1. © 2010 www.ithembapharma.comPage 1 Rethinking health innovation from the South - Some south driven initiatives Dr Chris Edlin December 14 th 2010

2. © 2010 www.ithembapharma.comPage 2 A Few Facts Commenced operations: June 2008 with 4 staff Finance: R30M ($4M) initial investment (government), a further R19M ($2.5M) raised through collaborations Only small molecule Chemistry company in South Africa (and Africa) International BoD and SAB Facility: 800m 2 of laboratory space with state-of-the-art analytical and synthesis equipment Equipment: 400MHz NMR, Agilent LCMS and prep HPLC, Genevac, CEM discover 12 µW

3. © 2010 www.ithembapharma.comPage 3 Location South Africa is GMT +2h Cape Town to iThemba = London to Rome

4. © 2010 www.ithembapharma.comPage 4 Malaria Phenotypic Screening Objective: Identify novel, low cost anti malarials Origin: iThemba networks and desire/need to generate project starting points. MMV capacity to screen Status: Screened novel compound collections (~42k) and identified relevant starting points Partners: MRCT (UK), MMV (CH), Eskitis (AU) Facilitator: iThemba The Future…….. Several series identified -Novel (MMV feedback, literature (TCAMS, St Jude) -Good Ligand Efficiency H2L effort initiated ADMET properties assessed -Members from series show good properties - Solubility, LogD, Microsomal Stability, Caco2 Submitted LOI and full proposal to MMV for funding support -Selected as one of final 6 (/60) for support

5. © 2010 www.ithembapharma.comPage 5 GSK – Pool for Open Innovation Extracts from a speech (Jan 2010) given by Andrew Witty (GSK CEO) at the Council on Foreign Relations in New York: “Since I took over at GSK I have been focused on changing the business model for the company to improve performance. But equally important is the imperative to earn the trust of society, not just by meeting expectations, but by exceeding them.” -Open lab ($8M seed funding) -Open source malaria hits -Patent pool -Collaborations “A collaboration has also been established with South Africa firm iThemba Pharmaceuticals. This work will help research and discovery into new medicines to treat tuberculosis”

6. © 2010 www.ithembapharma.comPage 6 GSK – Pool for Open Innovation Objective: Access and exploit IP in the “GSK pool for open innovation” for novel TB therapy Origin: Board of directors network Status: Signed MOU with GSK to access Pool, Announced by Andrew Witty (CEO) The Future…….. Transfer of two scientists Jan 2011 Future license opportunity (fee?)? Member of international consortium -Emory, Alnylam, GSK, BVGH, Harvard, MIT…….

7. © 2010 www.ithembapharma.comPage 7 Mtb Phenotypic Screening Objective: Identify novel, low cost Mtb inhibitors Origin: iThemba networks and desire/need to generate project starting points Status: 100k compounds being transported to UIC for phenotypic screening (MABA, LORA and cytotox.) Partners: MRCT (UK), UIC (USA), iThemba Facilitator: iThemba The Future…….. Screen output expected Jan/Feb 2011 -Shared effort UIC/iThemba -Shared IP (UIC/iThemba) - MRCT retain right for MoA determination Seek funding for initial H2L phase -GTB, NIH, Wellcome Trust….

8. © 2010 www.ithembapharma.comPage 8 Key Learnings Require strong and renowned advisors (figure heads) –Gives partner confidence in capabilities Require excellent network (international) –Use appropriate tools e.g. Linkedin Require tenacity –Email/telephone call per collaboration = ………..