol and serious adverse reactions including fatal reactions have been reported in low birth weight neonates and preterm neonates who received benzyl alcohol containing drugs intravenously. FORZINITY is not approved for intravenous use. Hypersensitivity reactions, including serious allergic reactions requiring emergency medical intervention, have been reported in patients receiving FORZINITY. Monitor patients for signs and symptoms of hypersensitivity reactions during treatment. ADVERSE REACTIONS Most common adverse reactions are injection site reactions. To report SUSPECTED ADVERSE REACTIONS, contact Stealth BioTherapeutics Inc. at 1-844-444-6486 or FDA at 1-800-FDA-1088 or . For more information about FORZINITY, please see the full US Prescribing Information. About Barth Syndrome Barth syndrome is an ultra-rare genetic condition characterized by mitochondrial abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Barth syndrome occurs primarily in males and is estimated to affect one in 1,000,000 males births or around 150 individuals in the United States. There are no EMA-approved therapies for patients with Barth syndrome. About Stealth BioTherapeutics Stealth BioTherapeutics' mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Stealth's commercial product, FORZINITY, was granted accelerated approval by the U.S. Food & Drug Administration (FDA) in September 2025 as the first FDA-approved treatment for Barth syndrome, as well as the first FDA-approved mitochondria-targeted therapeutic. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). Stealth is studying elamipretide in additional indications, including dry age-related macular degeneration and primary mitochondrial myopathy, and is developing its second-generation clinical-stage candidate, bevemipretide (SBT-272), for ophthalmic and neurological disease indications. Media Contact Ascent Strategic Communications Anna Stallmann [email protected] Investor Contact Precision AQ Austin Murtagh [email protected] Patient Advocacy [email protected] SOURCE Stealth BioTherapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

编者按：多肽药物已成为治疗多种疾病的重要手段。目前，全球已有约100款多肽药物获批上市，覆盖糖尿病、肥胖症、癌症及罕见疾病等多个领域，为患者带来了全新的治疗策略。然而与传统的小分子药物相比，多肽药物更为复杂，涉及非天然氨基酸（UAA）合成、肽库合成、偶联化合物合成、放大生产工艺开发以及制剂开发等多个环节，对专业技术能力提出了更高要求。为更好地满足全球合作伙伴在多肽药物研发方面的需求，药明康德旗下WuXi TIDES围绕多肽药物建立了一体化CRDMO平台，提供包括线性、环状和高度修饰的多肽，以及非天然氨基酸、连接子、毒素和多肽偶联物的合成服务，支持从药物发现、CMC开发到商业化生产的各个阶段。本文将回顾2025年第三季度多肽领域的最新进展，并介绍药明康德的一体化CRDMO平台如何高效解决该领域药物开发过程中的诸多挑战。 GLP-1类多肽疗法持续进展 在2025年第三季度，GLP-1类多肽疗法在代谢与心血管领域持续进展。今年8月，美国FDA加速批准Wegovy（2.4 mg司美格鲁肽）用于联合低热量饮食及增加体能活动，治疗伴有中度至重度肝纤维化（2期或3期）的非肝硬化型代谢功能障碍相关脂肪性肝炎（MASH）成人患者。根据新闻稿，Wegovy为首个获批用于治疗MASH的GLP-1疗法。今年9月，欧洲药品管理局（EMA）人用药品委员会（CHMP）批准更新诺和诺德旗下Rybelsus（口服司美格鲁肽）的标签，使其成为欧盟范围内在2型糖尿病治疗中证实具有心血管获益的首个口服GLP-1受体激动剂。该批准主要根据3b期临床试验SOUL的结果。分析显示，在标准治疗基础上加用口服司美格鲁肽可使主要不良心血管事件（MACE）风险较安慰剂显著降低14%。诺和诺德在7月也向EMA提交了Wegovy 7.2 mg新剂量的监管申请，旨在为肥胖患者进一步提升体重管理效果。  与此同时，礼来（Eli Lilly and Company）也公布了其GIP/GLP-1双重受体激动剂Mounjaro（tirzepatide）与GLP-1受体激动剂Trulicity（dulaglutide）相较，在2型糖尿病合并动脉粥样硬化性心血管疾病成人患者中的疗效。临床3期研究结果显示，Mounjaro在降低主要不良心血管事件风险方面不劣于Trulicity，后者曾在REWIND研究中证实具有明确的心血管获益。Mounjaro也在这个季度获加拿大卫生部批准上市。根据新闻稿，该疗法是首个获得加拿大卫生部批准用于慢性体重管理的双重受体激动剂。 多肽疫苗在癌症领域取得成果 部分多肽疫苗也在癌症领域取得进展。例如，PDS Biotechnology旗下多肽癌症疫苗PDS0101（Versamune HPV）联合PD-1抑制剂Keytruda（pembrolizumab），在2期试验当中，作为一线疗法时，患者的中位总生存期达39.3个月（95% CI：23.9-尚未可估）。