Polycythemia Vera

2024年8月15-16日 苏州国际博览中心B-D馆展会背景CMC-China博览会以研发为主线，串联医药上下游，以圈友信任为纽带，帮助大家“寻找到下一个合作伙伴”，自2019年以来，我们已成功举办5届，影响力持续提升，在中国医药产业创新发展的大浪潮中，茁壮成长，已经成为国内生物医药领域大型博览会的知名品牌。企业邀约上海瀚合瑞——提供制剂项目从立项到获批的全流程药学CRO服务！欢迎莅临D馆-G05展位，现场参观，交流分享！↓↓扫描下方二维码免费报名重点项目推荐乌帕替尼缓释片规格：15mg、30mg、45mg适应症：特应性皮炎、类风湿关节炎、银屑病关节炎、溃疡性结肠炎等研发进展：小试阶段巴瑞替尼片规格：2mg、4mg适应症：类风湿关节炎、斑秃、特应性皮炎等研发进展：小试阶段非奈利酮片规格：10mg、20mg适应症：二型糖尿病相关的慢性肾病研发进展：中试阶段奥匹卡朋胶囊规格：25mg、50mg适应症：左旋多巴/卡比多巴的辅助治疗，适用于帕金森病(PD)的“off”期发作研发进展：原料药&制剂联合开发，小试阶段甲苯磺酸奥马环素片&注射用甲苯磺酸奥马环素规格：片剂150mg、注射剂100mg适应症：社区获得性细菌性肺炎感染、急性细菌性皮肤和皮肤结构感染研发进展：小试阶段来特莫韦片&来特莫韦注射液规格：片剂240mg、480mg、注射剂240mg：12ml适应症：用于接受异基因造血干细胞移植（HSCT）的巨细胞病毒（CMV）血清学阳性的成人受者[R+]，以及肾脏器官移植后(供者CMV血清阳性/受者CMV血清阴性)的成人患者的CMV感染和CMV病的预防研发进展：原料药&制剂联合开发，验证批阶段注射用硫酸艾沙康唑&硫酸艾沙康唑胶囊规格：注射剂 372mg、胶囊剂186mg适应症：治疗成人侵袭性曲霉病和毛霉菌病，属于第三代唑类抗真菌药研发进展：原料药&制剂联合开发，完成验证批地屈孕酮片规格：10mg适应症：用于治疗内源性孕酮不足引起的疾病，如痛经、子宫内膜异位症、继发性闭经、月经周期不规则、功能失调性子宫出血、经前期综合症、先兆性流产和黄体不足所致不孕症等研发进展：原料药&制剂联合开发，中试阶段阿帕他胺片规格：60mg适应症：是首款用于治疗非转移性去势抵抗性前列腺癌及转移性去势敏感性的前列腺癌的药物研发进展：原料药&制剂联合开发，完成预BE注射用卡非佐米规格：60mg适应症：难治性多发性骨髓瘤研发进展：原料药&制剂联合申报，评审中阿贝西利片规格：50mg、100mg、150mg适应症：早期乳腺癌；局部晚期或转移性乳腺癌研发进展：原料药&制剂联合开发，中试阶段妥卡替尼片规格：50mg、150mg适应症：乳腺癌研发进展：原料药&制剂联合开发，中试阶段注射用盐酸美法仑规格：50mg适应症：多发性骨髓瘤研发进展：小试阶段磷酸芦可替尼片规格：5mg、15mg、20mg适应症：用于中危或高危的原发性骨髓纤维化（PMF）、真性红细胞增多症继发的骨髓纤维化（PPV-MF）或原发性血小板增多症继发的骨髓纤维化（PET-MF）的成年患者研发进展：完成预BE部分已有开发经验制剂品种关于上海瀚合瑞上海瀚合瑞医药科技有限公司：提供制剂项目从立项到获批的全流程药学CRO服务，具备经验丰富的的固体制剂、液体制剂和半固体制剂研发团队，以及完备的工艺和分析研发设备。截至目前，团队已完成近200个仿制药和多个创新药制剂研发服务， 多个仿制药项目取得国内首仿或前三仿。可与兄弟单位一起，为客户提供制剂和原料药研发的一站式整体解决方案，包括药学研究、临床研究（含生物等效性研究）、委托加工落户、备案登记/申报注册、MAH 持证管理、上市后技术支持等。 集团技术服务平台01制剂技术平台固体制剂、注射剂、外用制剂全流程开发难溶性药物给药技术固体分散体技术高难度复方制剂开发平台MAH合作/品种转让02原料技术平台仿制药与创新药原料药工艺开发创新工艺路线设计不对称合成、金属催化偶联、致突变杂质研究分析方法开发和验证03临床技术平台临床药代动力学生物等效性临床实验方案设计及研究运营、覆盖Ⅰ-Ⅳ期及真实世界分析检测服务联系我们1330658606013625705846↓↓扫描下方二维码免费报名关于CMC-China博览会

On track to achieve 250 patient enrollment target in March 2024 Top-line data for 32-week primary endpoint expected in the first quarter of 2025 NEWARK, CA / ACCESSWIRE / March 26, 2024 / Protagonist Therapeutics, Inc. ("Protagonist" or the "Company") announced it has randomized 241 patients in the ongoing Phase 3 VERIFY clinical trial evaluating rusfertide in polycythemia vera ("PV") as of today and expects to meet the trial's 250 patient enrollment target by the end of March 2024. Eligible patients in screening as of March 31, 2024 will be randomized by the end of April 2024. The Company expects to announce top-line data for the study's 32-week primary efficacy endpoint by the end of the first quarter of 2025. "Currently available therapies provide suboptimal hematocrit and symptom control in patients with PV, and we believe rusfertide has the potential to meet these unmet medical needs. We are looking forward to the results of the VERIFY phase 3 study in anticipation of filing an NDA for rusfertide in 2025 together with our partner Takeda," said Arturo Molina, M.D., M.S., Protagonist's Chief Medical Officer. "We wish to thank the principal investigators and study staff for their dedication and contributions towards completing enrollment, and the patients who have agreed to participate in this important research study." The Phase 3 VERIFY trial (NCT05210790) is a global Phase 3 randomized, double-blind, placebo-controlled trial designed to enroll approximately 250 patients. The trial evaluates the efficacy, symptom burden and safety of once-weekly, subcutaneously self-administered rusfertide in patients with uncontrolled hematocrit who are phlebotomy dependent despite standard of care treatment. The trial enrolled patients in 20 countries across North and South America, Europe, Asia and Australia. This Phase 3 study along with the REVIVE Phase 2 trial and