Betrixaban

REDWOOD CITY, Calif., Sept. 25, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on the development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address diseases such as chronic spontaneous urticaria (CSU), lower to intermediate risk myelodysplastic syndromes (LR-MDS) as well as novel stem cell transplant conditioning regimens, today announced the appointment of Herb Cross, an experienced senior financial executive, as the company’s Chief Financial Officer (CFO). Jeet Mahal, who previously held both CFO and Chief Operating Officer (COO) roles, will continue as the company’s COO. “We are very pleased to welcome Herb, who brings more than twenty years of public company executive leadership experience to our team, as our new CFO,” said Ronald Martell, Chief Executive Officer of Jasper. “With multiple clinical studies of briquilimab in Chronic Spontaneous Urticaria, Low to Intermediate Risk MDS and hematopoietic stem cell transplant either ongoing or planned to start this year, we are taking the opportunity to strengthen our leadership team in preparation for continued growth. The addition of Herb also allows Jeet Mahal to focus on our increasing operational needs in his ongoing role as our COO.” "I am excited to join Jasper at an important time in its development, particularly given the growing set of compelling data supporting the continued development of briquilimab,” said Herb Cross. “I look forward to building on the company’s strong foundation and am eager to begin working with the leadership team to help realize briquilimab’s potential.” Mr. Cross brings more than two decades of experience in financial leadership roles at both public and private biotechnology companies. Before joining Jasper, he served as CFO at Atreca, leading its initial public offering and holding responsibility for all administrative functions, including finance, corporate communications, human resources and corporate services. Prior to Atreca, he was CFO at ARMO BioSciences, with responsibility for all administrative functions and business development, and led its initial public offering and served as CFO through the sale of the company to Eli Lilly in 2018. Mr. Cross also served as CFO and interim Chief Executive Officer at KaloBios Pharmaceuticals and was CFO at Affymax as well. Mr. Cross began his career at Arthur Andersen, LLP, and received a Bachelor of Science in Business Administration from the Haas School of Business at the University of California, Berkeley. He is a certified public accountant, currently inactive, in the state of California. Mr. Mahal joined Jasper in December 2019 from Portola Pharmaceuticals, where he worked for 11 years and held a number of positions of increasing leadership, most recently as Vice President, Business Development and Vice President, Strategic Marketing. He led the successful execution of multiple business development partnerships for Andexxa®, Bevyxxa® and cerdulatinib. He also played a key role in Portola Pharmaceuticals’ equity financings, including its initial public offering and multiple royalty transactions. Earlier in his career, he was Director, Business and New Product Development, at