The document summarizes the 2016 annual meeting of stockholders held on July 14, 2016. It includes a forward-looking statement noting risks and uncertainties in projections. The development pipeline outlines several investigational products in various phases of clinical trials for breast cancer, gastric cancer, gynecological cancers, and hematological conditions. An overview of the current status of NeuVax clinical trials in breast cancer is provided, along with collaborations. Summaries are also given for GALE-301 and GALE-401 clinical programs, corporate details, leadership, and motivation.
2. FORWARD LOOKING STATEMENT
This presentation contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. Such statements include, but are
not limited to, statements about future expectations, plans and prospects for the
development and commercialization of the Company's product candidates,
including patient enrollment in our clinical trials, present or future licensing,
collaborative or financing arrangements, expected outcomes with regulatory
agencies, and projected market opportunities for product candidates are subject
to a number of risks, uncertainties and assumptions, including those identified
under “Risk Factors” in the Company’s most recently filed Annual Report on Form
10-K and Quarterly Report on Form 10-Q and in other filings the
Company periodically makes with the SEC. Actual results may differ materially
from those contemplated by these forward-looking statements. The
Company does not undertake to update any of these forward-looking statements
to reflect a change in its views or events or circumstances that occur after the
date of this presentation.
2
3. DEVELOPMENT PIPELINE
Product Therapeutic Area Phase 1 Phase 2 Phase 3 BLA / NDA
Immunotherapy: Breast Cancer
NeuVax™ (nelipepimut-S)
Node-positive
HER2 IHC 1+/2+
NeuVax™ + Herceptin® Node-positive or node negative/triple
negative HER2 IHC 1+/2+
NeuVax™ + Herceptin® High risk, node-positive or negative,
HER2 IHC 3+
NeuVax™ Ductal Carcinoma in Situ (DCIS)
Immunotherapy: Gastric Cancer
NeuVax™ Gastric, HER2 IHC 1+/2+/3+
Immunotherapy: Gynecological Cancer
GALE-301 Ovarian & Endometrial
GALE-301 + GALE-302 Ovarian & Breast
Hematology
GALE-401 (Anagrelide CR) MPN-related thrombocytosis
*NeuVax is an investigational product. Efficacy has not been established. Herceptin is a registered trademark of Genentech.
Ongoing Planned
VADIS
3
2b
StoppedPRESENT
4. PRESENT CURRENT STATUS
IDMC recommend the trial stop for futility
• Study drug and all procedures have stopped
Investigation ongoing
• Compiled team with addition of outside experts and the Company
remains blinded to the data
• Operational elements under review include:
Database and data analysis (programming)
Randomization
Manufacturing and packaging elements such as filling, labeling and
kit assembly
• Biology and immunology
• Anticipated timeline: up to 3 months or longer
4
5. T-Cell
Activating Receptors Inhibitory Receptors
CD28
OX40
GITR
CD122
CD27
CD360
HVEM
CD137
CTLA-4
PD-1
TIM-3
BTLA
VISTA
LAG-3
IMMUNO-ONCOLOGY:
UNLOCKING THE POWER OF THE T-CELL
5
Checkpoint
inhibitors
Indirect Immune
Modulators
Co-stimulators
Immune Inhibitory
Enzymes
CAR T
Technology
TCR
Technology
6. T-Cell
Activating Receptors Inhibitory Receptors
