Building A Focused Oncology Business

Transcription

1 Building A Focused Oncology Business David Meek President, Oncology

2 Our Strategic Priority: To be at the forefront of patient-centric innovation, bringing life-changing cancer therapies to patients with limited options

3 *Excludes recently announced Oncaspar acquisition Building A Third Business In Oncology Key takeaways Strategically focusing on orphan diseases and targeted populations with significant unmet need and limited treatment options Continuing to bolster oncology portfolio of effective, differentiated and personalized treatment options in collaboration with leading R&D partners Leveraging existing global commercial capabilities and footprint, with heritage of expertise in patient services Accelerating momentum in building a targeted Oncology business Acquiring portfolio including Oncaspar, market-leading therapy for ALL, as well as next-generation formulations, new chemical entities and new indications; expected to close 2H 2015 Filed nal-iri (MM-398) in EU for pancreatic cancer post-gemcitabine Announced positive top-line Phase III data for Pacritinib for myelofibrosis Establishing A Focused Oncology Business With $500M+ In Sales By 2020* ($700M+ With Oncaspar)

4 Sources: IMS Institute 2014 and EvaluatePharma Baxalta Is Well-Positioned In Attractive Oncology Market Promising growth Designed for success Innovation is becoming more targeted Global market forecast of $100B+ in 2015, growing at a CAGR of 7% [ ] Orphan cancer drugs worth ~$50B in 2015 Global Cancer Market [2015] Other Cancers $100B+ Orphan Cancer Segment Focusing on differentiated therapies with molecules that have the highest probability of success Vertically-integrated Oncology structure: R&D, medical affairs and commercial teams Oncaspar portfolio acquisition will add experienced global commercial and clinical infrastructure, synergy with pipeline Team has been building for two years; expansion is already well underway to continue to add experienced and high-performing oncology associates Well developed, strong Go-To-Market platform for launch excellence Play to win approach with business development opportunities across multiple indications

5 *Excludes recently announced Oncaspar acquisition expected to close 2H 2015 Indications With Unmet Needs Will Drive Significant Growth In Oncology Baxalta Oncology Revenue* $ In Billions $1.5 $1.0 $0.5 $0.0 Oncaspar Other Therapies $0.1B ~$1.5B $0.7B+ $1.0B $0.5B ,000 Patients 51,000 Patients 90,000 Patients Acute Lymphoblastic Leukemia Pancreatic Cancer, Myelofibrosis, Acute Myeloid Leukemia, Metastatic Colorectal Cancer, Malignant Ascites (Ovarian) Chronic Lymphocytic Leukemia, Myelodysplastic Syndrome

6 Experienced Senior Management Team David Meek, President, Oncology Salim Yazji, M.D., Global Therapeutic Head Claude Knopf, Head of Business Development Steve Hamburger, Ph.D., Head of Regulatory Affairs Arup Pramanik, M.D., Head of Patient Access Torsten Hoof, Ph.D., Head of EMEA Expertise And Experience Covering A Comprehensive Spectrum Of Oncology Products

7 A Strong Heritage In Advocacy & Meeting Individual Patient Needs Leveraging leadership and expertise in rare diseases to develop more effective treatment options Utilizing expertise in bringing specialty medicines to targeted populations Oncology Addressing unmet needs with a differentiated portfolio of personalized, effective, lasting therapies Immunology Hematology Accessing transformational science in partnership with leading R&D companies Applying Proven Expertise In Hemophilia And Immunology To The Complex Requirements Of Oncology

8 Accessing Transformational Science In Partnership With R&D Companies Attract Partners By Offering: Expertise, responsiveness, flexibility and efficient decision-making Respect for the partner s science and patientcentered solutions Add Value Through: Critical functional guidance Dedicated oncology organization Collaborative approach to creating incremental value Finding Solutions For Success: Strategic flexibility for value-based deal structure and governance Respect for cultural differences and strengths A Preferred Strategic Partner For Driving Shared Value And Innovation

