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为罹患这一罕见病的患者群体实现新药可及 上海 2024年8月21日 /美通社/ -- 阿斯利康中国宣布，依托 "港澳药械通"政策，其罕见病领域创新药Strensiq（通用名：阿司福酶α注射液 / asfotase alfa）已获广东省药品监督管理局批准，正式引进大湾区药械通指定医疗机构，用于作为婴儿期发病的低磷酸酯酶症(hypophosphatasia, HPP)患者的长期酶替代疗法，以治疗该疾病的骨骼表现。此次大湾区先行获批认可了该药的临床应用先进性，内地患儿将无需出境便可在大湾区药械通指定医疗机构使用该药，缓解了中国患者亟需用药的难题。 低磷酸酯酶症是一种极其罕见的遗传性渐进式代谢紊乱疾病，患者因缺少碱性磷酸酶(alkaline phosphatase, ALP)，骨矿化和骨质素生成受到影响，致使骨骼生长和其强度出现问题，并引发其他并发症，可能面临骨折、骨痛、行走和呼吸困难、癫痫等问题，甚至造终身负担，严重影响生存质量 [1] 。 作为全球首个且唯一*获批用于治疗低磷酸酯酶症的酶替代疗法 [2] ，阿司福酶α注射液中的活性物质可以替代患者体内缺少的碱性磷酸酶，并作为长期酶替代治疗控制患者症状 [1] 。针对围产期、婴儿期或幼儿期发病低磷酸酯酶症的患者的四项前瞻性的开放标签临床试验，评估并证实了阿司福酶α注射液的临床疗效和安全性。经该药治疗的围产期/婴儿期发病的低磷酸酯酶症患者（37名）与历史对照的低磷酸酯酶症患者（48名）的总生存率对比的Kaplan-Meier分析发现，阿司福酶α注射液可明显改善患者生存率。研究显示，经阿司福酶α注射液治疗的患者1岁时生存率为95%，历史对照组患者生存率为42%；经阿司福酶α注射液治疗患者3.5岁时生存率为86%，历史对照组患者生存率为27%；经阿司福酶α注射液患者5岁时生存率为82%，而历史对照组患者生存率为27% [3] 。对于婴幼儿，阿司福酶α注射液有助于逆转矿质过少的症状 [4],[5],[6] 。在儿童患者中，阿司福酶α注射液可帮助患者实现低磷酸酯酶症相关佝偻病的完全或实质性治愈 [7] 。 酶替代治疗是国际公认的针对低磷酸酯酶症病因的有效治疗方法，尤其对于年幼和症状明显的患者，可被作为首选治疗方式 [8] ，然而，此前相关药物的可及性一直是困扰患者的一大难题。此次阿司福酶α注射液在大湾区的率先获批，是国内低磷酸酯酶症治疗的重大突破，为国内患有遗传性低磷酸酯酶症的儿童患者带来了新的治疗希望。 阿斯利康中国生物制药业务总经理，香港及澳门地区负责人林骁 表示："深耕中国逾30年，阿斯利康深刻洞察并理解中国患者未被满足的需求。此次，阿司福酶α注射液得益于‘港澳药械通'政策的支持先行获批引入大湾区，缩短了中国低磷酸酯酶症患者使用全球创新药物的用药时差。未来，我们将继续携手各方合作伙伴，让更多全球领先、患者亟需的创新药物通过大湾区这一桥梁加速引进国内，惠及更多中国患者。" 阿斯利康中国副总裁，罕见病事业部负责人胡轶清 表示："‘以患者为中心'，阿斯利康不仅积极投身并持续探索罕见病领域，还致力于联合多方为中国罕见病患者破解无药可用的困境，并为提升创新药物的可及性而努力。今天，我们非常高兴地看到阿司福酶α注射液在大湾区先行获批引入，让中国低磷酸酯酶症患者加速用上全球领先的药物。未来，阿斯利康也将持续高度重视低磷酸酯酶症及其他罕见病患者的未尽之需，多措并举，加速创新药品引入、可及，并联合各方力量支持建设健全中国罕见病诊疗的生态体系，让我们携手同行，点亮生命之光。" *截至2024年8月，来源于 www.fda.gov , www.ema.europa.eu , https://strensiq-hcp.com/ 声明：本资料涉及的药品和适应症尚未在中国内地获批，阿斯利康不推荐任何未被批准的药品使用。 关于低 磷酸酯酶症 低磷酸酯酶症是一种遗传性、慢性、进行性和危及生命的代谢性疾病，会对患者身体多个系统产生破坏性影响，导致衰弱或危及生命的并发症 [9] 。低磷酸酯酶症的特征是碱性磷酸酶（ALP）活性低和骨矿化缺陷，可导致骨骼破坏和畸形及其他骨骼异常，以及全身并发症，如肌无力和呼吸衰竭，导致婴儿早逝 [4] ,[ 8] ,[10] 。 关于阿斯利康 阿斯利康（LSE/STO/Nasdaq: AZN）是一家科学至上的全球生物制药企业，专注于研发、生产及营销处方类药品，重点关注肿瘤、罕见病以及包括心血管肾脏及代谢、呼吸及免疫在内的生物制药等领域。阿斯利康全球总部位于英国剑桥，业务遍布世界100多个国家，创新药物惠及全球数百万患者。更多信息，请访问 www.astrazeneca.com 。 关于阿斯利康中国 阿斯利康自1993年进入中国以来，专注中国患者需求最迫切的治疗领域，包括肿瘤、心血管、肾脏、代谢、呼吸、消化、罕见病、疫苗抗体及自体免疫等，已将40多款创新药物带到中国。阿斯利康中国总部及全球研发中国中心位于上海，并在无锡、泰州、青岛分别建立全球生产供应基地，已向近70个全球市场输送优质药品。近年来，公司分别在北京、广州、杭州、成都、青岛设立区域总部。阿斯利康还携手合作伙伴，通过打造包括中国智慧健康创新中心（CCiC）、国际生命科学创新园（iCampus）、阿斯利康中金医疗产业基金在内的创新"三驾马车"，构建多元化的国际创新健康生态圈，共同促进区域经济以及大健康行业的长足发展。今天，中国已经发展成为阿斯利康全球第 二大市场。 [1] Strensiq Hong Kong Prescribing Information (40mg), version Apr 2023 (Chinese) [2] Scott LJ. Asfotase Alfa: A Review in Paediatric-Onset Hypophosphatasia. Drugs. 2016 Feb;76(2):255-62. doi: 10.1007/s40265-015-0535-2. [3] Kaplan-Meier analysis of survival of patients with perinatal and infantile hypophosphatasia. Asfotase alfa–treated patients (studies ENB-002-08/ENB-003-08 and ENB-010-10) had a significantly better survival rate, calculated using the Kaplan-Meier product-limit estimate, compared with historical controls (study ENB-011-10; P .0001, Kaplan-Meier log-rank test). [4] Whyte MP, Greenberg CR, Salman NJ, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012;366(10):904-913. [5] Whyte MP, Simmons JH, Moseley S, et al. Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial. Lancet Diabetes Endocrinol. 2019;7(2):93-105. [6] Hofmann CE, Harmatz P, Vockley J, et al. Efficacy and safety of asfotase alfa in infants and young children with hypophosphatasia: a phase 2 open-label study. J Clin Endocrinol Metab. [7] Whyte MP, Madson KL, Phillips D, Reeves AL, McAlister WH, Yakimoski A, Mack KE, Hamilton K, Kagan K, Fujita KP, Thompson DD, Moseley S, Odrljin T, Rockman-Greenberg C. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016 Jun 16;1(9):e85971. doi: 10.1172/jci.insight.85971 [8] 刘敏,赵耘,巩纯秀. 低磷酸酶血症研究新进展[J]. 中华儿科杂志,2019,57(11):899-903 [9] Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013;10(suppl 2):380-388. [10] Fraser D. Hypophosphatasia. Am J Med. 1957;22(5):730-746.

