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KDDF-201312-10 Lead optimization of TU: A new hepatitis C virus drug candidate with a novel mechanism of action(Infectious Diseases, Chemical) [04.04.2014]


Development and Market Objectives

Institut Pasteur Korea (IP-K) identified TU a novel hepatitis C virus (HCV) inhibitor by phenotypically screening of small molecule libraries using infectious virions. Mechanism of action (MoA) studies demonstrated that IPK ES-1 inhibits at least early steps of the HCV life cycle. During structure-activity-relationship studies (SAR) a Lead molecule was identified with good antiviral efficacy and good DMPK properties.      
After Lead optimization our goal is to move towards a pre-clinical candidate, and together with an industrial partner we aim to enter pre-clinical stage to further develop a global novel drug candidate for a market size exceeding 15 billion USD annually.   

Unmet Medical Need & Target Patients

More than 200 million patients worldwide are chronically infected with HCV and are at risk of developing life-threatening liver diseases. There are no vaccines available yet and despite the recent approval of direct-acting antiviral agents (DAAs), the standard of care is a therapy with pegylated interferon-alpha (PEG-IFNα) and ribavirin (RBV), associated with an unsatisfactory sustained virologic response rates (SVR) of only 70-80 % and accompanied by serious side effects. Furthermore, DAAs have high economic burden to patients which will significantly limit access to therapy. In anticipation of potential therapy concerns like drug intolerability, viral drug resistance, etc. therapeutic options for patients and clinicians (personalized therapy) are needed.               
Targeted patients are all chronic HCV carrier by combinatorial therapy of IPK ES-1 together with other DAAs, and because of the MoA, our drug will be potentially particular suitable for pregnant women and liver transplant patients preventing mother to child transmission and re-infection of the new organ, respectively.


IPK ES-1 represents a new approach to the treatment of chronic hepatitis C. Identified by a phenotypic target free screening campaign IPK ES-1 is currently undergoing lead optimization by further improving already acceptable DMPK properties. Because IPK ES-1 has a novel MoA, inhibiting early and late steps in the HCV life cycle, in-depth characterization and confirmation of the putative molecular target envelope glycoprotein E1 is ongoing.

Intellectual Property

·         Patent filed: April 2014
·         Assignee: Institut Pasteur Korea
·         US provisional application No. 61/980,940: PCT
·         Title: Inhibitory molecules targeting HCV
·         Subject of invention: Composition of matter (chemical compounds) and their method of use (treatment of HCV)
·         Inventors: Windisch et al.

Competitive Advantages

·         IPK ES-1 is a first-in-class HCV inhibitor interfering with of early and late steps in the viral life cycle.
·         Outstanding antiviral potency.
·         Inhibition of all major HCV genotypes and its subtypes.
·         Synergy with selected DAAs in cell culture.
·         Accumulation in target organ (>40-fold accumulation in liver).


Infectious disease (HCV)

Research Period

Apr., 2014 ~ Jun., 2015


Institut Pasteur Korea (IPK)

Developmental Stage

Lead optimization

Additional Information

Contact Information

Address Company Name: Institut Pasteur Korea (IP-K)
WebSite Homepage: Contact Person: Marc P. Windisch
E-mail: Contact: +82-31-8018-8180

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