SphingoGene, Inc.Delaware C-Corporation

Small Molecule Platform for Improving Radiation Treatment of

Prostate and other Cancers

1

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

2

“My granddad died of prostate cancer. I have dedicated my thesis work which has led to our lead clinical compound to him.”

Joseph ChengMUSC MD/PhD candidate

SphingoGene Researcher

“Hurry up! The Baby Boom generation is getting prostate cancer!”

Ken BurgerAuthor of “Baptized in Sweet Tea”

Prostate Cancer Patient, Charleston, S.C.

Why Start with Prostate Cancer?

3

• 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

Prostate Cancer

4

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

5

Our Value Proposition

•Enhances Radiotherapy leading to more effective cancer treatment

•Fewer side effectsAchieve 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

6

Drug Target Stage of DevelopmentSPG 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 Drug

Efficacy established in rodent tumor xenograft pancreatic cancer models and cell lines

SPG 104 SK1 Inhibitor Clinical Efficacy in vitro and in vivo pending

• Clinical efficacy established in animal models of cancer at nM concentrations• Dose Escalation: No toxicity observed at effective doses and 20 X higher doses

Progress and Leads

Lead Small Molecule Candidates (of 40):

7

• SG105 (clinical lead) Significantly Reduces Tumor Size; in vivo mouse Xenograft Prostate Tumor Model

8

In Vivo Efficacy

4

5

6

7

8

9

10

0 10 20 30

Log 2

xeno

graf

t siz

e(%

of i

nitia

l vol

ume)

Days

Control (6)

IR only (10)

Vehicle (8)

Veh+IR (10)

LCL521 (10)

LCL+IR (10)

4

5

6

7

8

9

10

20 30 40 50 60 70 80 90 100

Log2

xen

ograft size

(% of initia

l volum

e)

Days

Control (6)IR only (10)Vehicle (8)Veh+IR (10)LCL521 (10)LCL+IR (10)

Log

2 Tu

mor

Siz

e(%

of i

nitia

l vol

ume)

Control (n=6)Radiation (Rad) Only (n=10)Vehicle Only (n=8)Vehicle + Rad (n=10)SPG105 Only (n=10)SPG105 + Rad (n=10)

• SPG105 Significantly Reduces Mortality; in vivo mouse Xenograft Prostate Tumor Model

9

0 25 50 75 1000

25

50

75

100

VehicleVeh+IRLCL521LCL+IR

ControlCtl+IR

Days

Perc

ent s

urvi

val

In Vivo EfficacyP

erce

nt S

urvi

val

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 A1Issued patent for SPG103; US8,093,393 B2Patent pending for SPG104; US 2012/0035268 A1

10

Prostate Cancer Market

11

• United States: 241,740 cases/year• Worldwide: 903,500 cases/year•Up to 50% will receive radiation therapy; 30% as a

first line treatmentTarget population for Phase 2a clinical trial15% are high risk patients with a significant chance of

relapse within 3 years

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

12

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

Other Markets

Estimated worldwide market projected in billions

13

Regulatory Path and TimelinesInvestigational 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:

14

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: $125,000Total: $2.34 Million of Non-dilutive funding

15

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

16

Exit Strategy•Potential acquirers/licensees are being

identified•For the company: multiple milestones after

licenses/acquisitions •Similar opportunities available for investors

17

• 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

Management Team & Advisors18

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

19

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

20