UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): September 28, 2012
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Sucampo Pharmaceuticals, Inc.
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Delaware
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001-33609
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30-0520478
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(State or Other Juris-
diction of Incorporation)
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(Commission
File Number)
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(IRS Employer
Identification No.)
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4520 East-West Highway, 3rd Floor
Bethesda, Maryland
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20814
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(Address of Principal Executive Offices)
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(Zip Code)
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Registrant’s telephone number, including area code: (301) 961-3400
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(Former Name or Former Address, if Changed Since Last Report)
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Item 7.01. Regulation FD Disclosure.
On September 28, 2012, Sucampo Pharmaceuticals, Inc. (“the Company”) will meet with analysts, investors and investment bankers and make a corporate update presentation and webcast at the Company’s Analyst Day in New York City, NY, that will include written communication comprised of slides. The slides from the presentation are being furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in this Item 7.01 and Exhibit 99.1 to this Form 8-K shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
99.1 The corporate update presentation slides dated September 28, 2012.
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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SUCAMPO PHARMACEUTICALS, INC.
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Date: September 28, 2012
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By: |
/s/ Thomas J. Knapp
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Name:
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Thomas J. Knapp | ||
| Title: | Executive Vice President, Chief Legal | ||
| Officer & Corporate Secretary | |||
Exhibit 99.1
Sucampo Analyst Day Le Parker Meridien, New York City September 28, 2012
Forward-Looking Statements This presentation contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements are based on management’s current expectations, and involve risks and uncertainties that may cause results to differ materially from those set forth in the statements. The forward-looking statements may include statements regarding product development, product potential, future financial and operating results, and other statements that are not historical facts. The following factors, among others, could cause actual results to differ from those set forth in the forward-looking statements: the impact of pharmaceutical industry regulation and healthcare legislation; Sucampo’s ability to accurately predict future market conditions; dependence on the effectiveness of Sucampo’s patents and other protections for innovative products; the risk of new and changing regulation and health policies in the US and internationally, and the exposure to litigation and/or regulatory actions. No forward-looking statement can be guaranteed and actual results may differ materially from those projected. Sucampo undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Forward-looking statements in this presentation should be evaluated together with the many uncertainties that affect Sucampo’s business, particularly those mentioned in the risk factors and cautionary statements in Sucampo’s Form 10-Q, May 10, 2012 and Form 10-K for the year ended Dec 31, 2011, which the company incorporates by reference
Welcome/Corporate Vision Ryuji Ueno, MD, PhD, PhD, Chairman, Chief Executive Officer, and Chief Scientific Officer September 28, 2012
Commercial-stage, global biopharmaceutical company since 1996 • 2 FDA-approved drugs based on our proprietary prostone technology . AMITIZA® (lubiprostone) in gastroenterology market . RESCULA® (unoprostone isopropyl) in ophthalmology market Prostone pioneers • Therapeutic potential 1st identified by Sucampo’s founders, Drs Ryuji Ueno and Sachiko Kuno Sucampo Snapshot: Prostone Pioneers Sucampo Mission To develop and commercialize prostone-based medicines to meet the major unmet medical needs of patients on a global basis ® Registered trademark of Sucampo
1. Expansion of AMITIZA franchise 2. Launch of RESCULA in US 3. Development of pipeline Sucampo Corporate Priorities
AMITIZA . Approved in Japan in June 2012 . Approved in UK in September 2012 . OIC sNDA accepted on September 19, 2012 (granted priority review) PDUFA expected late January 2013 RESCULA . US: Anticipate approval of revised label . EUROPE: Re-approval filings in EU and Switzerland • Research and Development . Pipeline prioritized • Emerging pipeline • Clinical stage Recent Progress Toward Our Priorities
Sucampo Management Team Ryuji Ueno, MD, PhD, PhD Chairman, Chief Executive Officer, Chief Scientific Officer, and Cofounder Peter Lichtlen, MD, PhD Senior Medical Officer and Vice President, European Operations Gayle Dolecek, PD, MPH Executive Advisor, Research and Development Silvia Taylor Senior Vice President , Investor Relations, Public Relations, and Corporate Communications Thomas J. Knapp Executive Vice President, Chief Legal Officer, and Secretary Stanley G. Miele President, Sucampo Pharma Americas and SVP, Sales and Marketing Cary J. Claiborne Chief Financial Officer Greg Deener Senior Vice President, Marketing Strategy and Implementation
Sucampo’s Prostone Technology GI Franchise Overview Ophthalmology Franchise Overview Pipeline Overview Global Commercial Update Financial Overview Q&A and Closing Remarks Lunch Agenda
Dr Ryuji Ueno Chairman, CEO, CSO Dr Peter Lichtlen Senior Medical Officer and Vice President, European Operations Dr Birgit Roerig Vice President, Pharmacology and Toxicology Taryn Joswick Vice President, Clinical Development Dr Glenn Noronha Vice President, Research and Development Stan Miele President, Sucampo Pharma Americas and SVP, Sales and Marketing Takashi Sekida Vice President, Research Planning and Business Development Dr Dipak Panigrahi Vice President, Medical Affairs Andrew Smith Vice President, Operations and Finance Cary Claiborne Chief Financial Officer Presenters
