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| 8-K - SUCAMPO PHARMACEUTICALS, INC. 8-K - Sucampo Pharmaceuticals, Inc. | a6567193.htm |
Exhibit 99.1
Corporate Update James J. Egan Chief Operating Officer Stanley G.Miele President, Sucampo Pharma Americas Jan Smilek Chief Financial Officer January 10, 2011
Forward-Looking Statement Forward-looking statements contained in this presentation are based on Sucampo’s assumptions and expectations concerning future events. They are subject to significant business, economic and competitive risks and uncertainties that could cause actual results to differ materially from those reflected in the forward-looking stattements. Sucampo’s forward-looking statements could be affected by numerous foreseeable and unforeseeable events and developments such as regulatory delays, the failure of clinical trials, the inability to fund drug development initiatives competitive products and other factors identified in the development initiatives, competitive products and other factors identified in the “Risk Factors” section of Sucampo’s Annual Report on Form 10-K and other periodic reports filed with the Securities and Exchange Commission. While Sucampo may elect to update these statements at some point in the future Sucampo specifically disclaim any obligation to do so, whether as a result of new information, future events or otherwise. In light of the significant uncertainties inherent in the forward-looking information in this presentation, you are cautioned not to place undue reliance on these forward-looking statements.
Sucampo: A Biopharmaceutical CompanySucampo: A Biopharmaceutical Company Sucampo: A Biopharmaceutical CompanySucampo: A Biopharmaceutical Company Rescula® FDA approved for lowering intra-ocular pressure (IOP) in glaucoma and ocular hypertension in patitientts who are intolerant off or insufficiently responsive to other IOP lIOP loweriing medications In-licensed US + Canadian development and marketing rights in April 2009 Awaiting FDA approval of label-enhancing supplemental NDA (sNDA) to re-launch in U.S. Designing trials for additional indications, based on partner’s breakthrough clinical results Amitiza® Only FDA approved drug for chronic idiopathic constipation (CIC) in adults Only FDA approved drug for irritable bowel syndrome with constipation (IBS-C) in adult women Marketing authorization approved (Nov 2009) in Switzerland for CIC indication Phase 3 trial in opioid-induced bowel dysfunction (OBD) to initiate late 2010 U.S + Canadian commercial rights held by Takeda, commercial rights in Japan held by Abbott A deep pipeline leveraging prostone technology, expertise Cobiprostone for prevention of NSAID-induced gastric ulcers in Phase 2 SPI-017 for peripheral arterial disease going into Phase 2 Additional prostones in preclinical development, such as SPI-3608 Strong financial position $110.7 million in cash and investments as of Sept. 30, 2010
Update: Sucampo Acquires Prostone Patent-Holding Company Sucampo Pharmaceuticals, Inc. (SPI) acquired Sucampo AG (SAG), a patent- holding company in December 2010 from SPI’s co founders holding company, in December 2010, from SPI's co-founders Secures control and ownership of the patents and other intellectual property underlying SPI’s current and future prostone products including Amitiza, cobibiprosttone, SPI-017, SPI-3608 and other compounds SPI 017 Advances SPI towards goal of becoming a fully integrated biopharma Eliminates future royalty and milestone payments to SAG from future sales and development milestone payments from current and future products as well as mandatory funding requirements of early-stage compounds in order to maintain rights Terms: Upfront payment of $28.1 million, and 7-year subordinated, unsecured promissory note of $51.9 million. Potential earn-out: if SPI receives any cash in connection with current arbitration against Takeda SAG holders will receive 15% connection with current arbitration against Takeda, SAG holders will receive 15%, up to a maximum of $40 million
