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Senesco Technologies, Inc.

Changing Cancer Therapy
Leslie J. Browne, Ph.D.
President & CEO
October 23rd, 2013
Safe Harbor Statement

Certain statements included in this presentation are forward-looking statements within the meaning of
the Private Securities Litigation Reform Act of 1995. Actual results could differ materially from such
statements expressed or implied herein as a result of a variety of factors, including, but not limited to:
the Company’s ability to recruit patients for its clinical trial, the ability of the Company to consummate
additional financings; the development of the Company’s gene technology; the approval of the
Company’s patent applications; the successful implementation of the Company’s research and
development programs and collaborations; the success of the Company's license agreements; the
acceptance by the market of the Company’s products; the timing and success of the Company’s
preliminary studies, preclinical research and clinical trials; competition and the timing of projects and
trends in future operating performance, the Company’s ability to comply with the continued listing
standards of the NYSE/MKT, as well as other factors expressed from time to time in the Company’s
periodic filings with the Securities and Exchange Commission (the "SEC"). As a result, this press
release should be read in conjunction with the Company’s periodic filings with the SEC. The forwardlooking statements contained herein are made only as of the date of this press release, and the
Company undertakes no obligation to publicly update such forward-looking statements to reflect
subsequent events or circumstances.

2
About Senesco

 Founded 1998
 Located Bridgewater, NJ
 9 employees
 Focused on cancer therapeutics
 Listed on OTC QB as SNTI
Senesco’s Gene Regulation
Platform Technology
 Senesco Technologies is a clinical stage biotech company specializing
in cancer therapeutics
 Our proprietary gene regulation technology has demonstrated the
ability to eliminate cancerous cells and protect healthy cells from
premature death
 The company is running a Phase 1b/2a trial with a product that treats
B-cell cancers (which include multiple myelomia and non-Hodgkins Bcell lymphomas)
 Trial sites include the Mayo Clinic and the Fred Hutchinson Cancer
Research Center
 The technology was developed over the last 15 years through the
discovery that the genetic pathway for cell growth control, targeted by
Senesco, is common to both plants and humans
Stock Basics & Financial Summary

Ticker:
Stock Exchange:
Recent Price:
Cash on Hand as of June 30, 2013, as adjusted
Current burn rate per quarter
Revolving secured credit line

SNTI
OTC QB
$3.30
$3,200,000
$1,200,000
$2,300,000

Capitalization Table as of October 21, 2013
Common Stock Outstanding
Preferred Stock
Options
Warrants Outstanding
Fully Diluted

3,067,000
232,000
278,000
283,000
3,860,000
5
Senesco’s Platform
A novel therapeutic
approach based on gene
regulation

6
Senesco’s Platform Targets
B-cell Cancers
 Multiple myeloma is an incurable cancer

of plasma cells – “B-cell” cancer
 ~65,000 patients in the US
 Median survival time is 2½ to 5 years

 Diffuse large B-cell lymphoma (DLBCL)
 Most commonly diagnosed lymphoma
 ~120,00 patients in the US
 ~ 50% of patients unresponsive to standard

therapy
 Mantle cell lymphoma (MCL)

All orphan drug indications

 ~30,000 patients in the US
 Aggressive tumor with poor outcomes

7
Multiple Myeloma: Large Market &
High Unmet Medical Need
Company

Drug®

2012 Sales

Takeda

Velcade

$2,100 MM

Celgene

Revlimid

$3,770 MM

Celgene

Thalomid

$387 MM

Onyx

Kyprolis

~$250 MM (1st year)

Multiple myeloma market projected at $6 billion by 2018
Turning on a Normal Regulatory
Process

 Lysine Protein – need to turn up
 Activates programmed cell death (apoptosis)
 Hypusine Protein – need to turn down
 Stimulates cell growth
 Senesco’s drug reprograms the cells to
recognize the death message by modulating
the levels of both proteins
9
Proteins Regulate Cell Growth
and Death
These two proteins act as a biological
switch to promote cell death or survival
amino
acid

Lysine

Hypusine

F5A
protein
protein

protein

Lysine form
stimulates cell death
aka “apoptosis”

amino
acid

Hypusine form
stimulates survival

 Lysine and hypusine proteins control cell death and
growth

10
Senesco’s Drug Candidate
Changes Levels of Both Proteins
Therapeutic Strategy: Our drug down-regulates the
growth message and up-regulates the death message so
inducing cancer cells to die
amino
acid

Lysine
Arginine

Hypusine

amino
acid

switch

Lysine form
Arginine form
stimulates cell death
aka “apoptosis”

