Our Story

EnePharma Inc., a small clinical-stage drug development company, was incorporated in Delaware in June, 2019 as a US subsidiary of StaGen Co., Ltd.  (Japan).  The company founder, Dr. Naoyuki Kamatani is an expert in statistical and bioinformatics analysis of the associations between genes and diseases. Among numerous publications, he has authored 34 Nature and Nature Genetics papers.  Based on that expertise, he has developed a combination drug to enhance cellular ATP (energy) for treating various diseases that are related to energy dysfunction, including Parkinson’s disease and mitochondrial disease.  StaGen has performed early phase 2 studies of Parkinson’s disease (n=26) and mitochondrial disease (n=2) patients in Japan, and obtained promising results. EnePharma is now raising capital to perform additional clinical trials in the US. In the future, EnePharma also aims to develop drugs to suppress aging (anti-aging drug) and elongate the life span.

Our Vision

We aim to develop ATP (energy) enhancing drugs to treat both rare diseases and common diseases caused by energy shortage. Among three important categories for life, i.e. things (molecules), information (genes) and energy (ATP), the last category (energy) has been ignored in medicine. Based on the vast knowledge of the associations between genes and diseases, Naoyuki Kamatani found that mitochondrial disease and neurodegenerative diseases including Parkinson’s disease are caused by energy shortage. Our combination drug, ATP enhancer was developed also based on the knowledge between genes and diseases. We believe that our drug will revolutionize the strategies to treat diseases in medicine.

Partnering

EnePharma is now looking to raise capital to perform clinical trials in the U.S. For partnering inquiries, please fill out your information and send it via email.

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News

Technology

EnePharma’s new drug is a combination of a xanthine oxidoreductase inhibitor (febuxostat) and inosine that enhances cellular ATP [ATP enhancer]. Since our drug is a combination of two active ingredients for which there is longterm experience in humans, we are targeting a 505(b)(2) pathway for speeding up the regulatory submission process with the FDA. We have evidence that it is effective to enhance cellular ATP from both pre-clinical and clinical studies.

Rationale

Energy (ATP) associated human genes comprise 8 % of all genes. So, many diseases must be caused by “energy shortage”, and there is large market potential for ATP enhancement therapy. Among rare diseases, mitochondrial disease is unequivocally caused by energy shortage, and among common diseases, mounting evidence from basic and clinical data strongly suggests that many of the neurodegenerative disorders including Parkinson’s disease are caused by energy shortage. By the intensive analyses of the association between genes and diseases, we have succeeded in designing a clever method to enhance the major purine compound ATP in humans by both providing more purine source and inhibiting purine degradation, i.e. by the administration of febuxostat and inosine.

In vivo disease studies in animals:  By the administration of febuxostat, the damage of mitochondria was suppressed. In transgenic model mice, progression of neurodegenerative disorders was suppressed.

Safety and efficacy supported by clinical data: Febuxostat and inosine have been co-administered to 65 subjects (18 healthy subjects and 47 patients), and the data supported the safety of this treatment. ATP and hypoxanthine were markedly increased in healthy subjects by the co-administration of febuxostat and inosine for 2 weeks but not by administration of either compound alone. In two patients with mitochondrial disease, co-administration of febuxostat and inosine for 2 weeks dramatically improved a biomarker of cardiac failure (BNP) in a mitochondrial cardiomyopathy patient and a biomarker of diabetes (insulinogenic index) in a mitochondrial diabetes patient. In patients with Parkinson’s disease, significant improvement (P = 0.0146) of the MDS-UPDRS Part III score with more than a minimal clinically important difference (MCID = -3.25) was shown in 26 patients treated for 8 weeks.

Current status of the development: Three leading professors in the US have agreed to become scientific advisory board members, and we are now preparing for pre-IND meetings with the FDA for mitochondrial disease and Parkinson’s disease based on our preclinical and clinical data.

Leadership

Naoyuki Kamatani, MD, PhD
Chief Executive Officer

Naoyuki Kamatani founded EnePharma Inc. in 2019 and has been Director of StaGen Co., Ltd. (Japan) since 2008.

Dr. Naoyuki Kamatani has extensive experience in genetics, drug R&D, and translational research. He worked with Dennis A Carson on research of MTAP deficiency in human cancers and proposed the first personalized anti-cancer therapy in the world. He led the clinical development of the anti-gout drug feuboxostat developed by Teijin Pharma, Japan.  Recently, he has developed a conceptual framework called the “Six-layer structure for genomics” (Kamatani N. Six-layer structure for genomics and its applications. J Hum Genet 2016 Mar;61:267-270.) to facilitate identification of genes that impact human phenotypes and diseases, potentially leading to novel therapeutic targets.  This framework along with the deep consideration into the relationship between entropy and energy in biology led to development of the ATP enhancing drug that is being developed by EnePharma Inc.

Previous affiliations: Director of the Laboratory for Statistical Analysis at the RIKEN Center for Genomic Medicine (CGM) from 2003 to 2011, Director of CGM, RIKEN from 2010 to 2011 and Professor of the Institute of Rheumatology at Tokyo Women’s Medical University from 1996 to 2008.

 

Yoshi Shima, LL.M.
Chief Officer, Legal & IP

Secretary at EnePharma, Inc. concurrent with Executive Officer in charge of Legal & IP at StaGen Co., Ltd., the parent company of EnePharma. He has been working as a corporate counsel and negotiator at several business entities, such fields as pharmaceutical, semiconductor, chemical, IT and banking. Concurrently with Head of Business Risk Management Office at MTI Ltd. (Tier 1 at TSE: 9438)

Previous affiliations: Head of Corporate Reorganization Office, Elpida Mermoy, Inc.; Head of Legal Office, Nihon Schering K.K.; and Corporate Counsel, Polyplastics Co., Ltd.