galena biopharma company presentation
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ONCOLOGY FOCUSED IMMUNOTHERAPY COMPANY
Targeting cancer survivorship
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 target revenue from the sales of the Company’s products, the future expectations, plans and prospects for the development and commercialization of the Company's product candidates, including patient enrollment in our clinical trials, and 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.
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ONCOLOGY FOCUSED BIOPHARMACEUTICAL COMPANY
u Pioneering secondary prevention in cancer survivors • Redefining the standard of care • Targeted therapies for prevention of cancer recurrence
u 1st in class vaccines targeting targeting HER2 and Folate Binding Protein in breast and gynecological cancers
u Focused in large markets in areas of major unmet medical need • Phase 3, PRESENT, breast cancer clinical trial ongoing under SPA
u Franchise of mid-stage trials ongoing or planned
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FIRST-IN-CLASS, TARGETED IMMUNOTHERAPY PIPELINE
4
Harnessing the power of the
immune system in the adjuvant setting
u Exploits specificity of natural immune surveillance
u Adjuvant patients have healthy immune systems
u Systemic protection
Goal is to prevent recurrence
u Recurrences are almost always fatal
u Minimal toxicity and improved safety profile
u Boosters provide long term protective effect
Well-validated targets
u HER2
u Folate binding protein (FBP)
Current Programs
u NeuVax™ (nelipepimut-S)
• Breast: HER2 1+, 2+ and 3+, DCIS
• Gastric trial planned
u GALE-301 & GALE-302
• Ovarian
Adjuvant Setting = Minimal Residual Disease
DEVELOPMENT PIPELINE
Product Therapeutic Area Phase 1 Phase 2 Phase 3 BLA / NDA
Immunotherapy: Breast Cancer
NeuVax™ 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
PRESENT
*NeuVax is an investigational product. Efficacy has not been established. Herceptin is a registered trademark of Genentech.
Ongoing Planned
VADIS
5
Adds ~10k patients >$3B
Combo: High risk, HER2 3+
NEUVAX: SIGNIFICANT U.S. COMMERCIAL OPPORTUNITY
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adds ~10k patients >$2.5B
Combo Node Pos or Neg HER2 1+, 2+
HLA-A2, A3, A24, A26
PRESENT 50-60k patients
>$2B
Source: Global Data 2015/Medtrack. Pricing estimates based on a 20% premium to the current average annual price of Herceptin® (U.S. Dollars).
Targeting HER2
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NEUVAX™ (nelipepimut-S)
What it is
8
NEUVAX: HER2 IMMUNODOMINANT PEPTIDE
u NeuVax contains the immunodominant peptide derived from the extracellular region of the HER2 protein
u Peptide (aa 369-377) immunotherapy administered as intradermal injection
u MHC Class I: HLA A2/A3
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K I F G S L A F L
ELICITS A STRONG CD8+ T-CELL RESPONSE
u NeuVax binds to antigen presenting cells (APCs)
u NeuVax stimulates APCs to activate CD8+ cytotoxic T lymphocytes (CTLs)
u CTLs rapidly replicate to seek out and destroy HER2 expressing tumor cells and micro-metastases
u Booster series maintains long term immunologic response
10
Sources: Peoples GE, et al (2005) JCO, 23(300, 7536-7545; Mittendorf EA, et al (2006) Surgery, 139(3): 407-418. Peoples, et al, ASCO 2012 Poster Presentation
0.4
1.8
0.7 0.5
0.0
0.5
1.0
1.5
2.0
2.5
% N
euVa
x sp
ecifi
c C
D8+
T c
ells
NeuVax Specific CD-8 CTLs: Pre-, Post, Mean and Long-Term (6 months)
Pre Max Mean Long-Term
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
11
Checkpoint inhibitors
Indirect Immune Modulators
Co-stimulators
Immune Inhibitory Enzymes
CAR T Technology
TCR Technology
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
12
Checkpoint inhibitors
Indirect Immune Modulators
Co-stimulators
Immune Inhibitory Enzymes
T-Cell
CD28 OX40
GITR
CD122
CD27
CD360
HVEM
CD137
CTLA-4
PD-1
TIM-3
BTLA
VISTA
LAG-3
Activating Receptors Inhibitory Receptors
NEUVAX STIMULATES T-CELL PROLIFERATION AND EXPANSION
13
T cells
Checkpoint inhibitors