F5A
protein
protein

protein

Hypusine form
promotes survival

 Replace hypusine with arginine
 Switch from survival to cell death

11
Our Drug Uses Nanotechnology

1. RNAi suppresses pro-survival
hypusine form
2. DNA plasmid makes stable death
message under control of B-cell
specific promoter
3. Polymer (PEI) forms nanoparticle
to protect RNA and DNA and
deliver death message to target

SNS01-T nanoparticle
~ 40 x 70 nM

12
Pre-Clinical Results
Efficacy in Cancer Cell Lines
and In Vivo Models
 Our drug platform modulates and has broad
activity in most cancer cell lines tested in vitro
 Efficacy in multiple in vivo disease models
including melanoma (B16-F0) and lung (A549)
cancer*
 Efficacy in blood cancer models in mice
 85-95% growth inhibition in B-Cell cancers
 Synergy with bortezomib and lenalidomide

* Gene Ther Mol Biol 12, 207-218 (2008)
14
>90% Inhibition of Human Multiple
Myeloma Tumors in Mice

* p < 0.05 (n = 3)
** p < 0.01 (n = 3)
*** p < 0.001 (n = 3)

Last Injection

*

*** ***

** ***

**

**

*** ***

*** ***

***

***

15
Tumor Growth Control

Human multiple myeloma tumors reduced
up to 95% by SNS01-T treatment
Significantly Improved Survival
in Multiple Myeloma Model
EX31-RPMI 8226:Survival proportions

RPMI 8226
Multiple Myeloma
Tumor Growth Inhibition
102 Day Survival

100

***

90

Percent survival

80

**

70
60
50

End of treatment

40

1. Lenalidomide 50mg
20% (p < 0.05)
2. SNS01-T 0.375mg
60% (p < 0.01)
3. SNS01-T+ LEN 50
100% (p < 0.001)

**

30
20
10
0
0

10

20

30

40

50

60

70

80

90

100

110

Days of Treatment
Control Nanoparticle
SNS01-T
LEN 50
SNS01-T +LEN 50

* p < 0.05; ** p < 0.01; *** p < 0.001
(compared to control group; Logrank test)

 SNS01-T is
better alone
 Combined
even better
17
Clinical Trial Status
Phase 1b/2a B-cell Cancer Study
Design
 Open-label, multiple-dose, dose-escalation
 Twice-weekly IV infusions for 6 weeks
Endpoints
 Safety and tolerability
 Pharmacokinetics
 Tumor response
 Time to relapse or progression
Clinical Sites
 Mayo Clinic, U Arkansas, Hackensack UMC, U West
Virginia, Seattle Cancer Care Alliance
19
Phase 1b/2a Interim Results
from Groups 1 & 2
 10 patients enrolled in groups 1 and 2
 Good tolerability and stable disease at lowest doses
 2 patients had stable disease at weeks 3 & 6
 1 patient remained stable 4 weeks post dosing
 No drug-related severe adverse events
 No dose-limiting toxicities
20
Recruitment Continuing at Next
Dose Level

 Phase 1b/2a group 3 is ongoing at 0.2 mg/Kg
• 0.2 mg/Kg is efficacious dose level in cancer
models in mice
• 3 patients need to be completed

21
Clinical Development Plans

 Group 3 results by 4Q-2013
 Topline results of Phase 1b/2a in 1H-2014
 Phase 2 initiation planned for 2H-2014
• Focus on multiple myeloma, mantle and diffuse large
B-cell lymphomas

22
Corporate
Board of Directors

Harlan W. Waksal, M.D.

Chair, Former COO, Imclone Systems

Christopher Forbes

Vice Chair, Forbes Media, LLC

John N. Braca

Exec Director Controller, Iroko Pharma

Leslie J. Browne, Ph.D.

Pres & CEO, former CEO Pharmacopeia

Warren Isabelle, CFA

Founder, Ironwood Investment Mgmt

Thomas C. Quick

Former Vice Chair, Quick & Reilly/Fleet

David Rector

Principal David Stephen Group

Ruedi Stalder

Former Exec Board Member Credit Suisse

John E. Thompson, Ph.D.