CD28
OX40
GITR
CD122
CD27
CD360
HVEM
CD137
CTLA-4
PD-1
TIM-3
BTLA
VISTA
LAG-3
LACK OF REACTIVE T-CELLS MAY RENDER SOME
TOOLS INEFFECTIVE IN MANY CANCERS
6
Checkpoint
inhibitors
Indirect Immune
Modulators
Co-stimulators
Immune Inhibitory
Enzymes
8. NOVEL DEVELOPMENT STRATEGY:
SECONDARY PREVENTION IN CANCER SURVIVORS
8
RECEIVES
PRIMARY
TREATMENT
• Surgery
• Chemotherapy
• and/or Radiation
Disease free
“survivor”
Breast: HER2, 1+/2+
25% recurrence rate in 3 yrs
No FDA Approved
targeted therapies
Breast: HER2, 3+ High
Risk
20% recurrence rate
DECLARED
TO PREVENT
RECURRENCE /
METASTATIC DISEASE
Breast: Ductal Carcinoma in
Situ
8-10% progression to invasive
Ovarian Cancer
~50% recurrence rate in 1 yr
No FDA Approved
targeted therapies
• Watch &
Wait, or
• Repetitive
therapies
TOLD
9. NEUVAX: DEVELOPMENT COLLABORATIONS
Phase Treatment
HER2
Status
Indication Trial Status
Protocol
Defined
# of Patients
Collaborations
3
Single agent
PRESENT
Study
1+, 2+
BREAST
Node Positive
HLA A2+, A3+
STOPPED
700
(enrolled 758)
2b
Combination
with
trastuzumab
1+, 2+
BREAST
Node Positive or High
Risk Node Negative
HLA A2+, A3+,
A24+, A26+
Enrolling
U.S. only
33 centers
300
2
Combination
with
trastuzumab
3+ high
risk
BREAST
Node Positive
HLA A2+, A3+
Enrolling
U.S. only
28 centers
100
2
Single agent
VADIS Study
1+,
2+,3+
BREAST
Ductal Carcinoma in
Situ (DCIS)
HLA A2+
Recruiting
U.S. only
4 centers
48
2 Single agent
1+,
2+,3+
GASTRIC
HLA A2+, A3+
Planned
India Only
50
9
11. GALE-301 & GALE-302:
CURRENT CLINICAL DEVELOPMENT
11
Phase Treatment Cancer Type
Target
Indication
Current
Status
# of Enrolled
Patients
1/2a GALE-301
Ovarian,
Endometrial
HLA A2+
Ovarian Enrolled 51
1b
GALE-301 &
GALE-302
Ovarian, Breast
HLA A2+
Ovarian /
Breast
Enrolled 39
12. GALE-301: OPTIMAL DOSE GROUP SHOWS
PRELIMINARY EFFICACY
Source: Peoples, et. al, Poster Presentation, American Society of Clinical Oncology 2015 12
Phase 1/2a trial ongoing
Phase 1: Determined optimal dose and
demonstrated safety and potent immune
response
Phase 2a Preliminary data:
• At 16 months median follow-up:
Overall recurrence rate was 44.8% in
the VG versus 54.5% in the CG
(p=0.58),
Recurrence rate of 23.5% in patients
who received booster inoculations.
• Two year DFS estimate in 1000 mcg dose
group is 73.5% vaccine vs. 38.1% control
(p=.03)
• GALE-301 plus GM-CSF is well tolerated
and elicits a strong in vivo immune
response with primarily Grade 1 and
Grade 2 toxicities
Estimated 24 months Disease Free Survival by
Dosing Cohort
14. GALE-401
ANAGRELIDE CONTROLLED RELEASE (CR)
Anagrelide
•Active ingredient
•Reduces the elevated platelet count and the risk of thrombosis in patients with
myeloproliferative neoplasms (MPNs)
•MPNs are hematological malignancies in which the bone marrow cells develop and function
abnormally
Immediate
Release
•Approved for the treatment of patients with thrombocythemia, secondary to MPNs
•IR formulation can cause unacceptable side effects believed to be Cmax-related and has largely
limited the use due to early treatment withdrawal
GALE-401
•Controlled Release (CR) formulation may decrease the frequency or severity of side effects
•Phase 2, Proof-of-Concept Trial Results
•Well tolerated with primarily Grade 1 and 2 toxicities
•Efficacy compares favorably to historical anagrelide IR
