Investor Presentation

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1 Investor Presentation 2018 GW Pharmaceuticals plc All Rights Reserved

2 Disclaimers 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, 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 of Sativex and Epidiolex. 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 20-F 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. GW Pharmaceuticals plc Investor Presentation 1

3 GW S VISION is to be the global leader in prescription cannabinoid medicines, developing and commercializing pharmaceutical products which address clear unmet needs GW Pharmaceuticals plc Investor Presentation 2

4 GW Pharmaceuticals RESEARCH DEVELOPMENT COMMERCIALIZATION 19+ years experience in researching and developing novel Rx cannabinoid medicines >500 person in-house team with expertise in bringing drugs to regulatory approval in US and ROW 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 NDA & MAA applications accepted for review US commercial launch preparations well advanced EU commercial infrastructure build ongoing Increased scale commercial manufacturing in place for Epidiolex launch GW is a World Leader in Cannabinoid Science GW Pharmaceuticals plc Investor Presentation 3

5 Epidiolex (cannabidiol or CBD) Treatment-Resistant Epilepsy Overview Extensive CBD pre-clinical research since 2007 showing in vivo and in vitro efficacy Positive data from three Phase 3 placebo-controlled trials in initial target orphan indications of Dravet syndrome and Lennox-Gastaut syndrome (LGS) Publication of Dravet Phase 3 trial results in The New England Journal of Medicine and first LGS Phase 3 results in The Lancet NDA submission accepted for priority review with planned PDUFA goal date of June 27, 2018 EMA submission accepted for review, expected decision in Q Novel mechanism of action in epilepsy - CBD does not act through cannabinoid (CB) receptors or sodium channels - Prime targets are GPR55 and adenosine reuptake, TRPV1 Significant patient and physician interest with over 2,000 patients now treated with Epidiolex Real-world experience in Epidiolex use - Data from >500 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 Epidiolex (cannabidiol) is an investigational product and not approved for any indication in any country GW Pharmaceuticals plc Investor Presentation 4

6 Significant Unmet Need in Epilepsy 2.2 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 Sources: Sander JW, Epilepsia. 1993;34(6):1007; Picot et al, 2008 ; Kwan P, Brodie MJ. N Engl J Med. 2000;342: ; Kwan P, Brodie MJ, CNS Spectr. 2004;9(2):110 3 Picot et al, 2008; Kwan P, Brodie MJ. N Engl J Med. 2000;342: ; Kwan P, Brodie MJ, CNS Spectr. 2004;9(2):110, Morgan Stanley research (2015) GW Pharmaceuticals plc Investor Presentation 5

7 The Epidiolex Story: 3.5 Years from IND to NDA Submission Q4 12 First child treated with Epidiolex Q2 14 FDA grants GW IND Formal GW CBD development program begins Q Phase 3 studies in Dravet and LGS started and enrolled Q LGS 1 topline results TSC Phase 3 trial started Q AES: Dravet 1 LGS 1 Q Dravet trial results published in NEJM Dec 2017 Epidiolex MAA submission Feb 2017 Epidiolex MAA accepted for review Q nd LGS trial results published in NEJM #1 Q1 14 EAPs (Expanded Access Programs) initiated Q4 14 Dravet 1 Part A started Q Dravet 1 topline results Q LGS 2 topline results Q AAN: LGS 2 Oct 2017 Epidiolex NDA for Dravet and LGS Dec 2017 NDA accepted for review: mid-2018 PDUFA Jan 2018 LGS trial results published in The Lancet 2,000 patients have been treated with Epidiolex GW Pharmaceuticals plc Investor Presentation 6

8 Epidiolex Expanded Access Program FDA authorized, physician-sponsored expanded access program (EAP) initiated in early Children and young adults with multiple etiologies, all with 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 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 AES 2017 EAP Median % Reduction in Seizures All Patients with TRE Convulsive Seizures Total Seizures Patients with LGS and Dravet Syndrome Only Convulsive Seizures Total Seizures GW Pharmaceuticals plc Investor Presentation 7

9 Epidiolex Clinical Program

10 Physician Sponsored GW Sponsored Epidiolex Phase 3 Program Overview Dravet Syndrome Lennox-Gastaut Syndrome Tuberous Sclerosis Complex Phase 3 (n=120) Positive Phase 3 (n=186) Fully Recruited NDA MAA Phase 3 (n=171) Positive Phase 3 (n=225) Positive Phase 3 (n=210) Recruiting PDUFA NDA submission accepted for Priority Review 27 June 18 PDUFA MAA submission accepted Feb 18 with expected decision in Q Childhood Epilepsy Syndromes Ongoing Treatment Data from >1,000 patients Other Epilepsies >97% of completers continued into Open Label Extension GW Pharmaceuticals plc Investor Presentation 9

