Best Practices: From Benchtop to IND

Slide 1: From Benchtop to IND May 13th 2008 Wendy Hill, Gap Strategies Valentia Lee-Brotherton, Ashuren Health Sciences Peter Pekos, Dalton Chemicals

Slide 2: Challenges to the Emerging Biotech Company  Lack of Resources  Lack of Experience in Drug Development  Clearly Defined Vision  Manufacturing/CMC/Quality Issues  Investor Pressure Date: May-14-08 Confidential Information – Not for Distribution Slide 1

Slide 3: Management of Product Development Nonclinical Pharmacology (Preclinical) And POC Safety Studies Testing Development Plan Regulatory Manufacturing Affairs Clinical Planning

Slide 4: The Early Development Program - I  Establish your development plan BEFORE embarking on preclinical development  Make sure you understand the physiology of your targeted disease and your compound and design your preclinical POC and studies accordingly  Seek advice from those who have experience  Know the Regulatory Expectation  The Toxicology Program is not a box-checking exercise Date: May-14-08 Confidential Information – Not for Distribution Slide 3

Slide 5: The Early Development Program - II  Co-ordinate Toxicology with Clinical (design, data) and Manufacturing (logistics)  Identify the target organs / systems of the drug and characterize the toxicity  Characterize the dose-response curve (NOAEL, MTD, TI, etc.)  Assess the systemic exposure  Understand impact on Clinical Development Date: May-14-08 Confidential Information – Not for Distribution Slide 4

Slide 6: The IND or CTA Submission for FIH Trials Present a high-quality submission: avoid Regulatory “Red Flags” and address reviewer’s expectations  Content  Format  Quality and quantity of data to support the proposed trial  NOAEL, MABEL, MTD, STD, Human starting dose  Quality of product (controls, stability, characterization, testing)  Clinical protocol safe to initiate? Date: May-14-08 Confidential Information – Not for Distribution Slide 5