EpiTherapeutics ApS Integrating Instant Jchem in the Discovering Organization of a Biopharmaceutical Startup ChemAxon UGM, Budapest May 2012 Thomas Boesen, PhD Director of Drug Discovery

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Agenda

Brief company overview

Use of IJC as an integrated tool in our Discovery Process

Further Development

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• Founded October 2008 on seminal research by world-leaders in oncology and epigenetics, Professor Kristian Helin and colleagues of BRIC, Copenhagen. Exclusive collaboration agreement with BRIC and right of first refusal to new IP from BRIC

Executive Summary

• $ 9.1 mill seed investment by Seed Capital, Novo Seeds, Lundbeckfond Ventures, Astellas Ventures and Merck Serono Ventures

• 3-year research collaboration agreement signed with Abbott December 2010

• Key targets are histone demethylases and histone methyltransferases with the potential for creating truly innovative cancer treatments

• Most progressed program (GASC1) is in early lead optimization with several compounds in the single digit nM range and inhibition of cellular GASC1 activity. Good PK data

• Preparing for Series A financing of USD 25M and seeking pharma partnerships to apply insight within epigenetic tumor biology

• 17 employees in Copenhagen/New Jersey (14 PhDs), 8 FTEs in Canada (PhDs)

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EpiTherapeutics focuses on two epigenetic enzyme families

DNA Methylation

• CpG Methylation/Demethylation?

Histone Modifications

• Acetylation/Deacetylation of Histones • Methylation/Demethylation of Histones

• Other Modifications of Histones

5’ 5’

3’ 3’

Deoxycytidine 5-Methyl-cytidine

DNMT

DNDM?

e-N-Methyl-Lysine

e-N-Acetyl-Lysine Lysine

Lysine

KAT

KDM

HDAC

KMT

The focus of EpiTherapeutics

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GASC1 Summary

Cancer GASC1

amplification

Excess H3K9 demethylation

Androgen receptor

activation

Expression of oncogenes

GASC1 inhibitors

Genomic instability

Overriding senescence

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Current activities include programs targeting both HDMs and HMTs

Pipeline – status of current internal projects

• GASC1 (HDM)

Assay Development HTS Hit-to-Lead Lead Optimization

• PLU1 (HDM)

Status of current collaborations

• X, Y, Z… Details from collaboration have not been disclosed

• DOT1L (HMT)

Assay Development HTS

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Agenda

Brief company overview

Use of IJC as an integrated tool in our Discovery Process

Further Development

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What we do

SAR: Structure Activity Relationship

Compound Design

Compound Synthesis /Purchase

Compound Testing

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How we are organized

Cpd characterization Screening

Target validation In vitro biology

Other CROs • ADME • PK • Compound characterization DATA • Protocols • Test results

Project Leadership Project Management

8 FTEs (Ph.D.)

Chemistry CRO • Synthesize compounds for

testing DATA • Compound identity and

structure • Analytical data • Amounts • Procedures and notebooks

Chemical vendors DATA • Compound identity and

structure • Analytical data • Amounts

Internal laboratories • Screening • Functional cell assays • Target validation DATA • Protocols • Test results

The database becomes the “common language” of chemist and biologists

Software specifications

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• Ease of use • No in-house programming

expertise • Users with different

background

• Compatible

• OS • Third party software

• Scalable

• Reliable/proven

• Versatile • Innovative organization

Database

End User interface

Design of Database Specifications

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• Traceability • Integrated with our data management setup • Compound batch and experiment level

• Versatile

• Types of data • A variety of assays and experiments

• Security

• User level • Project level

• Primary Key

• Compound Number

• Tool to enable SAR

Basic Tables

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Pivot – SAR table

Structure •Unique structure for

each EPT number

Project •User level security

/access

Secondary Assays •Dose response

screening

Primary Assays •Single point

screening

Batch

Batch table

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Secondary Assays Table

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Structure removed

SAR Table

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Structure removed

What it takes to maintain the database

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User level

• Scientists easily upload data • Data can easily be QC’ed • Data is easily accessed by Scientists

Administrator level

• Pivoting requires MySql programming • Time consuming • Complex • Performance

Agenda

Brief company overview

Use of IJC as an integrated tool in our Discovery Process

Further Development

Further Possible Development of our IJC setup

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Data Reporting

• Graphs • Correlations

• Build in Functions

Exporting options

• Overviews on slides (SAR) or in documents

Thank you for your attention!