Rescula:A Differentiated Ophthalmic Drug Rescula: A unique mechanism of action Rescula activates Maxi K channels in neurons and contractile cells Rescula activates Maxi K channels in neurons and contractile cells Lowers IOP by increased outflow of aqueous humor through trabecular meshwork and uveoscleral pathway Increases both retinal and choroidal components of ocular blood flow to Increases both retinal and choroidal components of ocular blood flow to optic nerve Maintains visual field in glaucoma patients; inhibits apoptosis of retinal neurons and ischemia-induced degeneration of optic nerve fibers in non- neurons and ischemia induced degeneration of optic nerve fibers in non clinical studies Leads to future opportunities in retinal diseases Leads to future opportunities in retinal diseases Dry Age-related Macular Edema (dry AMD) Diabetic Macular Edema (DME) Retinitis Pigmentosa (RP)
Rescula: Phase 2 Clinical Trial Design – Retinitis Pigmentosa Design of Phase 2 Trial A multi-center, randomized, double-blind, three parallel group, placebo- controlled trial conducted by R-Tech Ueno (RTU) at 6 Japanese sites Enrolled 112 mid-to late-stage Retinitis Pigmentosa (RP) patients with visual acuity of 0 5 or more in a narrow visual field acuity of 0.5 or more in a narrow visual field Patients received either one or two drops of active drug or placebo twice a day for 24 weeks Primary endpoint: change from baseline in the mean retinal sensitivity of the Primary endpoint: change from baseline in the mean retinal sensitivity of the central 2-degrees of the ocular fundus as measured with an MP-1 microperimeter Secondary end points included – Change in retina sensitivity measured by Humphrey perimeter (10-2) – Visual acuity – Contrast sensitivity – Health-related Quality of Life (measured by VFQ-25)
Rescula:Results of Phase 2 Clinica lTrial* Rescula met primary endpoint (change from baseline in retinal sensitivity, as measured byy MP-1)) with statistically significant,, dose-dependent improvement in visual function in the high dose group vs. placebo group – No severe adverse effects – Sub-analysis showed these changes from baseline in retinal sensitivity of more than 4dB: Increased Decreased sensitivity Placebo 15.2% 21.2% Low dose 7.9% 15.8% High dose 18.4% 2.6% – Secondary endpoint, changes in retinal sensitivity from baseline to Week 24, as measured by Humphrey perimeter, met with statistical significance in high dose group compared to placebo dose group compared to placebo * R-Tech Ueno press releases of June 3, 2010 and July 15, 2010
Rescula: In-Licensed from R-Tech Ueno Sucampo licensed Rescula’s US and Canadian rights from R-Tech Ueno (RTU) in April 2009 and the right of first refusal to additional indications for which RTU April 2009 and the right of first refusal to additional indications for which RTU develops Rescula RTU is responsible for clinical and commercial supply of Rescula to Sucampo Sucampo paid an upfront payment of $3 million to RTU and is responsible for additional milestone payments Sucampo responsible for development, regulatory and commercialization activities and expenses in the U.S. and Canada
Rescula: Current Status Rescula eye-drops are a prostone-based drug, not a prostaglandin FDA-approved for lowering of intra-ocular pressure (IOP) in primary open- angle glaucoma (POAG) and ocular hypertension patients who are intolerant of or are insufficiently responsive to other IOP lowering medications; not currently available in U.S. Sucampo submitted data developed after Rescula’s FDA approval in 2000 in Sucampo submitted data developed after Rescula s FDA approval in 2000 in an sNDA (August 2009) and amended sNDA in December 2010 Will complete label discussions with FDA before finalizing US launch plans
