Download Anthrax Vaccine Program - Corporate-ir

Vijay B. Samant
President and Chief Executive Officer
UBS Global Life Sciences Conference
September 24, 2003
Safe Harbor
This presentation contains forward-looking statements that
are subject to risks and uncertainties that could cause actual
results to differ materially from those set forth in the forwardlooking statements, including whether any product
candidates will be shown to be safe and efficacious in clinical
trials and the risks set forth in the company’s filings with the
Securities and Exchange Commission. Actual results may
differ materially from those projected. These forward-looking
statements represent the company’s judgment as of the date
of this presentation. The company disclaims, however, any
intent or obligation to update these forward-looking
statements.
Vical Investment Highlights
• Vaccine company – patented non-viral DNA
•
delivery technology
Broad product pipeline:
3 independent, 7 partnered programs
• Phase II product: melanoma
• Infectious disease vaccine programs: CMV, anthrax
• Key partnerships:
•
•
Merck, Merial, Aventis, Corautus Genetics, NIH
Extensive vaccine expertise
Strong cash position - $98 million
Vical’s Strategy
• Develop new vaccines using patented non-viral
•
•
•
•
DNA delivery technology
Focus in-house on infectious disease targets
• No current vaccine
• Obtain proof of efficacy in small trials
Outlicense targets requiring major resources
Advance Allovectin-7® lead cancer product
Leverage vaccine manufacturing expertise
Product Pipeline
OTHER
INFECTIOUS DISEASES
PRECLINICAL
PHASE I
PHASE II
DNA Vaccine
CMV
Vical
DNA Vaccine
ANTHRAX
Vical
HIV Vaccine
AIDS
Animal Health
UNDISCLOSED
Malaria Vaccine
MALARIA
DNA Vaccine
WEST NILE Vical / VRC
DNA Vaccine
EBOLA
Allovectin-7®
MELANOMA
VEGF-2
ANGIOGENESIS (CAD)
Corautus
Growth Factor
ANGIOGENESIS (PAD)
Aventis Pharma
Merck
Merial
Vical / US Navy
Vical / VRC
Vical
Allovectin-7® for Melanoma
• Plasmid DNA containing two genes
• HLA-B7 and 2-microglobulin
• Triggers rejection of tumor
• Restores immune recognition of tumors
• Formulated with DMRIE/DOPE
• Pro-inflammatory
• Stage III or IV melanoma
• Potential use for earlier-stage disease
• Potential application for other solid tumors
Allovectin-7®
High-Dose Phase II Trial
• Multi-center, single arm study
• Completed enrollment of 127 high-dose patients
• Similar patient population to low-dose (10 µg)
•
•
•
Phase II trial
Dose escalation (up to 2000 µg)
Multi-tumor injections (up to 5 lesions/patient)
Interim results from March 2003
• First 91 high-dose patients
• Response rate: 13%
• Excellent safety and tolerability
• Encouraging survival
®
Allovectin-7
Status
• Data maturing for high-dose trial
• 60 of first 91 patients: still alive in March
• 11 of 12 responders: still alive in July
• 7 of 12 responders: disease had not progressed in July
• Median duration of response: 6.4 months in July
• Continued excellent safety and tolerability
• <2% of patients with Grade 3 adverse events
• Review mature data with FDA in 7-9 months
Infectious Disease Vaccine
Development Timelines
Conventional vaccines
Chicken pox vaccine – Varivax
HPV
Rotavirus
MMRV “combination vaccines”
Pentavalent “combination vaccines”
FluMist
Approval years
33
(1962-1995)
14
(1992-2006)
15
(1992-2007)
18
(1989-2007)
12
(1991-2003)
27
(1976-2003)
DNA vaccines
Rapid synthesis/preclinical testing of vaccine
Simpler process scale-up issues
Streamlined bio-analytical testing plan
No advantage in clinical timelines
Integration of clinical/mfg. consistency lots
Potential reduction in size of safety database
Construction/startup of mfg. facilities
Potential years saved
4-6
3-5
3-5
0
2-4
1-3
3-5
NIH Vaccine Research Center
Key Partner in DNA Vaccines
• Established production orders
• Ebola
• West Nile Virus
• HIV
• New large-scale manufacturing agreement
• Option for additional vaccine targets
• Investment in infrastructure
CMV Vaccine Program
• About CMV
• Herpes virus – infects 50-85% in the US by age 40
• High risk groups
• Current therapy inadequate
• NO vaccine available
• Advantages of Vical’s vaccine
• Ability to harness antibody and T-cell responses
• Non-infective - a MUST for immunosuppressed patients
• Proof of efficacy in transplant patients
• Small clinical trials
CMV Vaccine Strategy