Technology Platform and Portfolio Introduction Peter Lichtlen, MD, PhD, BBA, Senior Medical Officer and Vice President of European Operations September 28, 2012
General background on Sucampo’s prostone platform technology Prostones: potent and selective ion-channel activators Sucampo’s IP position on prostones (>580 patents) Introduction of Sucampo’s clinical pipeline Overview
Sucampo: THE Prostone Company Sucampo Pharma Ltd Japan Sucampo Pharma Europe Ltd UK Sucampo Pharma Americas, LLC US Sucampo AG Switzerland Sucampo Pharmaceuticals, Inc. See Reference 1
Prostones: . Functional fatty acids naturally occuring in the human body . Ion-channel activators . Physiological mediators of restoration of cellular homeostasis and tissue regeneration • Clinical safety profile of prostones is excellent, as demonstrated by the clinical safety record of AMITIZA in GI and RESCULA in ophthalmology • Clinical potential of prostones is broad and applicable to various therapeutic fields beyond GI and ophthalmology Sucampo Has Pioneered the Field of Prostones Sucampo is the only company developing and commercializing prostone compounds globally See Reference 1
Unique mechanism of action Re-establishing dysregulated cellular homeostasis using naturally occuring (physiological) repair mechanisms Direct activation of critical ion channels: ClC-2-chloride and BK-potassium channels, respectively Low nanomolar-picomolar EC50s Prostones’ General Pharmacodynamic Effects See Reference 1
Proprietary Platform Technology: Sucampo’s Prostones Are Highly Potent Ion-Channel Activators AMITIZA RESCULA Fluid Secretion Barrier Repair and Mucosal Protection Anti-Inflammatory Activity ClC-2-Chloride Channel Activation BK-Potassium Channel Activation Prevents neuronal cell death Smooth Muscle Relaxation Nerve Stabilization Prostones See Reference 1
Discovery engine based on proprietary prostones Robust pipeline with 2 successful products Focus on chronic diseases that significantly affect patient’s quality of life Novel MOA 1st-in-class therapies Addressing unmet medical needs Prostones: Normalizing Physiological Processes Discovery Research and Development Commercial
Sucampo’s Clinical Pipeline CLINICAL FOCUS STAGE OF CLINICAL DEVELOPMENT COMPOUNDS DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 FILED AMITIZA CC Japan Launch 4Q12 AMITIZA CIC UK Approval 3Q12 AMITIZA OIC US 3Q12 AMITIZA OIC UK/CH 4Q12 AMITIZA Pediatric Constipation FPFV 1Q13 RESCULA POAG/OH US Launch 4Q12 RESCULA POAG/OH EU 4Q12 Cobiprostone Oral Mucositis 4Q12 SPI-017 IV Spinal Stenosis 4Q12 SPI-3608 PO Spinal Stenosis 4Q12 Successfully completed Approval pending Projected start or submission
1. Sucampo data on file References
Prostone Mechanism of Action Technology and Therapeutic Potential Birgit Roerig, PhD, Vice President of Pharmacology and Toxicology September 28, 2012
Mechanism of action ClC-2-channel activation BK-channel activation Therapeutic potential Conclusions Overview
Ion channels: integral cell components that regulate flow of specific ions in and out of cells . Ion-flow regulation is key to cell function, eg, metabolic processes and cell survival Prostones: . Directly activate BK-potassium and ClC-2.type chloride channels with high potency and selectivity . Do not increase production of intracellular 2nd messengers, eg, cGMP and cAMP . Do not increase intracellular calcium concentration . Efficacious at low doses with excellent safety profile Prostones Possess Unique MOA as Potent Activators of Specific Ion Channels ClC-2-Chloride Channel BK-Potassium Channel See Reference 1 Double-Barrel Structure Open: Hyperpolarization Low PH Cell Volume Increase Prostone Binding Site Prostone Binding Site
Prostones: Mechanisms of Action ClC-2-Channel Activation BK-Channel Activation Suppression of Pro-Inflammatory Cytokine Expression
Increases fluid secretion Maintains chloride homeostasis Repairs epithelial barriers, eg, intestinal epithelium, bloodbrain and -retinal barriers via restoration of tight junction complexes Prostones: Mechanisms of Action ClC-2-Channel Activation
ClC-2-Channel Activation Promotes Intestinal Fluid Secretion Lubiprostone increases intestinal fluid secretion without causing electrolyte imbalance See Reference 1 Chloride Sodium Fluid Fluid Na+ Cl–
Epithelial barriers restrict the diffusion of microscopic objects of Intestinal barrier: IBS, IBD Blood Brain Barrier: Alzheimer’s disease, Schizophrenia, Epilepsy, MS Retinal Blood Barrier: ME, AMD (CNV) Barrier Function in Body See References 1, 3
Tight Junctions Between Epithelial Cells Form the Structural Basis of Epithelial Barriers See References 1, 3
Control Uninjured ileal mucosa Injured Ischemic injured ileal mucosa Treated Lubiprostone-treated ischemic injured ileal mucosa Occludin distribution diffuse with predominant intracellular localization Occludin localized predominantly to apical TJs Prostones Protect Mucosal Barrier Function: Recovery of Barrier Function in Ischemic Injured Porcine Intestine Occludin localized to apical intercellular junction region (white arrows) See Reference 6
Mucosal-to-Serosal 3H-Mannitol Fluxes Prostones Accelerated Recovery of Disrupted Barrier Function Ischemia Control (noninjured) 3H-Mannitol Flux (mM/cm2.h) 0.0 0.1 0.2 0.3 0.4 0.5 0.6 * Ischemia + Lubiprostone (1 .M) See Reference 6
Neuroprotection via hyperpolarization of excitable membranes Prevention of neuronal cell death Relaxes smooth muscle cells (vasculature, trabecular meshwork) Increases microvascular circulation and blood supply (intraocular circulation, peripheral circulation) Stabilizes mitochondrial membrane potential Antagonizes vasoconstrictors, eg, endothelin-1 Prostones: Mechanisms of Action BK-Channel Activation
Neuroprotection Via Hyperpolarization of Excitable Membranes Mean no. of cells in ganglion cell layer (GCL; n = 4) Analysis of variance revealed statistically significant difference among the 3 groups (P = 0.004; unpaired t tests; *P <0.01, †P <0.05) No reduction in no. of GCL cells seen when unoprostone was given. Increases Survival of Excitatory and Inhibitory Neurons Regulation of Neuronal Excitability, Normalized Synaptic Circuit Function No. of Cells in GCL, Per Millimeter Unoprostone + ET-1 ET-1 Control BK Stimulation Reduces Cell Death BK Stimulation BK Stimulation Protects Retinal Ganglion Cells 70 60 50 40 30 20 10 See References 7-8
