Galena presentation 14 mar 16

37
ONCOLOGY FOCUSED IMMUNOTHERAPY COMPANY Targeting Cancer Survivorship

Transcript of Galena presentation 14 mar 16

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 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

LATE-PHASE, ONCOLOGY FOCUSED IMMUNOTHERAPY COMPANY

Targeted, 1st in Class therapies

for prevention of cancer

recurrence

Focused in large markets in

areas of major unmet medical

need

• Phase 3, PRESENT, breast cancer

clinical trial ongoing under SPA

Pioneering immunotherapy

technology for cancer

• Induce, activate and cause

proliferation of Cytotoxic T-Cells

• Proven Mechanism of Action

through Expansion of Tumor

Specific CTLs

3

0

5

10

15

20

25

0.5 1.5 2.5 3.5 4.5 5.5 6.5 7.5 8.5 9.5 10.5

Year

Hazard

of

recu

rren

ce b

y y

earl

y i

nte

rval Total

Node 0

Node 1-3

Node (4+)

Tumour size (<1cm)

Tumour size (1.1-3cm)

Tumour size (>3cm)

ER+

ER-

Premen

Postmen

Source: Early Breast Cancer Trialists’ Collaborative Group. Lancet. 1998;351:1451 ; Update of Houghton.

J Clin Oncol. 2005; 23(16S):24s. Abstract 582 Saphner et al., J Clin Oncol. 14: 2738-2746, 1996

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

PRESENT

*NeuVax is an investigational product. Efficacy has not been established. Herceptin is a registered trademark of Genentech.

Ongoing Planned

VADIS

4

2b

Adds ~10k patients

>$3B

Combo:

High risk, HER2 3+

NEUVAX:SIGNIFICANT U.S. COMMERCIAL OPPORTUNITY

5

adds ~10k patients

>$2.5B

Combo Node Pos or Neg

HER2 1+, 2+

HLA-A2, A3, A24, A26

PRESENT50-60k 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).

REDEFINING THE STANDARD OF CARE

6

NOVEL DEVELOPMENT STRATEGY:

SECONDARY PREVENTION IN CANCER SURVIVORS

7

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

DECLAREDTO 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

PREVENTING RECURRENCE: UNMET MEDICAL NEED

NEUVAX PHASE 3 PRESENT TRIAL DEMOGRAPHIC:

Node positive, Stage 2a - 3a, HER2 1+/2+, HLA A2/A3

Local or Metastatic recurrent disease =

8

Poor prognosis and/or Death

Patients have a ~25% Recurrence Rate

Prevention of recurrences saves lives!

Sources: 1 http://www.cancer.org/cancer/breastcancer/detailedguide/breast-cancer-survival-by-stage; 2 Sledge GW Jr: Curing Metastatic Breast Cancer. J Oncol Pract 12:6-10, 2016

5 year survival rate of metastatic cancer = 22%1

“Low Tumor volume” equates to improved overall survival 2

Occult tumor cells micrometastasis macrometastisis metastatic disease

UNIQUELY POSITIONED

14.5 million cancer survivors in US (NCI

Cancer Survivorship)

• Projected to 19 million survivors in 2024

Increase in survival due to decades of

productive research, improved

screening/prevention, and effective

treatments

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

Galena peptide vaccines – NeuVax and

GALE-301 are uniquely positioned to

maintain survivorship

9Source: DeSantis CE et al. CA Cancer J Clin 2014: 64:252-271

CANCER IMMUNOTHERAPY WITH INNOVATIVE TECHNOLOGY

Overcoming Cancer by

Activating and Expanding

Cytotoxic T-Cells

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FIRST-IN-CLASS, TARGETED IMMUNOTHERAPY PIPELINE

11

Harnessing the

power of the

immune system in

the adjuvant setting

Exploits specificity

of natural immune

surveillance

Adjuvant patients

have healthy

immune systems

Systemic

protection

Goal is to prevent

recurrence

Recurrences are

almost always

fatal

Minimal toxicity

and improved

safety profile

Boosters provide

long term

protective effect

Well-validated

targets

HER2

Folate binding

protein (FBP)