根据新闻稿，该类患者在接受标准治疗联合化疗时已公布的最佳中位总生存期为17.9个月。而Elicio Therapeutics旗下靶向突变KRAS的多肽疫苗ELI-002在1期AMPLIFY-201研究中，在中位随访19.7个月时，将胰腺癌和结直肠癌患者的死亡风险降低77%，复发风险降低88%。 多肽疗法在罕见病领域获得突破 此外，多肽疗法在本季度也于罕见病领域有许多突破。今年7月，美国FDA批准Apellis Pharmaceuticals旗下双环肽疗法Empaveli（pegcetacoplan）扩展适应症，用于治疗C3肾小球病（C3G）和原发性免疫复合物膜增生性肾小球肾炎（IC-MPGN），以减少患者的蛋白尿。根据新闻稿，该疗法是获FDA批准用以治疗这两类罕见肾病的首款疗法。此外，Stealth BioTherapeutics所开发的Forzinity（elamipretide）也在今年9月迎来FDA批准，成为巴思综合征全球首款获批疗法。 其他领域的进展 其他领域方面，强生（Johnson & Johnson）与Protagonist Therapeutics也已向美国FDA递交新药申请（NDA），寻求批准其联合开发的口服多肽疗法icotrokinra，用于治疗12岁及以上中度至重度斑块状银屑病（PsO）成人与儿科患者。默沙东（MSD）在今年9月宣布在研口服大环肽enlicitide decanoate在3期试验中，能够显著降低高胆固醇血症患者的低密度脂蛋白胆固醇（LDL-C）水平。根据新闻稿，该疗法是在3期试验中显示能显著降低LDL-C水平的首个口服PCSK9抑制剂。 商业研发合作进展 今年9月，辉瑞（Pfizer）与Metsera达成总额达数十亿美元的收购协议。此次收购将使辉瑞获得后者旗下具差异化的口服与注射型肠促胰岛素和胰淀素类候选药物以及联合疗法，这些项目在有效性与安全性方面具备“best-in-class”潜力。Unnatural Products（UNP）与argenx则在7月达成一项多靶点战略合作研究项目，旨在发现和开发针对“不可成药”疾病靶点的口服大环肽药物。该合作将利用UNP公司的专有药物发现平台，生成针对argenx公司所选定的多个靶点的高效、高选择性且可口服的大环肽分子。 综上，2025年三季度多肽疗法多线跃进：GLP-1版图延伸至MASH，肿瘤多肽疫苗在延长生存与降低复发方面表现亮眼，多项罕见病亦迎来首款获批疗法，口服多肽赛道持续升温。随着临床证据不断积累与产业协同加速推进，多肽疗法有望在代谢、肿瘤及罕见病等领域实现更多里程碑。 WuXi TIDES高效赋能合作伙伴开发复杂多肽偶联药物 药明康德旗下WuXi TIDES平台支持各类多肽药物从发现、CMC开发到商业化生产的各个阶段，致力赋能全球客户推动创新疗法问世。以下案例将展示WuXi TIDES的一体化平台如何高效推动合作伙伴复杂多肽偶联药物的开发进程。 在该案例中，客户所开发的一款多肽偶联药物结构复杂，由两个短环肽片段和一个长线性多肽组成，合成步骤多达87步。初期合成方案的总体产率不足0.1%，难以满足临床试验的需求。同时，该药物所需的3种UAA原料供应紧张，若从外部采购可能严重延误研发进度。 面对上述挑战，WuXi TIDES团队提出了多项针对性解决方案。针对UAA原料短缺问题，团队充分发挥自身生产UAA的能力，迅速开发出可行的合成工艺，并成功制备了超过3公斤的UAA原料，从源头保障了项目的顺利推进。这一内部合成策略不仅降低了对外部供应商的依赖，还显著优化了进度管理，大幅缩短整体项目周期。 与此同时，WuXi TIDES团队致力于提升多肽合成效率。考虑到传统固相合成方法合成超过50个氨基酸的多肽时，往往面临效率与产率下降的挑战，WuXi TIDES团队采用了“片段合成+化学连接”策略，将目标多肽拆分成多个适合固相合成的小片段，分别合成后再通过化学连接形成完整多肽分子。这一策略将总体产率从不足0.1%提升至3%，并使多个团队能够同时进行片段合成，显著加快了项目推进速度。 在复杂冗长的合成路线中，每一个细节都可能影响最终质量与产率。为进一步优化工艺，WuXi TIDES团队对多个关键步骤的合成和纯化条件进行了反复筛选与优化。在放大生产阶段，团队专门设计了定制的纯化材料，使杂质水平下降80%，最终产率提升20%。得益于UAA合成团队、多肽合成团队和分析团队的高效执行及并行作业，WuXi TIDES团队仅用4个月即完成了工艺开发与优化，7个月内交付了两批各100克的GLP样品，随后又在3个月内顺利生产出1千克GMP级原料药，产率达3%，纯度达97.4%。项目整体进度比合作伙伴预期提前4个月，为临床试验的及时启动提供了强有力保障。 化学合成能力之外，WuXi TIDES还可赋能多肽药物从临床前到商业化阶段的口服和注射制剂的开发和生产。口服制剂平台支持包括片剂、胶囊、脂质体、粉末、颗粒和口服液等各类剂型的开发和生产；注射剂平台则支持多种剂型和灌装形式的无菌制剂生产。展望未来，WuXi TIDES团队将持续依托其一体化CRDMO平台，助力全球合作伙伴充分发挥多肽疗法的巨大潜力，为患者带来更多创新药物。 CRDMO: Q3 2025 Review of Peptide Therapeutics Peptide drugs have emerged as an important therapeutic modality for a broad spectrum of diseases. To date, approximately 100 peptide-based medicines have received global regulatory approval, providing new treatment options for conditions such as diabetes, obesity, cancer, and rare diseases. However, compared to traditional small-molecule drugs, peptides present greater structural complexity. Their development requires advanced capabilities in areas such as unnatural amino acid (UAA) synthesis, peptide library design, conjugation chemistry, scale-up process development, and formulation support. To address these challenges and better support global partners, WuXi TIDES, an integral part of WuXi AppTec, has established an integrated CRDMO platform. The platform offers comprehensive synthesis services for linear, cyclic, and highly modified peptides, as well as UAAs, linkers, payloads, and peptide conjugates. Covering the full development spectrum from drug discovery and CMC development to commercial-scale manufacturing, the platform