Indirect Immune Modulators
Co-stimulators
Immune Inhibitory Enzymes
T cells
T cells
T cells
T cells
T cells
T cells
T cells
T cells
T cells
Where it fits
14
UNIQUELY POSITIONED
u 14.5 million cancer survivors in US (NCI Cancer Survivorship)
• Projected to 19 million survivors in 2024
u Increase in survival due to decades of productive research, improved screening/prevention, and effective treatments
u Survival leads to patients living longer
• 64% alive after 5 years of diagnosis • 41% alive after 10 years of diagnosis • 15% alive after 20 years or longer
u Galena peptide vaccines – NeuVax and GALE-301 are uniquely positioned to maintain survivorship
15 Source: DeSantis CE et al. CA Cancer J Clin 2014: 64:252-271
REDEFINING THE STANDARD OF CARE
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RECEIVES PRIMARY TREATMENT
• Surgery
• Chemotherapy
• Radiation
Slight evidence of disease
Disease free “survivor”
HER2 Status Standard of Care
HER2, 3+ (20-25% of patients)
Multiple, including Herceptin®
HER2, 1+/2+ (50-60% of patients)
No FDA approved therapies
HER2, 3+ High Risk (20-25% of patients)
• PRESENT trial
• NeuVax + trastuzumab
No FDA approved therapies • NeuVax +
trastuzumab
BREAST CANCER PROGRAMS
Phase Treatment HER2Status Indica6on TrialStatus
ProtocolDefined
#ofPa6entsCollabora6ons
3SingleagentPRESENTStudy
1+,2+ NodePosi6veHLAA2+,A3+
Enrolled13countries~140centers
700(enrolled758)
2bCombina6on
withtrastuzumab
1+,2+
NodePosi6veorHighRiskNodeNega6ve
HLAA2+,A3+,A24+,A26+
EnrollingU.S.only34centers
300
2Combina6on
withtrastuzumab
3+highrisk
NodePosi6veHLAA2,A3+
EnrollingU.S.only30centers
100
2 SingleagentVADISStudy
1+,2+,3+
DuctalCarcinomainSitu(DCIS)HLAA2+
Planned4U.S.sites 48
17
PRIMARY PREVENTION Expansion potential for safe vaccine in DCIS
METASTATIC DISEASE Expansion potential in combination with checkpoint inhibitors /immune modulators
NEUVAX: ACROSS THE BREAST CANCER TREATMENT SPECTRUM
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PROOF OF CONCEPT: Established in population with no standard of care treatment options
SECONDARY PREVENTION
IDEAL SETTING: Adjuvant treatment in patient population with no evidence of disease
MOST ADVANCED: PRESENT is the largest and only Phase 3 breast cancer vaccine trial
Where it stands
19
SN-33 PHASE 2 HER2 IHC 1+/2+ (N=45)
20
Source: 2012 San Antonio Breast Cancer Poster, Mazanet, et al.
PHASE 3, PRESENT TRIAL
Prevention of Recurrence in Early-Stage, Node Positive Breast Cancer with Low to Intermediate Her2 Expression with NeuVax Treatment u Trial being run under FDA-approved SPA
u Enrollment completed in April 2015 (n=758) • Adjuvant breast cancer patients, Node Positive, HER2 1+/2+, HLA A2/A3+
u Patient friendly regimen via intradermal injection • Primary Vaccine Series – injection once a month for 6 months • Booster Series – injection once every 6 months
u Upcoming Key Milestones • Interim safety/futility analysis: 2Q16 • Final Endpoint: 2018
21
PHASE 3 PRESENT TRIAL PER SPA
1 2 3 4
Interim analysis by DSMB at n=70 events
Endpoint DFS at n=141 events /
36 months
Dosing by Month + 1 booster dose every 6 months thereafter
5 6
Adjuvant breast cancer patients, randomized 1:1
§ Double blind
§ Node positive
§ HLA A2/A3+
§ HER2 IHC 1+/2+
§ Stratified by stage, type of surgery, hormone receptor, and menopausal status
§ Enrollment complete: n=758 Patients
Study Population + GM-CSF
Placebo + GM-CSF
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Prevention of Recurrence in Early-Stage, Node Positive Breast Cancer with Low to Intermediate Her2 Expression with NeuVax Treatment
NEUVAX FRANCHISE EXPANSION OPPORTUNITIES
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Breast Cancer HER2 1+/2+
Development Priority
1°
Breast Cancer HER2 1+/2+/3+ w/Herceptin
Gastric Cancer HER2 1+/2+/3+
Ductal Carcinoma in Situ (DCIS)
Breast Cancer Node Negative, HER2 1+/2+
Bladder Cancer
Prostate Cancer
Non Small Cell Lung Cancer
Breast Cancer HER2 1+/2+/3+ w/Checkpoint Inhibitor
Colorectal Cancer
Ovarian Cancer
2° 3°
Note: Boxed programs are underway or planned.
