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HOUSE_OVERSIGHT_024907

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COWEN COLLABORATIVE INSIGHTS February 25, 2019 Epidiolex appeared to be well tolerated in the trial. 94% of patients on 20 mg/kg Epdiolex and 84% of patients on 10 mg/kg Epidiolex had an adverse event, compared to =a 72% of placebo patients. 88% of 20 mg/kg patients and 89% of 10 mg/kg patients deemed their adverse events to be mild or moderate. The most common AEs on 20 mg/kg were somnolence, decreased appetite, diarrhea, upper respiratory infection, pyrexia, vomiting and nasopharyngitis. For 1Omg/kg the most common AEs were somnolence, decreased appetite, upper respiratory infection, diarrhea, and status epilepticus. None of the cases of status epilepticus on 10 mg/kg were deemed treatment-related. Thirteen patients on 20 mg/kg Epidiolex had an SAE, of which five were considered treatment related; and 13 patients on 10 mg/kg Epidiolex had an SAE, of which 2 were considered treatment related, compared to 8 patients on placebo. Elevations in ALT/AST levels were observed in 11 patients in the 2O0mg/kg group and 2 patients in the 1Omg/kg group; 10 of the 13 patients were also on valproic acid. Five Epidiolex patients withdrew due to the elevations, but none of the patients met the criteria for drug-induced liver injury. GW noted that overall 10 mg/kg seemed to be somewhat better tolerated; 6 patients on 20 mg/kg Epidiolex and 1 patient on 10 mg/kg Epidiolex discontinued treatment due to adverse events, compared with 1 patient on placebo. There were no deaths in the trial. The results of the trial were published in NEJM in May 2018. Following the release of Phase III datasets from 1 Dravet and 2 LGS trials, a key area of controversy among investors (though not physicians) had been the drug-drug interaction between Epidiolex and clobazam, and in particular whether Epidiolex was effective in patients who were not also taking clobazam. This was put to rest at AES 2017 when GW presented a pooled analysis of the two Phase Ill LGS trials evaluating Epidiolex’s efficacy with

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