Upload
others
View
1
Download
0
Embed Size (px)
Citation preview
©2018 GW Pharmaceuticals plc | All Rights Reserved
Investor PresentationNovember 2018
FORWARD-LOOKING STATEMENTS
This presentation contains forward-looking statements that are based on our management’s beliefs and assumptions
and on information currently available to management. Forward-looking statements include information about our
current expectations for future events, including potential results of operations, the timing of clinical trials, the timing
of regulatory filings and approvals, the timing and outcomes of regulatory or intellectual property decisions, demand
for our commercially available products and products in development and the clinical benefits, safety profile and
commercial potential and potential pricing of Sativex®, Epidiolex®, and any product candidates. These forward-
looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that could
cause our actual results, performance or achievements to be materially different than any future results, performance
or achievements expressed or implied by the forward-looking statements. Forward-looking statements represent our
management’s beliefs and assumptions only as of the date of this presentation. You should read our most recent
Annual Report, as filed on Form 10-K with the Securities and Exchange Commission, including the Risk Factors set
forth therein and the exhibits thereto, and our subsequent filings with the Securities and Exchange Commission,
completely and with the understanding that our actual future results may be materially different from what we expect.
Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to
update the reasons actual results could differ materially from those anticipated in the forward-looking statements,
even if new information becomes available in the future.
Disclaimers
GW Pharmaceuticals plc Investor Presentation 1Nov 2018
GW’s VISION is to be the global leader in prescription
cannabinoid medicines, developing and commercializing
pharmaceutical products which address clear unmet needs
Nov 2018 GW Pharmaceuticals plc Investor Presentation 2
GW Pharmaceuticals
Nov 2018 3
• 14 cannabinoids evaluated in pre-clinical
research
• >80 peer-reviewed publications
• Cannabinoids exported to >35 countries
for research
• >50 Phase 2/3 clinical trials
• >55,000 patient-years of human safety
data
• Sativex® approved in >25 countries (ex-
US) for relief of MS spasticity
• Epidiolex® approved by FDA (DEA
Schedule V)
• Launched in U.S. November 1
• MAA under review by EMA (in EU)
• US commercial launch team in place
• EU commercial infrastructure build
ongoing
• Increased scale commercial
manufacturing in place
RESEARCH DEVELOPMENT COMMERCIALIZATION
20years
experience in researching and
developing novel Rx
cannabinoid medicines
~500 person
in-house
teamwith expertise in
bringing drugs to
regulatory approval in
US and ROW
World Leader in Cannabinoid ScienceGW Pharmaceuticals plc Investor Presentation
Epidiolex® (cannabidiol) oral solution
Treatment-Resistant Epilepsy Overview
• Positive data from all four Phase 3 placebo-controlled trials in initial target orphan
indications of Dravet syndrome and Lennox-Gastaut syndrome (LGS)
• Two Phase 3 studies published in The New England Journal of Medicine and one
Phase 3 study in The Lancet
• Epidiolex approved by FDA on June 25, 2018, moved into Schedule V by DEA
• EMA submission accepted for review, expected decision in Q1 2019
• Novel mechanism of action in epilepsy
• “Real-world” experience in Epidiolex use
- Data from >1,000 patients in compassionate use program show treatment
effect and safety across a range of severe epilepsy types
• Active lifecycle management plan, including additional target orphan indications
Nov 2018 4
Epidiolex® (cannabidiol) is an investigational product outside of the United States of America
GW Pharmaceuticals plc Investor Presentation
Epidiolex launched in
the U.S. on November
1st 2018 – now
available by
prescription
• 3.4 million US patients with epilepsy (~470,000 children)
- ~ One third of patients are pharmacoresistant with seizures persisting
despite multiple antiepileptic drugs (AEDs)
• Childhood-onset epilepsy
- Multiple distinct orphan syndromes, almost none with a specific indicated
therapy
- Many syndromes highly resistant to currently available AEDs
- Seizures continue into adulthood
• Dravet syndrome and LGS represent two of the most difficult-to-treat
epilepsy syndromes
