GW Pharmaceuticals plc. Investor Presentation August 2014

Transcription

1 GW Pharmaceuticals plc Investor Presentation August 2014

2 Forward Looking Statements and Disclaimer This presentation contains forward-looking statements. Some of the matters discussed concerning our operations and financial performance include estimates and forward-looking statements within the meaning of the Securities Act and the Exchange Act. These forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that could cause our actual results of operations, financial condition, liquidity, performance, prospects, opportunities, achievements or industry results, as well as those of the markets we serve or intend to serve, to differ materially from those expressed in, or suggested by, these forward-looking statements. These forward-looking statements are based on assumptions regarding our present and future business strategies and the environment in which we expect to operate in the future. These statements are based on our current expectations and projections about future events and will be regarded by terminology such as believe, may, will, estimate, continue, anticipate, intend, expect and similar words are intended to identify estimates and forward-looking statements. Estimates and forward-looking statements speak only at the date they were made, and we undertake no obligation to update or to review any estimate and/or forward-looking statement because of new information, future events or other factors, except as required by applicable law. Estimates and forward-looking statements involve risks and uncertainties and are not guarantees of future performance. Our future results may differ materially from those expressed in these estimates and forward-looking statements. Because of these uncertainties, you should not make any investment decision based on these estimates and forward-looking statements. Although we believe that our plans, intentions and expectations are reasonable, we may not achieve our plans, intentions or expectations. You should read this presentation and the documents that we reference and have filed as exhibits to the Annual Report on Form 20-F that we have filed with the Securities and Exchange Commission completely and with the understanding that our actual future results, levels of activity, performance and achievements may be different from what we expect and that these differences may be material. We qualify all of our forward-looking statements by these cautionary statements. 2

3 GW Pharma Overview World leading position in development of plant-derived cannabinoid therapeutics Proprietary cannabinoid product platform targeting broad range of disease areas Commercialized product, Sativex Approved in 26 countries (ex-u.s.) for MS Spasticity Cannabinoid orphan program in pediatric epilepsy FDA Orphan Drug designation for Epidiolex in Dravet and Lennox-Gastaut syndromes Promising data from Expanded Access INDs in treatment-resistant children/young adults IND open in Dravet, Fast Track designation, Phase 2/3 trial due to commence H2 14 GW retains global commercial rights Two Phase 3 opportunities in U.S. for Sativex Cancer Pain: Phase 3 data expected towards end Trials funded by U.S. partner MS Spasticity: Phase 3 IND open; Phase 3 expected to commence early 2015 Promising clinical stage cannabinoid product pipeline Orphan (glioma/nhie) and other diseases (Type 2 diabetes, ulcerative colitis) 3

4 Our Proprietary Cannabinoid Product Platform: Overview Cannabis plant is unique source of >70 cannabinoid molecules Only THC is known to cause psychoactive effects Cannabinoids capable of targeting diseases across therapeutic areas Endocannabinoid system, TRP channels, adenosine uptake, serotonin receptors GW s novel proprietary plant chemotypes target selected cannabinoids CBD, THC, CBC, CBG, CBN, THCV, CBGV, CBDV, THCA, CBDA etc In-house formulation, processing, manufacturing and regulatory expertise Exclusivity via 46 patent families, know-how, complex formulations Specialized field provides substantial barriers to entry Overlay of 24 Sativex Chromatograms 4

5 Unpartnered GW owns global rights Partnered Our Pipeline PHASE 3 DATA AROUND END 2014 PHASE 3 IND OPEN, SPA ONGOING PHASE 2/3 IND, FAST TRACK, ORPHAN DESIGNATION ORPHAN DESIGNATION PHASE 2 TRIAL TO COMMENCE END 2014 / EARLY 2015 PHASE 1b/2a TRIAL UNDERWAY NEXT STEP: APPLY FOR ORPHAN DESIGNATION PHASE 2b TRIAL UNDERWAY PHASE 2 DATA H PHASE 2a TRIAL UNDERWAY 5