• Tiered approach for clinical development
• Rapid advancement - transplant indication
• Substantial downstream commercial potential - universal
indication
• Focus on transplant patients
• Trial Design
• Vaccinate donor to boost immunity
• 4 and 2 weeks prior to transplant
• Vaccinate recipient to provide further boost
• 4 - 6 weeks after transplant
• Monitor for signs of disease
CMV Vaccine Status
• Expert advisory panel in place
• Development pathway defined
•
•
•
•
•
•
• Consensus with FDA
IP freedom to operate
Basic constructs defined and manufactured
Preclinical trials started
Phase I by year-end
Proof of efficacy trial size defined
Key clinical centers selected
Anthrax Vaccine Program
• About anthrax
• Caused by toxins from spore-forming Bacillus anthracis
• Treated with antibiotics (i.e. Cipro)
• Vaccine is antibody-mediated
• Bivalent plasmid DNA vaccine
• Animal Rule: opportunity for rapid approval
• Concept to clinic in 24 months
Anthrax Vaccine Strategy
• Target effectiveness equivalent to
•
•
•
•
licensed vaccine
Potential to improve cross-protection
Reduced number of doses vs. licensed vaccine
• Shorter time to protection
Manufacturing well-characterized product
Stockpiling advantage due to stability
Rabbit Challenge Results
Anthrax Vaccine Program
Group
# Alive/
# Challenged
Letx Neut Titer
(Range)
Mortality Range
(Days)
PA DNA - 3 injections
8/8
2,560-20,480
All Alive
PA DNA - 2 injections
8/8
640-1,280
All Alive
PA DNA - 3 injections
DMRIE/DOPE
8/8
1,280-10,240
All Alive
PA + LF DNA - 3 injections
8/8
2,560-5,120
All Alive
LF DNA - 3 injections
5/9
320-1,280
Days 4-7
AVA - 2 injections
4/4
640-2,560
All Alive
Vector Controls
0/5
0
Days 2-3
Naïve Rabbits
0/12
ND
Days 2-4
All DNA vaccines formulated with Vaxfectin
except for one DMRIE/DOPE group.
Anthrax Spore Challenge Dose: 50-250 LD50.
Anthrax Vaccine Status
• Proof of concept completed in mice
• Challenge study completed in rabbits
•
•
•
•
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• Demonstrated 100% protection
Pre-IND meeting with FDA in December 2002
Manufactured initial clinical vaccine supplies
Phase I by year-end
Government funding of development
Agreement - approval based on Animal Rule
External Collaborations
• Merck
• HIV, cancer
• $23 million received to date
• Merial
• Animal health vaccines
• $7 million received to date
• NIH
• Ebola, West Nile Virus, HIV
• $7 million in grants to date
External Collaborations
• Aventis Pharma
• Angiogenesis - PAD
• $1.5 million received to date
• Corautus Genetics
• Angiogenesis - CAD
• Minority ownership
• U.S. Navy
• Malaria
• $5 million received to date
Nearly $40
million in
revenues
to date from
collaborations
Financial Information
(in millions, except per share data)
Revenues
Expenses
Write-down of investment
Loss from operations
Net investment income1
Net loss
Net loss per share
Cash, cash equivalents and
marketable securities
2Q03
$ 0.6
8.1
0.5
(7.5)
0.5
$ (6.9)
$ (0.34)
2Q02
1H03
1H02
$ 2.4 $ 1.5 $ 4.0
8.4
16.2
16.1
0.5
(6.0)
(15.2) (12.2)
1.0
1.2
1.9
$ (5.0)
(13.9) $ (10.2)
$ (0.25) $ (0.69) $ (0.51)
$
98 $ 123
Management
Depth of Vaccine Expertise
Vijay Samant
President and CEO
Merck
David Kaslow, M.D.
Chief Scientific Officer
Merck, NIH
Alain Rolland, Pharm.D., Ph.D. Valentis, Ciba-Geigy
Product Development
Tom Evans, M.D.
Clinical Development
UC - Davis
Milestones
Launch CMV and anthrax vaccine development programs
Manufacturing agreement with NIH
Encouraging Allovectin-7® interim data at ASCO
$5.7 million SBIR grant for anthrax vaccine program
Extension of Merck agreement to cancer
Multiple patent issuances - U.S. and Europe
• Presentations at scientific conferences
•
•
•
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 ICAAC
• World Vaccine Congress
Start Phase I study of anthrax vaccine (4Q03)
Start Phase I study of CMV vaccine (4Q03)
Startup of new manufacturing facility (1Q04)
Full Allovectin-7® high-dose cohort data (2Q04)
Vical Investment Summary
• Vaccine company – patented non-viral DNA
•
delivery technology
Broad product pipeline:
3 independent, 7 partnered programs
• Phase II product: melanoma
• Infectious disease vaccine programs: CMV, anthrax
• Key partnerships:
•
•
Merck, Merial, Aventis, Corautus Genetics, NIH
Extensive vaccine expertise
Strong cash position - $98 million