Amitiza Answers Unmet Medical Needs Represents a major market opportunity More than 14 million (CIC and IBS-C) office visits each year in U.S. Offers proven safety and efficacy for long-term usage – Efficacy + tolerability are similar for both genders + across age groups for CIC – 90% of nausea events diminish after first week of use – Competing products recommended for short-term use only Provides quick and predictable relief of symptoms Provides quick and predictable relief of symptoms – Between 57%-63% of CIC patients respond within 24 hours and remain responsive – IBS-C patients were twice as likely to achieve overall response than those receiving placebo Differentiated mechanisms of action – In CIC, Amitiza activates chloride ion channels, promoting fluid secretion In IBS-C, Amitiza activates chloride ion channels and promotes mucosal barrier protection
Amitiza: Chronic Idiopathic Constipation* Phase 3 pivotal trial design 2 multicenter trials, both randomized, parallel-group, enrolled 479 patients Administered 24 mcg gel capsule of Amitiza or placebo twice daily 4 week treatment period preceded by 2 week baseline pperiod Entry criteria: modified Rome II criteria for functional constipation Primary efficacy endpoint: change from baseline in number of spontaneous bowel movements ((SBMs)) after 1 week of treatment Secondary endpoints included: SBMs at weeks 2, 3 and 4 Percentage of patients with a SBM within 24 hours of first dose Percentage of patients with a SBM within 24 hours of first dose Time to first SBM Barish CF. Dig Dis Sci 2010; 55: 1090-1097 Johanson JF et al Am J Gastroenterol. 2008:103:170-177
Amitiza: Chronic Idiopathic Constipation* Phase 3 Trials Results Amitiza met the primary endpoint with statistical significance (p<0.0001), as Amitiza patients experienced statistically significantly greater mean numbers of SBMs at Week 1 as compared to placebo patients (5.5 / 5.9 vs. 3.5 / 4.0) Secondary endpoint results: In each week of the trials In each week of the trials, Amitiza patients had significantly higher frequency of Amitiza patients had significantly higher frequency of SBMs at all weeks except week 2 Significantly higher percentage of Amitiza patients experienced a SBM within 24 hours of first dose as compared to placebo (57-61.3% vs. 32-37%) Time to first SBM was significantly shorter in Amitiza patients than with placebo Amitiza approved by FDA for treatment of CIC in adults with no upper age limit in January 2006 with no upper age limit in January 2006 * Barish CF, et al Dig Dis Sci 2010; 55: 1090-1097 and Johanson JF, et al Am J Gastroenterol. 2008:103:170-177
Phase 3 Results in CIC: Amitiza* + Zelnorm** Phase 3 Results in CIC: Amitiza* + Zelnorm** Amitiza Mean Number of SBMs/Week 24 mcg bid Placebo Study 0131 5.69 3.46 Study 0232 5.89 3.99 Zelnorm Zelnorm SBMs/Week 2 mg bid 6 mg bid Placebo Study 2301 1.6 2.0 0.9y Study 2302 2.0 1.9 1.0 * Barish CF, et al Dig Dis Sci 2010; 55: 1090-1097 **Zelnorm FDA GI Adv Cmte Briefing Document Johanson JF et al Am J Gastroenterol. 2008:103:170-177
Amitiza: Irritable Bowel Syndrome with Constipation* Design of Amitiza’s two pivotal Phase 3 trials for IBS-C 2 multicenter trials identically designed, randomized, parallel-groups 2 multicenter trials identically designed, randomized, parallel groups 1,171 U.S. patients received 8 mcg Amitiza gel capsule or placebo twice daily 12 week treatment period following a 2 week baseline period Entry criteria: all patients met Rome II criteria for Constipation-Predominant IBS To measure relief, patients responded to a weekly question: “How would you rate your relief of IBS symptoms over the past week compared to how you felt before you entered the study?” 7-point scale used to rate relief: “significantly relieved,,” “moderately relieved,,” “a little bit relieved,” “unchanged,” “a little bit worse,” “moderately worse,” “significantly worse” Endpoint Primary endpoint was percentage of overall responders in drug and placebo groups An overall responder was a monthly responder for at least 2 of the 3 months of the study * Drossman DA, Chey WD, Johanson JF et al, Aliment Pharmacol Ther 2009 Feb;29(3):329-41
