Top Banner

Click here to load reader

Galena presentation 28 nov 16

Jan 06, 2017

ReportDownload

galenabio

  • HEMATOLOGY &

    ONCOLOGY FOCUSED

    COMPANY

    November 28, 2016

  • 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 Companys 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

  • DIVERSIFIED PIPELINE

    Diversified pipeline with multiple mid-to late stage clinical trials

    HEMATOLOGY

    GALE-401 (Anagrelide Controlled Release)

    Targeting MPNs

    Phase 3 Initiation Q2, 2017

    IMMUNOTHERAPY

    NeuVax (nelipepimut-S)

    Targeting HER2

    Multiple mid-stage trials ongoing & planned

    IMMUNOTHERAPY

    GALE-301/GALE-302

    Targeting Folate Binding Protein

    Early stage trials ongoing

    3

  • DEVELOPMENT PIPELINE

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

    Hematology

    GALE-401 (Anagrelide CR) Essential Thrombocythemia

    Immunotherapy: Breast 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)

    Immunotherapy: Gastric Cancer

    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.

    Ongoing Planned

    VADIS

    4

    2b

  • GALE-401

    Anagrelide Controlled

    Release (CR)

  • ANAGRELIDE

    Anagrelide suppresses megakaryocytopoiesis by inhibiting PDE III-

    dependent and PDE III-independent mechanisms

    No DNA damaging or cytotoxic effect

    Immediate release version indicated for the treatment of patients with

    thrombocythemia, secondary to myeloproliferative disorders to reduce

    the elevated platelet count and the risk of thrombosis, and to ameliorate

    associated symptoms including thrombo-hemorrhagic events

    Approved to treat Myleoproliferative Neoplasms (MPNs)

    Only drug approved to treat Essential Thrombocythemia (ET)

    6

  • GALE-401 (Anagrelide Controlled Release)

    GALE-401 is a proprietary, controlled release (CR) formulation of anagrelide

    FDA approved product with known mechanism of action

    Advantageous 505(b)2 regulatory path to be confirmed with the FDA

    Strong IP through 2029

    Six trials conducted to date

    Five Phase 1 studies in healthy volunteers

    Phase 2 pilot study in patients with MPNs

    Potential Clinical Benefits

    Potentially faster onset of action

    Consistent efficacy

    Indication of improved tolerability vs anagrelide IR

    Multiple life cycle management opportunities

    7

  • GALE-401: PHASE 1 TRIAL RESULTS

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

    Anagrelide CR

    Plasma Levels

    pg

    /mL

    Anagrelide CR Platelet Lowering

    Res

    ult

    s Reduces Cmax

    Maintains Area Under the Curve (AUC)

    Lowers peak plasma concentration

    Maintains Platelet Lowering

  • GALE-401: PHASE 2 PILOT STUDY RESULTS

    9

    0.0%

    5.0%

    10.0%

    15.0%

    20.0%

    25.0%

    30.0%

    Vaccine Control

    % o

    f S

    ub

    jec

    ts

    Recurrence

    24.0% 13.3%

    Source: ASH 2015 Poster Presentation, Verstovsek et al.

    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)

    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

    Figure 2. Platelet Response

    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

    Study Days

    N=

    Avg. daily dose

    0

    200

    400

    600

    800

    1000

    1200

    1400

    Mean Platelet Count ( SD)

    Pla

    tele

    t C

    ou

    nt

    (x10

    9/L

    )

    Figure 2. Platelet Response

    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

    Study Days

    N=

    Avg. daily dose

    0

    200

    400

    600

    800

    1000

    1200

    1400

    Mean Platelet Count ( SD)

    Pla

    tele

    t C

    ou

    nt

    (x10

    9/L

    )

    Figure 2. Platelet Response

    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

    Study Days

    N=

    Avg. daily dose

    0

    200

    400

    600

    800

    1000

    1200

    1400

    Mean Platelet Count ( SD)

    Pla

    tele

    t C

    ou

    nt

    (x10

    9/L

    )

    Figure 2. Platelet Response

    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

    Study Days

    N=

    Avg. daily dose

    0

    200

    400

    600

    800

    1000

    1200

    1400

    Mean Platelet Count ( SD)

    Pla

    tele

    t C

    ou

    nt

    (x10

    9/L

    )

  • 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)

  • ADVANTAGES OF ANAGRELIDE CONTROLLED RELEASE (CR): GALE-401

    11

    Anagrelide IR^ GALE-401*

    Benefits

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

    Wider - 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 for faster onset of action

    Doses per day 2 to 4 times a day 2 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)

    MPN 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

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

  • Essential Thrombocythemia (ET)

    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 pulmo

Welcome message from author
This document is posted to help you gain knowledge. Please leave a comment to let me know what you think about it! Share it to your friends and learn new things together.