BK Stimulation Relaxes Vascular Smooth Muscle to Increase Ocular Blood Flow Relaxation of Smooth Muscle Cells Partial antagonism of endothelin 1-induced vasoconstriction in the human choroid by topical unoprostone isopropyl. BK Stimulation Relaxes Trabecular Meshwork to Decrease Intra-ocular Pressure See Reference 9
Prevents inflammation-induced structural and functional changes Anti-inflammatory, immunomodulatory, and antinociceptive activity Suppression of Pro-Inflammatory Cytokine Expression
Prostones reduced expression of pro-inflammatory cytokines, eg, IL1-., TNF-., Inf-., IL-6, IL-12, as well as COX2 Anti-inflammatory potential of prostones enhances therapeutic potential for disease states with strong inflammatory component, eg, IBS and oral mucositis Prostones Reduced Inflammation and Expression of Pro- Inflammatory Cytokines in Chemically Induced Colitis Model Scale bar; 0.1 mm Control DSS DSS + Lubiprostone DSS-induced inflammation in C57/B6J mouse cecum in presence or absence of Lubiprostone DSS, dextran sodium sulfate. See Reference 10
Prostone Therapeutic Potential Neuronal Survival, Normal Circuit Function Hyperpolarization Schizophrenia, Alzheimer’s Disease BK Channel Hyperpolarization Relaxes Trabecular Meshwork Glaucoma Central Nervous System Ophthalmology Gastrointestinal/ Digestive BK Channel Hyperpolarization Relaxation of Vascular Smooth Muscle, Increased Blood Flow Spinal Stenosis ClC-2 Channel Tight Junction Repair BK Channel See References 6, 11-12 Restores Epithelial Barrier IBS, Oral Mucositis
Prostones are naturally occurring compounds that act locally to restore normal function in cells/tissues Prostone-based drugs use natural pathways to restore physiologic function Since prostones reactivate normal cell processes, safety profile of these compounds is excellent, as demonstrated by clinical safety record of AMITIZA® and RESCULA® Prostones have novel MOA as activators of BK-potassium and ClC-2.type chloride channels Large variety of potential target indications due to novel MOA Conclusions ® Registered trademark of Sucampo
1. Sucampo data on file 2. Bazan NG. Prostaglandins Leukot Essent Fatty Acids. 2009;81(2-3):205-11 3. www.bio.davidson.edu 4. Inst. Clin. Physiology: research topics 553 x 385 | 77.1 KB www.charite.de 5. Nighot PK, Blikslager AT. Am J Physiol Gastrointest Liver Physiol. 2010;299:G449-56 6. Blikslager AT, Ueno R. Am J Physiol Gastrointest Liver Physiol 2007;292:G647-G56 7. Sugiyama T, et al. Arch Ophthalmol. 2009;127(4):454-9 8. Hashimoto T, Lewis DA. Int Rev Neurobiol. 2007;78:109-31. Review 9. Polska E, et al. Arch Ophthalmol. 2002;120:348-52 10. Tokumasu et al. Japanese Biochemical Society. 2011 [abstr] 11. Llobet A, Gasull X, Gual A. Understanding trabecular meshwork physiology: a key to the control of intraocular pressure? News Physiol Sci. 2003;18:205-9. 12. Hashimoto T, Lewis DA. Deciphering the disease process of schizophrenia: the contribution of cortical GABA neurons. Lewis Int Rev Neurobiol. 2007;78:109-31. Review References
GI Franchise Taryn Joswick, BS, PMP, Vice President of Clinical Development September 28, 2012
AMITIZA® global approval status MOA in treatment of GI diseases Key features of product differentiation: efficacy and safety New and future indications Overview ® Registered trademark of Sucampo
Global AMITIZA Approvals and Regulatory Filings Japan CC (2012) UK CIC (2012) OIC (target filing 4Q12) US CIC (2006) IBS-C (2008) OIC sNDA Priority Review (2013) Switzerland CIC (2009) OIC (target filing 4Q12) AMITIZA has been used for >6 y with 6 million prescriptions by patients suffering from chronic idiopathic constipation and irritable bowel syndrome with constipation See Reference 1
AMITIZA Mechanism of Action: ClC-2 Ion-Channel Activation and Fluid Secretion Highly selective activation of ClC-2 channels in intestinal lumen Chloride efflux followed by passive efflux of sodium into small intestine Enhanced intestinal fluid secretion without alteration of serum electrolyte levels See Reference 1
AMITIZA Mechanism of Action: Restores ClC-2-Mediated Barrier Disease, injury, stress, or medications such as NSAIDs can damage epithelial barrier Disorganized tight junctions and resulting intestinal permeability may be involved in pathogenesis of IBS ClC-2 activation by AMITIZA enhances restoration of tight junctions and reduces intestinal permeability caused by stress or ischemia Cl– Apical membrane Intracellular Extracellular Tight junction Basolateral membrane CIC-2 Channel Prostone binding site See Reference 1
Chronic Idiopathic Constipation (CIC) . Affects ~14%-16% of adult population globally 33M in US (14%),2 41M in EU 5 (16%),2 15M in Japan (14.3%)3 – CC . Accounts for 92,000 hospitalizations/yr in US4 . Severe constipation is associated with increased cardiovascular risk in women5,6 Irritable Bowel Syndrome (IBS) . Affects ~15% of adult population globally, 1/3 of whom have IBS with constipation (IBS-C)7 12M in US, 11M in EU7,8, 3M in Japan7,9 . Direct and indirect costs of IBS care in US: $20 billion/yr7 . Patients with IBS consume >50% more healthcare resources than those without IBS10 Sucampo: Leader in Gastrointestinal Disease Medication Development See References 2-10
Differentiating Between Chronic Idiopathic Constipation and Irritable Bowel Syndrome With Constipation IBS-C CIC Reduced Bowel Frequency Abdominal Pain and Discomfort See Reference 11
No gender restriction in CIC Approved for use in women with IBS-C in US No black box warning Rapid onset in CIC: 57%-63% of patients respond within 24 h Proven long-term safety profile in CIC and IBS-C No limitation on duration of use in US, Japan, and Switzerland AMITIZA: Effective 1st-Line Therapy for CIC and IBS-C See Reference 1
Long-term Efficacy Positive Long-term Treatment Response: Phase 3 Studies of AMITIZA 8 µg BID in IBS-C Month 0 5 10 15 20 25 30 35 40 Treatment Responders, % 1 2 3 4 5 6 7 8 9 10 11 12 See Reference 1
Substantial Abdominal Pain Improvement in IBS-C Patients Reporting at Least Severe Abdominal Pain at Baseline* % Improvement Placebo BID (n = 94) Lubiprostone 8 µg BID (n = 183) P Value† =10 53.9% 61.9% <0.0001 =20 40.1% 49.6% <0.0001 =30 24.2% 35.1% <0.0001 =40 14.5% 23.7% <0.0001 =50 9.4% 16.7% <0.0001 =60 4.7% 12.7% <0.0001 *LOCF analysis; †P value from CMH test. See Reference 12
