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Oncology Focused Immunotherapy Company
- 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 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. 2
- 3. 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 3
- 4. 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+ • Gastric trial planned u GALE-301 (FBP) • Ovarian • Endometrial Adjuvant Setting = Minimal Residual Disease
- 5. NOVEMBER 2015 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
- 6. Adds ~10k patients >$3B Combo: High risk, HER2 3+ NEUVAX: SIGNIFICANT U.S. COMMERCIAL OPPORTUNITY 6 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).
- 8. What it is 8
- 9. 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 9 K I F G S L A F L
- 10. 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 %NeuVaxspecificCD8+Tcells NeuVax Specific CD-8 CTLs: Pre-, Post, Mean and Long-Term (6 months) Pre Max Mean Long-Term
- 11. 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
- 12. 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
- 13. 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
- 14. Where it fits 14
- 15. 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 15Source: DeSantis CE et al. CA Cancer J Clin 2014: 64:252-271
- 16. REDEFINING THE STANDARD OF CARE 16 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
- 17. BREAST CANCER PROGRAMS Phase Treatment HER2 Status Indica6on Trial Status Protocol Defined # of Pa6ents Collabora6ons 3 Single agent PRESENT Study 1+, 2+ Node Posi6ve HLA A2+, A3+ Enrolled 13 countries ~140 centers 700 (enrolled 758) 2b Combina6on with trastuzumab 1+, 2+ Node Posi6ve or High Risk Node Nega6ve HLA A2+, A3+, A24+, A26+ Enrolling U.S. only 34 centers 300 2 Combina6on with trastuzumab 3+ high risk Node Posi6ve HLA A2, A3+ Enrolling U.S. only 30 centers 100 2 Single agent VADIS Study 1+, 2+, 3+ Ductal Carcinoma in Situ (DCIS) HLA A2+ Planned 4 U.S. sites 48 17
- 18. PRIMARY PREVENTION Expansion potential for safe vaccine: e.g., DCIS METASTATIC DISEASE Expansion potential in combination with checkpoint inhibitors / immune modulators NEUVAX: ACROSS THE BREAST CANCER TREATMENT SPECTRUM 18 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
- 20. SN-33 PHASE 2 HER2 IHC 1+/2+ (N=45) 20 Source: 2012 San Antonio Breast Cancer Poster, Mazanet, et al.
- 21. 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
- 22. 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 22 Prevention of Recurrence in Early-Stage, Node Positive Breast Cancer with Low to Intermediate Her2 Expression with NeuVax Treatment
- 23. NEUVAX FRANCHISE EXPANSION OPPORTUNITIES 23 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.
- 24. GALE-301 & GALE-302 Targeting Folate Binding Protein 24
- 25. GALE-301: FOLATE BINDING PROTEIN (FBP) 25Source: 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
- 26. GALE-301 & GALE-302: CURRENT CLINICAL DEVELOPMENT 26 Phase Treatment Cancer Type Target Indica6on Current Status # of Enrolled Pa6ents 1/2a GALE-301 Ovarian, Endometrial HLA A2+ Ovarian Enrolled 51 1b GALE-301 & GALE-302 Ovarian, Breast HLA A2+ Ovarian Enrolled 39
- 27. GALE-301: PHASE 1/2a TRIAL 1 2 3 4 Dosing by Month + 1 booster dose every 6 months thereafter 5 6 Disease free (NED) ovarian and endometrial a[er SoC Rx § Node posi6ve § HLA A2+ - Vaccine § HLA A2- Control § N = 51 Study Popula6on 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
- 28. GALE-301: OPTIMAL DOSE GROUP SHOWS PRELIMINARY EFFICACY 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
- 29. GALE-301 (E39) & GALE-302 (E39’): PHASE 1b TRIAL Disease free (NED) ovarian and breast cancer a[er SoC Rx § Node posi6ve § HLA A2+ (Vaccine) § HLA A2- (Control) § Post menopausal § N = 39 Study Popula6on GALE-301 + GM-CSF x 6 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-CSF x 3 à GALE-301 + GM-CSF x 3 GALE-301+ GM-CSF x 3 à GALE-302 + GM-CSF x 3 29 1 2 3 4 Dosing by Month + 1 booster dose every 6 months x2 5 6
- 30. 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
- 32. GALE-401 ANAGRELIDE CONTROLLED RELEASE (CR) 32 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
- 33. GALE-401: PHASE 2 PILOT STUDY PRELIMINARY RESULTS 33Source: EHA 2015 Poster Presentation, Verstovsek et al. u Well tolerated with primarily Grade 1 and 2 toxicities in n=16/18 u Efficacy compares favorably to historical anagrelide IR • Platelet response: § ORR = 78% (14/18) § CR = 39% (7/18) § PR = 39% (7/18) u Median time to response was 5 weeks (range, 3–10) u Median duration of response has not yet been reached
- 35. 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 35Please see full prescribing information at www.abstral.com and www.zuplenz.com.
- 36. 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 • Report Final Phase 2 data 36
- 37. LEADERSHIP TEAM 37 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
- 38. 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 (24 November 15) ~$240 million 38
- 39. 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