REDWOOD CITY, Calif., May 08, 2024 (GLOBE NEWSWIRE) — Coherus BioSciences, Inc. (Coherus; Nasdaq: CHRS) today announced that the Cancer Research Institute (CRI) and its Immunotherapy Platform Study in Platinum-Resistant High-Grade Serous Ovarian Cancer (IPROC) Drug Selection Committee (DSC) have selected LOQTORZI (toripalimab-tpzi), anti-PD-1 antibody, to explore in combination with ENB-003, a first-in-class small molecule inhibitor of endothelin B receptor (ETBR), for the treatment of drug-resistant cancers in the iPROC platform study. Endothelin B receptor is implicated in tumorigenesis and tumor immune suppression for several solid tumors, including melanoma, ovarian, and pancreatic cancers. “The iPROC DSC is pleased to have access to this next-generation PD-1 inhibitor and advance this combination supported by preclinical and clinical data in this study in ovarian cancer,” said John Stagg, Ph.D., Professor, Faculty of Pharmacy at the University of Montreal and Principal Investigator, Centre Hospitalier de l’Université de Montréal (CHUM) and its affiliated Cancer Institute of Montreal. Dr. Stagg continued, “The iPROC study DSC brings together a group of scientific experts within the CRI community to select promising combinations and rapidly advance them to clinical trials. Importantly, the DSC is not confined by a pipeline but looks to work with companies with drugs that have a strong data package. For this multiparty platform study to succeed, it requires science-driven, highly collaborative, and engaged companies. Coherus and ENB Therapeutics are two such partners, and we are excited to advance this clinical study.” “CRI is an established and preeminent organization leading immunotherapy discovery and advancement through its global network of scientific experts, and we are excited that the CRI network has selected LOQTORZI for this study to gain more insight into the potential clinical benefit that LOQTORZI might have when combined with novel mechanisms such as ENB Therapeutics’ ETBR blocker, particularly in this underserved patient population,” said Rosh Dias, M.D., Chief Medical Officer at Coherus. “There is a large body of clinical evidence for LOQTORZI, including positive phase 3 studies published in top-tier scientific journals, showing efficacy and safety in multiple tumor types,” said Jay Campbell, Managing Director, Clinical Accelerator and Venture Fund at CRI. “This level of scientific evidence is what we are looking for when selecting immunotherapies to bring into our network, with the goal of advancing clinically meaningful solutions to patients. We are grateful to have the opportunity to collaborate with Coherus and ENB Therapeutics on this study and excited to be working with them on this novel combination that we hope will lead to improved outcomes in these patient populations.” Dr. Sumayah Jamal, CEO of ENB Therapeutics, added, “We are honored to be collaborating with CRI and Coherus to further accelerate the development of innovative immunotherapy solutions and expand the pool of patients benefitting from this approach to harness the immune system to fight cancer.” Under the terms of a clinical supply agreement with the Cancer Research Institute (CRI), Coherus will supply toripalimab-tpzi for combination treatment with ENB-003 to be investigated in the iPROC platform study. About the IPROC Platform Trial The IPROC clinical trial uses an adaptive platform