Current

Programs

NeuVax™

(nelipepimut-S)

• Breast: HER2

1+, 2+ and 3+;

DCIS

• Gastric trial

planned

GALE-301 &

GALE-302

• Ovarian

Adjuvant Setting = Minimal Residual Disease

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

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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

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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

OUR VACCINES STIMULATE T-CELL

PROLIFERATION AND EXPANSION

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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

GALE-301

NEUVAX™ (nelipepimut-S)

Targeting HER2

NEUVAX: HER2 IMMUNODOMINANT PEPTIDE

NeuVax contains the immunodominant peptide derived from the extracellular region of the HER2 protein

Peptide (aa 369-377) immunotherapy administered as intradermal injection

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

NeuVax binds to antigen presenting cells (APCs)

NeuVax stimulates APCs to activate CD8+ cytotoxic T lymphocytes (CTLs)

CTLs rapidly replicate to seek out and destroy HER2 expressing tumor cells and micro-metastases

Booster series maintains long term immunologic response

Demonstrated inter- and intra-antigenic epitope spreading

17Sources: 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

eu

Va

xsp

ecific

CD

8+

T c

ells

NeuVax Specific CD-8 CTLs: Pre-, Post, Mean and Long-Term (6 months)

Pre Max Mean Long-Term

POSITIVE SAFETY PROFILE

NeuVax is well-tolerated in multiple clinical trials

Phase 1/2 showed predominantly Grade 1/2 Adverse Events caused by GM-CSF(n=53)

• Injection site reactions in nearly all patients demonstrating the activated dendritic cells

• Systemic toxicities caused by GM-CSF

Fatigue (64%)

Headache (42%)

Myalgia/Other Pain (30%)

August 2015

• Independent Data Monitoring Committee (IDMC) recommended to the Company that it can reduce the cardiac toxicity monitoring in its Phase 3 PRESENT clinical trial

18Sources: Choy, et al, poster presentation 33rd Annual Chemotherapy Foundation

Symposium: Nov 2015; Mittendorf- Annals of Oncology 25: 1735–1742, 2014

NEUVAX: SN-33 PHASE 2 HER2 IHC 1+/2+

19Source: 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

Trial being run under FDA-approved SPA

Enrollment completed in April 2015 (n=758)

• Adjuvant breast cancer patients, Node Positive, HER2 1+/2+, HLA A2/A3+

Patient friendly regimen via intradermal injection

• Primary Vaccine Series – injection once a month for 6 months

• Booster Series – injection once every 6 months

Upcoming Key Milestones

• Interim safety/futility analysis: 2Q16

• Primary Endpoint: 2018

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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

PRESENT INTERIM ANALYSIS

70 Events Confirmed by the EAC

• Endpoint Adjudication Committee confirms 70 events

• Independent team of 2 Oncologists and 1 Radiologist

Galena Compiles

Data

• Prepares a detailed review on 70 patients with events and overall safety data set (n=758)

• Submits to IDMC

IDMC Evaluates

• Evaluates 70 patients with events and overall safety data set (n=758)

• Makes recommendation on futility and continuation of trial

Interim Analysis Results

•Estimated timing: End of Q2

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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

NEUVAX: DEVELOPMENT COLLABORATIONS

Phase Treatment HER2 Status

Indication Trial StatusProtocol Defined

# of PatientsCollaborations

3Single agentPRESENT

Study1+, 2+

BREASTNode PositiveHLA A2+, A3+

Enrolled13 countries~140 centers

700(enrolled 758)

