Evolocumab

Samir R. Patel, M.D., appointed as Chief Executive Officer Abizer Gaslightwala appointed to Board of Directors BOSTON and LONDON, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX) today announced the appointment of Samir R. Patel, M.D., as Chief Executive Officer, effective December 16, 2024. Dr. Patel has served as interim Chief Executive Officer since May 2024. Additionally, the Company announced it has appointed Abizer Gaslightwala to its Board of Directors, effective December 16, 2024. Michael Grissinger, a member of the Board of Directors, provided notice of his resignation on December 16, 2024. “Since beginning my role as interim CEO in May 2024, we have worked diligently to close the merger with Peak Bio, which we accomplished in November, and position Akari for continued growth and value creation. Looking ahead, we are focusing our efforts and resources to streamline operations and execute on our portfolio prioritization – our potentially best-in-class ADC platform. I am pleased to solidify my role with the Akari team and look forward to building momentum and driving shareholder value in the near and long term,” commented Dr. Patel, Chief Executive Officer of Akari. About Samir R. Patel, M.D. Dr. Patel currently serves as founder and principal of PranaBio Investments, LLC, a firm providing consulting, strategic advisory, and investment services for small cap biotechnology companies. He is also a consultant to GE Global Research, GE’s innovation engine that is creating novel products and solutions across several sectors including biomanufacturing and biotechnology. He has more than 20 years of experience in life sciences including founding SPEC Pharma, LLC, a company that develops and manufactures injectables used in human and veterinary applications. Previously, Dr. Patel held multiple roles in Medical Affairs with Centocor, Inc. (now Johnson & Johnson Innovative Medicine, part of Johnson & Johnson). He holds multiple patents, has been an author on several publications and has been an investigator in numerous clinical research studies. He has served on the boards of several public companies, including Rezolute Bio. Dr. Patel received his medical degree from the Medical College of Ohio and completed his internal medicine internship, residency, and rheumatology fellowship at The University of New Mexico School of Medicine Affiliated Hospitals. About Abizer Gaslightwala Mr. Gaslightwala is a well-established biotechnology / pharmaceutical industry leader with a demonstrated track record of success spanning over 25 years in the development and commercialization of novel medicines across a range of companies and therapeutic areas. Mr. Gaslightwala currently serves as the Senior Vice President and Franchise Head for Oncology at Jazz Pharmaceuticals, where he has full responsibility for a portfolio of products spanning both solid and hematological malignancies that have total annual sales of $1B. These brands include ZIIHERA® for HER2+ cancers, ZEPZELCA® for small cell lung cancer, and RYLAZE®, DEFITELIO®, and VYXEOS® for a range of hematological malignancies. “We are pleased to welcome Abizer to our Board of