Retrotope Appoints Rick E Winningham as Chairman of Board of Directors in Advance of Key Clinical Trial Data Readouts

Retrotope在关键临床试验数据读出之前任命Rick E Winningham为董事会主席

2021-02-12 16:46:42 BioSpace

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Data Readout from Potentially Pivotal Phase 2/3 INAD Trial Expected 1H ‘01; Phase 2 ALS and Pivotal Phase 2/3 Friedreich’s Ataxia Trial Readouts Expected by Year-End LOS ALTOS, Calif., Feb. 11, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced the appointment of Rick E Winningham as chairman of the company’s board of directors. Mr. Winningham, who currently serves as chairman and chief executive officer (CEO) of Theravance Biopharma, Inc., brings more than three decades of drug discovery, development, regulatory and commercialization experience in the global biopharmaceutical industry to his role as chairman. A member of the board of directors of the Biotechnology Industry Organization (BIO), his addition provides Retrotope with access to valuable leadership and expertise as it continues its transformation into a late clinical-stage drug development company with multiple clinical trial data readouts anticipated in 2021. Mr. Winningham has served as chairman of the board of Theravance Biopharma (NASD: TBPH), a biopharmaceutical company focused on organ-selective medicines, since 2013, and the company’s CEO since its spin-off from Theravance, Inc. (renamed Innoviva, Inc.) in 2014. Starting in 2001, and up until the Theravance Biopharma spin-off, he served as CEO of Theravance, Inc., where he was also chairman of the board from 2010 to 2014. From 1997 to 2001, Mr. Winningham was president of Bristol-Myers Squibb’s Oncology/Immunology/Oncology Therapeutics Network (OTN), while also serving as the company’s president of global marketing from 2000 to 2001. Over a fifteen-year period beginning in 1986 with BMS and its predecessor, Bristol-Myers, Mr. Winningham held various U.S. and global management positions. Mr. Winningham has led the global development and commercialization of medicines across multiple therapeutic areas including oncology, cardiovascular, anti-infective, antiviral, respiratory, gastrointestinal and neuroscience. “I am excited to join the Retrotope board as it transitions into a late clinical-stage biopharmaceutical company. By leveraging one of the most unique research platforms in the industry in pursuit of reducing cellular degeneration, Retrotope has created a broad pipeline of promising first-in-class programs aimed at some of the most serious and underserved degenerative diseases of our time, including orphan neurodegenerative conditions with no treatment options at all,” said Mr. Winningham. “2021 promises to be an important year with significant clinical progress as the company reads out data from three studies of its lead development candidate RT001. Additionally, Retrotope continues to make excellent progress advancing its second development candidate, RT011, toward the clinic as a potential treatment for dry age-related macular degeneration.” Retrotope’s novel drug discovery and development platform is focused on combatting the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). While all healthy human tissues undergo this physiological process of cell degeneration and repair, it is well-established that a wide range of serious degenerative diseases are precipitated when the LPO process becomes out of balance. Polysaturated fatty acids (PUFAs), which make up cell and mitochondrial membranes throughout the body and are vital to healthy cellular function, are the target of the LPO process due to their inherent instability. Free radicals in the body exploit the instability of PUFAs to trigger chain reactions that drive LPO hyperactivity. Retrotope’s novel platform technology is