Drug Development Support Program

Outline of the Program

KOINOBORI provides tangible support for new drug marketing authorization in Japan by bridging various gaps from the exploration phase to the development phase of drug discovery, to establish effective therapies as soon as possible.

1. Exploration of promising developmental compounds and therapies

We investigate and analyze research and development trends, related papers, patents, and business information, and other literature, that relate to compounds under clinical development for mitochondrial diseases overseas. In addition, we investigate compounds and therapies that have been shown to be applicable to mitochondrial disease. By doing this, we explore which compounds we will give support for further research (referred to as our ‘supported compounds’).

2. Support for clinical developmental work

To promote research and development, we provide practical support for promising supported compounds in clinical trials and collaborative research of mitochondrial diseases.

3. Support for research and development costs

We support the research and development costs of supported compounds by introducing them to investors or pharmaceutical companies. KOINOBORI supports compounds that are considered more promising by offering direct financing.

Actual Activities

Supoprt of EPI-743   edison logo

We promoted the start of the clinical study of supported compound EPI-743 in Japan.

To provide tangible support for drug development for mitochondrial encephalomyopathy (MELAS), KOINOBORI conducted research on superior candidate compounds based on information from research institutions, including pharmaceutical companies, biotech start-up companies, and universities around the globe, and then focused on EPI-743. Edison Pharmaceuticals (Edison) is advancing the clinical development of EPI-743 in the U.S. and Europe for clinical use in the treatment of Leigh's disease and Friedreich's Ataxia, among other diseases, and we approached Edison for cooperation.

At the outset of the EPI-743 clinical study at the National Center of Neurology and Psychiatry (NCNP), one of the nation’s central facilities that develops treatment for and diagnoses mitochondrial diseases, KOINOBORI supported the smooth assembly of a partnership between Edison and NCNP, as well as one between doctors and researchers. As a result, we initiated the early exploratory clinical trial of EPI-743 for MELAS (UMIN000010783). We also provided financial support for this clinical study, from the funds raised by the members, by paying a part of the cost to Edison as a subsidy.

T Trial ID UMIN000010783
Registration date 2013/05/23
Official scientific title of the study Exploratory Clinical Trial of EPI-743 in Japanese MELAS Syndrome Patients
Condition MELAS (mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes)
National Center of Neurology and Psychiatry, Japan
Public contact National Center Hospital, National Center of Neurology and Psychiatry UMIN (University Hospital Medical Information Network)

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