Certepetide

▎药明康德内容团队报道 刚刚过去的国际罕见病日，再次提醒我们全球数亿罕见病患者的困境。然而，2024年对于罕见病领域来说，却是一个充满希望的年份。在全球范围内，罕见病新药的研发和上市再次迎来突破。 据公开数据统计，2024年美国FDA批准了至少33款罕见病新药。在中国，2024年至少有17款罕见病新药获批上市，覆盖了Wilson病、阵发性睡眠性血红蛋白尿症、血友病、“渐冻症”、杜氏肌营养不良等十多种罕见病类型。这些新药为无数患者和家庭带来了新的希望。 罕见病药物的研发正在全球范围内加速推进。2024年，美国FDA授予了超过88个新药项目孤儿药资格。这些药物涵盖了杜氏肌营养不良、原发性胆汁性胆管炎、家族性地中海热、肝豆状核变性等多种罕见病类型。其中，来自中国公司的至少35个新药项目获得了这一资格，创下了近年来新高。 根据世界卫生组织的统计，全球约有6000多种罕见病，患者总数超过3亿人。然而，目前只有不到5%的罕见病有治疗方法。随着科学技术的进步和产业投入的加大，罕见病新药研发迎来前所未有的活跃期。数据显示，近5年来FDA批准的新药中，超过50%用于治疗罕见疾病和罕见癌症。 图片来源：123RF 罕见病药物的研发离不开创新科技的推动。从药物类型来看，2024年获得FDA孤儿药资格的新药涵盖了小分子、抗体、多肽、AAV基因疗法、CAR-T细胞疗法、反义寡核苷酸疗法、抗体偶联药物（ADC）以及干细胞疗法、溶瘤病毒等多种类型。 其中，小分子药物39项，占比超过40%。这其中，不乏一些中国产业人士熟知的在研产品。例如，百济神州的BTK抑制剂泽布替尼拟治疗膜性肾病；和誉医药的小分子FGFR4抑制剂依帕戈替尼拟治疗肝细胞癌；加科思的KRAS G12C抑制剂戈来雷塞拟治疗胰腺癌；华昊中天的优替德隆注射液拟治疗乳腺癌脑转移等。这些药物的研发不仅展示了科学技术的进步，也为全球罕见病患者提供了更多潜在治疗选择。 以多肽、寡核苷酸、细胞和基因疗法为代表的新分子药物，正在成为罕见病治疗的重要方向。例如，Lisata Therapeutics公司的环肽疗法certepetide拟用于治疗胆管癌；中美瑞康的小激活RNA药物RAG-18用于拟治疗杜氏肌营养不良；Ionis Pharmaceuticals的反义寡核苷酸疗法Tryngolza拟用于降低家族性乳糜微粒血症综合征患者的甘油三酯。这些新药的研发，标志着罕见病新药研发已进入了全新的新分子时代。 然而，罕见病药物的研发并非易事。患者群体小、研发成本高、技术难度大，一直是行业面临的重大挑战，需要创新生物医药生态圈的共同努力，来应对挑战。例如，合同研发和生产机构（CXO）也是推动罕见病药物创新的重要力量。 作为创新的推动者、客户信赖的合作伙伴以及全球健康产业的重要贡献者，药明康德多年来通过其一体化、端到端的CRDMO新药研发平台，通过技术创新、高效协同和全链条服务，为罕见病新药研发提供了强有力的支持，助力合作伙伴为全球罕见病患者带来更多的治疗选择。根据公开数据统计，在2024年FDA批准的33款罕见病新药中，至少有5款获得了药明康德的研发助力。而在获得FDA孤儿药资格的88款新药项目中，药明康德支持了其中的19款，占比达到21.6%。这相当于，每5款孤儿药新药项目中，就有1款获得了药明康德的支持。 罕见病药物的研发是一条漫长而复杂的道路，从实验室的早期发现到最终惠及患者，需要跨越无数技术难关和挑战。在这一过程中，药明康德凭借其一体化的技术平台和全链条服务，从药物发现、临床前研究到工艺开发、生产制造，为合作伙伴提供了全方位的支持。 罕见病药物的研发往往始于对疾病机制的深入理解。公开信息显示，药明康德生物学业务早在2016年就上线了罕见病平台，专注于罕见病药物研发的早期阶段。该平台覆盖了罕见病药物研发的全路线，提供包括罕见病动物模型构建、动物繁育，以及体内、外的药理药效研究一站式服务。该平台快速构建了A型血友病、镰状细胞性贫血、脊髓性肌萎缩症、杜氏肌营养不良症等几十种罕见病的动物模型，涵盖了血液疾病、代谢类疾病、免疫类疾病和精神类疾病等研发热点领域，助力罕见病药物的药理评价。 罕见病药物的研发不仅需要科学突破，还需要高效的工艺开发能力，以应对临床阶段的复杂挑战。药明康德子公司合全药业在这一领域展现了强大的技术实力。基于质量源于设计（QbD）的理念，合全药业的研发团队能够快速推进药物工艺的开发和安全放大，在保障产品质量的同时，显著降低了成本。 另外，当以寡核苷酸、多肽为代表的新分子药物成为罕见病治疗的重要方向时，药明康德也早有布局，其旗下专注于寡核苷酸和多肽及相关化学偶联药物的新分子业务平台——WuXi TIDES，展现出强大的赋能能力，为罕见病药物开发提供了重要支持。 以寡核苷酸药物为例，siRNA药物因其靶向性差、稳定性低等问题，研发难度较大。WuXi TIDES平台通过N-乙酰化半乳糖胺（GalNAc）共价连接技术，有效解决了siRNA的递送难题，成为行业主流解决方案。该平台还具备全面的寡核苷酸偶联能力、定制化GalNAc分子合成能力以及一体化CMC服务，能够在短时间内将候选化合物推进至IND阶段。 例如，WuXi TIDES平台曾仅用10个月就将一款用于治疗罕见病的siRNA-GalNAc候选药物推进至IND阶段，展现了其高效的技术执行力和协同能力。这种快速推进的能力，为罕见病药物的研发赢得了宝贵的时间。目前，WuXi TIDES 正在赋能20多种用于罕见病治疗的新药开发，包括复杂偶联药物，如寡核苷酸-多肽偶联、PDC及RDC等，更多患者有望从新分子疗法中获益。 此外，药明康德测试事业部也持续赋能客户开展罕见病新药研发IND工作和非临床研究工作。 这些平台共同构成了药明康德在罕见病药物研发领域的强大引擎，助力合作伙伴为全球罕见病患者带来更多的治疗选择。目前，药明康德正在积极支持全球更多合作伙伴罕见病新药在研管线的推进。 药明康德的赋能不仅限于技术支持，还体现在其全球化的合作网络中。通过与全球药企、生物技术公司和研究机构的紧密合作，药明康德持续助力罕见病新药走向世界。例如，在2024年获得FDA孤儿药资格的来自中国公司的35个新药项目中，药明康德就支持了其中5款。 罕见病药物的研发，是一场与时间的赛跑。每一个新药的诞生，都意味着无数患者和家庭的命运可能被改写。2024年，科学创新和全球合作在罕见病药物研发中的突出表现，为全球罕见病患者带来了新的希望。未来，随着科技的进步和产业的持续投入，罕见病治疗的曙光将越来越明亮。我们期待，更多的“孤儿药”能够走出实验室，走进患者的生活，为他们点亮希望之光。 本文来自药明康德内容团队，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。转载授权及其他合作需求，请联系wuxi_media@wuxiapptec.com。 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。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。

Strategic collaboration harnesses GATC’s proprietary Artificial Intelligence-powered, validated Multiomics Advanced Technology™ platform to rapidly optimize and derisk drug discovery and development, thereby accelerating and economizing the traditional drug development process. Lisata’s investigational product, certepetide, to be the subject of a comprehensive AI-analysis to determine optimum disease targets and development strategies. GATC to leverage Lisata’s experience and operational excellence in translational medicine and drug development to advance their drug candidate pipeline products into clinical trials. BASKING RIDGE, N.J. and IRVINE, Calif., March 05, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, and GATC Health Corp (“GATC”), a leading tech-bio company leveraging artificial intelligence (“AI”) to transform drug discovery, today announced the consummation of the first step of an intended multi-part strategic agreement. Under terms of the agreement, the two companies will leverage Lisata’s experience and expertise in drug development together with GATC’s proprietary AI-powered and validated Mutiomics Advanced Technology™ (“MAT”) platform to derisk, optimize, and accelerate drug development, creating opportunities with a higher probability of success. As the first step in the collaboration, GATC’s MAT AI platform will analyze Lisata’s investigational drug, certepetide, to identify optimized and derisked