Galena presentation 9 feb 17

HEMATOLOGY &

ONCOLOGY FOCUSED

COMPANY

February 9, 2017

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, Quarterly Report on Form 10-Q and in Current Reports on Form 8-K 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

DIVERSIFIED PIPELINE

Diversified pipeline with multiple mid-to late stage clinical trials

HEMATOLOGY

•GALE-401 (Anagrelide Controlled Release)

•Targeting MPNs

•Phase 3 ready in ET patients

IMMUNOTHERAPY

• NeuVax™ (nelipepimut-S)

• Targeting HER2

• Multiple Phase 2 clinical trials ongoing in breast cancer IMMUNOTHERAPY

•GALE-301/GALE-302

•Targeting Folate Binding Protein

•Early stage trials completed

3

DEVELOPMENT PIPELINE

PRODUCT THERAPETIC AREA PHASE 1 PHASE 2 PHASE 3 BLA / NDA

Hematology

GALE-401 (Anagrelide CR) Essential Thrombocythemia

Immunotherapy: Breast & Gastric Cancer

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)

NeuVax™ Gastric, HER2 IHC 1+/2+/3+

Immunotherapy: Gynecological Cancer

GALE-301 Ovarian & Endometrial

GALE-301 + GALE-302 Ovarian & Breast

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

Completed Planned

4

2b

VADIS

Ongoing

GALE-401

Anagrelide Controlled

Release (CR)

ANAGRELIDE

Anagrelide immediate release (IR) approved by the FDA to treat

Myleoproliferative Neoplasms (MPNs)

• Indicated for the treatment of patients with thrombocythemia,

secondary to myeloproliferative disorders to reduce the elevated

platelet count and the risk of thrombo-embolic events

• Only drug approved to treat Essential Thrombocythemia (ET)

Anagrelide suppresses megakaryocytopoiesis by inhibiting

PDE III-dependent and PDE III-independent mechanisms

No DNA damaging or cytotoxic effect

6

GALE-401:ANAGRELIDE CONTROLLED RELEASE (CR)

A proprietary, controlled release (CR) formulation of anagrelide

• 505(b)2 regulatory path allows for abbreviated submission package and potentially faster approval timelines

• Strong IP through 2029

Six trials conducted to date

• Five Phase 1 studies in healthy volunteers

• Phase 2 pilot study in patients with myeloproliferative neoplasms (MPNs)

Potential Clinical Benefits

• Consistent efficacy

• Potentially faster onset of action and indication of improved tolerability compared to anagrelide IR

• More convenient treatment regimen

• Favorable PK profile

Multiple life cycle management opportunities

7

Res

ult

s Reduces Cmax

Maintains Area Under the Curve (AUC)

Lowers peak plasma concentration

Maintains Platelet Lowering

GALE-401 PHASE 1 TRIALS

8Multiple Phase 1 studies in n=98 healthy volunteers; Agrylin is a registered trademark of Shire.

Anagrelide CR Platelet LoweringGALE-401 Median Cmax

Anagrelide IR Median Cmax

GALE-401: PHASE 2 PILOT STUDY FINAL RESULTS

9Source: Verstovsek et al, Final Results of Anagrelide Controlled-Release (Gale-401) Safety, Efficacy and Pharmacokinetics

in Subjects with Myeloproliferative Neoplasms (Mpn)-Related Thrombocytosis, ASH 2015 Poster Presentation.

Well tolerated with primarily Grade 1 and 2 toxicities in n=14/18

Efficacy compares favorably to historical anagrelide IR

• Platelet response:

ORR = 83.3% (15/18)

CR = 61.1% (11/18)

PR = 22.2% (4/18)

• 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

Safety profile indicates a potential benefit for GALE-401

compared to anagrelide IR

GALE-401 DEMONSTRATES IMPROVED AE PROFILES IN KEY CATEGORIES

Related Adverse Events (AEs)GALE-401*

(N=18)n (%)

AGRYLIN^

(n=942)%

Cardiac 6 (33) 42

General# 5 (27.8) 83

Gastrointestinal 9 (50) 92

Respiratory, thoracic and mediastinal 2 (11) 18

Skin and subcutaneous tissue 2 (11) 14

Musculoskeletal and connective tissue 1 (6) 6

Nervous system 9 (50) 65

Vascular 3 (16) <5

Hepatobiliary 2 (11) <5

Blood and Lymphatic 1 (6) <5

Number of AEs/patient 2.3 3.3

10Not a head-to-head trial. *GALE-401 related AE data from Phase 2 study; ^Anagrelide IR data from the product label. #General AEs referred to fatigue, peripheral edema, and malaise

