Jefferies 2015 Health Care Conference

Transcription

1 Jefferies 2015 Health Care Conference NASDAQ: ARNA Craig M. Audet, PhD J u n e 1, Sr. VP, Operations & Head of Global Regulatory Affairs

2 Forward-Looking Statements This presentation includes forward-looking statements that involve a number of risks and uncertainties, including statements about our and others focus, mission, goals, strategy, plans, timelines, milestones and expectations; the potential, opportunity, significance, value, safety, efficacy, indication, research and development (R&D), advancement, differentiation, status and related timing, results, regulatory activities and commercialization with respect to BELVIQ or lorcaserin, R&D programs and GPCR drugs in general; need for new treatments, addressing such need and impacting lives; commercializing BELVIQ, including with respect to focus, strategy, pricing, reimbursement, sales/prescriptions, supply, marketing, sales force, and outreach and awareness; collaborations, including related strategy, goals, progress, potential, activities, commitment, revenues, payments and expectations; financial guidance; intellectual property; our ability to discover and develop compounds and supply and commercialize drugs; and other statements that are not historical facts, including statements that may include words such as may, will, intend, plan, expect, potential or other similar words. For such statements, we claim the protection of the Private Securities Litigation Reform Act of Actual events or results may differ materially from expectations, and you are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the time they were made. Factors that could cause actual results to differ materially from such statements include, without limitation, risks related to commercializing drugs, including regulatory, manufacturing, supply and marketing issues and BELVIQ s or lorcaserin s availability and use; cash and revenues generated from BELVIQ; our revenues are based in part on estimates, judgment and accounting policies, and incorrect estimates or disagreement regarding estimates or accounting policies may result in changes to guidance or previously reported results; the timing and outcome of regulatory review is uncertain, and lorcaserin may not be approved for marketing when expected or ever in combination, for another indication or using a different formulation or in any other territory for any indication; regulatory decisions in one territory may impact other regulatory decisions and business prospects; reimbursement and pricing decisions; risks related to relying on collaborative arrangements; payments, if any, from collaborators; the entry into or modification or termination of collaborative arrangements; unexpected or unfavorable new data; nonclinical and clinical data is voluminous and detailed, and regulatory agencies may interpret or weigh data differently and reach different conclusions than we or others, request additional information, have additional recommendations or change their guidance or requirements; information related to R&D may not meet regulatory requirements or otherwise be sufficient for (or we or a collaborator may not pursue) further R&D, regulatory review or approval or continued marketing; our and third parties intellectual property rights; the timing, success and cost of R&D; study and trial results are subject to different interpretations and may not be predictive of future results; trials and studies may not proceed at the time or in the manner expected or at all; having adequate funds; and satisfactory resolution of litigation or other disagreements. Additional factors that could cause actual results to differ materially from those stated or implied by our forward-looking statements are disclosed in our SEC filings. We disclaim any intent or obligation to update these forward-looking statements, other than as may be required under applicable law. PROPRIETARY 2

3 Arena s Mission Embracing the Challenge of Improving Health by Bringing Innovative Medicines Targeting G Protein-Coupled Receptors to Patients PROPRIETARY 3

4 Arena: Embracing Innovation; Impacting Lives Our STRATEGY Discover, Develop and Commercialize Novel Oral Compounds Our FOCUS Targeting G Protein-Coupled Receptors Technologies and Expertise Our PORTFOLIO Commercial First-in-Class Clinical Stage Development Programs BELVIQ Lorcaserin HCI for Chronic Weight Management LORCASERIN Lifecycle Management PIPELINE Internally Discovered, Novel Drug Candidates PROPRIETARY 4

5 GPCR Development-Stage Programs PROGRAM/INDICATION IND Phase 1 Phase 2 Phase 3 NDA Phase 4 Once-Daily Formulation LORCASERIN LIFECYCLE MANAGEMENT* CLINICAL STAGE PIPELINE* Smoking Cessation Co-Admin w/phentermine CVOT/Conversion to T2D/MACE+ Ralinepag - Vascular (PAH) APD334 - Autoimmune (IBD) APD371 - Pain & Fibrotic Disease Temanogrel - Thrombotic Diseases Nelotanserin - Neurological Diseases Development Expense Shared or Funded by Collaborators Collaboration with Ildong for South Korea Collaboration with Roivant Preclinical GPCR Research Targets Include CV, CNS and Metabolic *See full presentation for program status PROPRIETARY 5