designed to stabilize PUFAs and down-regulate LPO in order to protect membranes from degeneration. Retrotope has created a pipeline of first-in-class new chemical entity (NCE) drug candidates that are being developed for a number of diseases ranging from orphan neurodegenerative indications to large market degenerative diseases, all of which are triggered or accelerated by LPO. RT001, the company’s lead development candidate, is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) that has been safely administered to more than 100 patients, spanning more than 1,000 patient months. The company’s second development candidate, RT011, is an isotopically stabilized, synthetic docosahexaenoic acid (DHA) that is advancing toward clinical development. RT001 Clinical Program Highlights: Infantile Neuroaxonal Dystrophy (INAD) – The most advanced clinical program in the company’s pipeline, Retrotope has completed dosing and expects data to read out from its potentially pivotal Phase 2/3 trial of RT001 in the first half of 2021. INAD is an ultra-rare, fatal, infantile genetic neurological disorder that is eligible for Rare Pediatric Disease (RPD) designation and a RPD voucher from the U.S. Food and Drug Adminstration (FDA). Friedreich’s Ataxia (FA) – Enrollment was completed in the ongoing pivotal Phase 2/3 trial of RT001 in FA in late 2020. Data from this trial is expected to read out by the end of 2021. Proof-of-concept for RT001 in FA was demonstrated in the company’s previously completed Phase 1/2 placebo-controlled trial. FA is a rare, debilitating, life-shortening pediatric neurodegenerative disease. Like INAD, FA qualifies for RPD designation and a RPD voucher from FDA. Amyotrophic Laterial Sclerosis (ALS) – A Phase 2 clinical trial of RT001 is planned in patients with ALS, with first patient dosing expected in the first quarter of 2021 and data read out anticipated by the end of 2021. This trial is being informed by the company’s single-arm expanded access program study for RT001 in ALS, which treated 18 patients for up to 24 months and demonstrated a slowing of disease progression when compared to historical placebo responses from a separate ALS clinical trial. Progressive Supranuclear Palsy (PSP) – A Phase 2 clinical trial of RT001 is planned in patients with PSP, with first patient dosing expected in the first half of 2021 and data read out anticipated in 2022. To date, RT011 has been administered to three PSP patients through an expanded access program, with all having received treatment for more than 27 months. For all three of these patients, disease progression significantly slowed or reversed at 12 months and 24 months according to standard severity scales used in the measurement of the disease. RT011 Preclinical Program Highlights: Dry Age-Related Macular Degeneration (AMD) – Retrotope expects to file an Investigational New Drug (IND) application for RT011 in mid-2021 and initiate first-in-human clinical studies in dry AMD soon thereafter. Unlike most other dry AMD treatments in development, RT011 is an oral drug, offering key administration advantages. Promising preclinical results have suggested the ability of RT011 to protect retinal cells and preserve photoreceptors in various AMD-related animal models. Dry AMD is an early stage of AMD prior to a patient’s advancement to wet AMD and the ultimate onset of vision loss. It affects more than 7.5 million Americans and more than 20 million adults in the western world. Treatments for this condition are limited and there are no approved oral therapeutic options, providing a significant market opportunity for RT011. About Retrotope Retrotope is a clinical-stage biopharmaceutical company focused on the development of first-in-class therapies for degenerative diseases ranging from orphan neurodegenerative