Nausea rated as mild-moderate by 89% and 96% of CIC and IBS-C patients, respectively, who experienced nausea . >93% of patients reporting nausea experienced only 1 event over course of treatment with AMITIZA In placebo-controlled, 12-wk IBS-C trials, diarrhea reported by 7% of AMITIZA patients vs 4% of placebo patients In IBS-C exposure up to 1 yr, dropout due to diarrhea accounted for <2% of patients AMITIZA Safety Profile: Clinical Trials in Patients With CIC or IBS-C AMITIZA has excellent tolerability and safety profile as demonstrated in clinical studies See References 1,13
No serious safety concerns have arisen in postmarketing use of AMITIZA Safety in clinical-use setting has been a problem for other CIC and IBS-C medications, leading to withdrawal of marketing applications Labeled risk-benefit ratio for AMITIZA is well supported by postmarketing safety profile from 6 million prescriptions over 6 yr AMITIZA Postmarketing Safety See Reference 1
Estimated 7.1M non-cancer chronic opioid users in US14 Most common reason for discontinuation of opioid therapy Mu-opioid-receptor agonist compounds under development may have cardiac safety concerns AMITIZA does not act on opiate receptors or inhibit analgesic activity of opioid therapy Opioid-Induced Constipation: Medical Significance and Unmet Need See Reference 14
Overall Spontaneous Bowel Movement (“SBM”) Response in OIC Patients n = 439 n = 353 18.6 21.7 26.9 32.5 0 5 10 15 20 25 30 35 ITT Completer Placebo Lubiprostone P = 0.035* P = 0.018* Proportion of Responders, % . = 8.3 . = 10.8 *Statistically significant (P =0.05) See Reference 15
0 10 20 30 40 50 60 70 80 Placebo Lubiprostone Proportion of Patients With SBM 48 h 24 h 12 h 8 h 4 h Time to SBM Onset in OIC Subjects Median time to 1st SBM (in brackets), P = 0.019 P = 0.002 P = 0.017 P = 0.021 P = 0.016 P = 0.022 [38.50 h] [24.25 h] See Reference 15
4-wk study of AMITIZA in 124 pediatric patients aged 3-17 yr with chronic constipation >86% of patients successfully completed 4 wk of treatment . Low discontinuation rate (6.5%) due to AEs Statistically significant improvements from baseline reported at each treatment week and overall for: . Stool frequency . Straining and pain with SBMs . Stool consistency Open-Label Evaluation of AMITIZA in Pediatric Constipation Placebo-controlled and long-term studies of AMITIZA in pediatric constipation patients will initiate in early 2013 See Reference 16
Well positioned to serve expanding population of patients with CIC and IBS-C . 6 million prescriptions used over past 6 yr with favorable benefit-risk profile Near-term goals . Seek approval for OIC indication in US and submit labeling applications for OIC abroad . Expand global approvals and launches for AMITIZA worldwide . Develop and seek approval for AMITIZA in pediatric constipation Currently unmet medical need; no approved prescription medications . Develop liquid formulation of AMITIZA for long-term care market . Evaluate potential of AMITIZA for new indications, such as mixed irritable bowel syndrome Summary and Outlook for AMITIZA
1. Sucampo data on file 2. Suares et al. Am J Gastroenterol. 2011 3. Kantar Health Epi database http://epidb.khapps.jp 4. Lembo et al. Sleisenger and Fordtran’s Gastrointestinal and Liver Disease. 2010 5. Salmoirago-Blotcher et al. Am J Med. 2011 6. Talley et al. Am J Gastroenterol. 2001 7. Saito et al. Am J Gastroenterol. 2002 8. Muller-Lissner S et al. Digestion. 2001 9. Kubo et al. Neurogastroenterol Motil. 2011 10. Hulisz D. J Manag Care Pharm. 2004 11. Brandt LJ et al. Am J Gastroenterol. 2005;100(suppl 1):S5-S21 12. Joswick et al. Digestive Disease Week, 2012 13. AMITIZA Package Inserts (US and UK) 14. Camilleri M. Clin Systemat Rev. 2011;106:835-42 15. Jamal et al. DDW 2012 16. Hyman et al. NASPGHAN, 2009 References
Ophthalmology: RESCULA® Glenn Noronha, PhD, Vice President of Research & Development September 28, 2012 ® Registered trademark of Sucampo
Focus is on diseases with degenerative drivers Glaucoma and ocular hypertension . Retinitis pigmentosa (RP) UI is a docosanoid-type prostone compound Overview of Ophthalmology: RESCULA Unoprostone isopropyl (UI) RESCULA HO HO O COOCH(CH3)2 CH3 H H H H See Reference 1
Glaucoma is a group of ocular diseases with various causes that ultimately are associated with a progressive optic neuropathy leading to loss of vision Glaucoma is an age-related disease Glaucoma is the second leading cause of bilateral blindness worldwide It will affect an estimated 79.6 million people worldwide by 20202 Reduction in intra-ocular pressure (IOP) is currently the only modifiable risk factor for patients with glaucoma and ocular hypertension Glaucoma: Unmet Medical Need TM, trabecular meshwork. See References 1-2
New therapies with unique MOAs provide physicians with options and add potential advantages for treating patients UI targets IOP reduction plus protection of cells in eye via BKchannel activation and, therefore, provides potential for longer term benefit RECULA is Unique BK channel Hyperpolarization Relaxation of TM Increases in outflow via conventional pathway TM, trabecular meshwork. See Reference 1
Aqueous humor . Formed in ciliary processes from arterial blood . Secreted to posterior chamber . Reaches anterior chamber by crossing pupil Unoprostone – BK Channels – Trabecular Outflow BK channels are found in the trabecular meshwork (TM)2 Unoprostone reduces IOP . Activation of BK channels hyperpolarizes the cell and leads to relaxation of the TM . Resulting in increased outflow via conventional pathway (through the TM) See Reference 2 Anterior Chamber
In glaucoma, there is damage to optic nerve and loss of retinal ganglion cells; untreated, progressive visual field loss and eventually blindness result IOP lowering . UI, by activating BK channels, increases aqueous humor outflow via relaxation of TM (conventional pathway) Prevention of cell death . Through activation of BK channel, UI protects retinal ganglion and other retinal cells in eye from destruction Unoprostone Demonstrates Potential Benefits Beyond IOP Lowering See Reference 1
UI protected photoreceptors from constant light–induced damage in vivo2 UI increased ganglion cell survival in vitro1 Cell-Death Protection: Unoprostone Increases Ganglion Cell Survival In Vivo and Photoreceptor Survival In Vitro Intravitreal UI, .g 0 1 2 3 4 0.6 1.2 3.8 8.0 Degree of photoreceptor rescue (0–4+) (n =6) (n =5) (n =8) (n = 8) No. of Cells in GCL/mm * † 0 10 20 30 40 50 60 70 80 Sham Vehicle Unoprostone Vehicle Vehicle + Endothelin UI + Endothelin See References 4-5