6 BELVIQ for Chronic Weight Management First-in-class molecule discovered by Arena MOA: Selective activation of 5-HT 2C receptors in hypothalamus are believed to decrease food consumption and promote satiety Large addressable patient population Overweight (BMI 27) with at least one weightrelated medical condition Obese (BMI 30) Launched in US by Eisai in June 2013 Launched in S. Korea by Ildong in February 2015 PROPRIETARY 6

7 Eisai Collaboration BELVIQ sales force 90 internal and 230 contract sales representatives promoting BELVIQ focusing on high-prescribers Eisai marketing program Pay no more than $75 savings card Drive patient demand with focus on increasing prescription refill rates Increase access by improving insurance coverage Eisai s goal Make BELVIQ the weight loss agent of choice of physicians and obese and overweight patients PROPRIETARY 7

8 BELVIQ Launched in South Korea Arena received $3.0 million milestone payment in 1Q15 Arena net sales of $2.2 million in 1Q15 ~12,770 one-month prescriptions through February 24 - March 31 Ildong estimates 617,044 tablets were prescribed in April; ~10,284 one-month prescriptions PROPRIETARY 8

9 Steady Prescription Growth BELVIQ QUARTERLY TRx US South Korea 110K 143K 148K 182K 44K 59K 77K Q3 Q4 Q1 Q2 Q3 Q4 Q ~865K TRxs Filled as of 5/22/15 PROPRIETARY 9

10 BELVIQ ROW Update Mexico & Brazil Review ongoing Taiwan Bridging study (6 months) needed; study began enrolling in April Study is intended to enable filing for marketing approval once completed Israel Review ongoing European Union Meeting requests filed with MHRA & MPA Meetings expected in 3Q15 Resubmission planned for end of March 2016 PROPRIETARY 10

11 BELVIQ Lifecycle Management Update BELVIQ XR May provide additional market differentiation Increased patient convenience and compliance NDA submission planned for later this year Coadministration with Phentermine Meeting request filed with FDA Meeting expected in 3Q15 Smoking Cessation Multiple commercial scenarios being assessed PROPRIETARY 11

12 Ralinepag for Vascular Disease

13 Ralinepag: Novel, Oral Prostacyclin Receptor Agonist to Treat Pulmonary Arterial Hypertension (PAH) Stable, orally bioavailable, highly selective, non-prostanoid drug candidate Unique pharmacokinetic profile expected to provide low peak-to-trough ratio more closely approximating continuous i.v. infusion, thereby providing potential for optimal clinical outcome Differentiated pharmacologic profile based upon robust IP receptor potency and in vitro target cell effects (smooth muscle relaxation and proliferation, platelet inhibition) Improved receptor coverage given long half-life Lower peak-to-trough ratio may improve tolerability t ½ (hr) Treprostinil 4.5hrs 1 Selexipag 8 hrs 2 Ralinepag ~20-26 hrs 3 1 Tapson, et al. Am J Respir Crit Care Med (2009) 2 Lang, et al Expert Opin Pharmacother (2014) 3 Arena Pharmaceuticals, Inc Plasma Conc. (ng/ml) 10 Adverse Events Long T½ low peak to trough Short T½ high peak to trough Long T½ modified release Time (hr) Note: modeling based on published and internal data Therapeutic Range PROPRIETARY 13

14 Ralinepag: Phase 2 Trial for PAH OBJECTIVES AND STUDY DESIGN Randomized, double-blind, placebo-controlled ~60 patients with WHO group 1 PAH; WHO/NYHA functional class II-IV symptomology 22 week-study including dose titration period followed by an open-label extension Primary outcomes: change in pulmonary vascular resistance, change in 6-min walk distance PROPRIETARY 14

