Merck Serono (Beijing) Pharmaceutical R&D Co., Ltd.

//   开栏语 生物医药行业面临着前所未有的挑战与机遇。这些挑战促使各大生物制药公司更加聚焦于临床试验的战略布局、效能提升及资源优化配置，以确保在激烈的内卷中保持领先地位。自9年前的“722”自查核查风暴以来，中国临床试验质量得到了显著提升。然而，每年开展实施的2323项新药临床试验，不仅为审评机构、核查部门、申办方、研究机构和受试者带来了新的发展机遇，同时也伴随诸多挑战。 受礼来全球高级副总裁、药物发展及医学事务中心负责人王莉博士的启发，同时在默克雪兰诺（北京）医药研发有限公司项目管理副总监张文博老师、科霸生物公司临床及医学副总裁李宾老师、爱恩康临床医学总监李弈老师以及研发客副总裁储旻华老师的指导下，我们策划了“临床运营在中国”专栏，并将于8月16日在上海与太美医疗科技携手举办“出海、提效、临床运营新方向——卓越临床战略与运营高峰论坛”。 我们希望通过对行业的深入探访和观察，从IND审评流程、人类遗传资源管理、临床试验设计、伦理审查与合规、AI在临床试验的应用、药物警戒、与CRO跨公司协作、现场核查及未来临床试验新趋势等方面，为业界一线工作者提供有价值的视角和思考。“临床运营在中国”专栏第一篇，将探讨近期热议的话题：IND审批可能减半至30个工作日。（冬蕾） 在临床试验启动环节，进一步缩短IND审批时限是否真实需要？《北京市支持创新医药高质量发展若干措施(2024)》（以下简称《若干措施》）提出的“IND审批时限从60日压缩至30日”的试点备受行业关注和讨论。 研发客采访业内专家们获得的看法是，IND审评时间缩短，对加速临床研究的启动是积极的，但需综合衡量审评资源，进一步扩充人力资源、优化审评流程和简化申报资料。 而是否仅靠IND提速就能提升试验启动效率？如何做到临床试验启动的真正提速？研发客采访多位从事注册的业内专家，他们认为实践中仍存在制约临床试验效率的节点，同样值得关注。同时，专家们建议，只有做到IND审评、GCP立项、伦理审查、人类遗传资源审查、合同审阅等各项流程平行或部分前置进行，才能做到临床试验启动的真正提速。 审评资源要跟上 2018年试行、2019年被正式纳入《药品管理法》开始实施的60日默示许可制，对药品IND审评加速明显。研发客根据NMPA历年药品审评报告统计，2018~2023年间，IND申请受理数量和审结数量的年均增长率分别达31%和22%。 默许制的制度设计在一定程度上借鉴了美国等国外经验，美国FDA的新药IND时限是30天。如果进一步缩短中国的药品临床试验审批时限，中国药品监督管理研究会会长张伟认为，时间缩短至与FDA一样的30天，将加速中国同步参与全球临床研究、尤其是早期临床试验的步伐。 但从实践操作层面，业内人士提出一些考虑。某位国内大型药企注册事务部负责人表示，IND审评时限缩短需量力而行。他通过个人总结以及实践工作经验发现，近三年IND审评审批平均时限约48日左右，如果提速到30日，现有审评力量多方面有不可承受的可能，例如工作强度、审评质量等，对占用其他注册申请特别是上市许可申请资源尤其令人担忧。 中国药科大学药品监管科学研究院执行院长邵蓉，在今年5月泰格医药与研发客联合举办的2024药政法规科学年会（以下简称“法规年会”）上也提到，“应全面回顾评价60天默示许可的落实情况，全面考量CDE的审评人员数量、审评能力、审评资源和审评任务量，不能一味地为了所谓的速度而忽略CDE面临的困难”。 如果要进行IND时限缩短的试点，如何在实操层面提升能力、优化流程帮助提速？ 受访专家们一致认为需要增加人力资源的配备。元羿生物执行副总裁、药政事务负责人左珺在法规年会上提出，目前CDE人力资源很紧张，如果将IND时限进一步缩短，需要国家增加审评资源，否则会挤压NDA的审评资源。 同时，左珺还提出，可以通过进一步简化流程来减轻审评工作量。例如IND批件中要求的沟通交流内容，可以考虑通过一般技术问题咨询关联IND受理号的方式进行，而不是通过II类会议提交的方式。II类会议时限是60日，会拉长临床试验启动的战线。 对IND申报资料，专家们也提出了简化建议。目前IND申报资料是按照ICH《M4：人用药物注册申请通用技术文档（CTD）》，勃林格殷格翰大中华区人用药品注册事务负责人夏鲲表示，这相当于一个新药注册申请（NDA）的申报资料格式要求，资料提交的复杂性相对较高，对IND申报带来了一定的困难和挑战。 某位国内大型药企注册事务部负责人表示，中美在IND申报流程上大体相同，区别在技术要求的细节，这些细节的要求多是不成文、经验性的，如药学研究批次要求、稳定性长短等。对比中美临床试验申请资料清单可发现，CDE对技术要求、特别是证明性文件要求较多。 因而借鉴FDA的IND申报模式，简化申报资料要求，或可作为下一步优化工作的思考方向或者着力点。 此外，IND的审评共包括临床研究、药学以及非临床研究的审评。专家们建议，探索在IND申报之前就针对药学和非临床研究部分进行沟通交流，将这两部分的审评前置，或可能实现时限的进一步缩短。 实际制约研究启动的节点 “IND提速之后，临床试验流程或能进一步加快，但还需要找到实际制约试验启动的节点。”左珺在法规年会上这样提到。 国家药品监督管理局药品审评中心在Nature发表了题为Trends in innovative drug development in China的文章，其分析结果显示，制约临床试验启动的因素不仅限于IND审评时间。文章以2015年7月药政改革作为节点，改革后（2015年7月至2020年12月）的IND获批时间比改革前阶段（2010年1月至2015年6月）缩短了414天（之后87天，之前501天），但IND获批后、第一例受试者入组的时间反而延长了，改革后比改革前多了69天（之后328天、之前269天）。 那么，临床试验启动提速还需要从哪些环节入手？夏鲲表示，企业在申请伦理委员会审查时，时常会被要求提供临床试验批件（CTA），目前还有相当数量的机构不接受伦理前置。如果能做到GCP立项及医学伦理审查同步开展、推进中心伦理/伦理互认的实施，将CTA、伦理审查及人遗行政许可技术评审平行化进行，将大大提高临床试验启动速度。 此外，试验合同审阅和签订也是启动中耗时较多的关键步骤，如果临床试验机构允许申办者在递交IND后就可以启动合同协商和签署，依托行业共识，加快合同审核流程或可提升临床试验启动效率。夏鲲坦言，对跨国药企而言，药品进口也是目前启动的限速步骤之一，近年来国家药检所和海关等相关部门也在积极探索进口流程简化和相关文件出台的可行性。 “只有在临床试验的全链条建设上形成合力，才能真正实现IND审评时限以及临床试验进程有价值和意义的缩短。”张伟总结到。 从《若干措施》的具体细则中能看到，已开始试点优化临床试验的启动流程。其中提到，“支持药物临床试验申办方基于基本研发资料，在向国家药品监督管理局提交临床试验申请前与临床试验机构进行工作对接，并在递交临床试验申请时同步向临床试验机构提交材料，实现立项审查、伦理审查、合同审查同步开展”。 《若干措施》还明确，将临床试验启动整体用时压缩至28周以内。