Amitiza: Phase 3 IBS-C Overall Responder Rate*Amitiza: Phase 3 IBS-C Overall Responder Rate* Amitiza: Phase 3 IBS-C Overall Responder Rate*Amitiza: Phase 3 IBS-C Overall Responder Rate* Overall 8mcg bid Placebo p valueOverall Responders 8 mcg bid Placebo p value Study ‘431 13.8% 7.8% 0.029 Study ‘432 12.1% 5.7% 0.023 Pooled 13.0% 6.8% 0.001 Amitiza approved by FDA for treatment of IBS-CAmitiza approved by FDA for treatment of IBS C in adult women in April 2008 *Drossman DA, Chey WD, Johanson JF et al, Aliment Pharmacol Ther 2009 Feb;29(3):329-41
Study 4 mg 12 mg Placebo 4 mg 12 mg Placebo ‘301 28.8 26.2 20.5 38.8 38.4 30.2 p value 0.056 0.116 0.033 0.033 ‘307 25.5 26.5 28.2 38.3 42.2 37.0 p value 0.703 0.703 0.837 0.284 ‘351 29.4 26.2 22.1 38.9 45.7 33.3 l 0 200 0 370 0 314 0 016 16 Amitiza: Drossman DA et al, Aliment PharmacolTher 2009 Feb;29(3):329-41 Zelnorm: FDA GI AdvCmteBriefing Document p value 0.200 0.370 0.314 0.016 Study 4 mg 12 mg Placebo 4 mg 12 mg Placebo ‘301 28.8 26.2 20.5 38.8 38.4 30.2 p value 0.056 0.116 0.033 0.033 ‘307 25.5 26.5 28.2 38.3 42.2 37.0 p value 0.703 0.703 0.837 0.284 ‘351 29.4 26.2 22.1 38.9 45.7 33.3 l 0 200 0 370 0 314 0 016 16 Amitiza: Drossman DA et al, Aliment PharmacolTher 2009 Feb;29(3):329-41 Zelnorm: FDA GI AdvCmteBriefing Document p value 0.200 0.370 0.314 0.016 IBS-C phase 3 Results: Overall Responders Amitiza* + Zelnorm** Study 8 mcg bid Placebo Amitiza ‘431 13.8 7.8 p=0.029 ‘432 12.1 5.7 p=0.023 Original Responder Definition Original Responder Definition Changed Responder Definition Changed Responder Definition Zelnorm Zelnorm
Amitiza: Further Opportunities OBD DatafromSuccessfulPhase3OBDTrial* Amitiza: Further Opportunities OBD Data from Successful Phase 3 OBD Trial*DatafromaSuccessfulPhase3OBDTrial*Data from a Successful Phase 3 OBD Trial* Amitiza: Further Opportunities OBD DatafromSuccessfulPhase3OBDTrial* Amitiza: Further Opportunities OBD Data from Successful Phase 3 OBD Trial*DatafromaSuccessfulPhase3OBDTrial*Data from a Successful Phase 3 OBD Trial* Management of Opioid-induced Bowel Dysfunction in non-malignant pain (OBD) patients non malignant pain (OBD) patients Reported results of Amitiza phase 3 trial (OBD0631) at DDW 2010 Patients taking lubiprostone achieved a statistically significant (p=0.02) greater increase in the mean number of SBMs per week in 8 of the 12 weeks of the trial as compared to placebo patients The percentage of patients who achieved a SBM within 24 hours and 48 hours was significantly higher with lubiprostone as compared to placebo (p=0.0126 at 24 hours,, and p=0.0360 at 48 hours)) (p p Statistical significance was achieved for the overall change from baseline in constipation-associated symptom secondary endpoints * DDW 2010, Abstract #780958
Amitiza: Another phase 3 trial of Amitiza for OBD Initiated*Amitiza: Another phase 3 trial of Amitiza for OBD Initiated* Amitiza: Another phase 3 trial of Amitiza for OBD Initiated*Amitiza: Another phase 3 trial of Amitiza for OBD Initiated* Sucampo initiated a phase 3 trial of Amitiza for OBD in December 2010 Sucampo and Takeda to share costs 50-50 Phase 3 trial design: Almost the same protocol as used in the phase 3 trial reported at DDW 2010DDW 2010, except exclusion of patients on methadone pain therapy A randomized, placebo-controlled, multi-center trial in the U.S. and Europe One 24 mcg gel capsule of lubiprostone or placebo twice each day One 24-mcg gel capsule of lubiprostone or placebo twice each day 12 week treatment period Permitted concomitant pain medications include: fentanyl, morphine and oxycontin Goal is to enroll 420 patients Primary endpoint: change from baseline in SBM frequency at Week 8 without reduction in dose of study pain medication *Sucampo press release, Jan. 7, 2011