15 Ralinepag: IP Agonist Market Opportunity EARLY STAGE DISEASE Oral Therapies Dominate First Line Treatment Oral Monotherapy Oral Combos LATE STAGE DISEASE Inhaled/SC/IV PDE5 Inhibitors: +$500 Million Endothelin Receptor Antagonists: +$2 Billion Soluble Guanylate Cyclase (sgc) Stimulator: ~$100 Million Potential advancement to earlier lines of therapy with oral formulations Launched Prostacyclins: +$1 Billion An oral, IP receptor agonist with a long half-life could move treatment earlier in the treatment paradigm PROPRIETARY 15

16 Ralinepag: IP Agonist Market Opportunity Once-Daily, Oral Drug that Approximates Continuous Therapeutic Exposure of I.V. Infusion Convenience of Administration (Oral Drug) Symptomatic Disease Management (Vasodilation) Earlier Intervention in PAH Potential Disease Modification (Vascular Remodeling) PROPRIETARY 16

17 Ralinepag: Activity in Rat Monocrotaline Model of PAH Treatment with ralinepag lowered pulmonary artery pressure Pulmonary Artery Pressure p<0.01 mpap(mmhg) (9) (9) Sham (8) (5) MCT + Vehicle MCT + APD811 30mpk Monocrotaline (MCT) induced PAH in rats is a common model used to investigate potential therapies for PAH. MCT induces a significant increase in pulmonary artery pressure, medial thickness of small pulmonary arteries, and right ventricular hypertrophy. PROPRIETARY 17

18 RV/LV+S Ralinepag: Activity in Rat Monocrotaline Model Treatment with ralinepag blocked development of PAH & reduced pulmonary vascular and cardiac remodeling Wall Thickness % wall thickness (%) (9) Sham MCT MCT + APD811 p<0.01 (5) (5) (5) (9) Right Ventricle Weight p<0.001 * Vs. MCT +Vehicle * 0 Sham MCT + Vehicle MCT + APD811 30mpk 0.00 PROPRIETARY 18

19 APD334 for Autoimmune Diseases

20 APD334: Novel, Oral S1P 1 Modulator to Treat Autoimmune Diseases (Phase 1 MAD Results) Attributes of A Potential Best-in-Class Once-Daily, Oral Therapy Inhibiting Lymphocyte Trafficking Lymphocyte Reduction with Rapid Recovery Dose-dependent effect on lymphocyte count lowering in blood, with mean decreases from baseline up to 69% Recovery of lymphocyte counts to baseline within one week (half-life ~35 hours) Favorable Cardiovascular Safety Profile No clinically significant safety findings with respect to heart rate or rhythm Favorable Hepatotoxicity Safety Profile No clinically significant elevations in liver enzyme tests in any dose group PROPRIETARY 20

21 Incidence per 100,000 People Inflammatory Bowel Disease (IBD): Market Overview Disease Burden Chronic disease with sizeable economic burden: Over 1.6 million people (907,000 with ulcerative colitis and 780,000 with Crohn s disease) in the US Annual US economic costs estimated at +$31 billion Growing disease of the developed world, particularly urban areas and northern climates ~$9.0 billion global IBD market in Canada Denmark Iceland United Kingdom Ulcerative Colitis Crohn's Disease United States Unmet Need Novel, oral, efficacious maintenance therapy with fewer side effects ~48% of ulcerative colitis patients are in remission; 70% of patients with disease will have a reoccurrence in the next year ~50% of people with Crohn s disease will be in remission, or have mild disease, over the next five years; more than 50% of patients in remission will have a relapse over a year Severe Disease 1-2% Ulcerative Colitis: Disease Activity Moderate Disease 20% Mild Disease 30% In Remission 48% Preparing for Phase 2 study start in ulcerative colitis later this year. PROPRIETARY 21

22 IBD Treatment Algorithm Primary Market Research Suggests that S1P1 Receptor Modulator May be Prescribed to a Similar Patient Population as TNF-α Blockers Mild Mild Moderate Severe Aminosalicylates (5-ASAs) Corticosteroids Immunosuppressants and Anti-Integrins TNF-α Inhibitors S1P 1 Receptor Modulator PROPRIETARY 22

