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Small Molecule Platform Improving Radiation Treatment

SphingoGene, Inc.
Delaware C-Corporation

James S Norris PhD Interim CEO

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


Acid Ceramidase
Radiation Therapy

Prevents ceramide accumulation Allows escape from cell death

How our drugs work:

Cancer Cell Death


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


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


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.


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


Financial plan
Company Targets
Phase I Trial Phase IIa Trial

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


Exit Strategy
Potential acquirers/licensees are being identified For the company: multiple milestones after licenses/acquisitions

Similar opportunities available for investors


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,