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COWEN COLLABORATIVE INSIGHTS February 25, 2019 month and the average age of patients in the trial was 16 yrs, although 30% were 18 yrs or older. On the primary endpoint, 20 mg/kg Epidiolex produced a median reduction in monthly drop seizures of 42% compared to a reduction of 17% in patients taking placebo, p=0.0047. There was also a suggestion of a dose response in the data, with the lower 10 mg/kg/day dose of Epidiolex producing a median reduction in monthly drop seizures of 37%, p=0.0016. In both dose groups the difference between Epidiolex and placebo emerged during the first month of treatment and was sustained during the entire treatment period. GW disclosed that the trial's secondary endpoints, and a series of sensitivity analyses, confirmed the robustness of the results. Similar to Epidiolex's other Phase III studies, although patients on clobazam (51%) had some additional benefit, GW indicated that Epidiolex also showed efficacy in patients not on clobazam. Figure 125 GWPCARES3: Reduction In Drop Seizures Figure 126 GWPCARE3: Responder Analysis a ut ¥ 34 ' A a5 530 7 2 325 . Ba ; Fis 7 “os 25 = Treatment Period (Primary) Maintenance Period MCBD 20 mglig (n=78) spe 94 CBD 10 mgikg(n=73) ——tpef.005 PLE (n=78) Baseline median (@1, 3) drop seizure frequency: CBD 20 mg/kg: 88 (38, 162) CBD 10 mg/kg: 87 (41, 180) Placebo: 80 (48, 148) Estimated treatment differences vs placebo CBD 20 mg/kg: -21.6 (95% Cl: -34.8, -6.7) CBD 10 mg/kg: -19.2 (95% Cl: -31.2, -7.7) ‘% Patients Treatment Period ‘ps0.05 'p<0.01 tps0.001 70 Maintenance % Patients Period p<0.05 tpo0.08 #p20.001 » za 225% 250% @ CBD 20 mg/kg (n=/6) f t 40 = t t 30 ' 20 5 = 10 13 3 a 0 27% 225% 250% CBD 10 mg/kg (n=73) M@ PLB (n=76) 275% Source: GW Pharma, AAN 2017 90 Source: GW Pharma, AAN 2017 Additional data were presented at AAN 2017. Included in this presentation were several of the trial's secondary endpoints and a series of sensitivity analyses. All of these confirmed the robustness of the results. For example, the proportion of patients witha >50% reduction in seizure frequency was 40% for 20 mg/kg Epidiolex (p<0.001), 36% for 10 mg/kg Epidiolex (p<0.01), and 15% for placebo. The proportion of patients with a >75% reduction in seizure frequency was 25% for 20 mg/kg Epidiolex (p<0.01), 11% for 10 mg/kg (p<0.05) Epidiolex, and 3% for placebo. The proportion of patients who achieved seizure freedom was 7% for 20 mg/kg Epidiolex, 4% for 10 mg/kg Epidiolex, and 1% for placebo. Figure 127 GWPCARES3: Safety CBD 20 mg/kg (n=82) CBD 10 mg/kg (n=67) Placebo (n=76) m (%) m (%) n (%) Al-causality TEAEs TT (94) 56 (84) 55 (72) Treatment-related TEAEs 51 (62) 20 (30) 15 (20) TEAEs leading to withdrawal 6 (7) 1 (1.5) 1(1) Serious TEAEs 13 (16) 13 (19.4) 8 (11) Treatment-related serious TEAEs 5 (6) 2 (3) it) TEAESs reported in >10% of patients in any group by preferred term Somnolence 25 (31) 14 (21) 4 (5) Decreased appetite 21 (26) 11 (16) 6 (8) Dianhea 12 (15) FT (10) 6 (8) Upper respiratory tract infection 11 (14) 11 (16) 11 (75) Pyrexia 10 (12) 6 (9) 12 (16) Vomiting 10 (12) 4 (6) ‘9 (12) Nasopharyngitis 9 (11) 3 (5) 57) Status epilepticus 4(5) 7 (10) 3 (4) Source: GW Pharma, AAN 2017 COWEN.COM HOUSE_OVERSIGHT_024906