- Multiple seizure types, developmental delay, high risk of SUDEP
- Highly refractory even with current medications, polytherapy generally
required
• Physicians seek new treatment options
- Majority of physicians not satisfied with current therapeutic options
Significant Unmet Need in Epilepsy
GW Pharmaceuticals plc Investor Presentation 5
Sources: Sander JW, Epilepsia. 1993;34(6):1007; 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
3
Nov 2018
Source: 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)
The Epidiolex® Story: IND to FDA Approval in 4 Years
6
Q4 ‘12
First child
treated with
Epidiolex
Q1 ‘14
EAPs
(Expanded
Access
Programs)
initiated
Q2-4 ‘15
3 Phase 3
studies in
Dravet and
LGS started
and enrolled
Q2 ‘14
FDA grants GW
IND. Formal
GW CBD
development
program begins
2016
Positive topline
results in
Dravet & LGS
pivotal studies
Oct 2017
Epidiolex
NDA
submission
#1
2,000
patients have been treated with Epidiolex
Dec 2017
Epidiolex
Europe
submission
Q2 2017
Dravet trial
results
published
in NEJM
Jan 2018
LGS trial
results
published in
The Lancet
June 25, 2018
Epidiolex
approved by
the FDA(Sept. 28: Moved to
DEA Schedule V)
Q1 2018
2nd LGS trial
results
published in
NEJM
Nov 2018 GW Pharmaceuticals plc Investor Presentation
Nov 1 2018
Epidiolex
launched in
the U.S.
• FDA authorized, physician-sponsored expanded access
program (EAP) initiated in early 2014
- Children and young adults with multiple etiologies, all with
treatment-resistant epilepsy
- ~40 physician site EAPs and 6 US state sponsored EAP
programs
- Over 1,000 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
- All Children and Adults with Treatment-Resistant Epilepsies
(Bebin et al, 2017)
- Patients with LGS & Dravet syndrome (Laux et al, 2017)
- For both convulsive and total seizures, median % reductions
and ≥50%, ≥75% and 100% responder rates were consistent
across all time points
- CBD was generally well tolerated, adverse events (AEs) were
consistent with those seen in previous pivotal and expanded
access reports
Epidiolex® Expanded Access Program
GW Pharmaceuticals plc Investor Presentation 7Nov 2018
Patients with LGS and Dravet Syndrome Only
AES 2017 – EAP Median % Reduction in Seizures
Convulsive Seizures Total Seizures
Convulsive Seizures Total Seizures
All Patients with TRE
Epidiolex Clinical Program
Epidiolex® Clinical Program Overview
9
GW makes Epidiolex
available to the most
needy patients
through an
innovative
compassionate use
program
Nov 2018
Ph
ysic
ian
Sp
on
so
red
GW
Sp
on
so
red
Dravet
Syndrome
Lennox-Gastaut
Syndrome
Tuberous
Sclerosis
Complex
Childhood
Epilepsy
Syndromes
2015
Phase 3 (n=120) — Positive
Phase 3 (n=199) — Positive
Phase 3 (n=171) — Positive
Phase 3 (n=225) — Positive
Phase 3 (n=210) — Fully Recruited
Ongoing Treatment Data from ~ 1,000 patients — Other Epilepsies
FDA
Approval
FDA approval on
June 25th, 2018
Rescheduled by
DEA to Schedule V
Launched in the
U.S. on Nov 1, 2018
MAA submission
decision expected
in Q1 2019
2016 2017 2018
NDA MAA
2019
GW Pharmaceuticals plc Investor Presentation
Phase 3 Dravet 2 (Multiple Dose Cohorts) Trial Principal Efficacy Results
GW Pharmaceuticals plc Investor Presentation 10Nov 2018
49P=0.0069 44
P=0.0332
26
0
10
20
30
40
50
60
≥50%
Reduction in Convulsive Seizures Convulsive Seizure Responder Rates
Treatment Period
% r
eduction
% P
atients
CBD 20 mg/kg/day (n=67), CBD 10mg/kg/day (n=67), placebo (n=65)
Source: GW company press release – 26 November 2018
4649
27
0
5
10
15
20
25
30
35
40
45
50
Treatment Period (Primary)
P=0.0095P=0.0299
Phase 3 LGS 2 (Multiple Dose Cohorts) Trial Principal Efficacy Results
GW Pharmaceuticals plc Investor Presentation 11Nov 2018
62
40
25
63
36
11
43
15
3
0
10
20
30
40
50
60
70
≥25% ≥50% ≥75%
*
†
*
‡
‡
*
Reduction in Drop Seizures Drop Seizure Responder Rates
Treatment Period
Media
n %
reduction /
28 d
ays
% P
atients
*p<0.05†p<0.01‡p<0.001
†p<0.01
▪ 5 CBD 20 mg/kg, 3 CBD 10 mg/kg, and 1 placebo patient achieved drop seizure freedom during maintenanceSource: 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
42
47
3740
1719
0
5
10
15
20
25
30
35
40
45
50
Treatment Period(Primary)
Maintenance Period
CBD 20 mg/kg/day (n=76) CBD 10 mg/kg/day (n=73) Placebo (n=76)
†
†
†
†
Phase 3 Safety Profile
• Consistent adverse reaction profile across pivotal trials
• Most common AEs (>10% of patients) in Phase 3 trials include:
- Somnolence, decreased appetite, diarrhea, transaminase elevations, fatigue, malaise,
asthenia, rash, insomnia, sleep disorder and poor-quality sleep, and infections.