11 Median % Reduction Median % Reduction Phase 3 Dravet 1 Syndrome Trial Principal Efficacy Results p= Convulsive Seizures p= p= Total Seizures p= Treatment Period Maintenance Period 0 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 GW Pharmaceuticals plc Investor Presentation 10

12 Median % Reduction Median % Reduction Phase 3 LGS 1 (Single Dose Cohort) Trial Principal Efficacy Results p= Drop Seizures p= p= Total Seizures p= Treatment Period Maintenance Period 0 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 GW Pharmaceuticals plc Investor Presentation 11

13 Median % reduction / 28 days % Patients Phase 3 LGS 2 (Multiple Dose Cohorts) Trial Principal Efficacy Results Reduction in Drop Seizures Treatment Period (Primary) Maintenance Period p< * 62 Drop Seizure Responder Rates Treatment Period * GW Pharmaceuticals plc Investor Presentation % 50% 75% 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 * 11 * p<0.05 p<0.01 p<

14 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 Serious AEs consistent with the pattern of common AEs 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 and typically run routine liver tests Across the Phase 3 program, Epidiolex was generally welltolerated, with most AEs reported as mild or moderate GW Pharmaceuticals plc Investor Presentation 13

15 3rd Target Indication: Tuberous Sclerosis Complex (TSC) Selection of these indications driven by encouraging EAP data Tuberous Sclerosis Complex (~25,000 US TSC patients with treatment resistant seizures) - Single Phase 3 pivotal trial underway - TSC is a genetic disorder that results from a mutation in tumor suppressor genes 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%) Sources: Tuberous Sclerosis Alliance; Child Neurology Foundation; Infantile Spasms Project GW Pharmaceuticals plc Investor Presentation 14

16 Commercialization

17 Commercial Manufacturing Growing & Harvesting Drying & Processing Extraction Crystallisation (API) Drug Product Manufacture Bottling & Labeling Non-GMP GMP GW has operated under commercial Good Manufacturing Practices (GMP) licenses issued outside the US since 2005 Manufacturing scaled through in-house and 3 rd party facilities Further increases in scale anticipated in 2018 and beyond to meet anticipated global demand Regulatory evaluation same as small molecule, not botanical FDA pre-approval inspections completed with no form 483 citations GW Pharmaceuticals plc Investor Presentation 16

18 Epidiolex Commercial Exclusivity Orphan Designation 7 years in the US plus expected 6-month pediatric extension 10 years in the EU plus expected 2 years pediatric extension Intellectual Property Pursuing a patent strategy designed to have patents issued to protect lead drug candidate and other pipeline candidates beyond Orphan Designation Current portfolio includes multiple distinct patent families in the treatment of epilepsy and formulations To date, 8 patents granted & 2 Notices of Allowance issued by the USPTO A number of these patents expected to be listed in the Orange Book 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 Orphan Exclusivity Patent Protections Life-Cycle Management GW Pharmaceuticals plc Investor Presentation 17

19 Epidiolex Patent Portfolio (applications in public domain numerous patents filed and not yet available through USPTO website) Patent Primary Claim Status US 9,066,920 A method of treating partial seizure comprising administering CBD at a daily dose of at least 400mg. Granted 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 mg/kg/day or greater and the purity of the CBD is greater than 98%. US 9,949,937 US 9,956,183 US 9,956,186 US 9,956,185 US 15/449,402 US 15/449,535 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%. 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%. A method of reducing convulsive seizures in LGS with CBD, wherein the CBD dose is 10 mg/kg/day or greater and the purity of CBD is greater than 98%. A method of reducing convulsive seizures in Dravet syndrome with CBD, wherein the CBD dose is 10 mg/kg/day or greater and the purity of CBD is greater than 98%. A method of reducing drop seizure frequency in treatment-resistant childhood-onset epilepsy with CBD, wherein the CBD dose is between 5-35 mg/kg/day and the purity of CBD is greater than 98%. A method of reducing drop seizure frequency in a patient with Lennox-Gastaut syndrome, comprising administering to 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 Granted Granted Granted Granted Granted NoA NoA NoA: Notice of Allowance received by Company GW Pharmaceuticals plc Investor Presentation 18

20 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) At the time of NDA approval, FDA recommend the appropriate lower schedule based on the abuse liability data submitted as part of the NDA Following NDA approval (if received), DEA has up to 90 days to issue an interim final rule (IFR) to reschedule, after which Epidiolex can be marketed and dispensed GW is aiming for Epidiolex to be placed into Schedule IV - MARINOL (synthetic THC in sesame oil) Schedule III (1) Assessment of the Abuse Potential of Cannabidiol in Recreational Polydrug Users: A Randomized, Double- Blind, Controlled Trial, Sommerville et al, 2017, Greenwich Biosciences, Inc. GW Pharmaceuticals plc Investor Presentation 19