ADVANTAGES OF CR FORMULATION

11

Anagrelide IR^ GALE-401* Benefits w/CR Formulation

Therapeutic index# Limited - dose escalation to optimal effect is challenging

Larger - Possibility of achieving desired effect with lower dose

Pharmacokinetics (PK) • Half life• Cmax

• 2-3 hours• 4x GALE-401

Improved PK profile• 20 hours• 25% of IR

Onset of Action As early as 4 weeks As early as 1 week

Doses per day 2 to 4 times a day2 times a day Targeting 1x/day in future trials

Dosing regimen 2 to 10 mg per day Mean 2 mg per day

Safety Profile• Treatment Related AEs• # of AE/Patient

• 42.1%• 3.3

• 30%• 2.3

Not a head-to-head trial. ^Anagrelide IR data from the product label/Agrylin Package Insert. *GALE-401 profile from Phase 1 and 2 studies. #Therapeutic Index distance between therapeutic dose curve and toxic dose.

ESSENTIAL THROMBOCYTHEMIA (ET)

One of the major MPNs

Characterized by increased

number of platelets

• ET is a neoplastic stem cell disorder

causing dysregulated production of

large numbers of abnormal

megakaryocytes

Chronic condition

• Median Overall Survival: 14.7 years

• Up to 50% of patients may be

asymptomatic at presentation

Associated with vascular

complications

12

Arrows indicate

Megakaryocytes

ET has Larger Number

of Megakayocytes

Sources: Haematologica. 2009 June; 94(6): 865, Am J Hematology. 2008 May;83(5):359)

ET OVERVIEW

Diagnosis

• Chronic hematologic malignancy with no known cause

• Clinical presentation of symptoms

• Diagnostic tools

• Blood test

• Bone marrow biopsy

• Gene mutation test

Common Symptoms

• Headache

• Vision disturbances or migraines

• Dizziness or lightheadedness

• Coldness or blueness of fingers or toes

• Burning, redness, and pain in the hands and feet

Thrombotic Complications

• Stroke

• Transient ischemic attack (TIA)

• Heart attack

• DVT or pulmonary embolus

• Blood clotting in unusual locations

Risk Factors

• Women 1.5x more likely

• Patients >60 years old, with 20% <40 years

• Mutations

• JAK2 - 50%

• CALR ~25%

13Source: MPN Research Foundation

ET: CURRENT TREATMENT OPTIONS

Hydroxyurea

Other Therapies

•Generally initial treatment option

•Cytotoxic myelosuppressive drug (also reduces other blood cells)

• Increased risk of developing acute leukemia over long term

•Avoided in younger patients

•~25% of patients intolerant/refractory

• Anagrelide IR

• Interferon

• Busulfan

• Retry hydroxyurea

• Observation

14

PIVOTAL, PHASE 3 TRIAL

15

Failed or Intolerant to Hydroxyurea

GALE-401

(Anagrelide CR)

BEST AVAILABLE THERAPY

Anagrelide IR (sizable population)

Interferon

Busulfan

Retry hydroxyurea

Observation

Sources: Mehta et al, (2014) Epidemiology of myeloproliferative neoplasms in the United States, Leukemia & Lymphoma; Sever et al (2014)

Therapeutic options for patients with polycythemia vera and essential thrombocythemia refractory/resistant to hydroxyurea, Leukemia & Lymphoma

Targeting the reduction of platelets in ET patients

• Limited competition with very few agents in development

• US Prevalence: 135,000 - 175,000

Estimate up to 25% of those patients who fail or are intolerant to initial

treatment with hydroxyurea may be trial candidates

IMMUNOTHERAPY

NeuVax™ (nelipepimut-S)

GALE-301/GALE-302

NEUVAX™ (nelipepimut-S):ELICITS A STRONG CD8+ T-CELL RESPONSE

Contains the immunodominant peptide derived from the extracellular region of the HER2 protein

Binds to antigen presenting cells (APCs)