other completed studies will comprise the clinical data package planned for NDA filing. "2024 has been a very busy year for the rusfertide program, and we are pleased to announce our Phase 3 VERIFY trial is advancing as planned," said Dinesh V. Patel, Ph.D., the Company's President and CEO. "Rusfertide has performed very well in the Phase 2 REVIVE study as further confirmed by the long-term follow-up data showing the durability of hematocrit control. We look forward to data from the 52-week endpoint for VERIFY including the durability of response in the first half of 2025, and potentially filing an NDA in the fourth quarter of 2025. Dr. Patel continued, "We are in a very exciting phase for the Company as we anticipate a series of announcements and data disclosures in the second half of 2024 and throughout 2025, centering on our late stage partnered assets, as well as our fully owned discovery programs like Oral IL-17 antagonist, and other programs in hematology and metabolic diseases." About Protagonist Protagonist Therapeutics is a biopharmaceutical company with peptide-based new chemical entities (NCEs) rusfertide and JNJ-2113 (formerly PN-235) in advanced Phase 3 stages of clinical development, both derived from the Company's proprietary technology platform. Protagonist and JNJ scientists jointly discovered PN-235 (now known as JNJ-2113) as part of Protagonist's Interleukin-23 receptor (IL-23R) collaboration with JNJ and followed it through IND-enabling pre-clinical and Phase 1 studies, with JNJ assuming responsibility for further clinical development. Rusfertide, a mimetic of the natural hormone hepcidin, is the Company's lead drug candidate currently in a global Phase 3 development program. The randomized portion of the Phase 2 REVIVE study has been successfully completed, and results were published in The New England Journal of Medicine in February 2024. The open-label extension component of Phase 2 is ongoing, as is the global Phase 3 VERIFY study of rusfertide in polycythemia vera. Rusfertide is being co-developed and co-commercialized with Takeda pursuant to a worldwide collaboration and license agreement with Takeda announced in January 2024. More information on Protagonist, its pipeline drug candidates and clinical studies can be found on the Company's website at . Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding the potential clinical benefits of rusfertide, the timing of top-line data and other data disclosures. In some cases, you can identify these statements by forward-looking words such as "anticipate," "believe," "may," "will," "expect," or the negative or plural of these words or similar expressions. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, our ability to develop and commercialize our product candidates, our ability to earn milestone payments under our collaboration agreement with Johnson & Johnson, our ability to use and expand our programs to build a pipeline of product candidates, our ability to obtain and maintain regulatory approval of our product candidates, our ability to operate in a competitive industry and compete successfully against competitors that have greater resources than we do, and our ability to obtain and adequately protect intellectual property rights for our product candidates. Additional information concerning these and other risk factors affecting our business can be found in our periodic filings with the Securities and Exchange Commission, including under the heading "Risk Factors" contained in our most recently filed periodic reports on Form 10-K and Form 10-Q filed with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release. Investor Relations Contact Corey Davis, Ph.D. LifeSci Advisors +1 212 915 2577 cdavis@lifesciadvisors.com Media Contact Virginia Amann, Founder/CEO ENTENTE Network of Companies virginiaamann@ententeinc.com SOURCE: Protagonist Therapeutics, Inc. View the original press release on accesswire.com