enables efficient and flexible solutions tailored to each stage of the pipeline. Here we summarize key developments in the peptide space during Q3 2025 and share a case study that illustrates how WuXi TIDES helps accelerate progress in this dynamic area. Sustained Momentum for GLP-1–Based Therapeutics In the third quarter of 2025, GLP-1 peptide therapeutics continued to advance in the fields of metabolic and cardiovascular diseases. In August, the U.S. FDA granted accelerated approval to Wegovy (semaglutide 2.4 mg) for use in combination with a reduced-calorie diet and increased physical activity to treat adult patients with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis (stage 2 or 3). According to the company, Wegovy is the first GLP-1 therapy approved for the treatment of MASH. In September, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) approved an update to the label of Novo Nordisk’s Rybelsus (oral semaglutide), making it the first oral GLP-1 receptor agonist approved for type 2 diabetes in the European Union, with proven cardiovascular benefit. The decision was based on results from the Phase 3b SOUL trial, which demonstrated that adding oral semaglutide to standard therapy reduced the risk of major adverse cardiovascular events (MACE) by 14% compared with placebo. In July, Novo Nordisk also submitted a regulatory application to the EMA for a new 7.2 mg dose of Wegovy, aiming to further enhance weight management outcomes for patients with obesity. Meanwhile, Eli Lilly and Company reported data comparing its dual GIP/GLP-1 receptor agonist Mounjaro (tirzepatide) with the GLP-1 receptor agonist Trulicity (dulaglutide) in adults with type 2 diabetes and atherosclerotic cardiovascular disease. Results from a Phase 3 trial showed that Mounjaro was non-inferior to Trulicity in reducing the risk of major adverse cardiovascular events. Trulicity had previously demonstrated clear cardiovascular benefit in the REWIND trial. Mounjaro also gained approval from Health Canada this quarter, becoming the first dual receptor agonist authorized in Canada for chronic weight management. Breakthroughs for Peptide Vaccines in Oncology Peptide vaccines also achieved important milestones in oncology. PDS Biotechnology’s peptide-based cancer vaccine PDS0101 (Versamune HPV), in combination with the PD-1 inhibitor Keytruda (pembrolizumab), delivered a median overall survival (mOS) of 39.3 months (95% CI: 23.9–not estimable) as a first-line therapy in a Phase 2 trial. By comparison, previously reported standard therapy plus chemotherapy in this patient group achieved a best mOS of 17.9 months. Elicio Therapeutics also reported promising results for its mutant KRAS-targeted peptide vaccine ELI-002. In the Phase 1 AMPLIFY-201 study, at a median follow-up of 19.7 months, ELI-002 reduced the risk of death by 77% and recurrence by 88% in patients with pancreatic and colorectal cancers. Advances in Peptide Therapeutics for Rare Diseases Significant progress was also made in rare diseases this quarter. In July, the U.S. FDA approved Apellis Pharmaceuticals’ cyclic peptide therapy Empaveli (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria. According to the company, this is the first FDA-approved therapy for these two rare kidney disorders. In