1. GZR/EBR 100/50 mg qd N = 107 N = 52 W12W24W16 PlaceboGZR/EBR W28 > 18 years HCV infection Genotype 1, 4, 6 IBLD: sickle cell anemia,  -thalassemia, hemophilia A/B, von Willebrand disease Hemoglobin > 7 g/dl Treatment-naïve or experienced Compensated cirrhosis allowed * HIV co-infection allowed ** Randomisation 2 : 1 Double blind  Design C-EDGE IBLD: grazoprevir/elbasvir for HCV infected patients with inherited blood disorders (IBLD)  Objective –SVR 12 (HCV RNA < 15 IU/ml), by intention to treat analysis C-EDGE IBLD Hezode C., EASL 2016, Abs. SAT-128, J Hepatol 2016; 64:S753 * Fibroscan > 12.5 kPa, or FibroTest > 0.75 + APRI > 2 ** On stable ART with TDF or ABC, 3TC/FTC + RAL or DTG or RPV for ≥ 8 weeks, and CD4 > 200/mm 3, and undetectable HIV RNA

2. GZR/EBR N = 107 Placebo (deferred) N = 52 Female25% Black / White / Asian 18% / 76% / 6%17% / 77% / 6% Genotype 1a 1b 4 6 44% 43% 11% 0% 35% 52% 12% 2% HCV treatment-experienced50.5%48.1% Cirrhosis24.3%23.1% HIV co-infection5.6%7.7% IL28B CC25%17% IBLD Sickle-cell disease  -thalassemia Von Willebrand/hemophilia 17.8% 38.3% 43.9% 19.2% 38.5% 42.3% Baseline characteristics C-EDGE IBLD: grazoprevir/elbasvir for HCV infected patients with inherited blood disorders (IBLD) C-EDGE IBLD Hezode C., EASL 2016, Abs. SAT-128, J Hepatol 2016; 64:S753

3. Breakthrough, N0000 Relapse, N6411 Lost to follow-up/early discontinuation, N 1010 SVR 12, ITT mFAS, %94.391.597.891.7 Primary endpoint : SVR 12 (HCV RNA < 15 IU/ml), %, ITT * ITT, mFAS: exclusion of 1 patient (genotype 1b) who discontinued due to non compliance C-EDGE IBLD: grazoprevir/elbasvir for HCV infected patients with inherited blood disorders (IBLD) C-EDGE IBLD Hezode C., EASL 2016, Abs. SAT-128, J Hepatol 2016; 64:S753 100 75 50 25 0 95.5 91.5 95.7 91.7 All patientsGenotype 1aGenotype 1bGenotype 4 107474612 % N=

4.  Impact of baseline RAVs on efficacy in patients with genotype 1a (population sequencing with threshold of 20%) –NS5A RAVs at baseline in 4/47 (8.5%): SVR 12 = 1/4 –NS3 RAVS at baseline in 26/47 (55%) 22 with NS3 only: SVR 12 = 100% 4 with NS3 + NS5A RAVs: SVR 12 = 1/4 C-EDGE IBLD: grazoprevir/elbasvir for HCV infected patients with inherited blood disorders (IBLD) C-EDGE IBLD Hezode C., EASL 2016, Abs. SAT-128, J Hepatol 2016; 64:S753

5.  Relapses NS3 RAVsNS5A RAVs Patient characteristicsGenotype Baseline HCV RNA (IU/ml) Follow-up day of relapse At baselineAt failureAt baselineAt failure 43y, Thai female,  -thalassemia 1a9.07 x 10 5 56S122S/GI170V Q30L/Q, Y93Y/H Y93N 43y, white male, Factor VIII deficiency 1a2.02 x 10 6 28NoneA156T/ANone Q30R, L31M 60y, HIV+, white male, Factor VIII deficiency 1a4.29 x 10 6 27Q80K Y56H, Q80K, D168A Y93C L31M, Y93C 47y, white male, Factor VIII deficiency 1a4.51 x 10 6 58Q80K M28V M28A, Q30R 41y, white male, Factor VIII deficiency 1b2.24 x 10 6 86T54S, V55I T54S, V55I, R155L/R R30Q, L31M R30Q, L31M, Y93H 47y, black female, Sicke cell anemia 41.97 x 10 6 57D168E L28M, L30H L28M, L30H, H93C All patients were HCV treatment-naïve and had no cirrhosis C-EDGE IBLD: grazoprevir/elbasvir for HCV infected patients with inherited blood disorders (IBLD) C-EDGE IBLD Hezode C., EASL 2016, Abs. SAT-128, J Hepatol 2016; 64:S753

6. GZR/EBR, N = 107Placebo, N = 52 Serious adverse event2.8%11.5% Adverse event leading to discontinuation0%1.9% Adverse events in > 5% of patients Headache21.5%11.5% Fatigue16.8%7.7% Nausea8.4%15.4% Asthenia7.5%3.8% Abdominal pain6.5%3.8% Arthralgia6.5%5.8% Pyrexia5.6%0 Nasopharyngitis5.6%3.8% ALT 1.25 – 2.5 x ULN > 2.5 ULN 4.7% 0.9% 38.5% 26.9% AST 1.25 – 2.5 x ULN > 2.5 ULN 5.6% 1.9% 30.8% 26.9% Bilirubin Grade 3 Grade 4 11.2% 7.5% 17.3% 5.8% Alkaline phosphatase > 3 x ULN00 Adverse events and laboratory abnormalities, % C-EDGE IBLD: grazoprevir/elbasvir for HCV infected patients with inherited blood disorders (IBLD) C-EDGE IBLD Hezode C., EASL 2016, Abs. SAT-128, J Hepatol 2016; 64:S753

7.  Summary –12 weeks of GZR/EBR was highly efficacious in patients with inherited blood disorders and HCV genotype1 and 4 infection, and evidenced in various patient subgroups Cirrhosis HIV co-infection All IBLDs –Lower response in patients with genotype 1a infection with baseline NS5A RAVs –Tolerance was generally good No impact on measures of hematology and clotting or on the treatment of the underlying blood disorder C-EDGE IBLD: grazoprevir/elbasvir for HCV infected patients with inherited blood disorders (IBLD) C-EDGE IBLD Hezode C., EASL 2016, Abs. SAT-128, J Hepatol 2016; 64:S753