Directors. We believe the depth and breadth of his development and commercialization leadership and expertise amassed over the course of his career will provide valuable insight as we work to propel Akari to its next phase of growth. Additionally, we would like to thank Michael for his years of service on our board. His perspective and guidance have played a key role in getting the Company to where we are today,” added Dr. Patel. Mr. Gaslightwala added, “I'm thrilled to join the Akari board and have been extremely compelled by the Company’s unique ADC platform targeting the spliceosome, that has the potential to set a new standard of care for cancer patients. I look forward to working closely with the management team and other members of the board to advance this platform forward and potentially address areas of significant unmet need for cancer patients.” Prior to his current position at Jazz Pharmaceuticals, Mr. Gaslightwala has led and driven growth in several leadership roles at Amgen, Pfizer, and Johnson & Johnson across multiple brands including Kyprolis®, Vectibix®, Neulasta®, XGEVAf®, Repatha®, and ELIQUIS®. His experience spans business unit leadership, brand marketing, sales leadership, commercial pipeline planning, advanced analytics and insights, and business development. Mr. Gaslightwala also helped lead R&D strategic planning within the autoimmune/inflammation portfolio at Johnson & Johnson, as well as lead commercial planning for Remicade® and several novel pipeline molecules focused on rheumatoid arthritis, inflammatory bowel disease, psoriasis, and atopic dermatitis. Additionally, Mr. Gaslightwala advised several life science companies through his time at the Boston Consulting Group (BCG). Mr. Gaslightwala holds a BS in Chemical Engineering from Cornell University, and an MBA from the Sloan School of Management, and a MS in Chemical Engineering from MIT. About Akari Therapeutics Akari Therapeutics, Plc (Nasdaq: AKTX) is a biotechnology company developing advanced therapies for autoimmune, oncology and inflammatory diseases. Akari has two lead assets, investigational nomacopan and an antibody drug conjugate (ADC) platform. Nomacopan is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. The Company’s ADC platform includes novel toxins and linkers coupled with important cancer antibody targets. For more information about Akari, please visit akaritx.com. Cautionary Note Regarding Forward-Looking Statements This press release includes express or implied forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), about the Company that involve risks and uncertainties relating to future events and the future performance of the Company. Actual events or results may differ materially from these forward-looking statements. Words such as “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “future,” “opportunity” “will likely result,” “target,” variations of such words, and similar expressions or negatives of these words are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. Examples of such forward-looking statements include, but are not limited to, express or implied statements regarding: the business combination and