- Warnings & Precautions related to hepatocellular injury and somnolence & sedation are
included in the US Prescribing Information
• Low withdrawal rates seen throughout clinical program
• Elevations in hepatic transaminases have been reported, most commonly with
concomitant valproate; none met criteria for serious liver injury; all elevations
resolved–most while on treatment
- Physicians routinely monitor liver function in patients with epilepsy as there are a number
of approved AEDs that are associated with elevations in transaminases
- Monitoring recommendations and lower starting dose are included in US package
insert to help physicians manage potential liver risks
GW Pharmaceuticals plc Investor Presentation 12
Across the Phase 3
program, Epidiolex
was generally well-
tolerated, with most
AEs reported as mild
or moderate
Nov 2018
• Selection of this indication driven by encouraging EAP data
• Single Phase 3 pivotal trial now fully enrolled – data H1 2019
• sNDA H2 2019
• Tuberous Sclerosis Complex (~25,000 US TSC patients with
treatment-resistant seizures)
- TSC is a genetic disorder that results from a mutation in tumor
suppressor genes TSC1 or TSC2 and causes non-malignant tumors to
form in different organs (primarily the brain, eyes, heart, kidney, skin and
lungs)
- Of TSC patients with epilepsy, 70% experience seizure onset in their first
year of life
- Co-morbidities include: cognitive impairment (50%), autism spectrum
disorders (up to 40%), neurobehavioral disorders (over 60%)
3rd Target Indication:Tuberous Sclerosis Complex (TSC)
Nov 2018 GW Pharmaceuticals plc Investor Presentation 13
Source: Tuberous Sclerosis Alliance; Child Neurology Foundation; Infantile Spasms Project
• Rett syndrome is a rare, non-inherited, X-linked neurodevelopmental disorder
- Affects approximately 1 in 10,000 to 15,000 live female
births
- No approved treatments for Rett syndrome
- The condition affects predominantly females and results
in abnormal neuronal development and function in
affected children.
- The symptomatology of Rett is progressive, with early
onset from about 6–18 months of life, followed by a
rapid destructive phase at the age of 1-4 years.
• Clinical evidences from open label investigations in
patients with refractory epilepsy suggest that Epidiolex
may have beneficial effects on cognition, behavior and quality of life, which could benefit patients
with Rett syndrome.