development opportunities across various indications. GATC’s MAT AI simulates billions of complex systems biological interactions, predicting safety, efficacy, off-target effects, and clinical trial outcomes. Based on the outcome of GATC’s analysis, Lisata plans to optimize its Phase 3 development of certepetide in metastatic pancreatic ductal adenocarcinoma (“mPDAC”) while also identifying new indications with a high probability of development success. In a subsequent step in the collaboration, GATC’s MAT AI will identify drugs that can be used in combination with certepetide across a wide variety of indications, including those outside of oncology. Finally, the strategic collaboration contemplates the engagement of Lisata as an operational partner of GATC for the development of GATC’s own drug development candidates. To date, GATC’s MAT AI has contributed to the discovery of novel drug candidates targeting addiction, PTSD, diabetes, obesity, glioblastoma, and cognitive decline. In addition to the practical implications of enhanced and accelerated development, the collaboration is expected to reduce the risks traditionally associated with biopharmaceutical ventures and related investments. By leveraging GATC’s MAT AI platform, drug candidates with a higher probability of success are expected to be quickly identified, thus enabling more focused studies and truncated development timelines along with optimized capital management. “Lisata is delighted by the prospects of this agreement and is honored to be chosen as a strategic partner by GATC. It is noteworthy that GATC has been selected as the exclusive AI partner of Lloyd’s of London’s syndicate, Medical & Commercial International, and their marketing partner Acrisure, to support the underwriting of the first insurance-backed financial program to fund clinical trials. Additionally, we are excited by the plans for GATC to engage Lisata as a collaborator in the advancement of their own drug development candidates,” stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. “By combining our expertise with GATC and their cutting-edge AI technology, we are ensuring that Lisata’s and GATC’s development programs have the highest probability of success, allowing us the ability to conduct the most time- and capital-efficient studies to accelerate products to market for the benefit of patients, the medical community, and our shareholders.” Traditional drug discovery and development is a notoriously risky, expensive, and time-consuming process, often continually projected to take over a decade at a cost of billions of dollars to bring a single therapy to market. The high failure rate, with an estimated 1 in 10,000 identified potential drugs that make it to market, is a direct result of inaccurate target identification, unforeseen toxicity, poor efficacy, and a lengthy, complicated preclinical and clinical trial process. By applying GATC’s MAT AI, drug discovery and development is anticipated to be much faster and more efficient with a greater probability of success. “GATC’s validated technology inverts predicted clinical trial failures and provides an unbiased analysis of investigational new drugs, such as Lisata’s certepetide,” stated Jayson Uffens, Chief Technology Officer at GATC. “This enables a derisked and optimized development roadmap that can include additional indications and new intellectual property related to the drug candidate. We are also pleased to collaborate with Lisata to leverage their expertise for GATC’s pipeline of assets.” About Lisata Therapeutics Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s cyclic peptide product candidate, certepetide, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to selectively target and penetrate solid tumors more effectively. Lisata has already established noteworthy commercial and R&D partnerships based on its CendR Platform® technology. The Company expects to announce numerous milestones over the next 1.5 years and believes that its projected capital will fund operations into the second quarter of 2026, encompassing anticipated data milestones from its ongoing and planned clinical trials. Learn more about certepetide’s mechanism of action in our short film. For more information on the Company, please visit www.lisata.com. About Certepetide Certepetide (formerly LSTA1), an internalizing RGD (arginylglycylaspartic acid or iRGD), cyclic peptide product