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SphingoGene, Inc. Delaware C-Corporation

Small Molecule Platform for Improving Radiation Treatment of Prostate and other Cancers. SphingoGene, Inc. Delaware C-Corporation. Background on SphingoGene. Founded in 2006 by scientist-entrepreneurs at the Medical University of South Carolina (MUSC)

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SphingoGene, Inc. Delaware C-Corporation

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  1. Small MoleculePlatform 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 compoundto 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 withage • 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):

  8. In Vivo Efficacy SG105 (clinical lead) Significantly Reduces Tumor Size; in vivo mouse Xenograft Prostate Tumor Model Control (n=6) Radiation (Rad) Only (n=10) Vehicle Only (n=8) Vehicle + Rad (n=10) SPG105 Only (n=10) SPG105 + Rad (n=10) Log 2 Tumor Size (% of initial volume)

  9. In Vivo Efficacy SPG105 Significantly Reduces Mortality; in vivo mouse Xenograft Prostate Tumor Model Percent Survival

  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

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