Johnson & Johnson on the Xarelto® development and strategic marketing team. He started his career in the drug development laboratories at COR Therapeutics. Inducement Grant In connection with the appointment of Mr. Cross as its Chief Financial Officer, on September 22, 2023, Jasper granted Mr. Cross an option to purchase 550,000 shares of its voting common stock (the “Option”). The Option was granted pursuant to the Jasper Therapeutics, Inc. Amended and Restated 2022 Inducement Equity Incentive Plan, as approved by the compensation committee of Jasper’s board of directors on March 14, 2022 and as amended and restated on June 2, 2023, and was granted as an inducement material to Mr. Cross’ employment with Jasper in accordance with Nasdaq Listing Rule 5635(c)(4). The exercise price of the Option is $0.78. The Option will vest over four years, with 25% of the total number of shares vesting on September 22, 2024 and 1/48th of the total number of shares subject to the Option vesting monthly thereafter, subject in each case to Mr. Cross’ continued service to Jasper on each vesting date. Jasper is providing this information in accordance with Nasdaq Listing Rule 5635(c)(4). About Jasper Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic urticaria and lower to intermediate risk myelodysplastic syndromes (MDS) and as a conditioning agent for stem cell transplants for rare diseases such as sickle cell disease (SCD), Fanconi anemia (FA) and severe combined immunodeficiency (SCID). To date, briquilimab has a demonstrated efficacy and safety pro over 145 dosed patients and healthy volunteers, with clinical outcomes as a conditioning agent in SCID, acute myeloid leukemia (AML), MDS, FA, and SCD. For more information, please visit us at . Forward-Looking Statements Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding briquilimab’s potential, the development of briquilimab for CSU, LR-MDS and stem cell transplant conditioning and plans to commence multiple clinical studies of briquilimab, briquilimab’s further development and the strategic direction for briquilimab. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jasper’s product candidates may not be beneficial to patients or successfully commercialized; patients’ willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper’s business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jasper’s filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2022 and any subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper’s assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements. Contacts: John Mullaly (investors) LifeSci Advisors 617-429-3548 jmullaly@lifesciadvisors.com Jeet Mahal (investors) Jasper Therapeutics 650-549-1403 jmahal@jaspertherapeutics.com Lauren Barbiero (media) Real Chemistry 646-564-2156 lbarbiero@realchemistry.com