Saito et al. Nippon Ganka Gakkai Zasshi. 2006;110:717-22 . Long-term effects of UI monotherapy on IOP and visual field for ocular hypertension and primary open-angle glaucoma: visual field stabilized Ishida et al. Ganka Ophthalmology. 2005;47:1107-12 . Better visual-field data with UI (3/49 eyes; 6.3%) vs latanoprost (9/38 eyes; 26.0%) in head-to-head study UI demonstrated reduction in IOP in 2 well-controlled, randomized, triple-masked, comparator-controlled phase 3 studies run in US, Europe, and Israel by Ciba Vision Clinical Evidence for Advantages of Unoprostone in Glaucoma Patients Visual-field protection Lowering of IOP RESCULA is the first drug, with a new MOA, approved for patients with primary open-angle glaucoma and ocular hypertension by FDA since approval of prostaglandins See Reference 6
Mechanistic basis . Most forms of RP are caused by gene mutations in photoreceptors and other visual-cycle components leading to photoreceptor cell death . Retinal degeneration may be slowed by interfering with this cell-death mechanism RP begins with degeneration of rods, followed by progressive and irreversible death of cones leading to blindness Mutations initially damage rods; therefore, preservation of central cones is important goal for vision preservation in patients with mid-late-stage RP Retinitis Pigmentosa: Presentation and Manifestations See Reference 7
Phase 2 study of UI in 112 patients with RP Determine whether topical UI 0.15% has protective effect on central retinal function of RP in mid-late-stage patients Primary endpoint: change from baseline in retinal sensitivity in central 2o as measured by microperimetry Retinitis Pigmentosa Clinical Study 3 Arms Vehicle as placebo UI 0.15%, 1 drop bid UI 0.15%, 2 drops bid Microperimetry is a reproducible analytic technique used to assess local visual function of specific areas of retina correlated with anatomic data See Reference 7
Results High-dose group met primary endpoint: statistically significant (P = 0.018) increase in central retinal sensitivity threshold as measured by microperimetry Loss of retinal sensitivity most likely arises from loss of retinal cells Preservation of central retinal sensitivity in this study demonstrated in UI-treated patients and not in vehicle-treated patients This result illustrates that preservation of retinal cells is critical for preventing retinal damage and, potentially, in preserving vision See Reference 7
Placebo n=33 2-drops n=36 1-drop n=38 Mean ± S.E. 3-arm comparison Change in Social Life Function Score† • HRQOL measured by 22-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25, Japanese version) Secondary Endpoint: Health-Related Quality of Life (HRQOL) -10 -5 0 5 10 HRQOL measures were carried out by patients *P = 0.280 *P <0.001 *Williams’ test (1-tailed significance level 2.5%); †VFQ-25 Subscale Change Value for “Social Life Functions Due to Vision.” See Reference 7
Major degenerative diseases of the retina, eg, glaucoma and RP, have overlapping pathophysiology In patients with primary open angle glaucoma or ocular hypertension, RESCULA . Reduces IOP throughout the day, alone or in combination . Has a good systemic and ocular safety profile . Novel MOA: ion channel activator promotes aqueous humor outflow through the trabecular meshwork Clinically meaningful results have been achieved in both glaucoma and intraocular hypertension, and in RP Unoprostone in Ophthalmology
1. RESCULA PI 2. Quigley et al. Br J Ophthalmol 2006 Mar;90(3):262-7. 3. Cuppoletti et al, 2012 4. Sugiyama et al, 2009 5. Hayami and Unoki, 2001 6. Sucampo data on file 7. Yamamoto et al. Ophthalmol Ther 2012 References
Emerging Pipeline Peter Lichtlen, MD, PhD, BBA, Senior Medical Officer and Vice President of European Operations September 28, 2012
Selection process for transition of preclinical projects to clinical PoC Cobiprostone for prevention of oral mucositis (phase 1) SPI-017 IV (phase 2) and SPI-3608 PO (phase 1) for treatment of lumbar spinal stenosis (LSS) Overview PoC: proof of concept.
Early-Stage Clinical Pipeline Projects Exploratory IND Enabling Phase 1 Phase 2 Phase 3 Lumbar Spinal Stenosis: SPI-017 IV (BK-channel activation) Lumbar Spinal Stenosis: SPI-3608 PO (BK-channel activation) Oral Mucositis: Cobiprostone (ClC-2-channel activation)
Primary target indication: prevention of radiation-induced oral mucositis in head and neck cancer patients . Applicable formulation: spray Radiation-induced mucositis (RIM) is common toxicity with increasing frequency due to more intensive altered radiation fractionation . RIM can be treatment limiting, requiring opioid analgesia Oral Mucositis: Cobiprostone See Reference 1
Symptoms include: . Pain . Xerostomia . Dysphagia, including feeding-tube dependency . Dehydration . Infections . Potentially life-threatening aspiration Oral Mucositis: Cobiprostone See Reference 1
Pathogenesis of Oral Mucositis Ulceration Amplification Primary damage response Initiation See Reference 1-4 Healing
Golden Syrian Hamster (5-6 wk; ~80 g; n = 8/group) Mucositis induction with single dose of radiation (40 Gy/dose) administered on day 0 . Irradiation targeted left buccal pouch mucosa at rate of 2.0 Gy/min 4 treatment groups: cobiprostone (0.01, 0.03, 0.1 mg/mL) and vehicle (MCT) administered topically BID from day 0 to 28 (0.1 mL/site) Efficacy of Cobiprostone on Radiation-Induced Oral Mucositis Topical treatment with cobiprostone showed significant, dose-dependent protection from radiation-induced oral mucositis and reduction of days with severe oral mucositis (P <0.05) See Reference 5
Development Strategy in Oral Mucositis Cobiprostone: Spray Formulation Current Status Well tolerated in preclinical tolerability and toxicology studies Next Milestone Phase 1a study; 1st subject in 4Q12 (Japan) Start of phase 1b/2a in 3Q13 Target Population Head and neck cancer patients undergoing radiotherapy; primary target: prevention; secondary target: treatment Treatment Duration Up to 12 wk Potential Target Indication Prevention of radiation-induced oral mucositis; later label extension WW/Local Development Phase 1 study in Japan, Phase 1b/2a in US, followed by global development Competitors Mugard (device, approved under 510k) Palifermin (KGF) Benzydamin (not approved in US) WW: worldwide.
