Professional Documents
Culture Documents
June 2018
Picot et al, 2008; Kwan P, Brodie MJ. N Engl J Med. 2000;342:314-319; Kwan P, Brodie MJ, CNS Spectr. 2004;9(2):110, Morgan Stanley research (2015)
Q1 ‘14 Q4 ‘14 Q1 2016 Q3 2016 Q2 2017 Oct 2017 Dec 2017 Jan 2018
EAPs Dravet 1 Dravet 1 LGS 2 AAN: Epidiolex NDA accepted LGS trial results
(Expanded Part A topline topline LGS 2 NDA for review: published in
Access started results results for Dravet mid-2018 The Lancet
#1 Programs) and LGS PDUFA
initiated
2,000
patients have been treated with Epidiolex
June 2018 GW Pharmaceuticals plc Investor Presentation 6
Epidiolex® Expanded Access Program
• FDA authorized, physician-sponsored expanded access AES 2017 – EAP Median % Reduction in Seizures
program (EAP) initiated in early 2014 All Patients with TRE
treatment-resistant epilepsy
- ~20 physician site EAPs and 6 US state sponsored EAP
programs
- Over 1,100 patients approved for treatment by FDA
• Most recent EAP data presented in abstracts at AES 2017
- Long-term (96-week) Epidiolex safety and treatment effect data Patients with LGS and Dravet Syndrome Only
- All Children and Adults with Treatment-Resistant Epilepsies
(Bebin et al, 2017) Convulsive Seizures Total Seizures
>97% of
completers
Sponsored
continued into
Physician
Childhood
Epilepsy Ongoing Treatment Data from >1,000 patients — Other Epilepsies Open Label
Syndromes Extension
Median % Reduction
Median % Reduction
30 29
25
25
20
20
15
15 16
13 10
10 10
9
5 5
0 0
Treatment Period Maintenance Period Treatment Period Maintenance Period
20 mg/kg/day CBD (n=61)
Placebo (n=59)
Source: J. H. Cross, O. Devinsky, L. Laux, E. Marsh, I. Miller, R. Nabbout, I. Scheffer, E. Thiele, S. Wright, Cannabidiol (CBD) reduces convulsive seizure frequency in Dravet Syndrome: results of a
multi-centered, randomized, controlled study, Abstract No 2.362, 2016, American Epilepsy Society Annual Meeting
June 2018 GW Pharmaceuticals plc Investor Presentation 10
Phase 3 LGS 1 (Single Dose Cohort) Trial Principal Efficacy
Results
Drop Seizures Total Seizures
50
60 p=0.0004
45
p=0.0005 45
p=0.0096 40
50 41
Median % Reduction
p=0.0135 49
Median % Reduction
35
40 44
30
30 25
20
20 22 15
20 15
10 14
10
5
0 0
Treatment Period Maintenance Period Treatment Period Maintenance Period
20 mg/kg/day CBD (n=86)
Placebo (n=85)
Source: E. Thiele, M. Mazurkiewicz-Beldzinska, S. Benbadis, E. Marsh, C. Joshi, J. French, C. Roberts, A. Taylor, K. Sommerville, Cannabidiol (CBD) significantly reduces drop seizure frequency in
Lennox Gastaut syndrome: results of a multicenter, randomized, double blind, placebo-controlled trial, Abstract No 1.377, 2016, American Epilepsy Society Annual Meeting
June 2018 GW Pharmaceuticals plc Investor Presentation 11
Phase 3 LGS 2 (Multiple Dose Cohorts) Trial Principal Efficacy
Results
Reduction in Drop Seizures Drop Seizure Responder Rates
70 Treatment Period
50 † †p<0.01 * *
Median % reduction / 28 days
*p<0.05
45 †
60 62 63 †p<0.01
47
† ‡p<0.001
40 42 †
50
40
35 37 ‡
40 43 †
% Patients
30 40
25 36
30 ‡
20
25
15 19 20
17
10 15 *
10 11
5 3
0 0
Treatment Period Maintenance Period ≥25% ≥50% ≥75%
(Primary)
CBD 20 mg/kg/day (n=76) CBD 10 mg/kg/day (n=73) Placebo (n=76)
▪ 5 CBD 20 mg/kg, 3 CBD 10 mg/kg, and 1 placebo patient achieved drop seizure freedom during maintenance
Cannabidiol (CBD) Significantly Reduces Drop Seizure Frequency in Lennox-Gastaut Syndrome (LGS): Multi-Center, Randomized, Double-Blind, Placebo-Controlled Trial (GWPCARE3)
A.D. Patel, O. Devinsky, J.H. Cross, V. Villanueva, E. Wirrell, K. VanLandingham, C. Roberts, D. Checketts, S. Zuberi, American Academy of Neurology Annual Meeting