9 Focus On Diseases With High Unmet Needs & Limited Treatment Options Focus Cancers with limited treatment options Therapies that are first-in-class, best-in-class or standard of care Favorable competitive dynamics Target Well understood underlying mechanisms of disease Innovative biomarker diagnostics Technology New molecular entities, biologics, differentiated reformulations Immunotherapies Cancer metabolism and epigenetics Targeted therapies Our Business Development Process Yields A Sustainable, Diversified And Innovative Pipeline

10 Sources: WHO, Globocan 2014; EvaluatePharma March 12, 2015 & Decision Resources Cancers With Limited Treatment Options Are Lead Targets For Our Pipeline Assets Cancer Type & Description Est. # Patients Globally 2020 Opportunity Pancreatic Cancer, Post-Gemcitabine Myelofibrosis (MF) Acute Lymphoblastic Leukemia (ALL) Myelodysplastic Syndrome (MDS) Metastatic Colorectal Cancer Acute Myeloid Leukemia (AML) Represents only 3% of cancers but is the 4 th most common cause of cancer deaths worldwide A bone marrow disorder that disrupts normal blood cell production Cancer of the white blood cells, characterized by the overproduction and accumulation of cancerous, immature white blood cells known as lymphoblasts. A type of cancer in which the bone marrow does not make enough healthy blood cells Third most common cancer in worldwide; lack of effective treatments for patients who progressed in first and second line therapy Cancer of the myeloid line of blood cells, lowest survival rate of all leukemias 37,000 $3.0B 38,000 $1.8B 40,000 $1.9B 80,000 $1.7B 92,000 $0.6B 126,000 $0.6B Our Innovative Assets Will Have The Potential To Become Standard Of Care In Diseases Where Treatment Options Are Limited

11 *Acquisition of Oncaspar expected to close 2H 2015 Differentiated Portfolio With Strong Cadence Of New Pipeline Launches Expected Launch Dates Of Key Programs Baxalta Oncaspar Portfolio Acquisition* CTI BioPharma Merrimack Pharmaceuticals Oncaspar Lyophilized ALL [EU] nal-iri (MM-398) Pancreatic Cancer Post-gemcitabine [Japan] Oncaspar Lyophilized ALL [U.S.] Pacritinib AML [U.S. & EU] Oncaspar ALL [Japan] nal-iri (MM-398) 1 st line metastatic Pancreatic Cancer [EU] Imalumab (BAX 069) 3 rd line metastatic Colorectal [U.S.] nal-iri (MM-398) Pancreatic Cancer Post-gemcitabine [EU] Pacritinib MF [U.S. & EU] Pacritinib MF [Japan] Calaspargase pegol ALL [U.S.] nal-iri (MM-398) 1 st line Gastric Cancer [EU] Imalumab (BAX 069) Malignant Ascites [U.S.]

12 Sources: Decision Resources 2014 Exciting Growth Prospects Of Oncaspar Portfolio Acquisition Acute Lymphoblastic Leukemia Rare leukemia with 10,000 cases annually in U.S. and Europe Current cure rates of 90% in children, 50% in young adults and 30% in adults High relapse rate in older patients Oncaspar portfolio opportunity Market-leading 1st line therapy for children in the U.S. and Germany Improve survival & relapse rates in older patients Broaden geographic reach Expand portfolio with new formulations and indications Expected to close 2H 2015 # In Thousands ALL Incidence (U.S. & EU) (Number Of Patients) 0-17 yrs yrs > 37 yrs Incidence Treated with Oncaspar

13 Oncaspar Acquisition Will Accelerate Our Oncology Presence Established standard of care in children most convenient (every two weeks) Expanding into new markets: Europe, Japan and BRIC Expecting to seek label expansions outside the U.S. Enhancing therapy with less frequent dosing from novel chemical entity, calaspargase pegol Exploring new indications to extend survival in acute myeloid leukemia (AML) ~$500M ~$100M ~$200M Acquisition of Oncaspar expected to close 2H 2015

