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KDDF-201406-03 Development of lead candidates for non-alcoholic steatohepatitis and liver cirrhosis using natural compound analogues(Gastrointestinal Diseases, Chemical) [10.01.2014]

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Development and Market Objectives

Non-alcoholic steatohepatitis (NASH) and liver cirrhosis are prevalent and serious diseases, but effective treatment options have yet to be developed. In this study, we are focusing on the development of an innovative new therapeutic that will inhibit the generation and progression of NASH and cirrhosis.

Unmet Medical Need & Target Patients

Along with the rise in obesity due to the increasing popularity of the Westernized diet, non-alcoholic liver disease (NAFLD) is increasing. The prevalence of NAFLD is approximately 20% and it is the most common cause of liver dysfunction. Some forms of NAFLD appear in the form of NASH that involves the destruction of liver cells and can proceed to cirrhosis or liver cancer via liver fibrosis.
NASH is one of the most significant causes of cirrhosis and liver cancer in developed countries where viral hepatitis is decreasing. Approximately 20 percent of NASH patients progress to cirrhosis that leads to death, due to failure of the liver or malignancy. According to a recent meta-analysis, NASH, compared to simple fatty liver disease, has a 5.7-fold higher risk of death, which increases to 10-fold if accompanied with liver fibrosis.
However, there are no evidence-based treatments for the clinical effects on NAFLD available, especially therapeutics that can prevent the generation of NASH or the progression to cirrhosis.

Status

We found that a natural compound can inhibit the generation of NASH and the progression to cirrhosis via activation of NLRP3 inflammasome. With potency from at least 10-fold lower concentrations of the natural compound derived from structural analogs of that compound, 8 leading candidates have been identified that inhibit the activation of NLRP3 inflammasome. We are planning to select 2-3 optimized lead candidates through studies of:
1) Target validation
2) In vitro efficacy and in vitro DMPK/ toxicity
3) In vivo PK
4) In vivo efficacy

Intellectual Property

A patent application has been submitted

Competitive Advantages

Structural analogs of the natural compound, with potent efficacy confirmed by our researchers, are highly novel and excellent candidates for patent protection.
- Structurally, they belong to a class of novel compounds that have not been previously reported and related compounds have not been developed for therapeutic purposes for the treatment of NAFLD, NASH and liver cirrhosis.
- Accordingly, patent protection for the compounds’ structural aspects and their usage is highly likely, and activity of the lead candidate is significantly superior to the natural compound. Both Korean and international PCT applications are planned upon finalization of the lead substance.

Indication

Gastrointestinal Disease(NASH, Liver cirrhosis)

Research Period

Sep. 01, 2014 - Jan. 31. 2016

Company

Asan Medical Center

Developmental Stage

Lead Generation

Additional Information

Contact Information

Contact
Address Company Name: Asan Medical Center
WebSite Homepage: http:// Contact Person: Eun-Hee Koh
E-mail: ehk@amc.seoul.kr Contact: +82-2-3010-3248

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