candidate, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to target and penetrate solid tumors more effectively. Certepetide actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. Certepetide also has been shown to modify the tumor microenvironment resulting in tumors which are more susceptible to immunotherapies. We and our collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of emerging anti-cancer therapies, including immunotherapies and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing clinical trials designed to test its ability to enhance the effectiveness of standard-of-care chemotherapy for pancreatic cancer. Lisata is exploring the potential of certepetide to enable a variety of treatment modalities to treat a range of solid tumors more effectively. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.) as well as Orphan Drug Designation for glioma (U.S.) and osteosarcoma (U.S.). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.). About GATC Health Corp GATC Health Corp is a technology company revolutionizing drug discovery and development through its transformative AI platform and approach. The company’s validated and proprietary Multiomics Advanced Technology™ (MAT) simulates human biochemistry’s billions of interactions to rapidly create novel therapeutics, identify and confirm targets, accelerate development, and de-risk drug pipelines by predicting efficacy, safety, and off-target effects. For more information, visit www.gatchealth.com. Forward-Looking Statements This communication contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding the Company’s clinical development programs are forward-looking statements. In addition, when or if used in this communication, the words “may,” “could,” “should,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “predict” and similar expressions and their variants, as they relate to Lisata or its management, may identify forward-looking statements. Examples of forward-looking statements include, but are not limited to, the potential efficacy of certepetide as a treatment for patients with metastatic pancreatic ductal adenocarcinoma and other solid tumors; our beliefs about the potential uses and benefits of certepetide; statements relating to Lisata’s continued listing on the Nasdaq Capital Market; expectations regarding the capitalization, resources and ownership structure of Lisata; the approach Lisata is taking to discover and develop novel therapeutics; the adequacy of Lisata’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; and the difficulty in predicting the time and cost of development of Lisata’s product candidates. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: results observed from a single patient case study are not necessarily indicative of final results and one or more of the clinical outcomes may materially change following more comprehensive reviews of the data and as more patient data becomes available, including the risk that unconfirmed responses may not ultimately result in confirmed responses to treatment after follow-up evaluations; the risk that product candidates that appeared promising in early research and clinical trials do not demonstrate safety and/or efficacy in larger-scale or later clinical trials; the safety and efficacy of Lisata’s product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in Lisata’s clinical programs, Lisata’s ability to finance its operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of Lisata’s scientific studies, Lisata’s ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in Lisata’s markets, the ability of Lisata to protect its intellectual property rights; and legislative, regulatory, political and economic developments. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in Lisata’s Annual Report on Form 10-K filed with the SEC on February 27, 2025, and in other documents filed by Lisata with the Securities and Exchange Commission. Except as required by applicable law, Lisata undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events, or otherwise. Lisata Therapeutics Contact: Investors: Lisata Therapeutics John Menditto Vice President, Investor Relations and Corporate Communications Phone: 908-842-0084 Email: jmenditto@lisata.com Media: ICR Healthcare Elizabeth Coleman Account Supervisor Phone: 203-682-4783 Email: elizabeth.coleman@icrhealthcare.com This press release was published by a CLEAR® Verified individual.