Executive VP, R&D, FRS of Canada
24

Jack Van Hulst

Operating Partner, SK Capital Partners
Summary
World-class science and management team
Founder of ImClone, Dr. Harlan Waksal, is
Chairman of the Board
Near-term clinical trial results from Phase 1b/2a
study
6 billion dollar opportunity for multiple myeloma
alone
Many additional applications in solid tumors,
inflammation and Alzheimers

25
Corporate Information

Senesco Technologies, Inc.
721 Route 202/206, Suite 130
Bridgewater, NJ 08807
Phone: 908-864-4444
www.senesco.com
OTCQB: SNTI
IR: RedChip Companies, Inc
Dave Gentry
Phone: 800-RED-CHIP (733-2447), ext.104
Email: info@redchip.com
26

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Snti 102013

  • 1. Senesco Technologies, Inc. Changing Cancer Therapy Leslie J. Browne, Ph.D. President & CEO October 23rd, 2013
  • 2. Safe Harbor Statement Certain statements included in this presentation are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results could differ materially from such statements expressed or implied herein as a result of a variety of factors, including, but not limited to: the Company’s ability to recruit patients for its clinical trial, the ability of the Company to consummate additional financings; the development of the Company’s gene technology; the approval of the Company’s patent applications; the successful implementation of the Company’s research and development programs and collaborations; the success of the Company's license agreements; the acceptance by the market of the Company’s products; the timing and success of the Company’s preliminary studies, preclinical research and clinical trials; competition and the timing of projects and trends in future operating performance, the Company’s ability to comply with the continued listing standards of the NYSE/MKT, as well as other factors expressed from time to time in the Company’s periodic filings with the Securities and Exchange Commission (the "SEC"). As a result, this press release should be read in conjunction with the Company’s periodic filings with the SEC. The forwardlooking statements contained herein are made only as of the date of this press release, and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances. 2
  • 3. About Senesco  Founded 1998  Located Bridgewater, NJ  9 employees  Focused on cancer therapeutics  Listed on OTC QB as SNTI
  • 4. Senesco’s Gene Regulation Platform Technology  Senesco Technologies is a clinical stage biotech company specializing in cancer therapeutics  Our proprietary gene regulation technology has demonstrated the ability to eliminate cancerous cells and protect healthy cells from premature death  The company is running a Phase 1b/2a trial with a product that treats B-cell cancers (which include multiple myelomia and non-Hodgkins Bcell lymphomas)  Trial sites include the Mayo Clinic and the Fred Hutchinson Cancer Research Center  The technology was developed over the last 15 years through the discovery that the genetic pathway for cell growth control, targeted by Senesco, is common to both plants and humans
  • 5. Stock Basics & Financial Summary Ticker: Stock Exchange: Recent Price: Cash on Hand as of June 30, 2013, as adjusted Current burn rate per quarter Revolving secured credit line SNTI OTC QB $3.30 $3,200,000 $1,200,000 $2,300,000 Capitalization Table as of October 21, 2013 Common Stock Outstanding Preferred Stock Options Warrants Outstanding Fully Diluted 3,067,000 232,000 278,000 283,000 3,860,000 5
  • 6. Senesco’s Platform A novel therapeutic approach based on gene regulation 6
  • 7. Senesco’s Platform Targets B-cell Cancers  Multiple myeloma is an incurable cancer of plasma cells – “B-cell” cancer  ~65,000 patients in the US  Median survival time is 2½ to 5 years  Diffuse large B-cell lymphoma (DLBCL)  Most commonly diagnosed lymphoma  ~120,00 patients in the US  ~ 50% of patients unresponsive to standard therapy  Mantle cell lymphoma (MCL) All orphan drug indications  ~30,000 patients in the US  Aggressive tumor with poor outcomes 7
  • 8. Multiple Myeloma: Large Market & High Unmet Medical Need Company Drug® 2012 Sales Takeda Velcade $2,100 MM Celgene Revlimid $3,770 MM Celgene Thalomid $387 MM Onyx Kyprolis ~$250 MM (1st year) Multiple myeloma market projected at $6 billion by 2018
  • 9. Turning on a Normal Regulatory Process  Lysine Protein – need to turn up  Activates programmed cell death (apoptosis)  Hypusine Protein – need to turn down  Stimulates cell growth  Senesco’s drug reprograms the cells to recognize the death message by modulating the levels of both proteins 9
