Transcript a PowerPoint presentation detailing our

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Small Molecule Platform for
Improving Radiation Treatment
of Prostate and other Cancers
SphingoGene, Inc.
Delaware C-Corporation
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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
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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.
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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
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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
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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
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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
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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)
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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
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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
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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
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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
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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
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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:
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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
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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
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Exit Strategy
• Potential acquirers/licensees are being
identified
• For the company: multiple milestones after
licenses/acquisitions
• Similar opportunities available for investors
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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
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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
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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