2019/08/14

Session2-1 Delivering medicines that matter and maximizing pharma asset value through robust and efficient industry-academia collaborations

Hiroshi Nagabukuro PhD  ~ARTham Therapeutics Inc.~

[Summary]

Two years back, I was here to represent Takeda Pharmaceutical to introduce its entrepreneurship venture program, which supported Takeda employees to create their own new companies. The program resulted in launching 9 new companies. ARTham Therapeutics Inc. is the 9th one.
Our mission is to deliver medicine that matters for patients by approaching mainly two things. One is unmet medical needs. We identify and fulfill the makers' need wherever it is. The second one is pharma assets on the shelf, and we discover hidden value of such assets by drug repositioning approach. We initially approach pharma and in-license assets and grow the value of those asset in collaboration with academic institutions. We strongly believe that this is low risk strategy because pharma company has invested in the assets already. In addition, we can eventually trigger a clinical program immediately so that we can quickly progress its developmental stage. This can be done because of the management and our expertise. Dr. Sham Nikam has more than 30 years of experience in pharma R&D in the US, Europe and Japan, and I have been involved in drug discovery and development both in the US at Merck and in Japan at Takeda. Dr. Nikam and I have achieved multiple INDs as well as NDAs. Dr. Naoto Uemura who is a professor in Oita University Clinical Pharmacology and Therapeutics, has extensive experience in planning and executing early clinical development through his tenure at Merck in the US.
We licensed in 4 assets from Takeda upon the company launch. ART-648 is an orally available PDE4 inhibitor, which was initially developed for type II diabetes and is now being developed for nonalcoholic steatohepatitis (NASH). ART-001 is a selective PI3Ka inhibitor used to be developed for solid cancer. As there is a strong genetic association between the disease and the molecular target, we are developing this molecule for vascular malformations.
Greater than 200 million US dollars is the corporate value we would like to achieve within 2 years. With Series A funds as well as AMED grant, we have successfully completed both ART-648 phase 1 and phase 1 enabling studies for ART-001. We anticipate achieving human POC of ART-648 and phase 1 exit for ART-001 in 2020-2021 with Series B funds to be invested by the end of this year. By reaching these milestones, the corporate value will grow significantly.
NASH is a progressing disease of liver with multiple causes starting from hepatic fat deposition and inflammation. Development of fibrosis leads to cirrhosis and then hepatocellular carcinoma. As there is no way to cure such end-stage diseases except liver transplantation, it is critical to stop or reverse the disease at NASH stage. Because any pharmacological therapies are not available for NASH although 〜10 million people are suffered from the disease in the US, there is clear unmet medical needs and huge market opportunity. We do believe that PDE4 inhibition has a great possibility of success because none of phase 3 candidates having a different molecular mechanism does not necessary possess all aspect of pharmacological activities associated with NASH treatment including anti-steatosis, anti-inflammatory, anti-fibrotic, anti-diabetic and anti-obesity whereas PDE4 inhibitor cover all activities.
Working with Business School in University of Michigan, we project the revenue of ART-648, post-launch in 2025, as 1.5 billion US dollars if 3.8% of fair share is gained. The revenue will then be increased in 5 years by 2.4 billion dollars assuming a share of 5.8%.
We are running R&D with a robust collaboration with Japanese academia. We rely on the strong science in Japan and also cost efficiency by working with academic institutions while looking at global market for late stage development of our assets.
Delivering value for patients and their families and society is our goal.

[Profile]

HNphoto.jpg Dr. Hiroshi Nagabukuro is a co-founder, COO/CSO of newly launched ARTham Therapeutics Inc. He has strong background in pharmacology with >20 years' experience of drug discovery research in both Japan and US. Dr. Nagabukuro also holds special appointed professorship at Oita University Medical School.