23 S1P 1 Receptor Modulator Class Potential Multiple Products and Autoimmune Indications Could Expand Commercial Potential Over Time Similar to the TNF-α Blocker Class Rheumatoid Arthritis Rheumatoid Arthritis Ulcerative Colitis Psoriatic Arthritis Multiple Sclerosis Psoriatic Arthritis Psoriasis Ankylosing Spondylitis Ulcerative Colitis Ankylosing Spondylitis Crohn s Disease Psoriasis Crohn s Disease TNF-α Blockers ~$32 Billion (2014) 1 S1P 1 Receptor Modulators $? Billion 16X ~$2 Billion (2002) 2 $2.5 Billion (2014) 2 PROPRIETARY 23

24 APD371 for Pain

25 % reporting pain in prior month Pain: Market Overview Disease Burden 100 million people in US are burdened with persistent pain each year Pain is a leading cause of medically related work absenteeism ~>50 million workdays lost each year ~$ billion estimated annual US economic costs ~$35 billion in global pain management market in 2012 At least 1 in 3 Americans suffer from chronic pain Unmet Need Pain is often under treated Current treatment options do not provide adequate balance of safety and efficacy NSAIDs: modest efficacy with potential for increased risk of GI bleeding Opioids: stronger efficacy with potential for dependence/abuse and GI side effects COXIBs: narrow therapeutic benefit over NSAIDs with enhanced risk of CV adverse events With the prevalence of pain on the rise, the need for safe, well-tolerated and more-efficacious pain relievers is substantial According to NHANES data, prevalence of chronic pain in the US has been increasing, particularly in patients aged Ages 20 to 44 Ages 45 to 64 Ages 65 and over 10 NHANES NHANES NHANES Trends in Pain Prevalence: United States, PROPRIETARY 25

26 APD371: Novel, Oral Agonist of the Cannabinoid 2 (CB2) Receptor Initial Market Opportunity Additional Potential Attributes Need for non-opioid, non-nsaid approaches to pain Opioids: strong efficacy with potential for dependence/abuse and GI side effects NSAIDs (including COXIBs): modest efficacy with increased risk of GI bleeding, renal toxicity and CV events Fibrotic diseases Full agonist Selectively targeting CB2 receptor may provide pain relief without the liabilities of opiates and NSAIDs Status Phase 1 single-ascending trial dosing completed No adverse events that limited dose escalation up to 400mg Blood drug levels achieved greatly exceed the in vitro CB2 receptor activation constant [EC 50 ] Plan to initiate Phase 1 multiple-ascending dose study later this year PROPRIETARY 26

27 Nelotanserin: Novel 5-HT 2A Inverse Agonist for Behavioral and Neuropsychiatric Disturbances Nelotanserin Internally discovered inverse agonist of the serotonin 5-HT 2A receptor Previous trials support potential to treat a variety of neuropsychiatric conditions Development, Marketing and Supply Agreement Roivant Sciences: Granted exclusive worldwide rights to develop and commercialize nelotanserin Responsible for all development expenses Intend to initiate Phase 2 clinical trials in certain neurological diseases Arena Pharmaceuticals GmbH: Will manufacture clinical supply and commercial product to sell to Roivant Receive $4 million upfront payment; eligible for $41.5 million in milestone payments 15% of net sales and up to $60 million in purchase price adjustments PROPRIETARY 27

28 ARNA Investment Highlights Revenue stream from BELVIQ (lorcaserin HCl), indicated for weight management Strategy to create additional value with lorcaserin by pursuing lifecycle management opportunities Clinical-stage pipeline of novel, internally discovered compounds targeting large market opportunities Cash and cash equivalents of $241M at 3/31/15 PROPRIETARY 28

29 Arena s Mission Embracing the Challenge of Improving Health by Bringing Innovative Medicines Targeting G Protein-Coupled Receptors to Patients PROPRIETARY 29

30 Jefferies 2015 Health Care Conference NASDAQ: ARNA Craig M. Audet, PhD J u n e 1, Sr. VP, Operations & Head of Global Regulatory Affairs