21 Epidiolex Commercialization Status Global commercialization rights to Epidiolex fully-owned by GW Pharmaceuticals Group companies In the US we will use our subsidiary, Greenwich Biosciences Inc., to commercialize our products US Launch Preparations - Full commercial leadership team in place - Finalizing build-out of experienced team of medical affairs professionals, marketing and market access/payor experts, many of whom have strong epilepsy knowledge and experience - Active discussions in U.S. with a wide variety of payors and insurance programs - High-touch patient, payer and physician communication, education and distribution model EU commercial footprint now in place in five major European markets GW Pharmaceuticals plc Investor Presentation 20

22 Additional Programs, Financial Information and Upcoming Milestones

23 GW s Cannabinoid Platform: A Proprietary Growth Engine Beyond Epidiolex Cannabis plant is a unique source of >100 cannabinoid molecules 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 reacquired from Otsuka, plans underway to commence U.S. pivotal trial in MS spasticity GW Pharmaceuticals plc Investor Presentation 22

24 CBDV Program CBDV in Autism Spectrum Disorders - Pre-clinical research shows promising signals on cognitive/social endpoints as well as repetitive behavior - A physician-led, FDA authorized, expanded access IND to treat seizures associated with autism in 10 patients underway - An investigator-led 100 patient placebo-controlled trial in autism is also due to commence in Q Rett syndrome - Orphan Drug Designation from FDA for CBDV for the treatment of Rett syndrome - Open-label study in Rett Syndrome expected to commence in Q Phase 2 placebo-controlled trial expected to commence Q CBDV in epilepsy (GWP42006) - CBDV looks to be differentiated from CBD in four key ways: efficacy profile in seizure models, metabolic profile, pharmacological profile and has different physico-chemical characteristics - CBDV has shown anti-epileptic properties across a range of in vitro and in vivo animal models of epilepsy - Phase 2a placebo-controlled trial (n=162) in adult patients with partial-onset epilepsy failed to differentiate from placebo on the primary endpoint as both arms showed reductions in focal seizures of approximately 40% - CBDV was generally well-tolerated with the majority of CBDV patients experiencing adverse events of mild or moderate severity - GW will continue to explore potential development opportunities for CBDV in the field of epilepsy GW Pharmaceuticals plc Investor Presentation 23

25 Recurrent Glioblastoma Multiforme (GBM) Program THC:CBD investigational product candidate for the treatment of GBM - Orphan Drug Designation from FDA GBM is the most common and most aggressive type of malignant primary brain tumor 1,2 - Typically patients succumb to the disease approximately 15 months after diagnosis - Treatment for GBM remains challenging and additional safe and effective long-term treatment options are needed - Standard treatment is surgical resection, radiotherapy and concomitant adjunctive chemotherapy Phase 2 study completed - 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 3 - THC:CBD was generally well tolerated with treatment emergent adverse events leading to discontinuation in two patients in each group - The most common adverse events (three patients or more and greater than placebo) were vomiting (75%), dizziness (67%) nausea (58%), headache (33%), and constipation (33%) 1. National Brain Tumor Society web site. "Tumor Types." 2. American Brain Tumor Association. (2012) "Glioblastoma and Malignant Astrocytoma." 3. Data reported by GW Pharmaceuticals press release 7 February 2017 GW Pharmaceuticals plc Investor Presentation 24

26 Sativex - New U.S. Late Stage Pipeline Product Oromucosal spray of a formulated 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 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 GW Pharmaceuticals plc Investor Presentation 25

27 Key Financial Data Clean up ICON Cash at 31 March 2018 H cash outflow guidance ($10-$20m Capex for manufacturing expansion $487.2m $120m $140m Share Capital Current Options Fully Diluted ADS/m GW Pharmaceuticals plc Investor Presentation 26

28 Upcoming Milestones/Newsflow Expected Timing EPIDIOLEX REGULATORY NDA PDUFA June 27, 2018 EMA decision Q DEA rescheduling Q U.S. launch H EPIDIOLEX DATA 2nd LGS Phase 3 publication H Phase 3 tuberous sclerosis complex trial data H nd Phase 3 Dravet syndrome trial data H EPIDIOLEX EXCLUSIVITY Track One patent decisions Ongoing PIPELINE CBDV investigator-led placebo control trial in autism start Q CBDV open label in Rett syndrome trial start Q Sativex IND for pivotal Phase 3 study H CBDV Phase 2 Rett syndrome trial start Q GW Pharmaceuticals plc Investor Presentation 27

29 Thank You GW Pharmaceuticals plc NASDAQ: GWPH Stephen Schultz, VP Investor Relations