GALE-301 & GALE-302
Targeting Folate Binding Protein
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GALE-301: FOLATE BINDING PROTEIN (FBP)
25
0.0%
5.0%
10.0%
15.0%
20.0%
25.0%
30.0%
Vaccine Control
% o
f Sub
ject
s
Recurrence
24.0% 13.3%
Source: U.S. Ovarian Cancer http://seer.cancer.gov/statfacts/html/ovary.html
u Targeted cancer immunotherapy
u FBP is over-expressed (20-80 fold) in >90% of ovarian and endometrial cancers
u FBP has very limited tissue distribution and expression in non-malignant tissue making it an ideal immunotherapy target
u Current treatments are generic • Carboplatin and paclitaxel • High recurrence rate
u Most patients relapse with poor prognosis
GALE-301 & GALE-302: CURRENT CLINICAL DEVELOPMENT
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Phase Treatment CancerType TargetIndica6on
CurrentStatus
#ofEnrolledPa6ents
1/2a GALE-301Ovarian,
Endometrial HLAA2+
Ovarian Enrolled 51
1b GALE-301&GALE-302
Ovarian,BreastHLAA2+ Ovarian Enrolled 39
GALE-301: PHASE 1/2a TRIAL
1 2 3 4
Dosing by Month + 1 booster dose every 6 months thereafter 5 6
Diseasefree(NED)ovarianandendometriala[erSoCRx
§ Nodeposi6ve
§ HLAA2+-Vaccine
§ HLAA2-Control
§ N=51
StudyPopula6on
GALE-301+GM-CSF
100 µg GALE-301+GM-CSF
500 µg
GALE-301+GM-CSF
1000 µg
Phase 1 Dose Escalation
GALE-301+GM-CSF
Phase 2 Optimal Dose
Source: ClinicalTrials.gov Identifier: NCT01580696) 27
GALE-301: OPTIMAL DOSE GROUP SHOWS PRELIMINARY EFFICACY
0.0%
5.0%
10.0%
15.0%
20.0%
25.0%
30.0%
Vaccine Control
% o
f Sub
ject
s
Recurrence
24.0% 13.3%
Source: Peoples, et. al, Poster Presentation, European Cancer Congress 2015 28
Phase 1/2a trial ongoing u Phase 1: Determined optimal dose and
demonstrated safety and potent immune response
u Phase 2a Preliminary data in 1000 mcg dose group:
• At 12 months median follow-up:
§ Vaccine group: two clinical recurrences (13.3%) n=15
§ Control group: 12 recurrences (55%) n=22
• 2 year DFS estimate in 1000 mcg dose group is 85.7% vaccine vs. 33.6% control (p<.02)
• GALE-301 plus GM-CSF is well tolerated and elicits a strong in vivo immune response with primarily Grade 1 and Grade 2 toxicities
2 Year DFS Estimate by Dose Cohort
GALE-301 (E39) & GALE-302 (E39’): PHASE 1b TRIAL
Diseasefree(NED)ovarianandbreastcancera[erSoCRx
§ Nodeposi6ve
§ HLAA2+(Vaccine)
§ HLAA2-(Control)
§ Post menopausal
§ N=39
StudyPopula6onGALE-301+GM-CSFx6
Phase 1 Primary Vaccination Series (PVS)
Endpoints: • Immunologic
response assessed by local reaction (LR) after each vaccination
• Delayed-type hypersensitivity (DTH) reaction after completion
Source: ClinicalTrials.gov Identifier: NCT020196524
GALE-302+GM-CSFx3àGALE-301+GM-CSFx3
GALE-301+GM-CSFx3àGALE-302+GM-CSFx3
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1 2 3 4
Dosing by Month + 1 booster dose every 6 months x2 5 6
GALE-301 & GALE 302: DELAYED-TYPE HYPERSENSITIVITY
30