//  CDE《新药获益-风险评估技术指导原则》的出台，将提高我国新药审评决策的可预测性、一致性、透明度和清晰度，反映监管决策的获益-风险评估过程，最终科学做出批准药物上市的决定。在6月25日首次出台的《新药获益-风险评估技术指导原则》（以下简称《指导原则》）里，国家药品监督管理局药品审评中心（CDE）建议企业在新药上市申请中，依照获益-风险评估框架与CDE进行沟通交流，同时制定获益-风险评估框架与计划。据了解，申请人在临床研发之前所做的框架和计划，应在提交申请时向CDE提供获益-风险评估框架及相关内容。当企业制定了计划以后，开展的临床研究结果不但可用于研发决策，同时还可用于与监管机构沟通，或作为上市申报资料，为支持药品上市申请提供证据。对比欧美药监部门，中国虽然制定有关风险获益的指南起步较晚，但业内普遍认为，这一指南规范系统地指导了药物临床研发中对获益-风险评估的把握，为企业提供可参考的技术指导，指导药物研发。获益-风险评估框架包含什么获益-风险评估是药物临床开发、上市申请和上市后监管决策的重要考虑因素。此次，CDE的指导原则主要围绕获益-风险评估的概念、总体考虑、上市申请的获益-风险评估以及临床研发中的获益-风险评估计划以及其他需要关注事项加以介绍。其中，对企业最大的影响莫过于制定获益-风险评估的框架和计划。获益-风险评估框架和计划将发挥什么作用？指导原则认为，药物获益与风险的评估，不能仅通过直接对比有效性和安全性数据而得出结论。因此，制定结构清晰的获益-风险评估框架和计划，提供获益-风险评估中不同关键问题、相应证据和不确定性信息十分重要。具体而言，CDE对获益-风险评估的总体考虑主要有以下5个方面：首先，当药物带来具有临床意义的获益，并且该药物的安全性特征良好，且未发现严重的安全性风险时，新药的获益-风险评估较为直接明确。若新药存在已识别或潜在的严重安全性风险，如风险危及生命或有较高的不良反应和不良事件的发生率，获益-风险评估就十分艰巨。其次，适应症是评估药物获益是否大于严重风险时重要的信息，如新药拟申请适应症无可用治疗选择，或新药较现有治疗有明显优势时，患者或能接受更大的风险。第三，企业上市申请提交的证据，可以作为支持提供药物获益和风险评估的信息。主要来源包括非临床数据、临床数据、患者体验数据和流行病学数据等。第四，要通过良好的临床试验设计和实施，减少药物获益和风险中的不确定性。对于不确定性，CDE将综合考虑，科学评估药物的获益是否超过风险，以及是否需要进一步的处理措施。最后，收集患者体验数据有助于确定未满足的患者需求。近期CDE出台了一系列以患者为中心的技术指南也备受关注，体现出患者对获益-风险的偏好。强生旗下杨森制药中国研发中心代理负责人狄佳宁博士告诉研发客：“这个获益-风险框架让同样的客观证据和潜在不同的主观视角可以在同一个系统平台中进行探讨和评价，令获益-风险评价更加科学和逻辑。”获益-风险评估框架作为一种定性方法，包括了获益-风险评估中的重要考虑因素、支持性证据和不确定性，无论对监管部门还是企业都有非常清晰的指引。如果药物申请多个适应症，应结合每个拟申请的适应症单独进行获益-风险评估。前FDA审评员建议尽早准备《指南原则》要求企业在临床研发中要制定获益-风险评估计划，因为这是药物全生命周期获益-风险评估的重要部分，有助于指导药物研发。前美国FDA高级审评员、思路迪医药首席医学官肖申博士建议，尽早制定获益-风险评估计划将助力临床试验设计和实施事半功倍，研发人员要注意临床终点设计、终点指标的选取，提高药物对患者的获益。“在美国，企业在IND递交过程中，FDA往往要求公司写1~2页整体开发计划，需要企业提到风险-获益的计划与想法。企业如能制定计划，并依此与FDA沟通交流，往往会取得监管部门的认可和信赖。”肖申说。同时，还要全面收集临床前研究的安全数据，临床试验所有与不良反应的相关数据，判断个体与群体发生风险之间的比例与相关性。正如《指导原则》指出的包括历史性对照、流行病学数据和同类产品的数据（如果有的话）等都要考量。肖申还建议，申请人应在临床研发各个阶段与监管机构保持密切沟通，尤其在2期临床试验结束后3期临床试验开展前的沟通中，应对获益-风险评估给予高度重视，协助CDE作出最明智的决策与判断。贯穿药物全生命周期事实上，获益-风险评估是早已经是业内耳熟能详的概念和新药研发的实践方法。中国药品监督管理研究会会长张伟解释说，风险和获益评估是一个动态和复杂的过程，贯穿于药品全生命周期，需要综合考虑新药开发目的、设计、方法、人群、环境等，并根据不同阶段进行调整。《指导原则》的制定，对保护受试者、保证试验质量、促进医学发展具有重要意义。需要参与新药开发的各方和全社会共同提高对风险与获益评估的认识和能力，建立完善和规范的评估体系和流程。具体而言，风险分为试验风险和治疗风险，前者是由试验行为造成，后者是即使不参加临床研究也会承担的风险。风险的程度一般分为4级：最小风险、低风险、中风险和高风险。临床试验中应根据风险大小采取相应的风险控制措施，切实保护受试者安全。而获益是指参与临床试验而可能获得的益处，包括受试者获益和社会获益。受试者获益主要是指通过试验改善疾病状况或提高生活质量，或通过试验检查发现并预防潜在的疾病。社会获益主要是指通过试验结果，促进医学知识和技术的进步，为类似患者提供更有效或更安全的治疗或预防方法。狄佳宁认为，CDE发布的指南为药物研发中的获益-风险评价提供了清晰的框架和逻辑。同时，实际药物开发和评价往往不是“非黑即白”“非对即错”，需要全面权衡患者的偏好和体验，并且把握几点关键原则：药物获益-风险平衡是客观和主观评价的结合、是一套基于群体结论的标准、并且应反映药物对于已有治疗手段的相对特征。来自临床界的专家也同意这一观点。复旦大学附属肿瘤医院I期临床研究病房行政主任、肿瘤内科主任医师张剑教授认为，获益和风险其实没有绝对，只有相对。作为临床医生，通常会从病人角度多考虑，给病人选择当时的最优或较优治疗，其中包括一些已有一定疗效数据的创新药。肖申评价认为，《指导原则》体现了CDE对新药临床开发、审评过程的科学思考，对差异化的临床试验设计具有科学的指导意义。《指导原则》的出台，将提高我国新药审评决策的可预测性、一致性、透明度和清晰度，反映监管决策的获益-风险评估过程，最终科学做出批准药物上市的决定。Fortrea Japan临床开发部（原Labcorp Development Japan）、《医药研发达人》主编高野哲臣最后总结说，CDE近年来出台技术指南的速度之快、范围之广、内容之具体、力度之大，往往引发业内热议。国家药监局/CDE作为药品主管部门通过制定指南，正确引导近10~15年爆发式增长的新兴生物技术/制药公司的研发活动。很多时候，并非需要经过长时间充分讨论并得出结论才行动。CDE密切关注 ICH、美国、欧洲和日本药监局及产业界、学术界的动向，时刻保持思考，并形成自己的想法和主见发布每一份指南，展示了其审评实力。►关于各国监管部门对获益-风险的主要考虑放眼全球，有关获益-风险技术指南的制定由来已久（见下图），美国FDA早在2012年发布首个与获益-风险相关的技术指南。尽管中国CDE起步较晚，但我国审评机构在药物评价过程中对获益-风险平衡的关注正逐步提高。事实上，不同国家的监管机构对获益-风险的评估标准也可能不同。强生旗下杨森制药中国研发中心代理负责人狄佳宁博士认为，美国FDA和欧盟在具体案例评审上往往显示出不同的理念。例如，Dexience（贝曲沙班，预防静脉血栓栓塞）2017年6月被FDA批准，而基于同样数据的上市申请在2018年3月却被欧盟拒绝，主要原因是治疗组的出血事件导致监管无法确定获益大于风险。“获益和风险的平衡是药监机构对于新药批准的关键标准，同时由于结合了科学数据和主观评价而存在潜在的不同。”狄佳宁说。前美国FDA高级审评员、思路迪医药首席医学官肖申博士介绍说，在美国FDA，对不同治疗领域的不同病人群体，不同临床疗效终点和不同的药物安全特征都会做详细认真的具体分析和评估。必要时还需依托外部专家的帮助、指导。这也是FDA在不同治疗领域有几十个顾问委员会存在的重要基础。FDA的主要责任之一就是从疾病的专业角度协助监管机构评估药物的获益和风险，而这种评估是动态变化的。编辑 | 戴佳凌dai.jialing@PharmaDJ.com 总第1958期访问研发客网站可浏览更多文章www.PharmaDJ.com