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

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

18

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

CORRELATION BETWEEN HER2 & MHC-1

There is an inverse

correlation between

HER2 and MHC class I

HER2 overexpression is

associated with

decreased expression of

components of the

antigen processing/

presentation pathway

19

COMBINATION IMMUNOTHERAPY ENHANCES ANTIGEN PRESENTATION

Trastuzumab/HER2 complexes are internalized and

processed by proteasomes into short peptides

which are then presented on MHC class I molecules

PBMC from HER2/neu peptide, E75,

vaccinated patients efficiently recognize and

lyse trastuzumab-treated HER2/neu-

expressing tumor cell lines

20

Trastuzumab

HER2/neu

Breast

tumor cell

HER2/neu –derived peptide

presented on MHC-I

HER2/neu-

derived

peptide

20.0

25.0

30.0

35.0

40.0

45.0

50.0

55.0

60.0

Ave

rage

% C

yto

toxi

city

51

Cr

0 ug 10 ug 50 ug

* p=0.015

Trastuzumab

Hypothesis: Trastuzumab treatment will enhance

response to vaccination by making tumor cells more

visible to T-cells/immune system

Interim

Analysis

at 6 months

DFS

Standard of Care: Standard Herceptin

dosing every 3 weeks for 1 year

6 doses of NeuVax given every 3 weeks

starting with third dose of Herceptin

+ 1 booster

dose every

6 months

thereafter

+ Dosing to disease

progression;

36 mo follow up

Primary

Endpoint

DFS at

24 mos.

300 adjuvant breast cancer patients, randomized 1:1

Single blind (subject)

Node positive or high risk node negative

HLA A2/A3+

HLA A24/A26+

HER2 IHC 1+/2+

Stratified by nodal status and HER2 status

Study Population

NEUVAX+TRASTUZUMAB: HER2 1+/2+ PHASE 2 STUDY

GM-CSF

+ GM-CSF

21

NEUVAX: MULTIPLE SETTINGS AND COMBINATION STRATEGY

PhaseTreatment

HER2 StatusIndication Trial Status

TargetedEnrollmentCompletion

PlannedData

ReadoutsCollaborations

2bCombination

w/trastuzumabHER2 1+, 2+

BREAST Node Positive or High Risk Node

NegativeHLA A2+, A3+,

A24+, A26+

EnrollingU.S. only

33 centersn=300

Q2 2017

Q4, 2017Interim Analysis

1H, 2019Final Data

2

Combinationw/trastuzumab

high risk HER2 3+

BREASTNode PositiveHLA A2+, A3+

EnrollingU.S. only

28 centersn=100

Q4 20171H, 2019Interim Analysis

2

Single agentVADIS StudyHER2 1+, 2+,

3+

BREASTDuctal Carcinoma in Situ (DCIS)

HLA A2+

EnrollingU.S. only4 centers

N=48

2

Single agentHER2 1+, 2+,

3+GASTRIC

HLA A2+, A3+

PlannedIndia Only

N=50

22

GALE-301 (E39) & GALE-302 (E39’): TARGETING FOLATE BINDING PROTEIN

Sources: U.S. Ovarian Cancer http://seer.cancer.gov/statfacts/html/ovary.html;

Peoples, et. al, Poster Presentation, American Society of Clinical Oncology 201623

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

High unmet medical need in ovarian cancer patients

Relatively shorter development timelines

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: 73.5% vaccine vs 38.1% control (p=.03)

• GALE-301 + GM-CSF is well tolerated and elicits a strong in vivo immune response with primarily Grade 1 & 2 toxicities

Estimated 24 months Disease Free

Survival by Dosing Cohort

CORPORATE

OVERVIEW

24

LEADERSHIP TEAM

25

Bijan Nejadnik, M.D.

Executive VP, Chief Medical Officer

Jazz Pharmaceuticals, Johnson & Johnson,

Stanford, Johns Hopkins, UC Davis

Stephen Ghiglieri

Executive VP, Chief Financial Officer

MedData Inc., NeurogesX, Hansen Medical,

Inc., Oacis Healthcare Systems, Oclassen

Pharmaceuticals

Tom Knapp, Esq.

Interim General Counsel

Sucampo, Exemplar Law Partners,

NorthWestern Energy, Paul Hastings, The

Boeing Company

Remy Bernarda,

SVP, Investor Relations & Corporate

Communications

IR Sense, Hana Biosciences, Knight

Equity Markets, Bear Stearns, Goldman

Sachs

John Burns, CPA

VP, Finance & Corporate Controller

Pixelworks, Moss Adams

2017 MILESTONES

26

PROGRAM MILESTONEPROJECTED

DATE

GALE-401 (anagrelide CR)

Finalize Phase 3 Clinical Trial Protocol Q1

Determine Phase 3 initiation 2H

NeuVax™

(nelipepimut-S)

Complete enrollment in NeuVax/trastuzumab 1+/2+ CombinationTrial

Q2

Interim safety data presentation for NeuVax/trastuzumab 1+/2+ CombinationTrial

Q2

Complete enrollment in NeuVax/trastuzumab 3+ Combination Trial

Q4

Interim data analysis: NeuVax/trastuzumab1+/2+ Combination Trial

Q4

GALE-301GALE-302

Final GALE-301 data presentation Q1

THANK YOU

NASDAQ: GALE