Akcea and Pfizer Inc. Announce Licensing Agreement for investigative antisense therapy AKCEA-ANGPTL3-LRx


2019-10-10 10:40:45 BioSpace


Akcea and Ionis to earn $250 million license fee BOSTON, Mass. and NEW YORK, NY, October 7, 2019 – Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and Pfizer Inc. (NYSE:PFE), today announced that the companies have entered into a worldwide exclusive licensing agreement for AKCEA-ANGPTL3-LRx, an investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases. AKCEA-ANGPTL3-LRx is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver, a key regulator of triglycerides, cholesterol, glucose and energy metabolism. AKCEA-ANGPTL3-LRx is currently being evaluated in a Phase 2 study in patients with Type 2 diabetes, hypertriglyceridemia and non-alcoholic fatty liver disease (NAFLD). “AKCEA-ANGPTL3-LRx has the potential to treat people suffering from certain cardiovascular and metabolic diseases. Given the unmet medical need for this patient population and the broad market potential, we believe Pfizer’s expertise and breadth of experience in cardiovascular and metabolic diseases makes it well suited to accelerate clinical development of AKCEA-ANGPTL3-LRx, and to deliver it to patients in need of additional therapies for these life threatening diseases,” said Damien McDevitt, Ph.D., interim chief executive officer at Akcea. “Pfizer is committed to delivering breakthrough medicines to patients with unmet medical needs,” said Mikael Dolsten, Chief Scientific Officer and President, Worldwide Research & Development and Medical, Pfizer. “AKCEA-ANGPTL3-LRx is a novel therapy that will complement our clinical mid-stage internal medicine pipeline, and we believe that our deep expertise in cardiovascular and metabolic diseases will help allow this program to reach its maximum potential for patients.” Under terms of the agreement, Akcea and Ionis will receive a $250 million upfront license fee, which will be split equally between the two companies. Akcea will settle its $125 million obligation to Ionis in Akcea common stock. The companies are also eligible to receive development, regulatory and sales milestone payments of up to $1.3 billion and tiered, double-digit royalties on annual worldwide net sales following marketing approval of AKCEA-ANGPTL3-LRx. Future milestone payments and royalties will be split equally between Akcea and Ionis. Pfizer is responsible for all development and regulatory activities and costs beyond those associated with the ongoing Phase 2 study. Prior to regulatory filing for marketing approval, Akcea has the right, at its option to participate in certain commercialization activities with Pfizer in the U.S. and certain additional markets on pre-defined terms and based on meeting pre-defined criteria. This transaction is subject to clearance under the Hart-Scott Rodino Antitrust Improvements Act and