Findings from the admIRE study – freedom from atrial arrhythmia recurrence at 12 months – support the submission of the VARIPULSE™ Platform among U.S. patients IRVINE, Calif., March 25, 2024 /PRNewswire/ -- Biosense Webster, Inc., a global leader in cardiac arrhythmia treatment and part of Johnson & Johnson MedTechi, announced the submission of the VARIPULSE™ Platform for Premarket Approval Application (PMA) to the U.S. Food & Drug Administration (FDA). The submission was supported by results from the admIRE study, a prospective, multi-center, non-randomized trial. The VARIPULSE™ Platform is comprised of the VARIPULSE™ Catheter, a fully integrated variable-loop multielectrode catheter; the TRUPULSE™ Generator, a pulsed field ablation (PFA) generator; and the CARTO™ 3 System VARIPULSE™ Software, which provides full integration with the world's leading 3D cardiac mapping system. The VARIPULSE™ Platform is designed to enable pulmonary vein isolation with the versatility of a catheter loop, a simple generator user interface, and a mapping system that provides an intuitive, reproducible workflow with real-time visualization, contact indicator, and PF tagging mechanisms. "The VARIPULSE™ Platform, differentiated by its CARTO™ 3 System integration and mapping capabilities, is part of a versatile portfolio of PFA tools that Biosense Webster is developing to meet the varied needs of electrophysiologists and their patients," said Jasmina Brooks, President, Biosense Webster, Inc. "Based on the results from the admIRE clinical trial, we are confident that this innovation has the promise to deliver significant value and differentiated capabilities, and become an important tool for the treatment of AFib." Twelve-month outcomes data from the pilot phase of the admIRE study, which assessed the safety and efficacy of the VARIPULSE™ Platform among U.S. patients, were presented as a late-breaker at the 2024 AF Symposium.1 Among 20 patients who completed the 12-month follow-up visit, 100% achieved acute success from ablation procedures and 80% remained free from atrial arrhythmia recurrence at one year.1 No procedure or device-related primary adverse events were reported in the pilot phase of the study.1,ii For patients who had received ablation, median procedure and fluoroscopy times were 90.0 and 3.5 minutes, respectively, likely attributable to integration with the CARTO™ 3 System.1 Full admIRE study results will be presented later this year. AFib is the most common type of cardiac arrhythmia and affects more than 6 million people in the United States and nearly 38 million people worldwide.2,3 Approximately 1 in 4 adults over the age of 40 are at risk for developing AFib.4 Despite these projections, many people are unfamiliar with AFib symptoms, available treatment options, and the importance of early treatment to avoid disease progression.5 Catheter ablation is a safe and effective procedure when drugs don't work to help restore the heart's incorrect electrical signals, which cause an abnormal heart rhythm.6 Learn more about Biosense Webster's PFA research and clinical evidence here. About admIRE The admIRE study (Assessment of Safety and Effectiveness in Treatment Management of Atrial Fibrillation With the Biosense Webster IRE Ablation System) (NCT05293639) is a prospective, multi-center, single-arm study to demonstrate the safety and long-term