PRESS RELEASE
Metcela Resumes Phase 3 Clinical Trials of Cell Product JRM-001 for Patients with Functional Single Ventricle | Metcela Inc.
Metcela Inc. is pleased to announce that on January 31, 2025, we submitted and received acceptance for a clinical trial amendment notification to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan, following a quality consultation. This amendment allows us to resume the pivotal trial of JRM-001, an autologous cardiac stem cell (CSC) therapy for patients with functional single ventricle.
■Phase 3 Clinical Trial
Phase 3 trial of JRM-001 is designed to evaluate the efficacy and safety of an autologous cell product composed of CSCs for treating pediatric patients with functional single ventricle. This trial is based on two previous clinical studies (Phase 1: TICAP, Phase 2: PERSEUS) conducted by a research group at Okayama University and serves as a pivotal clinical trial for regulatory approval and commercialization.
JRM-001 has been selected for the Startup Type (ViCLE) of the Cyclic Innovation for Clinical Empowerment (CiCLE) by the Japan Agency for Medical Research and Development (AMED) (press release ). It is also designated under the Sakigake Designation System for Regenerative Medicine Product and as an Orphan Regenerative Medicine Product.
■ Resumption of This Clinical Trial
Japan Regenerative Medicine Co., Ltd., which merged with Metcela in 2022, initially submitted the clinical trial notification and began the trial in 2016. However, challenges were identified during the study, including interpatient variability in cell proliferation and the large volume of patient’s blood required for manufacturing. These issues required significant revision of the manufacturing process, leading to a temporary suspension of patient enrollment.
Following that, Metcela has repeatedly manufactured JRM-001 using cardiac tissues from multiple medical institutions under the same conditions as the clinical trial. Additionally, process verification studies were conducted by our contract manufacturing partner, establishing a robust manufacturing process.
Following a successful quality consultation with the PMDA and agreement on the revised manufacturing process, Metcela has submitted the trial amendment and patient enrollment is now underway.
To ensure a stable supply of JRM-001, Metcela has entered into a manufacturing agreement with Japan Tissue Engineering Co., Ltd., a pioneering regenerative medicine company with the most commercialized regenerative medicine products in Japan. This partnership will facilitate the reliable production of autologous regenerative therapy.