September, Stealth BioTherapeutics secured FDA approval for Forzinity (elamipretide), marking the world’s first approved treatment for Barth syndrome. Expanding Applications in Other Fields In addition, Johnson & Johnson and Protagonist Therapeutics have submitted a New Drug Application (NDA) to the U.S. FDA for their jointly developed oral peptide therapy icotrokinra, seeking approval for the treatment of adults and adolescents aged 12 years and older with moderate-to-severe plaque psoriasis (PsO). Merck (known as MSD outside of the U.S. and Canada) announced in September that its investigational oral macrocyclic peptide enlicitide decanoate achieved a significant reduction in low-density lipoprotein cholesterol (LDL-C) in a Phase 3 trial in patients with hypercholesterolemia. According to the company’s release, this marks the first oral PCSK9 inhibitor to demonstrate a significant LDL-C reduction in a Phase 3 study. Progress in Partnerships and Collaborations September also saw major business developments, including Pfizer’s acquisition agreement with Metsera. The deal grants Pfizer access to Metsera’s differentiated oral and injectable incretin and amylin candidates, as well as combination therapies, which hold best-in-class potential in both efficacy and safety. In July, Unnatural Products (UNP) entered into a multi-target strategic research collaboration with argenx to discover and develop orally available macrocyclic peptide drugs for previously “undruggable” disease targets. The collaboration will leverage UNP’s proprietary drug discovery platform to generate potent, selective, and orally bioavailable macrocyclic peptides against multiple targets selected by argenx. In summary, Q3 2025 saw peptide therapeutics advance on multiple fronts: the GLP-1 landscape expanded into MASH; peptide cancer vaccines showed compelling benefits in extending survival and reducing recurrence; several rare diseases welcomed their first approved therapies; and the oral-peptide continued to gain momentum. As clinical evidence builds and industry collaboration accelerates, peptide therapeutics are poised to deliver further milestones across metabolic, oncology, and rare disease fields. WuXi TIDES Empowering Global Innovation WuXi AppTec’s WuXi TIDES platform provides end-to-end support for peptide therapeutics—from discovery and CMC development through commercial manufacturing—helping global partners speed innovative therapies to patients. To demonstrate how this integrated model translates into execution, one representative case is outlined below. In this program, the candidate molecule comprised two short cyclic peptide fragments and one long linear peptide, requiring an intricate synthesis involving up to 87 steps. The initial synthetic route yielded less than 0.1% overall, insufficient to meet clinical trial demands. Additionally, the project was hampered by the limited availability of three critical UAAs, with external sourcing posing a risk of significant delays. To overcome these obstacles, the WuXi TIDES team implemented several targeted solutions. First, the team addressed the UAA supply bottleneck by leveraging its internal UAA synthesis capabilities. They quickly developed viable synthetic pathways and produced over 3 kilograms of UAAs, securing raw