other customary closing conditions. ABOUT AKCEA-ANGPTL3-LRx AKCEA-ANGPTL3-LRx is an investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases. This antisense medicine is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver, a key regulator of triglycerides, cholesterol, glucose and energy metabolism. AKCEA-ANGPTL3-LRx was developed using Ionis’ advanced LIgand Conjugated Antisense (LICA) technology platform. The potential therapeutic benefits of ANGPTL3 reduction are supported by the discovery that people with a genetic deficiency in ANGPTL3 have reduced levels of low-density lipoprotein cholesterol (LDL-C) and triglycerides, and a decreased risk of diabetes and cardiovascular disease1. In a Phase 1/2 study, patients treated with AKCEA-ANGPTL3-LRx achieved robust, dose-dependent reductions of ANGPTL3, triglycerides, LDL-cholesterol and total cholesterol with a positive safety and tolerability profile2. AKCEA-ANGPTL3-LRx is currently being evaluated in a Phase 2 study in patients with Type 2 diabetes, hypertriglyceridemia and non-alcoholic fatty liver disease (NAFLD). AKCEA-ANGPTL3-LRx was discovered by Ionis and has been co-developed by Akcea and Ionis. ABOUT AKCEA THERAPEUTICS, INC. Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) as well as advancing a mature pipeline of novel drugs, including AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, with the potential to treat multiple diseases. All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. TEGSEDI is approved in the U.S., E.U. and Canada. WAYLIVRA is approved in the E.U. and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Boston, Massachusetts. Additional information about Akcea is available at www.akceatx.com and you can follow us on twitter at @akceatx. Pfizer Inc.: Breakthroughs that Change Patients’ Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer. AKCEA FORWARD-LOOKING STATEMENT This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and the therapeutic and commercial potential of AKCEA-ANGPTL3-LRx and other products in development. Any statement describing Akcea’s goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of AKCEA-ANGPTL3-LRx or other of Akcea’s drugs in development is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Akcea’s forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Akcea’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea’s programs are described in additional detail in Akcea’s quarterly report on Form 10-Q and annual report on Form 10-K, which are on file with the SEC. Copies of these and other documents are available from the company. In this press release, unless the context requires otherwise, “Pfizer”, “Akcea,” “Company,” “Companies,” “we,” “our,” and “us” refers to Pfizer and/or Akcea Therapeutics. Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics®, TEGSEDI® and WAYLIVRA® are trademarks of Akcea Therapeutics, Inc. PFIZER DISCLOSURE NOTICE: The information contained in this release is as of October 7, 2019. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. This release contains forward-looking information about a worldwide exclusive licensing agreement among Pfizer, Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc., and AKCEA-ANGPTL3-LRx, an investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications may be filed in any jurisdictions for AKCEA-ANGPTL3-LRx; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether AKCEA-ANGPTL3-LRx will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of AKCEA-ANGPTL3-LRx; and competitive developments. A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com. Akcea Investor Contact: Kathleen Gallagher Vice President, Corporate Communications and Investor Relations (617)-207-8509 kgallagher@akceatx.com Bill Berry Berry & Company T: 212 253-8881 bberry@berrypr.com Lynn Granito Berry & Company T: 212 253-8881 lgranito@berrypr.com Pfizer Media Relations: Patricia Kelly 212-733-3810 patricia.kelly@pfizer.com Pfizer Investor Relations: Chuck Triano 212-733-3901 charles.e.triano@pfizer.com References JAMA Cardiol. 2018 Oct 1;3(10):957-966. N Engl J Med. 2017 Jul 20;3
Akcea 和 Ionis 将获得2.5亿美元的许可费 马萨诸塞州波顿.和纽约,纽约,2019年10月7日- Akcea Therapeutics , Inc .( NASDAQ : AKCA ),一家由 Ionis Pharmaceuticals , Inc .( NASDAQ : IONS )和辉瑞(Pfizer) Inc .( NYSE : PFE )控股的子公司,今天宣布,这些公司已签订了 AKCEA-ANPTL3-LRx 全球独家许可协议,正在开发一种用于治疗某些心血管和代谢疾病患者的研究反义疗法。 AKCEA-ANPTL3-LRx 的设计是为了减少肝脏中血管生成素样蛋白3( ANPTL3)的产生,这是甘油三酯、胆固醇、葡萄糖和能量代谢的关键调节因子。AKCEA-ANGEPTL3-LRx 目前正在对2型糖尿病、高脂血症和非酒精性脂肪肝( NAFLD )患者进行第2期研究。 “ AKCEA-ANPTL3-LRx 有可能治疗某些心血管和代谢疾病患者。鉴于该患者人群尚未得到满足的医疗需求以及广阔的市场潜力,我们认为辉瑞(Pfizer)在心血管和代谢性疾病方面的专业知识和丰富的经验使其非常适合加速 AKCEA-ANPTL3-LRx 的临床开发。Akcea 的临时首席执行官 Damien McDevitt 博士说:“我们将为需要额外治疗的患者提供治疗,以治疗这些危及生命的疾病。” “辉瑞致力于向医疗需求未得到满足的患者提供突破性药物,”辉瑞(Pfizer)全球研发和医疗首席科学官兼总裁 Mikael Dolsten 说。“ AKCEA-ANPTL3-LRx 是一种新的治疗方法,它将补充我们临床中期的内科管道,我们相信,我们在心血管和代谢性疾病方面的深厚专业知识将有助于该项目实现患者的最大潜力。” 根据协议条款, Akcea 和 Ionis 将获得2.5亿美元的前期许可费,这两家公司将平分。Akcea 将偿还其在 Akcea 普通股中对 Ionis 的1.25亿美元债务。在 AKCEA-ANPTL3-LRx 上市许可后,这些公司还有资格获得高达13亿美元的开发、监管和销售里程碑付款,以及每年全球净销售额的两位数专利使用费。未来的里程碑付款和特许权使用费将在 Akcea 和 Ionis 之间平均分配。辉瑞(Pfizer)负责所有的开发和监管活动以及与正在进行的第二阶段研究相关的成本。在提交上市批准的监管文件之前, Akcea 有权根据预先确定的条款和满足预先确定的标准,选择与辉瑞(Pfizer)在美国和某些其他市场参与某些商业化活动。 根据 Hart-Scott Rodino Antitrust Improvements Act 和其他惯例成交条件,本次交易需获得批准。 关于 AKCEA-ANPTL3-LRx AKCEA-ANPTL3-LRx 是一种正在开发的反义疗法,用于治疗某些心血管和代谢性疾病患者。这种反义药物的目的是减少生产血管生成素样3( ANPTL3)蛋白质在肝脏,一个关键的调节甘油三酯,胆固醇,葡萄糖和能量代谢。AKCEA-ANPTL3-LRx 是利用 Ionis 先进的 LIgand 共轭反义( LICA )技术平台开发的。降低 ANGEPTL3的潜在治疗益处得到了以下发现的支持:在 ANGEPTL3中有遗传缺陷的人降低了低密度脂蛋白胆固醇( LDL-C )和甘油三酯的水平,降低了糖尿病和心血管疾病的风险。在第1/2期研究中,接受 AKCEA-ANPTL3-LRx 治疗的患者获得了较强的剂量依赖性降低 ANPTL3、甘油三酯、低密度脂蛋白胆固醇和总胆固醇,具有良好的安全性和耐受性。AKCEA-ANGEPTL3-LRx 目前正在对2型糖尿病、高脂血症和非酒精性脂肪肝( NAFLD )患者进行第2期研究。AKCEA-ANPTL3-LRx 是由 Ionis 发现的,由 Akcea 和 Ionis 共同开发。 