6 Epilepsy Orphan Drug Program

7 Overview 6 years of GW pre-clinical research into cannabinoids in epilepsy Significant interest from U.S. specialists and patient groups in Epidiolex Epidiolex (CBD - cannabidiol) FDA expanded access INDs authorized for use of Epidiolex in ~395 children Initial 12 week clinical effect data disclosed on 27 patients Safety data disclosed on 62 patients Rolling data during H and 2015 as additional patients enter treatment FDA formal orphan development Orphan and Fast Track designations and Sponsor IND granted in Dravet syndrome Dravet syndrome Phase 2/3 trial expected to commence H Orphan designation in Lennox-Gastaut syndrome GWP42006 (CBDV - cannabidivarin) Phase 1 complete, Phase 2 expected to commence end 2014/early

8 Intractable Childhood Epilepsy US CHILDREN WITH EPILEPSY PREVALENCE OF 6.3/1000 DIAGNOSED 466,000 1 PHARMACORESISTANT EPILEPTICS 2 SEIZURES THAT PERSIST, DESPITE MULTIPLE AED TREATMENT 3 74 MILLION US CHILDREN 20% TARGET US POPULATION REFRACTORY EPILEPSY COMPOSED OF CLUSTER OF ELECTROCLINICAL SYNDROMES 93,000 Most syndromes have orphan sized populations Strategy is initially to target NDAs in Dravet and L-G syndromes Severe infantile-onset, drug-resistant syndromes Expanded access IND patients include range of syndromes [1] Russ et al, 2012 [2] Wirrel 2013 [3] Picot et al, 2008 [4] Dravet et al, 2012 [5] Trevathan et al, 1997 DRAVET SYNDROME UP TO 5% OF ALL CHILDHOOD EPILEPSIES IN THE FIRST YEAR OF LIFE 4 5,440 US and 6,710 EU patients LENNOX-GASTAUT SYNDROME 3 TO 4% OF CHILDHOOD EPILEPSY k US and 23-31k EU patients ADDITIONAL RELATED REFRACTORY EPILEPSIES 2. Intractable childhood epilepsy with generalized tonic-clonic seizures Infantile seizures with variable foci Doose syndrome Infantile spasms 8

9 Epidiolex Expanded Access INDs Physician Reported Treatment Effect Data

10 Expanded Access Studies Expanded access studies are uncontrolled, carried out by individual investigators, and not typically conducted in strict compliance with Good Clinical Practices, all of which can lead to a treatment effect which may differ from that in placebo-controlled trials. Data from these studies provide only anecdotal evidence of efficacy for regulatory review, contain no control or comparator group for reference and are not designed to be aggregated or reported as study results. Moreover, data from such small numbers of patients may be highly variable. Such information may not reliably predict data collected via systematic evaluation of the efficacy in company-sponsored clinical trials. Reliance on such information may lead to Phase 2 and 3 clinical trials that are not adequately designed to demonstrate efficacy and could delay or prevent GW s ability to seek approval of Epidiolex. Expanded access programs may provide supportive safety information for regulatory review. Physicians conducting these studies may use Epidiolex in a manner inconsistent with the protocol, including in children with conditions different from those being studied in GW-sponsored trials. Any adverse events or reactions experienced by subjects in the expanded access program may be attributed to Epidiolex and may limit GW s ability to obtain regulatory approval with labeling that GW considers desirable, or at all. 10

11 Introduction Expanded Access IND Data Treatment-resistant children and young adults (mean age 10.5 years) Epidiolex added to existing meds. Patients on average 2.7 other AEDs Clinical effect data presented on all 27 patients with 12 week data available: Wide range of treatment-resistant epilepsies Data includes all seizures (convulsive and non-convulsive) NYU Dr Devinsky (n=18), UCSF Dr Cilio (n=9) Additional analyses presented for largest sub-group - Dravet syndrome (n=9) Data presented on convulsive seizures only - FDA type of seizures for primary efficacy Treatment effect calculated consistent with FDA's recommended endpoint: % change in average 4 week seizure frequency throughout the 12 week treatment period compared with 4 week baseline Total safety database of 62 patients (35 additional patients from NY, SF and Boston who have yet to reach 12 weeks of treatment) 11