2bCombination

with trastuzumab

1+, 2+

BREAST Node Positive or High Risk Node Negative

HLA A2+, A3+, A24+, A26+

EnrollingU.S. only

33 centers300

2Combination

with trastuzumab

3+ high risk

BREASTNode PositiveHLA A2+, A3+

EnrollingU.S. only

28 centers100

2Single agentVADIS Study

1+, 2+,3+

BREASTDuctal Carcinoma in

Situ (DCIS)HLA A2+

PlannedU.S. only4 centers

48

2 Single agent1+,

2+,3+GASTRIC

HLA A2+, A3+Planned

India Only50

24

Targeting Folate Binding

Protein

GALE-301 &

GALE-302

GALE-301 & GALE-302

26Source: U.S. Ovarian Cancer http://seer.cancer.gov/statfacts/html/ovary.html

Targeted cancer immunotherapy

Folate Binding Protein (FBP) is over-expressed (20-80 fold) in >90% of ovarian and endometrial cancers

FBP has very limited tissue distribution and expression in non-malignant tissue making it an ideal immunotherapy target

Current treatments are generic

• Carboplatin and paclitaxel

• High recurrence rate

Most patients relapse with poor prognosis

GALE-301: OPTIMAL DOSE GROUP SHOWS PRELIMINARY EFFICACY

Source: Peoples, et. al, Poster Presentation, European Cancer Congress 2015 27

Phase 1/2a trial ongoing

Phase 1: Determined optimal dose and demonstrated safety and potent immune response

Phase 2a Preliminary data in 1000 mcg dose group:

• At 12 months median follow-up:

Vaccine group: 2 clinical recurrences (13.3%) n=15

Control group: 12 recurrences (55%) n=22

• Two 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 & GALE 302: PHASE 1DELAYED-TYPE HYPERSENSITIVITY

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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)

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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CORPORATE

OVERVIEW

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1st IN CLASS PROGRAMS WITH EXPANSION OPPORTUNITIES

Mid-stage clinical trials have proven T-cell generation

• NeuVax™ (nelipepimut-S) Phase 2 trial demonstrated 2% of the patient’s

T-Cells become CD8+, HER2 directed

• GALE-301 Phase 1/2: Two year DFS estimate in optimal dose group is

85.7% vaccine vs. 33.6% control (p<.02)

Targeting “high value” settings: Prevention of recurrence in breast

and ovarian cancer are areas of clear unmet medical needs

• No approved drugs for these women with limited late stage competition

Multiple trials ongoing as stand-alone therapies and in-combination

with other agents

Breast & Ovarian are just a start – HER2 and Folate Binding

Protein expressed in numerous cancer types

HER2

Breast

Gastric

Prostate

Non-Small Cell Lung

Bladder

Colorectal

Ovarian

Head & Neck

Folate Binding Protein

Ovarian

Endometrial

Breast

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LEADERSHIP TEAM

33

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

2016 MILESTONES

34

PROGRAM MILESTONEPROJECTED

DATE

NeuVax™

(nelipepimut-S)

Initiate DCIS trial March/April

PRESENT: Reach 70 events March/April

PRESENT: Interim analysis Q2

Combo H&N 1+/2+ Interim safety data Q4

Combo H&N 1+/2+ A24/A26 data Q4

GALE-301GALE-302

Present 301/302 booster data Q2

Present GALE-301 Phase 2a two year data Q4

GALE-401 (anagrelide CR)

Confirmation of 505(b)2 pathway 2H

Publish final Phase 2 report Q4

FINANCIAL OVERVIEW

Cash Position (as of 2/29/15) $38.2 million

Q1 Remaining Burn $2.5 - $3.5 million

Q2 Projected Burn $13 - $15 million

Includes legal settlement & fees ~$4-$5 million

2H Projected Quarterly Burn $9 - $11million

Debt (as of 12/31/15) $4.7 million

Shares Outstanding 182 million

Market Cap (9 March 16) ~$170 million

35

WHY WE’RE HERE

36Source: 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