LSS caused by degenerative change in lumbar spine; very common disease observed in growing aged population6 Specific treatment in Japan . Only approved medication for LSS is oral PGE1 analogue (limaprost alfadex: OPALMON®) . Prostaglandins associated with poor safety profile requiring careful, fractionated dosing Pharmacologic treatment in US and Europe (no formally approved medication)6 . NSAIDs: GI and renal AEs . Muscle relaxants: sedation, HRQOL . Tricyclic antidepressants: somnolence, dry eye/mouth, constipation, arrhythmia . Short-term oral opioids: drug addiction, constipation . Membrane-stabilizing convulsants (eg, carbamazepine): sedation, ataxia, psychosis • Long-term benefit from current pharmacologic treatment considered small7 • Few new pharmaceutical products under development8 Lumbar Spinal Stenosis: SPI-017 and SPI-3608 Unmet medical need for new safe and effective pharmacologic treatment See References 6-17
Rationale for Treatment of Lumbar Spinal Stenosis With BK-Channel Activator Potassium (BK)-channel activation Reduced calcium influx Relaxation of vascular smooth muscle cells Hyperpolarization of vascular smooth muscle cells Decreased action potential firing in ascending pain pathways Increase in pain threshold Hyperpolarization of spinal neurons Less pain, better walking performance Increased peripheral circulation and increased blood supply to spinal cord See Reference 5
SPI-017 Proposed Prostone Treatment for LSS Following Established Japanese Treatment Paradigm If no improvement within 2-3 mo after onset of LSS Surgery SPI-3608 PGE1 oral dosing Corset Consultation PGE1 infusion (hospitalization) Nerve block See Reference 19
Next project milestones . Phase 2 PoC study for SPI-017 IV, Phase 1 study for SPI-3608 PO BK-channel activation: acknowledged, emerging therapeutic approach to address neuropathic pain20 Significant (P <0.01) dose-dependent increase . Pain threshold (improvement of hyperalgesia) in rat dorsal root compression model . Walking distance (intermittent neurogenic claudication) in rat cauda equina compression model . Spinal-cord blood flow in rat cauda equina compression model Key Supportive Data See Reference 20 Metabolic stability of SPI-3608 > SPI-017 • Sucampo intends to develop BK-channel activators SPI-017 and SPI-3608
Development Strategy in LSS SPI-017 IV SPI-3608 PO Current status Well tolerated in phase 1a/b studies Well tolerated in preclinical studies Next milestone PoC study (1st patient in Jan 2013) 1st in human (1st subject in Dec 2012) Topline results: 4Q13 Completion phase 1a/b program: 4Q13 Target population Hospitalized patients (severe disease) Outpatients (mild/moderate disease) Treatment duration Short term (2 wk) Long term (chronic) Potential target indication LSS (severe) LSS (mild/moderate), if successful: - ev PAD (intermittent claudication) - ev cervical spondylosis, etc WW/local development Local . global Local . global (incl other potential indications) Competitors No competitor on market No competitor on market (US/EU)
1. Rosenthal and Trotti. Semin Radiat Oncol. 2009;19:29-34 2. Wu et al. Future Oncol. 2010:6:1751-70 3. Mosel et al. Anti-Cancer Drugs. 2011;22:607-12 4. Mattson. Database 2003, NCI; http://www.cancer.gov/aboutnci/servingpeople/snapshots/head-neck.pdf 5. Sucampo data on file 6. Kalichman et al. Spine. 2009;9:545-50 7. Tran et al. Can J Anaesth. 2010;57:694-703 8. Weinstein et al. N Engl J Med. 2008;358:794-810; 9. Ciol et al. J Am Geriatr Soc. 1996;44:285-90 10. Konno S. Sogo Rihabiriteshon 2009; 37(6): 509-15 (Japanese) 11. Matsudaira et al. Spine 2009;34:115-20; 12. Harrision and Plosker. Limaprost. Drugs 2007;67:109-18 13. IMS data. 14. Hsiang. Spinal Stenosis. http://emedicine.medscape.com/article/1913265-overview 15. 2008 North American Spine Society (NASS) issued evidence-based guidelines for the diagnosis and treatment of degenerative lumbar spinal stenosis 16. http://clinicaltrials.gov/ 17. Overdevest et al. BMC Musculoskeletal Disorders 2011;12:57. 18. IMS Midas 19. Matsuyama Y. (Orthopedic, Nagoya University), Nikkei Medical Online 2006.12 20. Chen et al. J Neurochem. 2009;110:352-62 References
AMITIZA® Commercial Update ® Registered trademark of Sucampo Stan Miele, Senior Vice President of Sales and Marketing, and President of Sucampo Pharma Americas, LLC Takashi Sekida, Vice President, Director of Research Planning and Business Development Andrew P. Smith, FCMA, Vice President, Operations and Finance September 28, 2012
This section contains the following updates regarding AMITIZA: . US Commercial Update . Japan Commercial Update . European Commercial Update Section Overview
AMITIZA® Commercial Update: US and Japan Stan Miele, Senior Vice President of Sales and Marketing, and President of Sucampo Pharma Americas, LLC September 28, 2012 ® Registered trademark of Sucampo
AMITIZA Users Are the Most Satisfied With Their Treatment and Twice as Satisfied as MiraLAX Users 32.3% 35.1% 18.1% 28.5% 15.6% 21.1% 27.5% 52.8% Enemas (n=65) Glycolax or Polyethylene glycol solution (n=77) Stool softeners (n=226) Fiber (n=284) Laxative other than Miralax (n=173) Probiotics (n=322) OTC MiraLax and similar store brands (n=160) AMITIZA (n=127) Satisfaction with Current Treatments AMITIZA also has highest persistency rate in category See Reference 1
1.1M 1.2M 1,000,000 1,050,000 1,100,000 1,150,000 1,200,000 1,250,000 TRx MAT July 2011 TRx MAT July 2012 261M 291M 250,000,000 260,000,000 270,000,000 280,000,000 290,000,000 300,000,000 $ MAT July 2011 $ MAT July 2012 TRx: +7% Dollars: +12% Positive Clinical Experience Translating to Consumption Growth: $291M on Annualized Basis See Reference 2
AMITIZA® Has 1%-2% Share of ~250M Units for Constipation 61.6% 37.1% 1.3% We estimate there are millions of dissatisfied patients with CIC and IBS-C % of Patients Treated With Constipation Medications OTC Rx Excluding AMITIZA AMITIZA See Reference 3