material availability and safeguarding project timelines. This internal manufacturing strategy not only reduced dependency on external suppliers but also significantly improved timeline control and shortened the overall project duration. Concurrently, efforts were directed at improving the efficiency of peptide synthesis. Given the challenges of efficiency and yield declines when traditional solid-phase peptide synthesis (SPPS) techniques are used for sequencing longer than 50 amino acids, the WuXi TIDES team adopted a "fragment synthesis + chemical ligation" strategy—breaking down the target peptide into shorter fragments suitable for SPPS, synthesizing them individually, and then linking them chemically into the full-length peptide. This approach improved the overall yield from under 0.1% to 3%, while enabling multiple teams to work in parallel, significantly accelerating development timelines. Given the complexity and length of the synthesis route, process optimization was critical. The WuXi TIDES team conducted extensive rounds of screening and refinement for key synthesis and purification steps. During scale-up, the team introduced customized purification materials that reduced impurity levels by 80% and improved yield by 20%. Through efficient execution and parallel activities between the UAA synthesis, peptide synthesis, and analytical teams, process development and optimization were completed in just four months. The WuXi TIDES team delivered two batches of 100 grams of GLP-grade material within seven months, followed by the production of 1 kilogram of GMP-grade API in an additional three months. The final process achieved a 3% yield with 97.4% purity. Overall, the project was completed four months ahead of schedule, ensuring timely clinical trial initiation with a secure and robust supply. Beyond peptide API synthesis, WuXi TIDES also supports both oral and parenteral drug product development and manufacturing for peptide drugs from preclinical to commercial stages. The oral solid platform supports a diverse array of dosage forms such as tablets, capsules, lipid formulations, powders, granules, and liquid-in-bottle, while the parenteral dosage platform accommodates various injectable drug forms and filling formats. Looking forward, the WuXi TIDES team will continue to leverage its integrated CRDMO platform to accelerate the development of peptide therapeutics. By enabling global partners to unlock the full potential of peptide drugs, WuXi TIDES is helping bring more innovative medicines to patients around the world. 参考资料： [1] FDA Approves Apellis’ EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older. Retrieved September 25, 2025 from https://investors.apellis.com/news-releases/news-release-details/fda-approves-apellis-empavelir-pegcetacoplan-first-c3g-and [2] Merck’s Investigational Oral PCSK9 Inhibitor Enlicitide Decanoate Met All Primary and Key Secondary Endpoints in Adults with Hypercholesterolemia in Pivotal CORALreef Lipids Study. Retrieved September 25, 2025 from https://www.merck.com/news/mercks-investigational-oral-pcsk9-inhibitor-enlicitide-decanoate-met-all-primary-and-key-secondary-endpoints-in-adults-with-hypercholesterolemia-in-pivotal-coralreef-lipids-study/ 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新