根据RDPAC对临床研究启动速度2023的调研结果，从临床研究方案定稿到第一例受试者入组，我国临床研究启动的时间平均为40周以上，而国际上平均水平为20周以内，能够看出我国在临床启动环节还有差距。 总结而言，IND审评与临床研究启动在整个药品上市进程中不可或缺且至关重要。在进一步缩短IND审评时限，并配备足够人力资源、简化审评流程和申报资料，同时做到CTA、人类遗传资源管理、研究中心伦理和合同审批的真正并行。在保证受试者安全的基础上，实现中国同步加入全球临床研究，加速创新产品在中国的研发和注册，尽早让中国的患者获益，是中国创新药企和药品监管部门共同期盼和实现的愿望。 下篇预告： 近年来，我国在合理利用人类遗传资源方面进行了系列改革。我们调研了RDPAC及多家跨国医药公司，探索其如何与监管部门有效沟通、明确流程并积累经验，未来我国人类遗传资源管理还有哪些改进空间，如何进一步完善审批机制并加强国际合作。 编辑 | 姚嘉 yao.jia@PharmaDJ.com  总第2160期 访问研发客网站，深度报道和每日新闻抢鲜看 www.PharmaDJ.com

SAN DIEGO, March 04, 2024 (GLOBE NEWSWIRE) -- Skye Bioscience, Inc. (OTCQB: SKYE) ("Skye" or the "Company"), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel classes of therapeutic drugs that modulate the endocannabinoid system, with a focus on obesity and ocular indications, is pleased to announce the appointment of Dr. Annalisa Jenkins, MBBS, FRCP, to the Company’s Board of Directors. Dr. Jenkins’ esteemed career spans more than 25 years in the global pharmaceutical, biotechnology and life science industry. She has broad experience in building and leading teams and developing strategies that have resulted in the advancement of new products across multiple disease areas from discovery through to clinical development, regulatory approval and commercialization worldwide. Dr. Jenkins currently provides board level and consulting services to organizations across the life science and healthcare industry. She previously served as President and CEO of Plaquetec Therapeutics and of Dimension Therapeutics, leading efforts to develop novel approaches in coronary vascular and rare diseases that included a significant worldwide partnership for a gene therapy approach to hemophilia A with Bayer Pharmaceuticals. Prior to these roles, Dr. Jenkins held senior leadership roles at Merck Serono Pharmaceuticals as Global Head of Research and Development and at Bristol-Myers Squibb as Senior Vice President Global Medical. Dr. Jenkins sits on a number of boards across the public and private sectors and as a trustee of nonprofit organizations globally, including AVROBIO, COMPASS Pathways, MEREO Biopharmaceuticals, AFFIMED, The British Heart Foundation, The King’s Fund, Cancer Research Horizons, Genomics England and Faster Cures at the Milken Institute. As a pharmaceutical thought leader, Dr. Jenkins routinely contributes publicly on leadership with purpose, social entrepreneurship, diversity and innovation. “We are delighted to have someone of Dr. Annalisa Jenkins’ caliber join the board of Skye,” said Punit Dhillon, CEO and Chair of Skye. “Dr. Jenkins' distinguished and diverse career goes back further than her notable positions in biotech and large pharmaceutical companies. With her astute understanding of pharmaceutical development, and her distinguished leadership in several existing and emerging life science hubs around the globe, we believe Dr. Jenkins’ guidance and presence will have a significant impact across Skye’s endeavors to develop and commercialize our novel classes of therapeutic drugs that are focused initially on obesity as well as primary open-angle