effectiveness of the VARIPULSE™ Platform when used for isolation of pulmonary veins in treatment of patients in the United States with symptomatic drug refractory paroxysmal AFib. Pulmonary vein isolation (PVI) was achieved using the VARIPULSE™ Platform. The study consisted of a pilot phase, which assessed initial device safety and effectiveness, and a pivotal phase, which assessed these against pre-specified performance goals. The primary safety endpoint was incidence of early onset (within seven days) primary adverse events; atrio-esophageal fistula (within 90 days); cardiac tamponade or perforation (within 30 days); and PV stenosis (within 12 months). Acute procedure success (defined as confirmed entrance block at the end of procedure) and freedom from documented atrial arrhythmia recurrence at 12 months were also assessed. About the VARIPULSE™ Platform The VARIPULSE™ Platform is Biosense Webster's Irreversible Electroporation ablation system. The fully integrated platform includes the VARIPULSE™ Catheter, TRUPULSE™ Generator, and CARTO™ 3 Mapping System VARIPULSE™ Service Pack Software. In January 2024, Biosense Webster announced that the VARIPULSE™ Platform received its first regulatory approval from the Japan Ministry of Health, Labour and Welfare for the treatment of symptomatic drug refractory recurrent paroxysmal AFib using PFA. In the U.S., the VARIPULSE™ Catheter and TRUPULSE™ Generator are currently investigational and are not approved by FDA. In Europe, the TRUPULSE™ generator received CE mark in late 2023 and the VARIPULSE™ Catheter received CE mark in February 2024. About Biosense Webster Biosense Webster, Inc. is the global market leader in the science and technology behind the diagnosis and treatment of cardiac arrhythmias. Part of Johnson & Johnson MedTech, the specialized medical-technology company is headquartered in Irvine, California, and works across the world to advance the tools and solutions that help electrophysiologists identify, treat, and deliver care. Learn more at biosensewebster.com and connect on LinkedIn and X, formerly Twitter. About Johnson & Johnson MedTechi At Johnson & Johnson MedTech,i we unleash diverse healthcare expertise, purposeful technology, and a passion for people to transform the future of medical intervention and empower everyone to live their best life possible. For more than a century, we have driven breakthrough scientific innovation to address unmet needs and reimagine health. In surgery, orthopedics, vision, and interventional solutions, we continue to help save lives and create a future where healthcare solutions are smarter, less invasive, and more personalized. Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding the VARIPULSE™ Platform clinical trials. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Biosense Webster, Inc. and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: uncertainty of regulatory approvals; uncertainty of commercial success; challenges to patents; competition, including technological advances, new products, and patents attained by competitors; product efficacy or safety concerns resulting in product recalls or regulatory action; changes to applicable laws and regulations, including global health care reforms; changes in behavior and spending patterns of purchasers of healthcare products and services; and trends toward healthcare cost containment. A further list and descriptions of these risks, uncertainties, and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at sec.gov, jnj.com or on request from Johnson & Johnson. Neither of Biosense Webster, Inc. nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.