Heart Failure iPSC-Derived Therapy Trial Doses First Patient

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Based on 4-week follow-up data from the first patient, an independent safety evaluation committee approved the trial’s continuation.

The first patient has been dosed in the phase 1/2 LAPiS clinical trial (NCT04945018), which is evaluating Heartseed and Novo Nordisk’s HS-001, an investigational allogeneic cell therapy, for the treatment of advanced heart failure.1

HS-001 is comprised of induced pluripotent stem cell (iPSC) derived cardiomyocyte spheroids which are intended to restore heart muscle and function through remuscularization and neovascularization. The cells are transplanted with a special administration needle and guide adapter to the diseased heart tissue via open-heart surgery along with a coronary artery bypass graft procedure. Heartseed noted that in preclinical research, iPSC-derived cardiomyocytes transplanted with gelatin hydrogel demonstrated the ability to improve heart function in a rodent model of heart failure.2 The company also announced that based on analysis of 4-week follow-up data from the first patient treated with HS-001, an independent safety evaluation committee approved the continuation of the trial.1

"As a cardiologist, I have been working for many years to realize cardiac regeneration medicine in order to treat patients with heart failure whose heart function has deteriorated to the point where they are unable to live their daily lives,” Keiichi Fukuda, chief executive officer, Heartseed, said in a statement regarding the news.1 “To achieve this goal, we established Heartseed and have continued our development with patient safety and benefit as our top priority. We are very pleased to announce that the first transplant of HS-001 has been successfully performed, which is a major first step in establishing a completely new treatment that I have long envisioned. I would like to express our deepest gratitude to all those who have contributed to this project."

The open-label, multicenter dose escalation trial is expected to enroll 10 patients aged 20 years to 80 years with severe heart failure with underlying ischemic heart disease. Participants are required to have a resting left ventricular ejection fraction (LVEF) less than or equal to 40% and a New York Heart Association (NYHA) cardiac function classification of grade 2 or higher at screening. Patients who are screened less than 1 month after the onset of myocardial infarction and patients with congenital heart disease or cardiac sarcoidosis will be excluded from the study. Additional inclusion and exclusion criteria are noted to exist.

Half of the participants in the trial will receive the study's low dose of HS-001 and half will receive the study’s high dose. After HS-001 transplantation, participants will receive immunosuppressants. The trial’s primary end point is the incidence of adverse events in the 26 weeks following treatment. Secondary end points include LVEF, myocardial wall motion, myocardial blood flow, myocardial viability, 6-minute walk distance, the Kansas City Cardiomyopathy Questionnaire, 5-level EQ-5D version, and an N-terminal Pro-brain Natriuretic Peptide test. The study is recruiting at multiple locations in Japan. It has an estimated completion date of March 31, 2024.

"Cell therapy holds the promise to offer truly disease-modifying treatments for people living with serious chronic disease.," Jacob Sten Petersen, corporate vice president of Cell Therapy R&D, Novo Nordisk, added to the statement.1 "We are very pleased that our partners from Heartseed have begun clinical testing of this innovative approach to treating heart failure. We aim to build on Heartseed's pioneering work in Japan and hope to bring a transformative treatment to people living with heart failure across the globe."

REFERENCES
1. Heartseed and Novo Nordisk announce first patient dosed in clinical study with HS-001 - a cell therapy designed to restore heart function in people with advanced heart failure. News release. Heartseed Inc. February 10, 2023. Accessed February 13, 2023. https://heartseed.jp/en/news/content.html#_edn5
2. Kawaguchi S, Soma Y, Nakajima K, et al. Intramyocardial transplantation of human ips cell–derived cardiac spheroids improves cardiac function in heart failure animals. JACC: Basic to Translational Science. 2021;6(3):239-254. doi: 10.1016/j.jacbts.2020.11.017

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