  • 10. Proteins Regulate Cell Growth and Death These two proteins act as a biological switch to promote cell death or survival amino acid Lysine Hypusine F5A protein protein protein Lysine form stimulates cell death aka “apoptosis” amino acid Hypusine form stimulates survival  Lysine and hypusine proteins control cell death and growth 10
  • 11. Senesco’s Drug Candidate Changes Levels of Both Proteins Therapeutic Strategy: Our drug down-regulates the growth message and up-regulates the death message so inducing cancer cells to die amino acid Lysine Arginine Hypusine amino acid switch Lysine form Arginine form stimulates cell death aka “apoptosis” F5A protein protein protein Hypusine form promotes survival  Replace hypusine with arginine  Switch from survival to cell death 11
  • 12. Our Drug Uses Nanotechnology 1. RNAi suppresses pro-survival hypusine form 2. DNA plasmid makes stable death message under control of B-cell specific promoter 3. Polymer (PEI) forms nanoparticle to protect RNA and DNA and deliver death message to target SNS01-T nanoparticle ~ 40 x 70 nM 12
  • 14. Efficacy in Cancer Cell Lines and In Vivo Models  Our drug platform modulates and has broad activity in most cancer cell lines tested in vitro  Efficacy in multiple in vivo disease models including melanoma (B16-F0) and lung (A549) cancer*  Efficacy in blood cancer models in mice  85-95% growth inhibition in B-Cell cancers  Synergy with bortezomib and lenalidomide * Gene Ther Mol Biol 12, 207-218 (2008) 14
  • 15. >90% Inhibition of Human Multiple Myeloma Tumors in Mice * p < 0.05 (n = 3) ** p < 0.01 (n = 3) *** p < 0.001 (n = 3) Last Injection * *** *** ** *** ** ** *** *** *** *** *** *** 15
  • 16. Tumor Growth Control Human multiple myeloma tumors reduced up to 95% by SNS01-T treatment
  • 17. Significantly Improved Survival in Multiple Myeloma Model EX31-RPMI 8226:Survival proportions RPMI 8226 Multiple Myeloma Tumor Growth Inhibition 102 Day Survival 100 *** 90 Percent survival 80 ** 70 60 50 End of treatment 40 1. Lenalidomide 50mg 20% (p < 0.05) 2. SNS01-T 0.375mg 60% (p < 0.01) 3. SNS01-T+ LEN 50 100% (p < 0.001) ** 30 20 10 0 0 10 20 30 40 50 60 70 80 90 100 110 Days of Treatment Control Nanoparticle SNS01-T LEN 50 SNS01-T +LEN 50 * p < 0.05; ** p < 0.01; *** p < 0.001 (compared to control group; Logrank test)  SNS01-T is better alone  Combined even better 17
  • 19. Phase 1b/2a B-cell Cancer Study Design  Open-label, multiple-dose, dose-escalation  Twice-weekly IV infusions for 6 weeks Endpoints  Safety and tolerability  Pharmacokinetics  Tumor response  Time to relapse or progression Clinical Sites  Mayo Clinic, U Arkansas, Hackensack UMC, U West Virginia, Seattle Cancer Care Alliance 19
  • 20. Phase 1b/2a Interim Results from Groups 1 & 2  10 patients enrolled in groups 1 and 2  Good tolerability and stable disease at lowest doses  2 patients had stable disease at weeks 3 & 6  1 patient remained stable 4 weeks post dosing  No drug-related severe adverse events  No dose-limiting toxicities 20
  • 21. Recruitment Continuing at Next Dose Level  Phase 1b/2a group 3 is ongoing at 0.2 mg/Kg • 0.2 mg/Kg is efficacious dose level in cancer models in mice • 3 patients need to be completed 21
  • 22. Clinical Development Plans  Group 3 results by 4Q-2013  Topline results of Phase 1b/2a in 1H-2014  Phase 2 initiation planned for 2H-2014 • Focus on multiple myeloma, mantle and diffuse large B-cell lymphomas 22
  • 24. Board of Directors Harlan W. Waksal, M.D. Chair, Former COO, Imclone Systems Christopher Forbes Vice Chair, Forbes Media, LLC John N. Braca Exec Director Controller, Iroko Pharma Leslie J. Browne, Ph.D. Pres & CEO, former CEO Pharmacopeia Warren Isabelle, CFA Founder, Ironwood Investment Mgmt Thomas C. Quick Former Vice Chair, Quick & Reilly/Fleet David Rector Principal David Stephen Group Ruedi Stalder Former Exec Board Member Credit Suisse John E. Thompson, Ph.D. Executive VP, R&D, FRS of Canada 24 Jack Van Hulst Operating Partner, SK Capital Partners
  • 25. Summary World-class science and management team Founder of ImClone, Dr. Harlan Waksal, is Chairman of the Board Near-term clinical trial results from Phase 1b/2a study 6 billion dollar opportunity for multiple myeloma alone Many additional applications in solid tumors, inflammation and Alzheimers 25
  • 26. Corporate Information Senesco Technologies, Inc. 721 Route 202/206, Suite 130 Bridgewater, NJ 08807 Phone: 908-864-4444 www.senesco.com OTCQB: SNTI IR: RedChip Companies, Inc Dave Gentry Phone: 800-RED-CHIP (733-2447), ext.104 Email: info@redchip.com 26