Applied Therapeutics to Host Virtual Rare Disease Forum - Mar 16, 2021


2021-03-16 21:30:07 BioSpace


NEW YORK, March 16, 2021 -- Applied Therapeutics, Inc. , a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced it will host a Virtual Rare Disease Forum on Tuesday, March 23rd, 2021 from 1:00pm to 3:00pm ET. The event will highlight Applied Therapeutics’ development programs in Galactosemia, SORD Deficiency, and PMM2-CDG. The agenda for the event will include: Update on ACTION-Galactosemia Clinical Development Program Overview of SORD Deficiency, presented by Stephan L. Züchner, MD, PhD, Professor and Department Chair of Human Genetics at the University of Miami Miller School of Medicine Update on SORD Deficiency Clinical Development Plans Overview of PMM2-CDG, presented by Joseph Muenzer, MD, PhD, Professor of Pediatric Genetics and Metabolism at University of North Carolina Children’s Research Institute Update on PMM2-CDG Clinical Development Program Plans “We’re excited to host this event to spotlight our clinical development programs in Galactosemia, SORD and PMM2-CDG,” said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. “We believe that AT-007 represents an important advancement for patients with these rare diseases, and our clinical development programs offer a unique opportunity to meaningfully impact patients’ lives.” An audio webcast of the presentation will be available live. More details will be available at leading up to the event. An archived version of the presentation will be available following the event. About Applied Therapeutics Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company’s lead drug candidate, AT-007, is a novel central nervous system penetrant aldose reductase inhibitor (ARI) for the treatment of Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2 clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced full data from the trial in April 2020. A pediatric Galactosemia study commenced in June 2020. The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications. To learn more, please visit and follow the company on Twitter @Applied_Tx. Investors: Maeve Conneighton (212) 600-1902 or Media:
2021年3月16日,纽约--应用治疗公司,一家临床阶段的生物制药公司,正在开发一系列针对高未满足医疗需求的有效分子靶点的新型候选药物,今天宣布,它将于2021年3月23日星期二下午1:00-3:00主办一个罕见病虚拟论坛。本次活动将重点介绍应用治疗公司在半乳糖血症、SORD缺乏症和PMM2-CDG方面的开发项目。活动议程包括: 行动-半乳糖血症临床发展计划最新进展 迈阿密大学米勒医学院人类遗传学系主任、教授Stephan L.Züchner博士介绍了SORD缺乏症的概况 SORD缺乏症临床发展计划的最新进展 PMM2-CDG概述,由北卡罗来纳大学儿童研究所儿科遗传学和新陈代谢教授Joseph Muenzer博士介绍 PMM2-CDG临床发展计划的更新 应用治疗学委员会创始人、首席执行官兼主席Shoshana Shendelman博士说:“我们很高兴主办这次活动,以突出我们在半乳糖血症、SORD和PMM2-CDG方面的临床开发项目。“我们认为,AT-007代表了这些罕见疾病患者的一个重要进步,我们的临床开发项目提供了一个独特的机会,对患者的生活产生有意义的影响。” 演示的音频网播将提供现场直播。更多详情请访问。活动结束后将提供演示文稿的存档版本。 关于应用治疗学 Application Therapeutics是一家临床阶段的生物制药公司,开发针对有效分子靶点的新型候选药物,以适应高未满足的医疗需求。该公司的主要候选药物AT-007,是一种新的中枢神经系统渗透醛糖还原酶抑制剂(ARI),用于治疗半乳糖血症,一种罕见的儿科代谢病。该公司于2019年6月启动了一项关键的1/2期临床试验,2020年1月在成人半乳糖血症患者中读出阳性的顶线生物标志物数据,2020年4月公布了该试验的全部数据。2020年6月开始了一项儿童半乳糖血症研究。该公司还在开发AT-001,这是一种新型强效ARI,用于治疗糖尿病心肌病或DbCM(一种致命的心脏纤维化)。本公司于2019年9月在DbCM启动第三阶段注册研究。临床前管道还包括AT-003,一种设计用于口服时穿过眼后的ARI,用于糖尿病视网膜病变的治疗,以及用于孤儿肿瘤适应症的新型双PI3k抑制剂的临床前开发。 欲了解更多信息,请访问,并在Twitter@applied_tx上关注该公司。 投资者: 梅夫·康尼伯顿 (212)600-1902或 媒体: 电子邮件