LEGEND EE = E39 (GALE-301) x 6 inoculations (n=12) EE’ = E39 (GALE-301) x 3 inoculations followed by E39’ (GALE-302) x 3 inoculations (n=14) E’E = E39’ (GALE-302) x 3 inoculations followed by E39 (GALE-301) x 3 inoculations (n=13)
R0 = baseline (pre-vaccination) RC1 = 1 month after completion of the PVS RC6 = 6 months after completion of the PVS and pre-booster
Source: Mittendorf et. al., Poster Presentation, Society of the Immunotherapy of Cancer 2015
GALE-401
Anagrelide Controlled Release (CR)
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GALE-401 ANAGRELIDE CONTROLLED RELEASE (CR)
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Indication & Current Treatment
u Active ingredient = anagrelide u Anagrelide reduces the elevated platelet count
and the risk of thrombosis u Immediate release (IR) version approved for the
treatment of patients with thrombocythemia, secondary to myeloproliferative neoplasms (MPNs) • MPNs are hematological malignancies in which the
bone marrow cells develop and function abnormally
u IR formulation can cause unacceptable side effects • Believed to be Cmax-related and has largely limited the
use due to early treatment withdrawal
u Controlled Release (CR) formulation may decrease the frequency or severity of side effects
u Phase 2, Proof-of-Concept, Trial Ongoing
u If successful, Galena will seek approval via the 505(b)(2) regulatory pathway
GALE-401
Source: Anagrelide Package Insert
GALE-401: PHASE 2 PILOT STUDY FINAL RESULTS
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0.0%
5.0%
10.0%
15.0%
20.0%
25.0%
30.0%
Vaccine Control
% o
f Sub
ject
s
Recurrence
24.0% 13.3%
Source: ASH 2015 Poster Presentation, Verstovsek et al.
u Well tolerated with primarily Grade 1 and 2 toxicities in n=14/18
u Efficacy compares favorably to historical anagrelide IR
• Platelet response: § ORR = 83.3% (15/18) § CR = 61.1% (11/18) § PR = 22.2% (4/18)
• Median time to response was 1 to 9 weeks (defined as platelet count ≤ 600 x109/L)
§ Anagrelide IR historical time to response ranged from 4 to 12 weeks
Figure2.PlateletResponse
1 8 15 22 29 36 43 50 57 64 71 78 85 99 113 127 141 155 169 197 225
18 18 18 18 18 18 15 14 14 14 13 13 13 13 13 13 12 12 11 11 11
1.0 1.4 1.8 1.9 2.0 2.2 2.4 2.5 2.4 2.2 2.0 2.1 2.1 1.9 1.8 1.9 2.0 1.9 1.8 1.9 1.8
StudyDays
N=
Avg.dailydose
0
200
400
600
800
1000
1200
1400 Mean Platelet Count (± SD)
Plat
elet
Cou
nt (x
109 /L
)
Figure2.PlateletResponse
1 8 15 22 29 36 43 50 57 64 71 78 85 99 113 127 141 155 169 197 225
18 18 18 18 18 18 15 14 14 14 13 13 13 13 13 13 12 12 11 11 11
1.0 1.4 1.8 1.9 2.0 2.2 2.4 2.5 2.4 2.2 2.0 2.1 2.1 1.9 1.8 1.9 2.0 1.9 1.8 1.9 1.8
StudyDays
N=
Avg.dailydose
0
200
400
600
800
1000
1200
1400 Mean Platelet Count (± SD)
Plat
elet
Cou
nt (x
109 /L
)
Figure2.PlateletResponse
1 8 15 22 29 36 43 50 57 64 71 78 85 99 113 127 141 155 169 197 225
18 18 18 18 18 18 15 14 14 14 13 13 13 13 13 13 12 12 11 11 11