AMITIZA Has Time-Tested Safety Profile and Positive Clinical Experience Valued by Physicians Criteria AMITIZA Linaclotide MOA ClC-2; mucosal barrier protection GC-C receptor Black box warning No Yes Long-term safety profile Established; 6 y, 6M prescriptions No Safety data in label for elderly with CIC Yes; lower nausea rates No; insufficient no. of subjects Primary side effect Nausea Diarrhea Efficacy in CIC and IBS-C Yes Yes Satisfied patients in “real world” Yes No Multisymptom benefit for CIC with abdominal discomfort/bloating in label Yes No Dosing BID with food and water qd =30 min before 1st meal See Reference 4
Translate time-tested safety profile and positive clinical experience to earlier use in treatment algorithm . Leverage increased category activity and managed-care status to increase AMITIZA sales . Mobilize patients to express their dissatisfaction to physicians and request AMITIZA . Continue education on unique MOA (mucosal barrier protection) of AMITIZA Growth Opportunities of AMITIZA in US With Current Indications
Moderate-severe OIC affects ~2.0M-2.5M patients . Currently no approved oral product for OIC . OIC patients are viewed as “difficult to treat” and are dissatisfied . Most OIC patients are treated by PCPs . PCPs welcome 1 medicine indicated for multiple causes of constipation FDA priority review action date: late January 2013 OIC Will Increase Potential Pool for AMITIZA and Strengthen Efficacy Positioning See References 5-8
Prevalence of CC • ~3.8% of Japanese population complain of constipation9 • Internists and gastroenterologists see 38.9 CC patients/mo on average10 • 81% of constipated patients who consult doctors are chronic10 Japan Constipation Market Potential patient population: =4 million Total prescription laxative market size in 2010: 38.6 billion Yen (approximately US $450M)11 See References 9-11
Japan US Leadership/share of voice at launch Yes No (Zelnorm on market) Key competitors at launch Magnesium oxide Zelnorm, PEG Prescription competitors on market at launch No Yes Linaclotide on market No Yes AMITIZA® access issues No restriction 17% of commercial lives have “prior authorization” 42% of part D lives have “step-edit” Favorable Market Situation in Japan Key Differences Between Japanese and US Markets See Reference 4 ® Registered trademark of Sucampo
1. Sucampo data on file – physician ATU 2. IMS MAT July 2012 compared with MAT July 2011 3. PBE physicians survey April 2012 4. Sucampo data on file 5. IMS Health 6. Verispan PDDA 7. Physician Interviews 8. ClearView Analysis 9. Comprehensive Survey of Living Conditions of the People on Health and Welfare, Ministry of Health, Labour and Welfare 10.Survey by Seed Planning Inc. 11.Constipation Market Survey by Seed Planning Inc. References
AMITIZA® Commercial Update: Japan Takashi Sekida, Vice President, Research Planning and Business Development September 28, 2012 ® Registered trademark of Sucampo
NDA approved 6/29/12 . Approved indication: chronic constipation (CC) excluding organic constipation . Dosage: 24 µg BID Expected launch date: mid-late November AMITIZA Japan Launch Schedule Jun July Aug Sep Oct Nov NDA approval Price negotiation Reimbursement price Launch
AMITIZA target population . Patients with CC who take medication Partnered with Abbott Japan Abbott Japan emphasis: . General internists and gastroenterologists . Some activities started in September Medical Reps Explanatory Meetings, Online Conferences, etc . Japan DDW seminar will be held in October . Abbott Japan will launch on price finalization (currently expected 4Q12) Will trigger $15M milestone payment to Sucampo Sucampo has retained co-promotion rights for Japan Marketing Plans in Japan
AMITIZA® Commercial Update: Europe Andrew P. Smith, FCMA, Vice President, Operations and Finance September 28, 2012 ® Registered trademark of Sucampo
CIC . Switzerland Product currently available but not reimbursed by insurers Conclude pricing / reimbursement discussions Q4-12 Launch contingent on pricing H1-13 . UK & Europe Approved with 2 week treatment restriction (UK) Sep-12 Seek possible label variation (UK) Q1-13 Anticipate approval via Mutual Recognition Procedure (MRP) in EU5 Q1-14 OIC File submission - UK & Switzerland Q4-12 Anticipate approval - UK & Switzerland Q4-13 Anticipate approval via MRP in EU5 Q3/Q4-14 AMITIZA® - Evaluating opportunities
RESCULA® Commercial Update ® Registered trademark of Sucampo Stan Miele, Senior Vice President of Sales and Marketing, and President of Sucampo Pharma Americas, LLC Dipak Panigrahi, MD, Vice President, Medical Affairs Andrew P. Smith, FCMA, Vice President, Operations and Finance September 28, 2012
This section contains the following updates regarding RESCULA: . US Launch . Medical Affairs Strategy . EU Filing Section Overview
RESCULA® Commercial Update: US Launch Stan Miele, President of Sucampo Pharma Americas, LLC September 28, 2012 ® Registered trademark of Sucampo
RESCULA was FDA-approved (2000) for the lowering of intraocular pressure (IOP) in primary open-angle glaucoma (POAG) and ocular hypertension (OH) in patients who are intolerant of or insufficiently responsive to other IOPlowering medications Label update expected 4Q2012: reflect current scientific understanding of mechanism of action and be approved for first-line treatment Sucampo plans to launch RESCULA in US by the end of 2012 RESCULA US Launch Overview
Over Past 3 Years, Category TRx Volume in US Has Increased 7% From 27.3M to 29.2M 2.0M 2.1M 2.2M 2.3M 2.4M 2.5M 2.6M TRx Volume Month TRx Volume Linear (TRx Volume) See Reference 1
RESCULA Launch Creative: US
RESCULA: Only Nonprostaglandin That Lowers IOP Throughout Day (12 h) With Excellent Systemic Safety Profile RESCULA ß-Blocker Alphagan-P Azopt Contraindicated in asthma/warning in COPD and diabetes No Yes No No Drug interactions in label No Yes Yes Yes Fatigue, muscle weakness, or drowsiness No Yes Yes No Caution in using antihypertensives No Yes Yes No Allergic reaction (10%-20%) No No Yes No Care exercised in driving motor vehicles or hazardous activities No No Yes No Bitter taste No No No Yes Recommended dosing BID BID/QD TID TID See References 2-5