study design that utilizes a single master protocol to evaluate multiple immunotherapy combinations. This allows multiple treatments to be evaluated in different groups of patients, or cohorts, from the same patient population. Such a study design offers flexibility in that different treatments can be evaluated in different cohorts, treatment regimens can be modified between cohorts, and treatment selection criteria can be customized for a specific cohort. The trial, titled Immunotherapy Platform Study in Platinum-Resistant High-Grade Serous Ovarian Cancer (IPROC) (NCT04918186), has two ongoing cohorts. About LOQTORZI (toripalimab-tpzi) LOQTORZI is an anti-PD-1 monoclonal antibody that blocks PD-L1 binding to the PD-1 receptor at a unique site with high affinity and activates anti-tumor immunity. LOQTORZI is indicated in the United States in combination with cisplatin and gemcitabine for first-line treatment of adults with metastatic or recurrent locally advanced nasopharyngeal carcinoma (NPC) and as a single agent for the treatment of adults with recurrent unresectable or metastatic NPC with disease progression on or after platinum-containing chemotherapy. For more information about LOQTORZI, including the U.S. Prescribing Information and important safety information, please visit www.loqtorzi.com. About ENB-003 ENB-003, a small-molecule drug candidate targeting the ETBR, is expressed in solid tumors and plays a role in tumorigenesis and immune evasion. ETBR, a G-protein-coupled receptor, is overexpressed in a variety of tumor cell types and plays a key role in tumor cell proliferation, invasion, epithelial-mesenchymal transition (EMT), and angiogenesis. It also plays a role in tumor immunosuppression and blocks T-cell trafficking. As reported in November 2023, preliminary results from the ENBOLDEN-101 demonstrated that ENB-003 in combination with KEYTRUDA® (pembrolizumab) demonstrated encouraging objective responses, disease control, and progression-free survival in patients with metastatic platinum refractory/resistant ovarian cancer (PROC). About the Cancer Research Institute The Cancer Research Institute, established in 1953, is the preeminent U.S. nonprofit organization dedicated to saving more lives by fueling the discovery and development of powerful immunotherapies for all cancers. Guided by a world-renowned Scientific Advisory Council that includes four Nobel laureates and 35 members of the National Academy of Sciences, CRI has invested over $517 million in support of research conducted by immunologists and tumor immunologists at the world’s leading medical centers and universities and has contributed to many of the key scientific advances that demonstrate the potential for immunotherapy to change the face of cancer treatment. Learn more at cancerresearch.org. About Coherus BioSciences Coherus is a commercial-stage biopharmaceutical company focused on researching, developing, and commercializing innovative cancer treatments. Coherus is developing an innovative immuno-oncology pipeline that is expected to synergize with its proven commercial capabilities in oncology. Coherus’ immuno-oncology pipeline includes multiple antibody immunotherapy candidates focused on enhancing the innate and adaptive immune responses to enable a robust antitumor immunologic response and enhance outcomes for patients with cancer. Casdozokitug is a novel IL-27 antagonistic antibody currently being evaluated in two ongoing clinical studies: a Phase 1/2 study in advanced solid tumors and a Phase 2 study in hepatocellular carcinoma. CHS-114 is a highly selective, competitively