related matters, including, but not limited to, post-closing operations and the outlook for the Company’s business; the Company’s targets, plans, objectives or goals for future operations, including those related to its product candidates; financial projections; future economic performance,; and the assumptions underlying or relating to such statements. These statements are based on the Company’s current plans, estimates and projections. By their very nature, forward-looking statements involve inherent risks and uncertainties, both general and specific. A number of important factors, including those described in this communication, could cause actual results to differ materially from those contemplated in any forward-looking statements. Factors that may affect future results and may cause these forward-looking statements to be inaccurate include, without limitation: the risk that Akari and Peak Bio may not realize the anticipated benefits of the Merger in the time frame expected, or at all; the ability to retain and hire key personnel; potential adverse reactions or changes to business relationships resulting from the Merger; the potential impact of unforeseen liabilities, future capital expenditures, revenues, costs, expenses, earnings, synergies, economic performance, indebtedness, financial condition and losses on the future prospects, business and management strategies for the management, expansion and growth of the combined business; uncertainties as to the long-term value of Akari’s American Depositary Shares (and the ordinary shares represented thereby), including the dilution caused by Akari’s issuance of additional American Depositary Shares (and the ordinary shares represented thereby) in connection with the Merger; risks related to global as well as local political and economic conditions, including interest rate and currency exchange rate fluctuations; potential delays or failures related to research and/or development of the Company’s programs or product candidates; risks related to any loss of the Company’s patents or other intellectual property rights; any interruptions of the supply chain for raw materials or manufacturing for the Company’s product candidates, the nature, timing, cost and possible success and therapeutic applications of product candidates being developed by the Company and/or its collaborators or licensees; the extent to which the results from the research and development programs conducted by the Company, and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; uncertainty of the utilization, market acceptance, and commercial success of the Company’s product candidates; unexpected breaches or terminations with respect to the Company’s material contracts or arrangements; risks related to competition for the Company’s product candidates; the Company’s ability to successfully develop or commercialize its product candidates; potential exposure to legal proceedings and investigations; risks related to changes in governmental laws and related interpretation thereof, including on reimbursement, intellectual property protection and regulatory controls on testing, approval, manufacturing, development or commercialization of any of the Company’s product candidates; the Company’s ability to maintain listing of its ADSs on the Nasdaq Capital Market. While the foregoing list of factors presented here is considered representative, no list should be considered to be a complete statement of all potential risks and uncertainties. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the SEC, copies of which may be obtained from the SEC's website at www.sec.gov. The Company assumes no, and hereby disclaims any, obligation to update the forward-looking statements contained in this press release. Investor Contact: JTC Team, LLCJenene Thomas908.824.0775AKTX@jtcir.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/110aebf9-2683-41c9-ba9d-bf06f7e42054

关注并星标CPHI制药在线 在当今竞争激烈的生物制药行业，开发重磅炸弹药物不仅仅是技术和科学的挑战，更是制药巨头在市场上获得持续竞争优势的关键所在。重磅炸弹是众多药物开发中"九天阊阖开宫殿，万国衣冠拜冕旒"的角色，它们能够迅速成为开发商利润增长的引擎。在财务回报方面，重磅炸弹药物不仅帮助企业巩固市场地位，更推动其在研发投入、人才吸引以及全球扩张等多个方面的进一步发展。 重磅炸弹药物也代表了企业创新能力的顶峰，彰显了其在未被满足的医疗需求方面的领先地位。成功的重磅炸弹药物能够提升公司品牌形象，增强患者和医生的信任，进而在药品市场上树立不可替代的竞争壁垒。因此重磅炸弹无疑是所有制药公司"会向瑶台月下逢"的梦想。 根据Evaluate Pharma的数据，在2014年至2023年间，FDA批准的487种药物中，预计有193种药物的峰值销售额超过10亿美元，也就是符合重磅炸弹的最低标准。这也意味着大约39%的新药预计将跨入重磅炸弹的行列。在这些药物中，预计有17种药物的高峰年销售额将超过100亿美元，42种药物的高峰年销售额将超过50亿美元，114种药物的高峰年销售额将超过20亿美元。 排除与COVID相关的产品，Evaluate Pharma预计未来销售峰值前五的药物将分别是默沙东的癌症药物Keytruda（312.8亿美元）、诺和诺德的肥胖症药物Wegovy（222.2亿美元）、赛诺菲/再生元的免疫疾病药物Dupixent（200.2亿美元）、强生的多发性骨髓瘤药物Darzalex（168.7亿美元）以及艾伯维公司的自身免疫疗法Skyrizi（161亿美元）。 阿斯利康的重磅之道 尽管"道可道，非常道"，但开发重磅炸弹的征途中仍然是有迹可循的，例如阿斯利康的Farxiga（dapagliflozin）。 Farxiga于2014年1月8日前首次获得FDA批准，用于治疗2型糖尿病。上市后Farxiga迅速获得重磅头衔，2023年实现了近60亿美元的销售额，预计今年的销售额将达到74亿美元。 但Farxiga的重磅头衔依靠的不仅仅是其背后卓越的科学。在"出门俱是看花人"的医药开发领域，商业成功的背后还需要众多因素的协同作用，其中最重要的一点就是必须存在一个真正的未满足的重大需求。将一种药物商业化并使其超过10亿美元的销售额是非常困难的。在医药开发这个"譬如朝露"般的大环境下，朝生暮死而未能达到商业开发计划的新药俯仰皆是。即使是被认为具有"重磅潜力"的药物，往往也是"蝴蝶梦中家万里"，最终未能达到预期。即便开发者发现了一个新的靶点，并且针对这一全新靶点开发一种新药，但这也并不能保证这款新药在市场上获取商业成功。例如当初意气风发的靶向β淀粉样蛋白的阿尔茨海默病药物Aduhelm，最终只落得一个出师未捷身先死的结局。Aduhelm曾经被苦于无药可治的阿尔茨海默病市场寄予厚望，并被预测将在2023年实现100亿美元的销售额，但事实却与期待大相径庭，Aduhelm最终在一片争议声中黯然退市。 虽然重磅炸弹的产生似乎是"可遇而不可求"，但其背后同样存在某些共性，例如针对多个适应症，上市三年内达到重磅标准等。在多个适应症获得批准是重磅药物的一个标志，例如默沙东的药王，肿瘤免疫疗法Keytruda就是最 好的例子。Keytruda于今年9月获得了第41次批准，而且这个数字看上去还要继续增加下去。 Farxiga的重磅之路同样遵循着针对多种适应症获批的路线。例如在2014年获得2型糖尿病适应症的批准后，Farxiga又相继获得了降低2型糖尿病患者心脏衰竭（2019年10月21日）、射血分数降低的心力衰竭（2020年5月6日）、慢性肾病（2021年4月30日）、降低心血管死亡和心力衰竭住院的风险（2023年5月9日）、10岁以上儿童的血糖控制（2024年6月12日）等多次FDA监管批准。尤其是慢性肾病和心力衰竭两项批准极大地推动了Farxiga的销售额。 扩大临床研究对象，对于扩展药物的现有标签意义重大，尽管这意味着更高的投入和成本。例如在心血管疾病领域，阿斯利康超越了Farxiga的安全性试验要求，研究了更广泛的患者群体，而不仅仅是那些高风险的不良事件患者，这也最 大程度地挖掘了Farxiga的适用范围和市场广度。通过深入研究这些数据，发现了Farxiga对于心脏的益处。这使得Farxiga 2020年成为首个获得批准用于特定心力衰竭患者的SGLT2抑制剂。勃林格殷格翰和礼来的Jardiance在一年后也获得了同样的批准。 众多的临床数据之中，Farxiga的开发者在观察不同亚组的数据时，也看到了它对肾功能的影响。这方面的发现与深耕最终促使了Farxiga针对慢性肾病的批准。广泛的临床数据使得这款最初的糖尿病药物标签扩展到了肾病领域，没有前瞻性的试验设计和广泛的患者群体是很难实现这样的监管批准的。甚至Farxiga的主战场也逐渐从糖尿病朝慢性肾病和心力衰竭转移，而诺和诺德与礼来的GLP-1药物针对这两个领域的开发尚未获得里程碑式成功，这种先驱性的开拓为Farxiga赢得了更多的市场独占机遇。阿斯利康在GLP-1的强力挑战下也选择将资源集中在他们认为更有机会的领域。Farxiga的电视广告目前主要围绕慢性肾病而非糖尿病展开。 图1. Farxiga联合疗法策略 联合疗法开发对于深挖重磅药物潜力至关重要。首先，通过不同作用机制的药物协同作用，联合疗法可以显著提高治疗效果，扩大药物的适应症范围，满足多种患者需求，从而增加市场潜力。此外，联合疗法还能延缓耐药性的出现，增强疗效持久性，提升患者的长期预后。这种策略不但延长了药物生命周期，还推动其在不同疾病领域中的广泛应用，进一步提升其商业价值和市场竞争力。在瞬息万变的市场环境中，阿斯利康也在动用联合疗法的力量继续拓展Farxiga的潜力。他们正在开发三款与Farxiga联合使用的产品，均在心血管和肾脏领域。