• Single Phase 3 pivotal trial expected to start in Q2 2019
4th Target Indication:Rett Syndrome
Nov 2018 GW Pharmaceuticals plc Investor Presentation 14
Commercialization
Commercial Manufacturing
16
Growing &
Harvesting
Drying & Processing
Crystallisation
(API)
Drug Product
Manufacture
Bottling & Labeling
GMP
Non-GMP
• GW has operated under current Good
Manufacturing Practices (cGMP) licenses
issued outside the US since 2005
• Manufacturing scaled through in-house and
3rd party facilities
• Further increases in scale anticipated in
2018 and beyond to meet anticipated global
demand
• FDA pre-approval inspections completed in
2018 with no Form 483 citations
Extraction
Nov 2018 GW Pharmaceuticals plc Investor Presentation
• Orphan Designation
• 7 years in the US plus expected 6-month pediatric extension (runs concurrent with 5-year exclusivity for new molecular entity)
• 10 years in the EU plus expected 2 years pediatric extension
• Intellectual Property
• Pursuing a strategy to have patents issued to provide protection beyond Orphan Designation
• Current portfolio includes multiple distinct patent families in the treatment of epilepsy and formulations
• To date, 12 issued patents with expiry to 2035
• 6 patents currently listed in the Orange Book (with 2 recent granted patents expected to follow)
• Additional patent applications expected as new data is generated
• Life-cycle management through new indications, formulation improvements, combinations and additional commercial geographies
• Proposed new formulations include liquid, solid dose, intravenous
Epidiolex® Commercial Exclusivity
GW Pharmaceuticals plc Investor Presentation 17Nov 2018
Patent
Protections
Orphan
Exclusivity
Life-Cycle
Management
Epidiolex® Orange Book Listable Patent Portfolio(applications in public domain – several additional patents filed, not yet available through USPTO website)
20Nov 2018
Patent Primary Claim Status
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
mg/kg/day or greater and the purity of the CBD is greater than 98%.
Granted
US 9,949,937 A method of reducing seizure frequency in Dravet syndrome with concomitant CBD and clobazam, wherein the CBD
dose is >10 mg/kg/day or greater and the purity of CBD is greater than 98%.
Granted
US 9,956,183 A method of reducing seizure frequency in LGS or Dravet syndrome with concomitant CBD and clobazam, wherein the
dose of clobazam is reduced and the purity of CBD is greater than 98%.
Granted
US 9,956,186 A method of reducing convulsive seizures in LGS with CBD, wherein the CBD dose is >10 mg/kg/day and the purity of
CBD is greater than 98%.
Granted
US 9,956,185 A method of reducing convulsive seizures in Dravet syndrome with CBD, wherein the CBD dose is >10 mg/kg/day and
the purity of CBD is greater than 98%.
Granted
US 15/449,402 A method of reducing drop seizures in Dravet syndrome with CBD, wherein the dose of CBD is about 20 mg/kg/day and
the purity of CBD is greater than 98%.
Granted
US 15/449,535 A method of reducing drop seizures in LGS with CBD, wherein the dose of CBD is about 20 mg/kg/day and the purity of
CBD is greater than 98%.
Granted
GW Pharmaceuticals plc Investor Presentation
• Epidiolex launched in the U.S. on November 1st, 2018
- Team actively promoting product to physicians
- Extensive communications campaign in place
• In the US we are using our wholly-owned subsidiary, Greenwich Biosciences
Inc., to commercialize our products
• Medical affairs organization in place for over two years, including 15 Medical
Science Liaisons (MSLs)
• U.S. sales organization fully recruited and trained, comprising two national
directors, eight regional managers plus 66 Neurology Account Managers
- ~5,000 target physicians
• Active engagement with U.S. payors ongoing
• Projected weighted average gross price in the first year of $32,500
• U.S. supply chain platform in place
Epidiolex® - U.S. Commercialization Status
GW Pharmaceuticals plc Investor Presentation 19Nov 2018
The Caregiver/Patient Campaign Features Actual EPIDIOLEX Patients w/Documentary Style Videos to bring their stories to life……
Before starting EPIDIOLEX, Cameron was having close to
60 drop seizures per day
Cameron’s Story
Jon was a happy, active child until complications from an operation led to LGS and
uncontrolled Seizures
Jon’s Story
Grace was diagnosed with Dravet Syndrome as in infant, experiencing as many as 400
atonic seizures a day
Grace’s Story
• We expect an outcome to the EMA regulatory review in Q1 2019
• Commercial leadership team consisting of experienced, epilepsy disease
experts fully recruited and in place
• Planning for launches in the five major European markets in 2019
- Significant progress in building out the local country organizations in these 5
markets
- Expected to included 17 sales professionals, 17 MSLs
- Second wave of countries expected to launch in 2020
• Progressing pricing and reimbursement, medical and pre-commercial activities
required to deliver a successful European launch
Epidiolex® - Europe Commercialization Status
GW Pharmaceuticals plc Investor Presentation 21Nov 2018
Additional Programs, Financial
Information and Upcoming Milestones
• Cannabis plant is a unique source of >100
cannabinoid molecules potentially capable of targeting
diseases across therapeutic areas and mechanisms
- TRP channels, adenosine re-uptake, serotonin
receptors, endocannabinoid system
• GW has a deep pipeline of additional cannabinoid
product candidates
• Focus on orphan pediatric neurologic conditions within
epilepsy, autism, NHIE, schizophrenia
• Existing physician relationships through Epidiolex
program
• U.S. development and commercialization rights to
Sativex fully owned by GW; plans underway to
determine FDA regulatory pathway for MS spasticity
GW’s Cannabinoid Platform:A Proprietary Growth Engine Beyond Epidiolex®
Nov 2018 GW Pharmaceuticals plc Investor Presentation 23
• Oromucosal spray of a formulated botanical extract of cannabis that contains the principal cannabinoids delta-9-
tetrahydrocannabinol (THC) and cannabidiol (CBD) in a 1:1 ratio
• Approved in >25 countries outside the United States for the treatment of spasticity due to Multiple Sclerosis (MS).