1.0 1.4 1.8 1.9 2.0 2.2 2.4 2.5 2.4 2.2 2.0 2.1 2.1 1.9 1.8 1.9 2.0 1.9 1.8 1.9 1.8
StudyDays
N=
Avg.dailydose
0
200
400
600
800
1000
1200
1400 Mean Platelet Count (± SD)
Plat
elet
Cou
nt (x
109 /L
)
Figure2.PlateletResponse
1 8 15 22 29 36 43 50 57 64 71 78 85 99 113 127 141 155 169 197 225
18 18 18 18 18 18 15 14 14 14 13 13 13 13 13 13 12 12 11 11 11
1.0 1.4 1.8 1.9 2.0 2.2 2.4 2.5 2.4 2.2 2.0 2.1 2.1 1.9 1.8 1.9 2.0 1.9 1.8 1.9 1.8
StudyDays
N=
Avg.dailydose
0
200
400
600
800
1000
1200
1400 Mean Platelet Count (± SD)
Plat
elet
Cou
nt (x
109 /L
)
CORPORATE OVERVIEW
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COMMERCIAL ASSETS
u Divestiture plan announced: Nov. 9, 2015
u Abstral® (fentanyl) sublingual tablets • Approved for breakthrough
cancer pain • SOLD: Nov 20, 2015
u Zuplenz® (ondansetron) oral soluble film • Approved for CINV, RINV, PONV • For Sale
35 Please see full prescribing information at www.abstral.com and www.zuplenz.com.
UPCOMING MILESTONES
NeuVax™
ü Enroll N=700 into PRESENT trial ü Complete enrollment in Phase 3 PRESENT trial • Initiate DCIS trial (1Q16) • PRESENT: reach 70 events (1Q16) • PRESENT: interim analysis (2Q16)
GALE-301 GALE-302
ü Report Top-Line Phase 2a clinical data ü Report 1-Year Phase 2a analysis ü Report GALE-301 + GALE-302 Phase 1b data
GALE-401 (anagrelide CR)
ü Report Top-Line efficacy and safety data ü Report Final Phase 2 data
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LEADERSHIP TEAM
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u Mark W. Schwartz, Ph.D., President & CEO Apthera, Bayhill Therapeutics, Calyx Therapeutics, Trega Biosciences, Incyte Genomics, DuPont Diagnostics
u Bijan Nejadnik, M.D., Executive Vice President, Chief Medical Officer Jazz Pharmaceuticals, Johnson & Johnson, Stanford, Johns Hopkins, UC Davis
u Ryan Dunlap, CPA, VP & CFO Moss Adams, Nike, KPMG, PricewaterhouseCoopers
u Remy Bernarda, SVP, Investor Relations & Corporate Communications IR Sense, Hana Biosciences, Knight Equity Markets, Bear Stearns, Goldman Sachs
u Gavin Choy, Pharm.D., SVP, Clinical Sciences & Operations Otsuka, Astex, SuperGen, Hana Biosciences, Gilead, Stanford University Medical Center, Department of Veteran Affairs
u Tom Knapp, Esq., Interim General Counsel Sucampo, Exemplar Law Partners, NorthWestern Energy, Paul Hastings, The Boeing Company
u Joe Lasaga, VP, Business Development & Alliance Management
Nektar Therapeutics, Rigel
u Pat Murphy, VP, Regulatory Affairs & Compliance Nektar Therapeutics, Bayhill Therapeutics, Berlex Laboratories, Serono, Parexel, Biogen
FINANCIAL OVERVIEW (as of September 30, 2015)
Cash & Cash Equivalents $34.8 million
Debt $5.7 million
Projected Quarterly Burn $9-$11 million
Shares Outstanding 162 million
Market Cap (7 December 15) ~$240 million
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WHY WE’RE HERE
39 Source: 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
THANK YOU
NASDAQ: GALE