Amitiza: Solid Cardiovascular Safety Data* Amitiza: Solid Cardiovascular Safety Data* No clinically significant QTc pprolongation was observed when healthyy volunteers were administered lubiprostone at a single 24 or 144 mcg dose No clinically significant QTc prolongation was reported when constipated patients were dosed daily for 3 weeks with varying doses of lubiprostone These findings indicate that lubiprostone treatment does not increase the risk of TdP associated with QTc prolongation Cumulative safety information for lubiprostone administered to patients with chronic constipation or irritable bowel syndrome with constipation are consistent No serious cardiac adverse events attributable to lubiprostone have been reported to date * Sprenger C, Copa A, Morganroth J, Panas R, Ueno R. Effect of lubiprostone, a unique agent for the treatment of chronic idiopathic constipation, on clinical electocardiographic results. Gastroenterology 2007; 132(4 Suppl 2): A-3225 [abstract S2136]
Key Terms of Agreements with Takeda Takeda shall exert best efforts to commercialize and market Amitiza in the U.S. and Canada to maximize net sales of Amitiza Currently covers two indications: CIC in adults and IBS-C in adult women Takeda holds right of first refusal to additional GI indications in US and Canada Takeda holds right of first refusal to additional GI indications in US and Canada Takeda records all U.S. sales, Sucampo receives a royalty Sucampo retains all other rights Takeda also has rights to Amitiza in Canada, but not yet launched Sucampo’s tiered royalty rate: 18% to 26% of annual net sales Sucampo reimbursed for majority of GI clinical development costs Sucampo has received a total of $150 million in upfront and development milestone payments as of Sept. 30, 2010
NetSalesofAmitizaSinceLaunchinApril2006 2006 $209 $193 $165 $155 $165 $23 2008 2009 2010 (9 Months) 20062006 20072007 2008 2009 2010 (9 Months)
Amitiza: Agreement with Abbott Japan A key element in Sucampo’s international growth strategy for Amitiza Abbott received exclusive rights to commercialize lubiprostone in Japan for CIC,, and right of first refusal for additional indications in Japan If successfully developed, Sucampo will supply finished product to Abbott Sucampo retains right to co-promote Amitiza in Japan and to develop mitiza for additional indications Sucampo has received a total of $22.5 million in upfront and milestone payments from Abbott, as of Sept. 30, 2010 Sucampo designed and managed the recently reported successful phase 3 efficacy trial and interim data from long-term safety trial in Japanese CIC patients
Amitiza: Future Opportunities in Japan Japanese Phase 3 efficacy trial * Primary efficacy endpoint reached statistical significance (p=0 001)* Primary efficacy endpoint reached statistical significance (p=0.001)* Double-blind, placebo-controlled multi-center trial, evaluated 124 patients Dose: Placebo or lubiprostone 24-mcg soft gel capsule, twice daily, for 28 days days Results filed with Japanese authorities in marketing application (Sept. 2010) Japanese Phase 3 long-term safety trial** Japanese Phase 3 long term safety trial An open-label, multi-center trial with 209 patients Dose: one lubiprostone 24-mcg gel capsule twice a day for 48 weeks Final results through Week 48 show lubiprostone is safe and well tolerated These safety results being added to Japanese marketing application Sucampo Press Releases: * August 5, 2010, ** Nov. 30, 2010
Future Opportunities: Cobiprostone -Phase 2 Results *Future Opportunities: Cobiprostone -Phase 2 Results * Future Opportunities: Cobiprostone -Phase 2 Results *Future Opportunities: Cobiprostone -Phase 2 Results * Percent of Patients With Ulcers at 12 Weeks Percent of Patients With Ulcers at 12 Weeks 40% 30% 30% 20 7% 20.7% Greatest reduction with 54 mcg compared to naproxen + placebo Naproxen + PlaceboNaproxen + Placebo Naproxen + Cobi 18 mcgNaproxen + Cobi 18 mcg Naproxen + Cobi 36 mcg Naproxen + Cobi 54 mcg *DDW 2010, oral presentation 780837 24