例如Farxiga与高血压候选药、醛固酮合酶抑制剂baxdrostat针对慢性肾病联合疗法（III期临床）、Farxiga与纤维化/高血压/肝硬化候选药、内皮素A受体拮抗剂zibotentan针对伴有高蛋白尿的慢性肾病的联合疗法（III期临床），以及Farxiga与慢性肾病/心力衰竭候选药、盐皮质激素受体调节剂balcinrenone针对伴有慢性肾病的心力衰竭的联合疗法（III期）（图1）。 反常规授权 在Medicare将Farxiga列为首批10种价格谈判药物之一后的几个月，阿斯利康后与Prasco合作推出了授权仿制药版本，这种提前授权的做法非常耐人寻味。 通常来说，授权重磅产品的做法发生在生命周期的末尾阶段，但阿斯利康却在仿制药出现前三年就将自己的关键产品授权出去。这一反常规举措是阿斯利康为应对价格压力和保持市场份额的战略行动。通过推出授权仿制药，阿斯利康可以在仍然保持对产品市场占有率和收入控制的情况下，提供Farxiga的低成本版本。这种方法有助于公司应对Medicare的价格谈判，并通过在低成本市场中建立立足点，可能减轻未来仿制药竞争的影响。此外，这也使阿斯利康能够在仿制药进入市场后，捕获可能会流失给仿制药竞争对手的市场份额。这也是一种"反者，道之动"的哲学思想在商战中的体现。 提前着手市场准入的筹备 获得FDA的监管审批只是成为重磅炸弹必经的一步，但这并不能保证成功，"行百里者半九十"的情形在药物开发领域已成常态。 在药品上市前，市场准入策略异常关键，它的设定将在很大程度上决定重磅炸弹的长成。药物商业化早期规划中应重点关注市场塑造、向利益相关者突出未被满足的需求和药物价值主张，尤其是在复杂的市场环境中。一些公司往往低估了市场准入的复杂性，导致药物上市后的失败。而制药业巨头不仅在追求重磅炸弹，还在追求超级重磅炸弹。但许多公司在市场准入上产生失误，导致药物上市后失去了获得批准之后的上升势头，很快陷入"兴尽而悲"的阶段。大约一半的公司在上市后失败，因为他们的市场准入策略不充分或基础薄弱。准入政策简单地说，就是要将合适的资源带给合适的人。这是一件听起来简单但一做就容易出错的事情，而且不同疾病的市场准入的筹备也不能一概而论。例如人们需要多少相关的教育？医生对于改变原有治疗方案的动力有多大？市场上的治疗惰性有多大？需要教育的目标群体有多大？他们喜欢通过什么方式接受教育？这都是一款重磅炸弹破蛹化蝶必须认真面对的问题。 前期如果没有做好这些工作，后期则很有可能反受其累。最糟糕的情况是，开发者没有合适的基础设施、没有充分的教育、没有支付方的支持就贸然进入市场，这就有可能无法领略"山顶千门次第开"的境界。 市场准入中常犯的错误是事到临头才开始准备。正常的情形是在预计FDA批准前的12至18个月就开始准备。虽然这看上去有些过于志得意满，但从成本效益角度来看确是划算而且必须的。提前进行市场准入准备的原因在于确保药物能够顺利进入市场并获得成功。这种准备涉及复杂的市场策略、定价、利益相关者教育以及支付方的支持等多方面内容。如果准备不充分或启动太晚，会导致上市后出现各种问题，例如市场推广不力、定价错误、支付方未能及时覆盖等。这些问题可能直接影响药物的销售、市场渗透和长期成功。 阿斯利康通常在II期临床阶段就开始着手准备市场准入方面的工作了。II期临床试验阶段是药物潜力逐渐显现的时期。阿斯利康在这一阶段就开始规划市场准入，反映了对药物成功上市的高度重视以及其具有前瞻性的市场战略。通过提早布局，阿斯利康可以更好地把握时间窗口，确保药物上市时的顺利推广。II期临床试验阶段的早期市场准入准备可以为药物的定价策略、保险覆盖范围以及与支付方的沟通留出充足时间，从而确保药物在上市时能够顺利纳入支付体系，减少定价失误或支付支持不足的风险。除此之外，提早准备有助于阿斯利康及时展开医生、患者和其他关键利益相关者的教育工作，逐步建立市场对新药的认知和接受度，在药物上市时迅速获得市场份额。特别是在创新药物可能改变治疗方式的情况下，提早与医生和支付方合作至关重要。 精准定价 定价也是公司孕育重磅炸弹努力过程中经常容易出错的地方，尤其是当年纷繁复杂的药物定价环境。很容易陷入"不审势即宽严皆误"的误区。 有些药物以错误的价格上市，然后开发商面对销售疲软的状况或者其它方面的压力不得不很快削减价格。例如安进的高胆固醇血症药物Evolocumab（Repatha）当初就不得不将其每年标价从14100美元降至5850美元，以应对对手的竞争和增加保险覆盖的需求。 内部预期通常是导致药物定价过高的原因。在预测资产潜力时，消除情感因素非常重要。导致公司对其内部资产估值过高的因素主要与情感和认知偏差有关。公司对自身研发的药物往往投入了大量的时间、资金和资源，因此它们在内部被视为"核心资产"。这种情感上的投入使得公司难以客观评估药物的实际市场价值，特别是在其商业潜力尚未被完全验证的早期阶段。此外，公司内部对这些药物有高度的期待，可能会忽视或低估市场上的现实挑战，例如竞争产品、支付方的接受度和医生的治疗惯性。还有一个因素是公司内部的反馈循环可能过于正向，缺乏独立或外部的客观评估。这种内向型的决策流程往往忽视了行业专家或市场分析的声音，导致资产的市场定位偏离实际需求或支付方的期望。最终，公司可能会因为高估药物的吸引力而在上市时遭遇市场接受度不足的问题。 总 结 在全球生物制药行业日益激烈的竞争中，开发重磅炸弹药物已不仅仅是技术创新的成果，更是企业实现可持续增长的战略核心。重磅药物通过精准捕捉未被满足的重大医疗需求，推动适应症拓展和联合疗法开发，成为提升市场竞争力和企业声誉的关键支柱。例如，阿斯利康的Farxiga不仅从糖尿病药物成功扩展至心力衰竭和慢性肾病领域，还通过创新商业模式应对仿制药的冲击，有效延长了其产品生命周期并稳固了市场地位。这一成功案例表明，重磅药物的开发不仅依赖于科学创新，更需要早期的市场准入准备、灵活的定价策略与深度的临床洞察相结合。未来，能够将科学突破与市场需求紧密结合的公司，将在这个快速变化的行业中获得长期的领先地位，并进一步推动全球医疗健康领域的变革与进步。 附录：超级重磅炸弹信息图 表1. 制药业巨头峰值销售额超35亿美元资产状况 表2. 阿斯利康超级重磅炸弹（峰值销售额超过35亿美元）资产状况 （点击小图看大图） 表3. 阿斯利康2024年Q1-Q3重磅炸弹销售 Ref. Dangi-Garimella, S. Amgen Announces 60% Reduction in List Price of PCSK9 Inhibitor Evolocumab. AJMC. 24. 10. 2018. Pecci, A. Want to launch a blockbuster? An AstraZeneca exec breaks it down. Pharma Voice. 16. 10. 2024. Billion Dollar Blueprint: The Journey to Blockbuster Status. Citeline. 2024. Drug launches reflect overall company performance. Deloitte. 25. 08. 2023. END 【智药研习社近期直播】 来源：CPHI制药在线 声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。 投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~