Product is sold via marketing partners
• Potential for multiple indications
Sativex® - New U.S. Late Stage Pipeline Product
24Nov 2018 GW Pharmaceuticals plc Investor Presentation
- 3 positive Phase 3 trials in MS spasticity
- >10 additional placebo-controlled trials completed in models of peripheral
and central neuropathic pain, other MS symptoms, rheumatoid arthritis
- Planning pivotal program in neuropathic pain/other neurological symptoms
• GW reacquired full rights to develop and commercialize Sativex in U.S.
(Dec 2017)
• Represents a new wholly-owned late-stage U.S. asset
• We expect to evaluate the optimal route to submit a NDA for MS
spasticity. Meeting with FDA expected to take place Q4 18
• CBDV
- Expanded access IND to treat seizures associated with autism in 20 patients underway
- Data on 5 patients to be presented at AES Annual Meeting – Initial data suggest CBDV exhibited AED properties with positive effects on general
autism scales (Source: Barnes AES abstract)
- Investigator-led 100 patient placebo-controlled trial in autism due to commence in Q4 2018
- Open-label study in Rett Syndrome expected to commence in Q1 2019
- Exploring next steps in epilepsy
• Schizophrenia (GWP42003)
- Positive top line results from exploratory Phase 2 placebo-controlled clinical trial in 88 patients with schizophrenia who had
previously failed to respond adequately to first line antipsychotic medications
- Potential for product with novel mechanism distinct from the dopamine D2 receptor augmentation of standard antipsychotics
• Glioblastoma (THC:CBD)
- Positive Phase 2 placebo-controlled data
- THC:CBD + TMZ had an 83% one year survival rate compared with 44% for patients on placebo (TMZ only) (p=0.042)
- Median survival for the THC:CBD group was over 662 days compared with 369 days in the placebo group
- THC:CBD generally well tolerated
Selected Additional Pipeline Candidates
25Nov 2018 GW Pharmaceuticals plc Investor Presentation
Key Financial Data
Nov 2018 GW Pharmaceuticals plc Investor Presentation 26
Clean up
ICON
Cash at 30 Sept 2018 $354.9m
Net cash from October financing $324.2m
Q ending 31 December cash outflow guidance $90m–$100m
Share Capital Current Options Fully Diluted
ADS/m 28.3 1.2 29.5
Upcoming Milestones/Newsflow
GW Pharmaceuticals plc Investor Presentation 27Nov 2018
Expected Timing
EPIDIOLEX REGULATORY
EMA decision Q1 2019
EPIDIOLEX DATA
American Epilepsy Society annual meeting Dec 2018
Phase 3 tuberous sclerosis complex trial data H1 2019
Presentation of Dravet 2 data at medical meeting H1 2019
EPIDIOLEX COMMERCIAL
U.S. launch update Q1 2019
European product launches 2019
PIPELINE
FDA meeting to determine Sativex NDA requirements Q4 2018
Epidiolex in Rett syndrome start H1 2019
CBDV investigator-led placebo control trial in autism start Q4 2018
CBDV open label in Rett syndrome trial start Q4 2018
Thank You
GW Pharmaceuticals plc
NASDAQ: GWPH
Stephen Schultz, VP Investor Relations
+ 1 401-500-6570