1 CBMTG 0601 Update 13-Feb-2009 Tampa, Florida

22 Key Information Pivotal Phase III trial comparing G-PB with G-BM (experimental arm) 230 recipient-donor pairs Sponsor: CBMTG Grant PI: Dr. Kirk Schultz Clinical Chair: Dr. Stephen Couban; Co-Chair: Dr. Jeff Lipton

33 Primary Objective Will we see less chronic GvHD with G-BM? Treatment Failure (Composite Endpoint): - extensive chronic GVHD - relapse - death (Time to the earliest of 3 events after HSCT)

44 Secondary Objectives Hematologic recovery Acute and chronic GVHD Donor and Recipient Quality of Life Laboratory analysis of graft characteristics Prospective economic analysis

55 Project History Grant first submitted July 2005 Received NIH funding April 2006 Investigator Meeting June 2006 First site activated January 2007 (CCMB)

66 Actual versus Projected Enrollment March 2008

7 Vancouver Winnipeg London HamiltonToronto Ottawa Montreal Quebec City Halifax 9 Canadian Sites 5 International Sites: Fred Hutch King Faisal Melbourne Auckland Adelaide

88 SiteState Depart Approval IRB ApprovalContract Site Activation Fred Hutch Jul-2008 King Faisal Nov-2008 Auckland City Hospital Royal Melbourne IMVS – Adelaide International Sites

9 Total enrollment = 61 February 2009

10 Cumulative Enrollment re-projected rate

11 Enrollment by Site (Feb-2009)

12 Re-projected Accrual Timeline Accrual to be completed by January 2012

13 Data Safety Monitoring Committee SAE’s are submitted to DSMC within 48 hours Formal review every 6 months (most recent Dec-08) Monitors accrual Requires updates on data submission compliance every 6 months 13

14 Data Submission Compliance Clinical Data Time since Randomization (in months) Number enrolled pairs are more than 6 months overdue Overall, data submission compliance is excellent Data current within number of months

15 Timelines for Data Submission Data FormSubmission Deadlines QuestionnairesSame day Section 3 to 6 Pre-HCT and graft product 1 month post HCT Section 7 and 8 Day 30 and Day month from day +100 Section 9 Follow-up 1 month from date of follow-up

16 What’s new? No more invoicing for per patient payments Electronic data capture on the horizon Collection of more GvHD data at Year 1 and Year 3 (NIH Consensus Data Collection Form)

17 All deaths to be reported as SAEs up until last follow-up (required by the DSMC) Data on immunosuppressive therapy (Section 9: Follow-Up) Revised Health Care Questionnaire

18 Consent and Enrollment Approach recipients first Quality of Life can be done prior to randomization if consent obtained Product can be cryopreserved if scheduling difficulties arise

19 Number Scheduled for Allo HCT versus Number on Study 9 Activated Sites (48% enrolled on average)

20 Summary Continued commitment necessary in order to complete accrual by 2012 Let’s try to submit data on schedule! Changes to SAE reporting are coming Sites are making a great contribution!