Guidelines Help Support Our Position The ophthalmologist should consider the balance between side effects and effectiveness in choosing a regimen of maximal effectiveness and tolerance to achieve the desired IOP reduction for each patient. Frequent dosing and side effects (such as depression, exercise intolerance, and impotence with topical ß-blockers) may affect adherence to therapy. See Reference 6
Changes over time in intra-ocular pressure (N = 23) ANOVA revealed no significant change (P = 0.41) RESCULA 0.12% Has Been Shown to Maintain IOP in Patients Intolerant of Prostaglandins Intra-ocular pressure, mm Hg 24 22 18 16 10 14 12 Before Switch M1 M2 M6 M9 Y1 Follow-up period IOP change in 23 eyes switched to RESCULA after mean 8 mo on prostaglandins, with mean initial IOP of 24.7 mm Hg and 17.2 mm Hg at treatment switch See Reference 7
~52% of total prescriptions will be covered with footprint of 40 sales personnel Frequency of message with targeted physicians resulting in trial and utilization Clinical sell and expected ability to convey MOA, efficacy highlights, and safety RESCULA Prescriber Target and Strategy Priority is to immediately visit pre-existing RESCULA prescribers currently still active We will follow with high-volume glaucoma specialists, then general ophthalmologists 2nd-tier targets will be designated: optometrists, focusing on higher volume prescribers and those offering full-service eye care. Former RESCULA Prescribers (~500) Glaucoma Specialists General Ophthalmologists Designated Optometrists
1. IMS NPA data, MAT June 2009 to MAT June 2012 2. Catalina presentation 2011 3. Timoptic Prescribing Information; 2005. Merck & Co. Inc., Whitehouse Station, NJ 4. Alphagan-P Prescribing Information. 2005. Allergan Inc, Irvine, CA 5. Azopt Prescribing information. 2000–2009. Alcon Laboratories Inc, Fort Worth, TX 6. American Academy of Ophthalmology Glaucoma Panel. Preferred Practice Pattern® Guidelines: Primary Open-Angle Glaucoma. 2010. 7. Goseki T et al. Jpn. J Clin Ophthalmol. 2006;60:1227-30 References
Medical Affairs Update Dipak Panigrahi, MD, Vice President, Medical Affairs September 28, 2012
Medical Affairs Support for RESCULA® A new story driven by an understanding of the science of prostones in the eye Novel MOA in eye driven by ion channels IOP reduction via enhanced outflow 1st agent in recent history impacting trabecular meshwork outflow MOA differentiated from classleading prostaglandin analogs ® Registered trademark of Sucampo
Medical Affairs National Structure 5 Regional MSL’s (PhD, MD, OD) 1 Senior MSL Manager
RESCULA® Commercial Update: EU Filing Andrew P. Smith, FCMA, Vice President, Operations and Finance September 28, 2012 ® Registered trademark of Sucampo
Previous approvals deregistered New Registration submission . Update of scientific understanding . Update of regulatory dossier & briefing package Q3-12 . Decentralized Process (DCP) Submission; Reference Member State (RMS) Denmark Q4-12 . Anticipated approvals Q4-13 RESCULA® - Registration
Financial Update Cary J. Claiborne, Chief Financial Officer September 28, 2012
Key Facts Trading Symbol SCMP (NASDAQ) Corporate Headquarters Bethesda, MD Stock Price (9/26/12), 52-wk Range $4.72, $8.50-$3.14 Shares Outstanding (9/26/12) 41.9 M (1 class of common stock) Daily Volume (90-d average at 9/26/12) 63,150 Market Capitalization (9/26/2012) $198 M Debt (6/30/12) $60.4 M Cash and Equivalents (6/30/12) $88.6 M Enterprise Value $169.8 M YTD Total Revenue (6/30/12) $31.1 M Full-time Employees (9/26/12) 111 Fiscal Year Ends December 31 Accounting Firm PricewaterhouseCoopers, LLP
Takeda Agreement . Takeda shall promote, market, and sell AMITIZA in US and Canada . Sucampo’s tiered royalty rate: 18%-26% of annual net sales . Sucampo earned $20M in upfront and $130M in development milestone payments as of 6/30/12 . Sucampo received $106M in reimbursement for R&D expenses from Takeda • Abbott Japan Agreement . Abbott Japan shall promote, market, and sell AMITIZA in Japan . Sucampo will sell product to Abbott Japan at discount to Abbott Japan’s approved reimbursement price . Sucampo earned $10M in upfront and $12.5M in development milestone payments as of 6/30/12 . Sucampo expected to earn $15M milestone payment on 1st commercial sale in Japan by Abbott Japan in 4Q12 Terms of Sucampo’s AMITIZA Agreements
AMITIZA®. Approved in Japan: June 2012. OIC sNDA application accepted by FDA for priority review: September 2012 RESCULA®. US: anticipate approval of sNDA for glaucoma indication (new label/updated MOA). EU: re-approval filings in EU and Switzerland Research and Development . Pipeline prioritized Financial/Corporate. Cash, cash equivalents, and investments Ended 2Q12 with $89M 1H 2012: net operating cash flow positive. Revenue 2Q $16.7M up 19%; 1H $31.1M up 19%. Dual class of common stock eliminated 2nd Quarter 2012 and Recent Highlights ® Registered trademark of Sucampo
Key Value Drivers . Filed OIC sNDA: Q312 . OIC filing accepted by FDA for priority review: 9/19/12 . Decision in Takeda arbitration resolved dispute US AMITIZA . Approved in Japan for CC: 2Q12 . Await pricing decision: 4Q12 . Launch: 4Q12 Japan . Approved in UK for CIC: 3Q12 . Submit OIC MAAs in UK and Switzerland EU . Pricing resolution: 4Q12 Switzerland RESCULA . Anticipate approval of sNDA for glaucoma indication (updated label) . Launch: 4Q12 US . Re-approval filings in EU and Switzerland EU . Completed . In Process
Closing Remarks Ryuji Ueno, MD, PhD, PhD, Chairman, Chief Executive Officer, and Chief Scientific Officer September 28, 2012
Prostones play an important role in the body Sucampo’s value proposition: . Proprietary prostone technology based on discoveries by Sucampo’s founders Strong patent portfolio (>580 issued) . Proven ability to get products approved globally . Upcoming product launches and growth in existing products . Deep and diverse pipeline Closing
