Presentation File 5112c3ed d0fc 4150 Bf1e 0287ac105ace

7/29/2019 Presentation File 5112c3ed d0fc 4150 Bf1e 0287ac105ace

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SphingoGene, Inc.Delaware C-Corporation

James S Norris PhD

Interim CEO

[email protected]

Small Molecule Platform

Improving Radiation Treatment

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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

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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

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 low uM concentrations

Dose Escalation: No toxicity observed at effective doses and 20 X higher doses

Progress and Leads

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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How our drugs work:

Radiation Therapy

CeramideCancerCell

DeathAcid

Ceramidase

Prevents ceramide accumulation

Allows escape from cell death

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How our drugs work:

Radiation Therapy

CeramideCancerCell

DeathAcid

Ceramidase

Prevents ceramide accumulation

Allows escape from cell death

SPG105Inhibits Acid CeramidasePotentiates Radiation

Induced Cancer Killing

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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

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Preclinical efficacy: prostate tumormodel exhibiting a durable cure

Log2T

umorSize

(%o

fin

itialvolume)

7

4

5

6

7

8

9

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0 2 4 6 8 10 12 14

Time (weeks)

SPG105 alone

Radiation alone

Radiation + SPG105


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Prostate Cancer Market

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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 localrelapse rate within 2 years


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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.

Additional spectrum of cancer patientstreated with radiation and candidates for

co-administration of SPG105


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Company fundingAwarded Anticipated

NCI research grant

$1.6M

Phase I, II NCI SBIR

$2.1M

NCI (STTR) grant

$432,000

Phase I NCI STTR

$346,792

ARRA supplement,$180,000

SCLaunch$200,000

SCLaunch STTR match

$125,000

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Financial plan

Company Targets From Investors

Phase I Trial $1,100,000

Phase IIa Trial $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

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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, [email protected]

Documents

Presentation File 5112c3ed d0fc 4150 Bf1e 0287ac105ace