positioned, cytolytic anti-CCR8 antibody currently in a Phase 1 study in patients with advanced solid tumors. CHS-1000, a novel ILT4-targeted antibody, is a preclinical candidate targeting immune-suppressive mechanisms via the pathway ILT4. Coherus markets LOQTORZI® (toripalimab-tpzi), a novel next-generation PD-1 inhibitor, UDENYCA® (pegfilgrastim-cbqv), a biosimilar of Neulasta®, and YUSIMRY® (adalimumab-aqvh), a biosimilar of Humira®. About ENB Therapeutics ENB Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, ETBR inhibitors, to overcome resistance to CAR-T in solid tumors and immune-based therapies such as immune checkpoint inhibitors. ETBR causes uncontrolled cancer growth, drives cancers to spread through the body, and prevents the immune system from detecting and killing cancer cells. ENB’s lead product candidate, ENB-003, specifically blocks the ETBR and can potentially drive the efficacy of CAR-T and anti-PD1 therapies in solid tumors. ENB-003 is being investigated in an ongoing Phase 1/2 clinical trial in collaboration with Merck. Forward-Looking Statements Except for the historical information contained herein, the matters set forth in this press release are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Coherus’ ability to identify synergies between its I-O pipeline and its commercial capabilities; Coherus’ expected initiating clinical trials for CHS-1000; Coherus’ expectations to be able to advance its candidates through clinical trials; and Coherus’ expectations that its immunotherapy candidates will enhance outcomes for patients with cancer. Such forward-looking statements involve substantial risks and uncertainties that could cause Coherus’ actual results, performance, or achievements to differ significantly from any future results, performance, or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the risks and uncertainties inherent in the preclinical and clinical drug development process; risks related to Coherus’ existing and potential collaboration partners; risks of Coherus’ competitive position; the risks and uncertainties of the regulatory approval process, including the speed of regulatory review and the timing of Coherus’ regulatory filings; the risks of competition; the risk that Coherus is unable to complete commercial transactions; and the risks and uncertainties of possible litigation. All forward-looking statements contained in this press release speak only as of the date of this press release. Coherus undertakes no obligation to update or revise any forward-looking statements. For a further description of the significant risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Coherus’ business in general, see Coherus’ Annual Report on Form 10-K for the fiscal year ended December 31, 2023 filed with the Securities and Exchange Commission on March 15, 2024, including the section therein captioned “Risk Factors” and in other documents Coherus files with the Securities and Exchange Commission. UDENYCA®, YUSIMRY®, and LOQTORZI®, whether or not appearing in large print or with the trademark symbol, are trademarks of Coherus, its affiliates, related companies, or its licensors or joint venture partners unless otherwise noted. Trademarks and trade names of other companies appearing in this press release are, to the knowledge of Coherus, the property of their respective owners. Neulasta® is a registered trademark of Amgen, Inc. Humira® is a registered trademark of AbbVie Inc. Coherus Contact Information: For Investors: Jami Taylor Head of Investor Relations IR@coherus.com For Media: Jodi Sievers VP, Corporate Communications media@coherus.com