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Small Molecule Platform Improving Radiation Treatment

SphingoGene, Inc.
Delaware C-Corporation

James S Norris PhD Interim CEO norrisjs@musc.edu

Management and advisors

James Norris, PhD
Board member and Interim CEO, Professor of Microbiology and Immunology, MUSC with >150 peer reviewed publications

Yusuf Hannun, MD
Board member and world renowned sphingolipid biochemist with >400 peer reviewed publications

David Haselwood, MBA, MPH (Berkeley, CA)

Member of the Board with over 1billion in M&A

Allen Conger, MBA (University of Chicago)

Acting CFO, experienced investment banker

Progress and Leads

Clinical efficacy established in animal models of cancer at low uM concentrations Dose Escalation: No toxicity observed at effective doses and 20 X higher doses
Drug SPG 105 SPG 103 SPG 104 Target AC Inhibitor Stage of Development Clinical lead; efficacy established in rodent tumor xenograft models and cell culture models of prostate and breast cancers

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

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

How our drugs work:

Cancer Cell Death

Ceramide

Acid Ceramidase
Radiation Therapy

Prevents ceramide accumulation Allows escape from cell death

How our drugs work:

Cancer Cell Death

Ceramide

Acid Ceramidase SPG105

Radiation Therapy Inhibits Acid Ceramidase Prevents ceramide accumulation Potentiates Radiation Allows escape from cell death Induced Cancer Killing

Enhanced radiotherapy of prostate cancer means:

Same clinical benefit with reduced radiation
Fewer side effects Greater preservation of sexual function and continence issues Reduced incidence of relapse Target mechanism of radioresistance

Reduced death rates

Preclinical efficacy: prostate tumor model exhibiting a durable cure

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Log 2 Tumor Size (% of initial volume)

9 8 7 6 5 4 0 2 4 6 8 Time (weeks) 10 12 14

SPG105 alone Radiation alone Radiation + SPG105

Prostate Cancer Market

United States: 241,740 cases/year

Worldwide:

903,500 cases/year

Up to 50% will receive radiation therapy;

Target population for Phase 2a clinical trial 15% are high risk patients with a significant local relapse rate within 2 years

Additional spectrum of cancer patients treated with radiation and candidates for co-administration of SPG105
Prostate 79-81 Gy in 40-45 fractions Pancreas 50.4-54 Gy in 28-30 fractions Melanoma 36-60 Gy in 6-30 fractions (big variability) Breast 50.4 - 60 Gy in 28-33 fractions Lung 60-70 Gy in 30-35 fractions Head and Neck 60-70 Gy in 30-35 fractions.

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Company funding
Awarded NCI research grant $1.6M NCI (STTR) grant $432,000 ARRA supplement, $180,000 SCLaunch STTR match $125,000 Anticipated Phase I, II NCI SBIR $2.1M Phase I NCI STTR $346,792 SCLaunch $200,000

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Financial plan
Company Targets
Phase I Trial Phase IIa Trial

From Investors
$1,100,000 $3,640,000

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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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Summary
Novel small molecule strategy for radiosensitization Addressable market is blockbuster scale if the drug becomes a standard of care with radiation therapy Potential return on investment is substantial (30-50x)

Contact Dr James S Norris, norrisjs@musc.edu