12 % of Patients All Patients with 12 Week Data (n=27) Responders - Total Seizures 70% 60% 50% 40% 48% (n=13) 41% (n=11) 30% 20% 22% (n=6) 15% (n=4) 10% 0% 50% >= 50% Responders Responder >= 70% 70% Responders >= 90% 90% Responders Seizure free Free at at 12 Weeks weeks Seizure free data = seizure free at weeks 8-12 Total Seizures = Convulsive and Non-Convulsive 12

13 % of Patients All Patients with 12 Week Data (n=27) Response Rates Total Seizures 70% 60% 50% 40% 37% (n=10) 30% 20% 15% (n=4) 22% (n=6) 15% (n=4) 11% (n=3) 10% 0% worse Worse % % decrease % % decrease % % decrease % % decrease Total Seizures = Convulsive and Non-Convulsive 13

14 % Seizure Reduction All Patients with 12 Week Data (n=27) % Reduction in Total Seizure Frequency 0% -10% mean median -20% -30% -40% -50% -60% -70% -80% -90% 44% 42% -100% FDA efficacy endpoint average 4 week seizure frequency over 12 weeks vs baseline Total Seizures = Convulsive and Non-Convulsive 14

15 % of Patients Dravet Syndrome Patients (n=9) Responders - Total Convulsive Seizures 70% 60% 56% (n=5) 50% 40% 30% 44% (n=4) 33% 33% (n=3) (n=3) 20% 10% 0% >= 50% Responders >= 70% Responders >= 90% Responders Seizure free Free at 12 Week weeks 12 FDA Assessment of Primary Efficacy in Dravet Convulsive Seizures Only Seizure free data = seizure free weeks

16 % of Patients Dravet Syndrome Patients (n=9) Response Rates - Convulsive Seizures 70% 60% 50% 40% 30% 20% 10% 11% (n=1) 11% (n=1) 22% (n=2) 22% (n=2) 33% (n=3) 0% worse Worse % % decrease % % decrease % % decrease % % decrease FDA Assessment of Primary Efficacy in Dravet Convulsive Seizures Only 16

17 % Seizure Reduction Dravet Syndrome Patients (n=9) % Reduction in Convulsive Seizures 0% -10% mean median -20% -30% -40% -50% -60% -70% -80% -90% 52% 63% -100% FDA efficacy endpoint average 4 week seizure frequency over 12 weeks vs baseline 17 FDA Assessment of Primary Efficacy in Dravet Convulsive Seizures Only

18 Safety Data (62 patients, 122 patient months treatment) 81% (50 patients) reported any adverse event, 80% mild or moderate Most common AEs all causes (10% or more of patients) - Somnolence 40% - Decreased appetite 11% - Fatigue 26% - Increased appetite 10% - Diarrhoea 16% No withdrawals due to AEs 1 withdrawal due to lack of clinical effect plus additional 2 patients being gradually withdrawn from treatment after 12 weeks due to lack of clinical effect Serious AEs reported in 7 patients (incl 1 death due to SUDEP). None deemed related to Epidiolex 18

19 Expanded Access IND Data Conclusions Promising signals of efficacy, especially in Dravet syndrome High proportion show >50% reduction in seizure frequency Seizure freedom in a portion of responders 80% of adverse events were mild or moderate; No withdrawals due to adverse events Data support decision to advance Epidiolex into formal development programs in pediatric epilepsy 19

20 CBDV GW Physician Sponsored EPIDIOLEX GW Sponsored Epilepsy: Expected Clinical Program Dravet Syndrome FDA Orphan Designation Awarded Phase 2/3 IND Open Ph 2 Part A (n=30) Ph 3 Part B (n=80) Phase 3 Lennox-Gastaut Syndrome FDA Orphan Designation Awarded Phase 3 IND Phase 3 Phase 3 Childhood Epilepsy Syndromes INDs ~395 patients Treatment Data Dravet / LGS / Other intractable epilepsies Refractory Epilepsies Phase 1 Complete Phase 2 20