Sucampo’s Clinical Product Opportunities Discovery Preclinical Phase 1 Phase 2 Phase 3 Filed Amitiza (lubiprostone) Opioid-induced bowel dysfunction (OBD) in non-malignant pain patients CIC in Japanese Patients (marketing application filed) CIC --Swiss marketing application approved, awaiting pricing negotiations outcome D ltd l d ti (d AMD) Rescula Lowering IOP in glaucoma and ocular hypertension patients intolerant of or insufficiently responsive to other IOP-lowering medications; sNDA filed, awaiting label discussions Cobiprostone Prevention of NSAID-induced ulcers Dry age-related macular degeneration (dry AMD) Chronic obstructive pulmonary disease (COPD) SPI-017 Pi h l A t i l Di Chronic obstructive pulmonary disease (COPD) Wound Healing Peripheral Arterial Disease
Prostones Fuel Sucampo’s Growth and Deep Product Pipeline Fatty Acids Prostones Amitiza (lubiprostone) Rescula (unoprostone isopropyl) Cobiprostone (SPI-8811) SPI-017 Other Prostones CIC (24 mcg) approved January 2006 FDA approved in 2000 Now planning re-launch in Reported phase 2 trial for prevention of NSAID-induced Planning phase 2 trial for peripheral arterial disease Several compounds selected for preclinical U.S. gastric ulcers p development IBS-C (8 mcg) approved April 2008 Phase 2 protocols for dry AMD under April 2008 dry AMD under development
Prostones Work As Potassium and Chloride Channel Activators Chloride channels activation Potassium channels activation Potential Therapeutic Targets Hyperpolarisation Cell Protection Fluid Secretion Tight Junction Produces Promotes Potential Therapeutic Targets Gastrointestinal ..OBD Cardiovascular ..Peripheral arterial disease (PAD) CNS ..StrokeProstones Work As Potassium and Chloride Channel Activators Prostones Work As Potassium and Chloride Channel Activators Chloride channels activation Potassium channels activation Potential Therapeutic Targets Hyperpolarisation Cell Protection Fluid Secretion Tight Junction Produces Promotes Potential Therapeutic Targets Gastrointestinal ..OBD Cardiovascular ..Peripheral arterial disease (PAD) CNS ..Stroke ..OBD ..Gastric ulcer ..Inflammatory bowel disease, etc. ..Peripheral arterial disease (PAD) ..Myocardial infarction ..Arterial hypertension, etc. ..Stroke ..Alzheimer’s disease ..Parkinson’s disease ..Epilepsy ..Amyotrophic lateral sclerosis (ALS) etc. ..Spinal stenosis Pulmonary ..Chronic obstructive Urology Ophthalmology ..Glaucoma ..Dry AMD ..Retinitis Pigmentosa ..Macular Edema Oncology ..Chemoprotection ..Secondary prevention (adjuvant) ..Chronic obstructive pulmonary disease (COPD) ..Asthma, etc. Urology ..Overactive bladder ..Erectile dysfunction, etc. Macular Edema
Sucampo’s Financial Results and Position (In millions, except per share data) 2007 2008 2009 2010 YTD As of Sept 30 (9 months) Product Royalty Revenue $27.5 $34.4 $38.3 $29.8 R&D Revenue* $59.4 $72.3 $24.0 $15.9 Total Revenue $91.9 $112.1 $67.4 $49.5 Net Income/(Loss) $13.2 $25.0 ($0.8) ($0.1) Earnings Per Share (diluted) Earnings Per Share (diluted) $0 35 $0.35 $0 59 $0.59 ($0 02) ($0.02) $0 0 $0.0 Cash and Investments $86.1 $121.5 $118.3 $110.7 *R&D Revenue includes reimbursement of clinical trial expenses, and revenue recognized from milestone payments for filing and approval of sNDA for IBS-C (in 2007 and 2008, respectively).
Sucampo’s 2010 Milestones Submit NDA in Japan with results of Amitiza in CIC program Submit NDA in Japan with results of Amitiza in CIC program v Report phase 3 efficacy trial results of Amitiza in Japanese CIC patients Complete phase 1 trial of SPL-017 for PAD in Japanese patients v Initiate pivotal phase 3 trial of Amitiza in OBD patients Initiate phase 2 trial of Rescula in dry AMD Plan Amitiza’s Swiss commercialization based on outcome of pricing negotiations
Corporate Update J. Egan Chief Operating Officer Stanley G.Miele President, Sucampo Pharma Americas President, Sucampo Pharma Americas Jan Smilek Chief Financial Officer January 10, 2011