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1 Small Molecule Platform for Improving Radiation Treatment of Prostate and other Cancers SphingoGene, Inc. Delaware C-Corporation 2 Background on SphingoGene • Founded in 2006 by scientist-entrepreneurs at the Medical University of South Carolina (MUSC) • Obtained exclusive worldwide rights to the intellectual property from MUSC 3 Why Start with Prostate Cancer? “My granddad died of prostate cancer. I have dedicated my thesis work which has led to our lead clinical compound to him.” Joseph Cheng MUSC MD/PhD candidate SphingoGene Researcher “Hurry up! The Baby Boom generation is getting prostate cancer!” Ken Burger Author of “Baptized in Sweet Tea” Prostate Cancer Patient, Charleston, S.C. 4 Prostate Cancer • Forms in male prostate gland • Most common cancer in men • Risk increases with age •In 2012: 241,740 men will be diagnosed 25,170 will die from the disease 5 How Our Platform Works •Ceramide levels increase during radiation therapy; leads to cancer cell death •Acid ceramidase (AC) and Sphingosine Kinase (SK) activity increase during radiation therapy in cancer cells •AC reduces ceramide levels, SK forms S1P, both permitting cancer cell survival •Our compounds inhibit AC or SK or mimic ceramide making radiation or other therapies more effective at inducing cancer cell death 6 Our Value Proposition • Enhances Radiotherapy leading to more effective cancer treatment • Fewer side effects Achieve same clinical benefit with reduced radiation • Better quality of life • Greater preservation of sexual function • Reduce incidence of relapse = Reduced overall treatment costs and reduced death rate • Small Molecules = Easy manufacturing and delivery 7 Progress and Leads •Clinical efficacy established in animal models of cancer at nM concentrations •Dose Escalation: No toxicity observed at effective doses and 20 X higher doses Lead Small Molecule Candidates (of 40): Drug Target Stage of Development SPG 105 AC Inhibitor Clinical lead; efficacy established in rodent tumor xenograft models and cell culture models of prostate and breast cancers SPG 103 Ceramide-like Efficacy established in rodent tumor xenograft pancreatic Drug cancer models and cell lines SK1 Inhibitor Clinical Efficacy in vitro and in vivo pending SPG 104 8 In Vivo Efficacy •SG105 (clinical lead) Significantly Reduces Tumor Size; in vivo mouse Xenograft Prostate Tumor Model Log2 size Log Log2 xenograft 2 Tumor Size (% of initial volume) (% of initial volume) 10 10 9 9 8 8 7 7 6 6 5 5 4 4 0 10 20 203030 40 50 60 70 80 90 100 DaysDays Control (6) IR only (10) Control (6) Control (n=6) Radiation (Rad) Only (n=10) IRVehicle only (10) (8) Vehicle VehicleOnly (8) (n=8) Veh+IR (10) Vehicle Rad (n=10) Veh+IR+(10) LCL521(10) (10) (n=10) SPG105 Only LCL521 SPG105 +(10) Rad (n=10) LCL+IR LCL+IR(10) 9 In Vivo Efficacy •SPG105 Significantly Reduces Mortality; in vivo mouse Xenograft Prostate Tumor Model Percent survival Percent Survival 100 75 Control Ctl+IR Vehicle Veh+IR LCL521 LCL+IR 50 25 0 0 25 50 Days 75 100 10 Patent Position • SphingoGene has filed broad patents around targets and various classes of compounds which can affect their targets • Lead Compounds: Worldwide Patent pending for SPG105 (clinical lead); US 2011/0251197 A1 Issued patent for SPG103; US8,093,393 B2 Patent pending for SPG104; US 2012/0035268 A1 11 Prostate Cancer Market • United States: 241,740 cases/year • Worldwide: 903,500 cases/year • Up to 50% will receive radiation therapy; 30% as a first line treatment Target population for Phase 2a clinical trial 15% are high risk patients with a significant chance of relapse within 3 years 12 Financial Assumptions and Forecast • Based on annual estimated US prostate cancer cases treated with radiation therapy • Market penetration expected similar to other cancer therapeutics • No increase in cases, no relapses • $8000 per treatment per patient (drug cost) Estimated worldwide market projected in billions 13 Other Markets Platform applicable to the majority of solid tumors and any cancer for which patients receive radiation therapy, including internal radiotherapy (brachytherapy). Approximate Incidence of other cancer markets (cases/year): •Lung: 1,600,000 •Breast: 1,380,000 •Pancreatic: 220,000 •Oral cavity: 263,900 •Brain: 237,913 Total: 3,701,813 cases/year Estimated worldwide market projected in billions 14 Regulatory Path and Timelines Investigational New Drug Application (IND) Filing in US: •Phase I: Prostate Cancer Patients undergoing primary radiotherapy •Primary Endpoint: Safety/Tolerability •Phase IIa: Prostate Cancer Patients undergoing primary radiotherapy •Primary Endpoint: Safety/Tolerability •Second Endpoint: Efficacy/biochemical relapse Overall Timeline to Exit: 15 Company Funding to Date • NIH/NCI (University) Program Project Grant: $1.6million • NIH Small Business Technology Transfer (STTR) Grant: $432,000 • ARRA stimulus package: $180,000 • South Carolina Research Authority (SCRA) start-up funds and SBIR match: $125,000 Total: $2.34 Million of Non-dilutive funding 16 Anticipated Funding • Phase I/II Small Business Innovative Research (SBIR) Grant (CA174027-01): $2,115,479 • Phase I STTR (CA177006-01): $346,792 • Up to $200,000 (SCRA) Total: $2.6 Million of Non-dilutive Funding 17 Exit Strategy • Potential acquirers/licensees are being identified • For the company: multiple milestones after licenses/acquisitions • Similar opportunities available for investors 18 Management Team & Advisors • James Norris, PhD, Chairman of the board and Interim CEO ▫ Professor, Department of Microbiology & Immunology ▫ Medical University of South Carolina (MUSC) • David Haselwood, Board Member & Business Advisor ▫ Experienced life science VC, entrepreneur & operator ▫ Burrill & Co, Roche, Proventys, Pharmasset, Primera • Yusuf Hannun, MD, Director of the Stony Brook University Cancer Center ▫ Joel Kenney Professor of Medicine, and the Vice Dean for Cancer Medicine ▫ World famous expert in sphingolipid biochemistry 19 SCIENTIFIC ADVISORS AND COLLABORATORS • Besim Ogretmen, Ph.D., Key expert on sphingolipid metabolism • Xiang Liu, MD, PhD, Key scientist and expert on acid ceramidase in cancer • Alicja Bielawska, Ph.D., Key chemist • Zdzislaw M. Szulc, PhD key chemist 20 Clinical Advisors • Thomas Keane, MD, Chairman of Urology, Medical University of SC • Michael Lilly, MD, Professor Department of Medicine, Hem-Onc, Medical University of SC • David Marshall, MD, Associate Professor, Radiation Oncology, Medical University of SC • Carolyn Britten, MD, Associate Professor, Department of Medicine, Hem-Onc, Medical University of SC