1. A Subsidiary of The Schnappi Corporation Team 5 Peter Chen Pasquale Cirone Crystal Gu April Haisu Ma Serina Xiaoqing Tang November 20, 2009 1

2. Executive Summary 2 Severe forms of inflammatory bowel disease (IBD) patients represent the best opportunities for Cure and GTX Safety, efficacy and innovative mechanism of action of GTX-001 can address the unmet needs of a profitable market The potential IBD market can support the cost of developing and licensing GTX-001 From our analysis, we recommend licensing GTX-001

3. 3 Epidemiology 910K 851K 1,035K 1,107K Source: Nguyen et al. 2007, US census, internal analysis

4. Unmet needs (Crohn’s Disease) 4

5. Unmet needs (Ulcerative Colitis) 5

6. Clinical Signals for Success 6 What signals (in preclinical and clinical studies) would indicate investigational agents can achieve the desired target product profile? Early Studies (Preclinical, PhaseI/II) GTX001; Pharmacokinetics, efficacy with multiple treatments GTX002; Toxicities are manageable upon scale-up. Later Studies Efficacy (Induction, Maintenance models) Crohn’s Disease Activity Index* Fistula Response Ulcerative Colitis Clinical Score** Rate of Onset Incidence of side effects (i.e., infections) Long-Term Efficacy Studies % Rollover Benefit over surgery IBD Questionnaire, QALY Note: * Best WR et al, Gastroenterology 70 (3): 439–44. 1976. ** Feagan et al., Gut 54: 782-8. 2005

7. Clinical Research Strategy (IBD) 7

8. Financial projection (GTX-001) Source: Case information, Nguyen et al. 2007, US census, internal analysis GTX-001 is expected to reach peak sales of $750 million in 5 years 8

9. Financial projection (GTX-002) Source: Case information, Nguyen et al. 2007, US census, internal analysis GTX-002 will not be able to generate enough sales to cover its major costs after it is launched 9

10. Source: Case information, internal analysis GTX-001 has positive rNPV in both base and high cases With the approval of expanded indications, GTX-001 is likely to create more value Assessment of Commercial Values 10

11. Final Recommendation Development of GTX002 for IBD is not recommended for CURE. Development of GTX001 for IBD is recommended. Base-market case represents a positive Risk-Adjusted NPV for CURE. Preclinical drug properties (drug target, safety etc) amenable for IND (assuming humanized MAB). Target market and indications are favored heavily by KOLs. Clinical Strategy proposed to mitigate technical and financial risks. 11

12. Appendix 12

13. Crohn’s Disease Activity Index 13 Clinical Readout Weighting factor Number of liquid or soft stools each day for seven daysx 2 Abdominal pain(graded from 0-3 on severity) each day for seven daysx 5 General well being, subjectively assessed from 0 (well) to 4 (terrible) each day for seven daysx 7 Presence of complications * *One point each is added for each set of complications: the presence of joint pains or frank arthritis inflammation of the iris or uveitis presence of erythema nodosum, pyoderma gangrenosum, or aphthous ulcers Anal fissures, fistulae or abscesses. Fever (> 100 °F) during the previous week. x 20 Taking Lomitil or opiates for diarrheax 30 Abdominal mass (0 as none, 2 as questionable, 5 as definite)x 10 Absolute deviation of Hematocrit from 47% in men and 42% in womenx 6 % deviation from standard weightx 1 Remission of Crohn's disease is defined as a CDAI of less than 150. Severe disease was defined as a value of greater than 450. Response defined as a fall of the CDAI of greater than 70 points. Best WR et al, Gastroenterology 70 (3): 439–44. 1976.

14. Rationale for GTX Products GTX001GTX002 Monoclonal Antibody (Humanized?) targeting VLA-1 (  1  1 integrin). -Effective 2mg/kg/2days (IV, rodents) -Tolerated at 6mg/kg (IV, rodents) Target: VLA1 VLA1 Expression: ***Endothelial Cells (extravasation) ***Memory and T H 1 T cells Other T cells NK, monocytes, macrophages Modulates adhesion, migration, proliferation, cytokine secretion and ECM remodeling. Small molecule inhibitor of I  B kinases (IKKs) -Effective 2mg/kg/2days (IV, rodents) -Tolerated at 50mg/kg (Oral, rodents) and 5mg/kg (IV, rodents). -Susceptibility to bacterial infections (side effect); Liver and kidney toxicities. -Significant efficacy with 5-ASA and/or steroids. Target: IkB Kinase(s) Phosphorylate IkB, which inhibits NF-kB pro- survival pathway (Appendix 20) Goldstein I, et al., J Clin Invest 112(9): 1444–1454. 2003 14

15. Key assumptions GTX-001GTX-002 Cumulative probability of success 70% # years to ramp-up 4 years COGS as % of sales 20%10% Discount rate10% Peak penetration 30-60%*10-30%* 15 Note: * Depending on target indications