Research Triangle Park, N.C., Jan. 05, 2024 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that it is initiating GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase 2 trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF).

北卡罗来纳州三角研究园，2024年1月5日（环球通讯社）--Asklepios BioPharmaceutical，Inc.（AskBio），一家基因治疗公司，作为拜耳公司的全资子公司独立运营，今天宣布它正在启动GenePHIT（基因磷酸酶抑制疗法），这是AB-1002（也称为NAN-101）治疗充血性心力衰竭（CHF）的2期临床试验。

GenePHIT is an adaptive, double-blind, placebo-controlled, randomized, multicenter trial to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy classified as New York Heart Association (NYHA) Class III Heart Failure who have been medically stable for at least 4 weeks.1 The advancement of AB-1002 into Phase 2 marks a significant milestone for this novel gene therapy for CHF and, if successful, could bring this important investigational therapy one step closer to treating patients with high unmet medical need.

GenePHIT是一种适应性，双盲，安慰剂对照，随机，多中心试验，评估单次冠状动脉内输注AB-1002对被归类为纽约心脏协会（NYHA）III级心力衰竭的成年人的安全性和有效性，这些患者在医学上稳定了至少4周。AB-1002进入第二阶段标志着这种新型CHF基因治疗的一个重要里程碑，如果成功，可以使这一重要的研究性治疗更接近治疗未满足医疗需求的患者。

GenePHIT will include between 90 and 150 adults with left ventricle ejection fraction between 15 and 35%, who continue to suffer from heart failure symptoms despite guideline recommended therapy. The primary efficacy endpoint at 52 weeks is a modified win ratio of several clinically meaningful assessments.1 “AskBio is excited to initiate GenePHIT under the leadership of Roger Hajjar, MD, Scientific Chair CHF, and Lothar Roessig, MD, Integrated Product Team Lead CHF,” said Jude Samulski, PhD, Co-Founder and Chief Scientific Officer, AskBio.

GenePHIT将包括90至150名左心室射血分数在15%至35%之间的成年人，尽管指南推荐治疗，他们仍继续患有心力衰竭症状。52周时的主要疗效终点是几项临床上有意义的评估的改进获胜率。AskBio联合创始人兼首席科学官Jude Samulski博士说：“AskBio很高兴在CHF科学主席Roger Hajjar和CHF综合产品团队负责人Lothar Roessig的领导下启动GenePHIT。”。

“We believe this trial will help determine the potential of AB-1002 as a treatment for one of the world’s most devastating diseases, and we look forward to learning more about this important investigational cardiac gene therapy. Our hope is that one day AB-1002 will potential.

“我们相信这项试验将有助于确定AB-1002作为世界上最具破坏性的疾病之一的治疗潜力，我们期待着更多地了解这一重要的研究性心脏基因疗法。我们希望有一天AB-1002将具有潜力。

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R. Jude Samulski, PhD

R、 Jude Samulski博士