21 Sativex for Cancer Pain and MS Spasticity, and other Pipeline Candidates

22 Sativex in Advanced Cancer Pain Overview Add-on treatment of persistent pain in people with advanced cancer who experience inadequate pain relief from optimized chronic opioid therapy 420k patients in the U.S. Substantial body of positive Phase 2 data Phase 2a study (n=177) published - Journal of Pain & Symptom Management Phase 2b study (n=360) published - Journal of Pain Long term extension study published - Journal of Pain & Symptom Management Phase 3 trials employ same key features as Phase 2b Patient population / 5-week treatment duration / Endpoint: 0-10 Pain NRS First two identical Phase 3 trials intended to form basis of NDA initial top-line data expected towards end of 2014 n = 380 per trial Third Phase 3 trial also underway not intended to be required for NDA All cancer pain trials and other U.S.-targeted studies funded by Otsuka 22

23 SCREENING RANDOMISATION END OF TREATMENT EXTENSION STUDY Two Core Phase 3 Trials: Trial Design (n=380) 35 DAYS 5-14 DAYS 14 DAYS n=190 Opioid + Sativex 3-10 sprays/day 5-14 days to obtain 3 day baseline period n=190 Opioid + Placebo 3-10 sprays/day Primary Efficacy Analysis Key Secondary Endpoints Change in Pain 0-10 NRS: Cumulative Proportion of Responders Analysis Sleep disturbance, opioid consumption, constipation Top-line Phase 3 data expected towards end of

24 Sativex in MS Spasticity Overview Approved in 26 countries (principally in EU) Indication: Treatment of MS spasticity in patients who have failed to respond to other anti-spasticity medication ECTRIMS 2013 Congress 70% in-market sales volume growth (Q vs Q3 2013) Phase 3 IND opened with FDA SPA process ongoing for single additional Phase 3 trial Subject to SPA, Phase 3 trial expected to commence early 2015 Attractive commercial opportunity in the U.S. >400,000 MS patients in the US - 80% affected by spasticity Current oral treatments afford only partial relief with unpleasant side effects 24

25 Other Pipeline Candidates: Significant Clinical Program Orphan Glioma Phase 1b THC:CBD trial underway (in recurrent glioblastoma) Safety cohort data expected in 2014 NHIE Expected next steps: Orphan designation application and IND Non-Orphan Ulcerative Colitis Phase 2 trial of GWP42003 extract due to report data H (n=62) Type 2 Diabetes Positive data from Phase 2a trial for GWP42004 GWP42004 Phase 2b dose ranging trial underway Schizophrenia GWP42003 Phase 2a trial underway 25

26 Financial Information

27 Key Financial Data $m Cash at 30 June 2014 ~288 Projected cash spend for year ended 30 Sept 2014 Opex and Capex no change to existing guidance (41) Share Capital Current Options Fully Diluted ADS/m Ordinary shares/m

28 Anticipated Newsflow Epidiolex in Orphan Epilepsy Timing Expanded access IND treatment data ongoing GW-sponsored Dravet Phase 2/3 trial start H GW-sponsored LGS IND H nd Phase 3 Dravet and 2 Phase 3 LGS trials Early 2015 GWP42006 (CBDV) Phase 2 trial start End 2014/Early 2015 Sativex for Cancer Pain Initial top-line Phase 3 trial data Around end 2014 Sativex for MS Spasticity US Phase 3 SPA H US Phase 3 trial start Early 2015 Pipeline Phase 2 Ulcerative Colitis trial data H Phase 1b Glioma trial safety cohort data 2014 Phase 2b GWP42004 Type 2 Diabetes trial completion H Phase 2a GWP42003 Schizophrenia trial completion H

29 Investment Highlights Commercialized product, Sativex Validated development and regulatory pathway 19,000 patient-years of safety data Approved in 26 countries (ex-u.s.) for MS spasticity Orphan program in pediatric epilepsy Promising data from Expanded Access INDs FDA formal development plans advancing rapidly GW retains global commercial rights Two significant near term Phase 3 U.S. opportunities for Sativex Anticipate cancer pain initial Phase 3 top-line results towards end of 2014 MS spasticity Phase 3 expected to commence early 2015 Deep product pipeline across multiple therapeutic targets Type 2 diabetes, ulcerative colitis, schizophrenia, cancer Unique global position in cannabinoids with substantial barriers to entry 29

30 GW Pharmaceuticals plc NASDAQ: GWPH AIM: GWP Stephen Schultz, VP Investor Relations