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15. ESSENTIAL THROMBOCYTHEMIA (ET):
CURRENT STANDARD OF CARE
15
• Generally first line therapy for ET
• Cytotoxic Myelosuppressive drug (reduces
other blood cells as well)
• Increased risk of developing acute leukemia
after long term; avoided in younger patients
• About 25% of patients are
intolerant/refractory
• Limited third line use
• Non cytotoxic drug
• Not used in most patients because requires
injection and has flu like symptoms
• Used mostly in pregnant women
• Generally second line
• Non cytotoxic drug
• Decreases platelets formation
• Not associated with increased risk of
leukemia
• Side effects: palpitations, headaches
• About one-third are intolerant to Anagrelide
• Hydroxyurea and/or Anagrelide
Treatment Failure
Sources: Leukemia and Lymphoma Society: Essential Thrombocythemia Facts Cervantes, F. Hematology 2011; 215-221
Hydroxyurea
Anagrelide IR
Interferon alpha
Unmet Need
17. FINANCIAL OVERVIEW
Cash Position (as of June 30, 2016) $19.6 million
Debt Financing (May 10, 2016) + $24 million (restricted cash)
Litigation Settlement (July 1, 2016) - $2.3 million
Projected Quarterly Burn $9 - $11 million
Shares Outstanding (as of March 31, 2016) 182 million
Market Cap (as of July 12, 2016) ~$75 million
17
July 2016 Financing $11.7 million (net proceeds)
Common Stock Issued 28,000,000 shares
18. 2016 MILESTONES
18
PROGRAM MILESTONE
PROJECTED
DATE
NeuVax™
(nelipepimut-S)
PRESENT: Achieve 70 Qualifying DFS Events ✓
Fast Track Designation ✓
PRESENT: Interim analysis Stopped
Initiate DCIS trial Q2
Combo H&N 1+/2+ Interim safety data Q4
GALE-301
GALE-302
Present 301/302 booster data ✓
Present GALE-301 Phase 2a two year data ✓
Orphan Drug Designation ✓
Present GALE-301 Biomarker & Dosing Data Q4
GALE-401
(anagrelide CR)
Present combined safety data ✓
Confirmation of 505(b)2 pathway 2H
Publish final Phase 2 report Q4
19. LEADERSHIP TEAM
19
Mark W. Schwartz, Ph.D., President & CEO
Apthera, Bayhill Therapeutics, Calyx Therapeutics,
Trega Biosciences, Incyte Genomics, DuPont
Diagnostics
Bijan Nejadnik, M.D., Executive Vice President,
Chief Medical Officer Jazz Pharmaceuticals,
Johnson & Johnson, Stanford, Johns Hopkins,
UC Davis
Remy Bernarda, SVP, Investor Relations &
Corporate Communications
IR Sense, Hana Biosciences, Knight Equity
Markets, Bear Stearns, Goldman Sachs
Gavin Choy, Pharm.D., SVP, Clinical Sciences &
Operations
Otsuka, Astex, SuperGen, Hana Biosciences,
Gilead, Stanford University Medical Center,
Department of Veteran Affairs
Tom Knapp, Esq., Interim General Counsel
Sucampo, Exemplar Law Partners,
NorthWestern Energy, Paul Hastings, The
Boeing Company
Joe Lasaga, VP, Business Development &
Alliance Management
Nektar Therapeutics, Rigel
Pat Murphy, VP, Regulatory Affairs &
Compliance
Nektar Therapeutics, Bayhill Therapeutics,
Berlex Laboratories, Serono, Parexel, Biogen
20. WHY WE’RE HERE
20
Source: San Antonio Express E75 vaccine's final tests start in S.A. By Don Finley, January 22, 2012;
Photo credit: Kin Man Hui/San Antonio Express-News/ZUMAPress
“I've had several friends
who've had (breast cancer)
and then…it came back
and they had to go through
treatment again. So this
would be wonderful, not to
have to come back.”
– First NeuVax Phase 3 patient