关于 AKCEA THERRAPEUTICS , INC . Ionis Pharmaceuticals , Inc .下属子公司 Akcea Therapeutics , Inc .是一家生物制药公司,专注于研发和商业化治疗严重和罕见疾病患者的药物。Akcea 正在将 TEGSEDI ®( inotersen )和 WAYLIVRA ®( volaneonersen )商业化,并开发一种成熟的新药管道,包括 AKCEA-APO ( a )-LRx 、 AKCEA-ANPTL3-LRx 、 AKCEA-APOCIII-LRx 和 AKCEA-TTR-LRx ,具有治疗多种疾病的潜力。这六种药物都是由反义疗法的领导者 Ionis 发现的,并且都是基于 Ionis 的专有反义技术。TEGSEDI 在美国、欧盟和加拿大获得批准。WAYLIVRA 在欧盟获得批准,目前正处于3期临床开发阶段,用于治疗家族性部分脂肪性营养不良( FPL )患者。Akcea 正在建设全球药品商业化的基础设施。Akcea 是一家总部位于马萨诸塞州波士顿的全球性公司。有关 Akcea 的更多信息,请访问 www.akcetx.com ,您可以在@ akcetx 上关注我们。 辉瑞(Pfizer)公司:改变患者生活的突破 在辉瑞(Pfizer),我们运用科学和全球资源为那些延长和显著改善他们生活的人提供治疗。我们努力为包括创新药物和疫苗在内的医疗保健产品的发现、开发和制造制定质量、安全和价值标准。每天,辉瑞(Pfizer)的同事们都在发达国家和新兴市场推动健康、预防、治疗和治疗,挑战我们这个时代最令人担心的疾病。与我们作为全球首屈一指的创新生物制药公司之一的责任一致,我们与医疗保健提供者、政府和当地社区合作,支持并扩大全球范围内获得可靠、负担得起的医疗保健的机会。150多年来,我们一直努力为所有依赖我们的人带来改变。我们经常在网站 www.pfizer.com 上发布对投资者可能很重要的信息。此外,为了了解更多信息,请访问 www.pfizer.com ,并在 Twitter 上关注我们,网址是@ Pfizer 和@ Pfizer _ News , LinkedIn , YouTube ,以及 Facebook 上的我们。com / Pfizer 。 AKCEA FORWARD-LOOKING 报表 本新闻稿包括有关 Akcea Therapeutics , Inc .业务的前瞻性陈述,以及 AKCEA-ANPTL3-LRx 及其他正在开发的产品的治疗和商业潜力。任何描述 Akcea 的目标、期望、财务或其他预测、意图或信念的声明,包括 AKCEA-ANPTL3-LRx 或 Akcea 正在开发的其他药物的商业潜力,都是前瞻性声明,应视为风险声明。此类声明存在一定的风险和不确定性,特别是在发现、开发和商业化药物过程中所固有的风险和不确定性,这些药物安全有效地用于人类治疗,并努力围绕这些药物建立业务。Akcea 的前瞻性陈述还涉及假设,如果这些假设从未实现或证明正确,可能导致其结果与这些前瞻性陈述所表达或暗示的结果存在重大差异。尽管 Akcea 的前瞻性陈述反映了其管理层的诚信判断,但这些陈述仅基于 Akcea 目前已知的事实和因素。因此,请注意不要依赖这些前瞻性陈述。这些风险和其他与 Akcea 计划有关的风险在 Akcea 关于10-Q 表格的季度报告和10-K 表格的年度报告中有更详细的描述,这些报告和其他文件的副本可从公司获得。 在本新闻稿中,除非上下文另有要求,“辉瑞”、“ Akcea ”、“公司”、“公司”、“我们”、“我们”和“我们”指辉瑞(Pfizer)和/或 Akcea Therapeutics 。 Ionis Pharmaceuticals ™是 Ionis Pharmaceuticals , Inc .的商标。 Akcea Therapeutics ®、 TEGSEDI ®和 WAYLIVRA ®是 Akcea Therapeutics , Inc .的商标。 PFIZER 披露公告:本新闻稿所载信息截至2019年10月7日。辉瑞(Pfizer)不承担因新信息或未来事件或发展而更新本新闻稿中包含的前瞻性声明的义务。 本新闻稿包含辉瑞(Pfizer)、 Akcea Therapeutics , Inc .和 Ionis Pharmaceuticals , Inc .与 AKCEA-ANPTL3-LRx 之间的全球独家许可协议的前瞻性信息, AKCEA-ANGEL3-LRx 是一种正在开发的用于治疗某些心血管和代谢性疾病患者的研究反义疗法,包括其潜在益处。这涉及实质性风险和不确定性,可能导致实际结果与此类声明所表达或暗示的结果存在重大差异。风险和不确定性包括(其中包括)研发固有的不确定性,包括满足预期临床终点、临床试验的开始和/或完成日期、监管提交日期、监管批准日期和/或启动日期的能力。以及不利的新临床数据和对现有临床数据的进一步分析的可能性;临床试验数据受到监管机构不同解释和评估的风险;监管机构是否会对我们的临床研究的设计和结果感到满意;是否和何时可以在任何司法管辖区提出 AKCEA-ANPTL3-LRx 的药物申请;是否和何时可以批准任何此类申请,这将取决于无数因素,包括确定产品的利益是否超过其已知的风险,以及确定产品的有效性,如果批准, AKCEA-ANPTL3-LRx 是否会在商业上成功地影响监管机构的决策;通过对标签程序产生影响可能影响 AKCEA-ANPTL3-LRx 的可用性或商业潜力的安全和/或其他事项;以及竞争发展。 有关风险和不确定性的进一步说明,请参见辉瑞(Pfizer)截至2018年12月31日止财政年度的10-K报表表格年度报告及其后续10-Q 表格报告,包括其标题为“风险因素”和“可能影响未来业绩的前瞻性信息和因素”的章节。以及表格8-K 的后续报告,所有这些报告都提交给美国证券交易委员会,可在 www.sec.gov 和 www.pfizer.com 上查阅。 Akcea 投资者联系人: Kathleen Gallagher 公司传讯及投资者关系副总裁 (617)-207-8509 kglallagher @ akcetx.com 比尔·贝瑞 Berry & Company T :21253-8881 bbery @ berypr.com Lynn Granito Berry & Company T :21253-8881 lgranito @ berrypt.com 辉瑞(Pfizer)媒体关系: Patricia Kelly 212-733-3810 帕特丽夏。kelly @ pfizer.com 辉瑞(Pfizer)投资者关系: 查克·特里亚诺 212-733-3901 charles.e.triano @ pfizer.com 参考文献 卡地尔.2018年10月1日;3(10): 英国医学博士.2017年7月20日;3