ENABLE 1 Study. To evaluate the safety and tolerability of eltrombopag Primary efficacy outcome: proportion of patients who achieve SVR

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1 Final Results of ENABLE 1, a Phase 3, Multicenter Study of as an Adjunct for Antiviral Treatment of Hepatitis C Virus -Related Chronic Liver Disease Associated With Thrombocytopenia N. Afdhal; G. Dusheiko; E.G. Giannini; P. Chen; K. Han; A. Moshin; M. Rodriguez-Torres; S. Rugina; E. Lawitz; M.L. Shiffman; G. Tayyab; F. Poordad; Y. Mostafa Kamel; A. Brainsky; J. Geib; S.Y. Vasey; R. Patwardhan; F.M. Campbell; D. Theodore Abstract LB 3 ENABLE 1 Study Objectives To assess the ability of eltrombopag to Increase platelet counts in patients with chronic HCV and thrombocytopenia Enable initiation of antiviral therapy Allow maintenance of antiviral therapy Increase SVR To evaluate the safety and tolerability of eltrombopag Primary efficacy outcome: proportion of patients who achieve SVR

2 Screening 45 Days Platelets <75K/µL Randomized Withdrawal Study Design START Part 1 (2-9 Wks) Open-Label Pre-Antiviral Treatment Phase 25 mg Platelets <9K/µL 5 mg Platelets <9K/µL Platelets 9K/µL 75 mg Platelets <9K/µL 1 mg Growth factor support allowed for anemia and neutropenia PEG-2a reduced or discontinued for thrombocytopenia /matched placebo could be titrated during Part 2 to maintain platelets 9K 2K/µL *24 weeks if HCV genotype 2/3, otherwise 48 weeks. Platelets 9K/µL Platelets 9K/µL Platelets <9K/µL Withdrawal Platelets 9K/µL Part 2 ( 48 Wks)* Double-Blind Antiviral Treatment Phase 2:1 Randomization (:) + PEG-2a + RBV OR + PEG-2a + RBV 24 Weeks Post-Therapy: SVR Assessment Results of Open-Label Therapy N=716 Initiated antiviral therapy, n (%) Failed to raise platelets >9,/µL 68 (95) 11 (2) Dose of eltrombopag that enabled initiation of antiviral therapy n (%) Cumulative % 25 mg 5 mg 75 mg 1 mg 451 (63) 176 (25) 39 (5) 14 (2)

3 Virologic Responses (ITT) P= =16.7% 95% CI: P< =14.8% 95% CI: P<.1 RVR EVR cevr ETR SVR 42 Virologic Response =1.7% 95% CI: P= =7.9% 95% CI: P= SVR by Platelet Count at Baseline and HCV RNA at Screening (ITT) =6.1% 95% CI: =8.4% 95% CI: =7.8% 95% CI: =8.% 95% CI: <5,/µL 5,/µL Platelet Count at Baseline* *Baseline is on/prior to Day 1 of Part 1/open-label phase. <8, IU/mL 8, IU/mL HCV RNA at Screening

4 Median Platelet Counts (ITT) Median Platelet Count (x1 3 /µl) % of patients from Part 1 entered Part 2 Part 1 Part 2 Follow-Up Threshold for dose reduction // // OLB 2 AVB Time Since Randomization (Weeks) Pbo Epag AVB, antiviral baseline; OLB, open-label baseline. PEG-2a Dose Reductions (ITT) >3 Number of Dose Reductions

5 Summary of Patients with Adverse Events AE Type, No. of * (n=232) (n=449) Any AE 226 (97) 43 (96) Any SAE 35 (15) 9 (2) Any fatal AE** 6 (3) 1 (2) Any drug-related AE 217 (94) 42 (94) Any AE leading to medication discontinuation 68 (29) 85 (19) Any AE leading to study withdrawal 7 (3) 11 (2) *Double-blind safety population. AEs on treatment + 3 days follow-up are reported except as noted. **On treatment + 6 months follow-up. Adverse Events of Special Interest AE Type, No. of * (n=232) (n=449) Thromboembolic 4 (2) 11 (2) Hepatobiliary Events suggestive of progressive liver disease ALT >3x ULN Malignancies Hepatocellular carcinoma Other Bleeding Variceal hemorrhage Gastrointestinal bleeding Ocular AEs Progression of pre-existing cataract** Incident cataract** 19 (8) 34 (15) 5 (2) 3 (1) 2 (<1) 59 (25) 2 (<1) 27 (12) 4 (2) 4 (2) *Double-blind safety population. AEs on treatment + 3 days follow-up are reported except as noted. **On treatment + 6 months follow-up. 59 (13) 67 (15) 13 (3) 13 (3) 83 (18) 8 (2) 9 (2) 53 (12) 21 (5) 17 (4)

6 ENABLE 2: Virologic Responses (ITT) =2.7% 95% CI: P< =9.1% 95% CI: P= RVR EVR cevr ETR SVR Virologic Response =13.1% 95% CI: P<.1 =6.% 95% CI: P= ENABLE 2: Preliminary Summary of Patients With Adverse Events AE Type, No. of * (n=252) (n=56) Any AE 235(93) 475 (94) Any serious AE (SAE) 37 (15) 99 (2) Any fatal AE** 4 (2) 19 (4) Any drug-related AE 225 (89) 453 (9) Any AE leading to medication discontinuation 7 (28) 115 (23) Any AE leading to study withdrawal 9 (4) 23 (5) Any hepatic decompensation, hepatocellular carcinoma, or death 2 (8) 74 (15) Any thrombotic/thromboembolic event 1 (<1) 22 (4) *Double-blind safety population. AEs on treatment + 3 days follow-up are reported except as noted. **On treatment + 6 months follow-up.

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