glaucoma and ocular hypertension.” Dr. Jenkins was commissioned by the British Royal Navy as a Surgeon Sub Lieutenant prior to graduating in medicine from St. Bartholomew’s Hospital in the U.K. Dr. Jenkins served as a medical officer in the British Royal Navy for nine years, achieving the rank of Surgeon Lieutenant Commander. About Skye Bioscience Skye is focused on unlocking the pharmaceutical potential of the endocannabinoid system to treat diseases with metabolic, inflammatory, and fibrotic processes. Backed by specialist life science investors, Skye's strategy leverages biologic targets with substantial human proof of mechanism for the development of first-in-class therapeutics with significant clinical and commercial differentiation. Skye plans to start a Phase 2 clinical trial in obesity in mid-2024 for nimacimab, a negative allosteric modulating antibody that inhibits peripheral CB1, comparing monotherapy and combination arms of nimacimab and a GLP-1R agonist. SBI-100 Ophthalmic Emulsion, a CB1 agonist, is being studied in a Phase 2 trial of patients with glaucoma and ocular hypertension, with data expected in Q2 2024. For more information, please visit: https://www.skyebioscience.com. CONTACT Investor Relationsir@skyebioscience.com858-410-0266 LifeSci Advisors, Mike Moyermmoyer@lifesciadvisors.com617-308-4306 FORWARD LOOKING STATEMENTS This release may contain forward-looking statements, including statements regarding the Company's product development programs, business strategy, and expected plans with respect to clinical trials, including the timing of patient enrollment and clinical trial data updates. Such statements and other statements in this press release that are not descriptions of historical facts are forward-looking statements that are based on management's current expectations and assumptions and are subject to risks and uncertainties. If such risks or uncertainties materialize or such assumptions prove incorrect, our business, operating results, financial condition, and stock price could be materially negatively affected. In some cases, forward-looking statements can be identified by terminology including "anticipated," "plans," "goal," "focus," "aims," "intends," "believes," "can," "could," "challenge," "predictable," "will," "would," "may" or the negative of these terms or other comparable terminology. We operate in a rapidly changing environment, and new risks emerge from time to time. As a result, it is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements the Company may make. Risks and uncertainties that may cause actual results to differ materially include, among others, our capital resources, uncertainty regarding the results of future testing and development efforts and other risks that are described in the Risk Factors section of the Company's most recent annual or quarterly report filed with the Securities and Exchange Commission. Except as expressly required by law, the Company disclaims any intent or obligation to update these forward-looking statements.