June 2018 GW Pharmaceuticals plc Investor Presentation 12
Phase 3 Safety Profile
• Consistent AE profile across pivotal trials, EAP and state programs. Most common
AEs (>10% of patients) in Phase 3 trials include:
- Somnolence, diarrhea, decreased appetite, fatigue, pyrexia, vomiting, lethargy, upper
respiratory tract infection, convulsion
Across the Phase 3
• Serious AEs consistent with the pattern of common AEs
program, Epidiolex
• Low withdrawal rates seen throughout clinical program was generally well-
• Elevations in hepatic transaminases have been reported, most commonly with tolerated, with most
concomitant valproate; none met criteria for serious liver injury; all elevations AEs reported as mild
resolved– most while on treatment or moderate
- Physicians routinely monitor liver function in patients with epilepsy as there are a number
of approved AEDs that are associated with elevations in transaminases and typically run
routine liver tests
Sources: Tuberous Sclerosis Alliance; Child Neurology Foundation; Infantile Spasms Project
US 9,522,123 A method of treating complex partial seizure comprising administering CBD at a daily dose of at least 400mg. Granted
US 9,474,726 A method of treating febrile infection related epilepsy syndrome (FIRES) comprising administering CBD) to a subject. Granted
US 9,956,184 A method of reducing seizure frequency in LGS with concomitant CBD and clobazam, wherein the CBD dose is 10 Granted
mg/kg/day or greater and the purity of the CBD is greater than 98%.
US 9,949,937 A method of reducing seizure frequency in Dravet syndrome with concomitant CBD and clobazam, wherein the CBD Granted
dose is 10 mg/kg/day or greater and the purity of CBD is greater than 98%.
US 9,956,183 A method of reducing seizure frequency in LGS or Dravet syndrome with concomitant CBD and clobazam, wherein the Granted
dose of clobazam is reduced and the purity of CBD is greater than 98%.
US 9,956,186 A method of reducing convulsive seizures in LGS with CBD, wherein the CBD dose is 10 mg/kg/day or greater and the Granted
purity of CBD is greater than 98%.
US 9,956,185 A method of reducing convulsive seizures in Dravet syndrome with CBD, wherein the CBD dose is 10 mg/kg/day or Granted
greater and the purity of CBD is greater than 98%.
US 15/449,402 A method of reducing drop seizure frequency in treatment-resistant childhood-onset epilepsy with CBD, wherein the NoA
CBD dose is between 5-35 mg/kg/day and the purity of CBD is greater than 98%.
US 15/449,535 A method of reducing drop seizure frequency in a patient with Lennox-Gastaut syndrome, comprising administering to NoA
the patient in need thereof cannabidiol (CBD), wherein the CBD has a purity of at least 98% (w/w) CBD and comprises
not more than 0.15% (w/w) Δ9-tetrahydrocannabinol (THC); and wherein the dose of the CBD is about 20 mg/kg/day
NoA: Notice of Allowance received by Company
June 2018 GW Pharmaceuticals plc Investor Presentation 18
Epidiolex® Rescheduling
• FDA approval necessitates that Epidiolex be moved
out of Schedule I once it has an “accepted medical
use”
Effect on Subjective Pharmacodynamic Measures(1)
• New placebo-controlled trial data presented at ECTRIMS 2017 – positive primary and secondary endpoints
• GW reacquired full rights to develop and commercialize Sativex in U.S. (December 2017). Previous license
agreement with Otsuka terminated
• Represents a new wholly-owned late-stage U.S. asset
• We expect to evaluate the optimal route to achieve a NDA and believe that this may require the conduct of an
additional single pivotal trial
ICON