indications to large market degenerative conditions. The company leverages its proprietary drug discovery platform to create novel, disease-modifying drugs designed to combat the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). It does so through the creation of isotopically stabilized synthetic versions of polysaturated fatty acids (PUFAs) that trigger the downregulation of the LPO process. The company’s lead development candidate, RT001, is in clinical development for a range of orphan neurodegenerative diseases including infantile neuroaxonal dystrophy (INAD), Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and progressive supranuclear palsy (PSP). In addition, the company is advancing its second development candidate, RT011, toward the clinic for the treatment of dry age-related macular degeneration (AMD). For more information, please visit www.retrotope.com.
预计1h'01可能关键的2/3阶段INAD试验的数据读出;第二阶段ALS和关键的2/3阶段Friedreich共济失调试验的读数预计在年底 加利福尼亚州洛斯阿尔托斯,2021年2月11日(环球新闻通讯社)--专注于开发治疗退行性疾病的新型一流疗法的临床阶段生物制药公司Retrotope今天宣布任命Rick E Winningham为公司董事会主席。Winningham先生目前担任Theravance Biopharma,Inc.的董事长兼首席执行官(CEO),他在全球生物制药行业拥有30多年的药物发现、开发、监管和商业化经验。作为生物技术工业组织(BIO)董事会成员,他的加入为Retrotope提供了宝贵的领导能力和专业知识,使其能够继续转型为一家后期临床阶段药物开发公司,预计将于2021年获得多项临床试验数据。 Winningham先生自2013年起担任专注于器官选择性药物的生物制药公司Theravance Biopharma(NASD代码:TBPH)的董事会主席,并自2014年从Theravance公司(更名为Innoviva公司)分拆出来后担任首席执行官。从2001年开始,直到分拆出Theravance Biopharma,他担任Theravance,Inc.的首席执行官,2010年至2014年,他还担任该公司的董事会主席。1997年至2001年,Winningham先生担任百时美施贵宝肿瘤学/免疫学/肿瘤治疗学网络(OTN)总裁,同时在2000年至2001年担任公司全球营销总裁。从1986年开始,在百时美公司及其前身百时美公司的15年时间里,温宁汉姆先生担任过各种美国和全球管理职位。Winningham先生领导了包括肿瘤、心血管、抗感染、抗病毒、呼吸、胃肠和神经科学在内的多个治疗领域的药物的全球开发和商业化。 “我很高兴能加入Retrotope董事会,因为它正在向一个晚期临床阶段的生物制药公司过渡。通过利用业界最独特的研究平台之一来寻求减少细胞退化,Retrotope已经创建了一系列有前途的一流项目,针对我们这个时代的一些最严重和服务不足的退行性疾病,包括完全没有治疗选择的孤儿神经退行性疾病,“Winningham先生说。“2021年有望成为重要的一年,随着该公司从其主导开发候选RT001的三项研究中读出数据,将取得重大临床进展。此外,Retrotope继续取得出色进展,将其第二个开发候选产品RT011推向临床,作为干性年龄相关性黄斑变性的潜在治疗手段。“ Retrotope的新药研发平台专注于对抗由脂质过氧化(LPO)引起的氧化应激和细胞变性。虽然所有健康的人体组织都经历了细胞退化和修复的生理过程,但众所周知,当LPO过程失去平衡时,就会导致广泛的严重退化性疾病。多饱和脂肪酸(Polysaturated fatty acids,PUFAs)是构成细胞和线粒体膜的重要物质,对健康的细胞功能至关重要,但由于其固有的不稳定性,是LPO过程的靶点。体内的自由基利用PUFAs的不稳定性引发连锁反应,从而驱动LPO过度活跃。Retrotope的新型平台技术旨在稳定PUFAs和下调LPO以保护膜不退化。 Retrotope已经建立了一个一流的新化学实体(NCE)候选药物的管道,这些药物正在开发用于多种疾病,从孤儿神经退行性适应证到大型市场退行性疾病,所有这些疾病都是由LPO触发或加速的。RT001,该公司的领先开发候选产品,是一种临床阶段的同位素稳定的合成亚油酸(LA),已安全地给100多名患者服用,时间跨度超过1000个患者月。该公司的第二个开发候选产品RT011,是一种同位素稳定的合成二十二碳六烯酸(DHA),正在向临床开发迈进。 RT001临床课程要点: 婴儿神经轴突营养不良症(INAD)--该公司正在进行的最先进的临床项目,Retrotope已经完成了剂量分配,并预计在2021年上半年从RT001的潜在关键的2/3期试验中读出数据。 INAD是一种极罕见、致命的婴儿遗传性神经疾病,符合美国食品和药物管理局(FDA)的罕见儿科疾病(RPD)指定和RPD凭单。 Friedreich's共济失调(FA)-在2020年末FA正在进行的RT001关键阶段2/3试验中完成了登记。这项试验的数据预计将在2021年底读出。在公司之前完成的1/2期安慰剂对照试验中演示了FA中RT001的概念验证。 FA是一种罕见的、使人衰弱、缩短生命的小儿神经退行性疾病。与INAD一样,FA有资格获得FDA的RPD指定和RPD凭证。 肌萎缩性侧索硬化症(ALS)-计划在ALS患者中进行RT001的2期临床试验,预计2021年第一季度首次给药,预计2021年底读出数据。该试验是由该公司的单臂扩展进入计划研究在ALS中的RT001提供的,该研究治疗了18名患者长达24个月,与单独的ALS临床试验的安慰剂反应相比,显示疾病进展减缓。 进行性核上性麻痹(PSP)-计划在PSP患者中进行RT001的2期临床试验,预计2021年上半年首次给药,预计2022年读出数据。到目前为止,RT011已经通过一个扩大的获取计划应用于三名PSP患者,他们都接受了超过27个月的治疗。对于所有这三个患者,根据用于测量疾病的标准严重程度,疾病进展在12个月和24个月时显著减缓或逆转。 RT011临床前计划要点: 干性年龄相关性黄斑变性(AMD)-Retrotope预计将于2021年中期提交一份针对RT011的研究性新药(IND)申请,并在此后不久启动干性AMD的首次人体临床研究。与大多数其他正在开发中的干性AMD治疗不同,RT011是一种口服药物,提供了关键的给药优势。有前途的临床前研究结果表明,RT011在各种AMD相关动物模型中具有保护视网膜细胞和保存光感受器的能力。 干性AMD是AMD的早期阶段,在患者进展为湿性AMD和最终视力丧失之前。它影响着750多万美国人和西方世界2000多万成年人。这种情况的治疗是有限的,并且没有经批准的口服治疗选择,这为RT011提供了一个重要的市场机会。 关于Retrotope Retrotope是一家临床阶段的生物制药公司,专注于开发一流的退行性疾病疗法,治疗范围从孤儿神经退行性指征到大型市场退行性疾病。该公司利用其专有的药物研发平台,创造出新的、可改变疾病的药物,旨在对抗由脂质过氧化(LPO)引起的氧化应激和细胞退化。它通过制造同位素稳定的合成型多饱和脂肪酸(PUFAs)来实现这一目的,这些合成型多饱和脂肪酸触发LPO过程的下调。该公司的主要开发候选产品RT001正在临床开发一系列孤儿神经退行性疾病,包括婴儿神经轴索营养不良(INAD)、弗里德赖希共济失调(FA)、肌萎缩侧索硬化症(ALS或卢格里格病)和进行性核上性麻痹(PSP)。此外,该公司正在推进其第二个开发候选者RT011,朝着治疗干性年龄相关性黄斑变性(AMD)的临床方向发展。 欲了解更多信息,请访问www.retrotope.com。

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