Results of joint research with the University of Tokyo published in the international scientific journal “Nature Communications”

2023.11.22

Axcelead Drug Discovery Partners Inc. (Headquarters: Fujisawa City, Kanagawa Prefecture; President & CEO: Nobuhiko Yamada; hereinafter “Axcelead DDP”) announces that the research results on the discovery of absorption pathways for essential nutrient “Choline” through a joint research conducted with Associate Professor Hisamitsu Hayashi and his research team at Graduate School of Pharmaceutical Sciences of University of Tokyo have been published in the international scientific journal “Nature Communications” released on November 21, 2023.

Associate Professor Hisamitsu Hayashi and his research team have been researching to overcome hepatic diseases in children. One of these diseases, progressive familial intrahepatic cholestasis type 1 (PFIC1), is a rare disease for which the causal gene has been found, but its pathogenic mechanism and fundamental treatment are unknown. To discover the mechanism, a metabolomic analysis involving lipid molecules was conducted at Axcelead DDP on samples from PFIC1 animal models and patients provided by the University of Tokyo. As a result, we found that PFIC1 could cause malabsorption of lysophosphatidylcholine, a precursor of the essential nutrient choline, in patients, followed by a deficiency of choline itself, leading to disease development. Furthermore, the University of Tokyo research team confirmed that oral choline supplementation in PFIC1 animal models eliminated liver symptoms. These results indicated that choline supplementation could be a treatmenteutic potential for PFIC1. Axcelead DDP is currently supporting the University of Tokyo research team to prepare for its clinical trial.

Masayuki Ii, PhD, CSO of Axcelead DDP, said ”We are very pleased to have achieved results leading to the development of a new medical treatment for PFIC1 through our joint research with the University of Tokyo. We believe that technologies for analysis of disease mechanism that Axcelead DDP has developed in its drug discovery to date has contributed to this achievement. We will continue to support the University of Tokyo research team to bring new treatments to patients as soon as possible by leveraging Axcelead DDP’s knowledge of more than 100 IND applications.”

About Axcelead DDP
Axcelead DDP is the first integrated drug discovery solutions provider in the pharmaceutical industry in Japan, having succeeded the drug discovery research capabilities of Takeda Pharmaceutical Company Limited and started its business in July, 2017. The company provides integrated services, from discovery of drug targets to optimization of small- and medium-molecule drug candidates, in which the company has particular expertise, and to the process of bridging the gap to clinical development.