14 ASCO GI, 2015: Expanded Analyses of NAPOLI-1; Poster A5, SEER data Pancreatic Cancer: Providing A New Treatment After Gemcitabine Metastatic Pancreatic Cancer (mpaca) Description & current treatment 3% of cancers worldwide; 4 th leading cause of cancer deaths 20% of all pancreatic cancer patients survive one year; 2% with recurring metastatic disease survive five years No approved therapy after gemcitabine containing treatment 70% of patients who undergo surgery receive gemcitabine as adjuvant therapy No 2 nd line standard of care Unmet need Earlier diagnosis and treatment Increased overall survival/improved quality of life (QoL) 70% of patients develop mpaca after initial surgery Effective treatment after gemcitabine with manageable side effects Currently there is no firmly established standard chemotherapy for patients after progression on 1 st line treatment. ESMO Guidelines 2012 Need For Effective Therapy After Gemcitabine For All Patients With Pancreatic Cancer

15 Source: Chen et al. Napoli-1. ASCO GI Poster A5 Pancreatic Cancer: Providing A New Treatment After Gemcitabine nal-iri (MM-398) Value proposition Treatment option after gemcitabine with proven survival nal-iri + 5-FU/LV overall survival 6.1 months, HR 0.67 (95% CI) nal-iri + 5-FU/LV progression free survival 3.1 months, HR 0.56 (95% CI) Engineered for sustained cytotoxic effects Potential An approved treatment plan for 2 nd line therapy: expansion opportunity to 1 st line metastatic pancreatic and gastric cancers nal-iri (MM-398) 110 nm Manageable adverse event and toxicity profile First And Only Therapy Showing Meaningful Survival In A Post-Gemcitabine Setting

16 Myelofibrosis: More Effective Disease Control Still Needed For All Patients Myelofibrosis (MF) Description & current treatment Bone marrow malignancy which impairs normal blood cell production ~30% of patients have thrombocytopenia (TTP platelet count <150,000/μL) and anemia Therapy targeting JAK 1/JAK 2 inhibition for patients with largely stable platelet counts Transfusions for TTP and anemia Treatment for enlarged spleen, e.g. hydroxyurea (estimated treatment benefit lasts 12 months) Unmet need Treatment for all patients regardless of platelet count Agents slow disease progression without significant myelosuppression Better safety profile reduces blood and systemic side effects Improved quality of life with symptom control and reduced splenomegaly Stem cell transplant for selected patients All Patients With Myelofibrosis Have Significant Unmet Need For More Effective, Safer Therapy Sources: Verstovsek V 2013, Pathophysiology of Myelofibrosis and Targeted Therapy in MF; FDA: Revised Prescribing Information July 2014

17 Pacritinib Achieves Primary Endpoint In Myelofibrosis Trial PERSIST-1 demonstrated positive results Trial demonstrated clinically and statistically significant 35% spleen volume reduction from baseline to Week 24 vs best available therapy (BAT) Population Phase III Data Pacritinib N=220 BAT N=107 p-value At Week 24 (ITT) 19.1% 4.7% Up to Week 24 (ITT) 23.6% 4.7% < Very few patients discontinued therapy or required a dose reduction due to treatment-related anemia or thrombocytopenia At Week 24 (Evaluable) 25% 5.9% % of patients in the BAT arm crossed over to receive pacritinib therapy JAK2 inhibition reduces spleen size in MF An Effective, Safe New Therapy Without Dosage Modification, Even At Low Platelet Levels Source: CTI 2015: 10K

18 Sources: CTI 2015: 10K, EvaluatePharma March 13, 2015 Pacritinib: Potential Breakthrough Therapy For All MF Patients Pacritinib value proposition New JAK2/FLT3 inhibitor free from treatment emergent myelosuppression 35% spleen volume reduction at 24 weeks Low discontinuation or dose reduction due to treatment-related anemia or thrombocytopenia Greater number of patients able to remain on therapy beyond one year vs current therapy Clinically significant reduction in red blood cell transfusion dependence compared to BAT Potential A new option for eligible patients in the EU (~14K), U.S. (~18K), Japan (~8K) Pacritinib as standard of care in first line options for MF Pacritinib kinome profile offers future development opportunities in AML/MDS, CMML, and PV $ In Billions $2.0 $1.0 $0.0 Global Myelofibrosis Market ~20% CAGR Received Fast Track Designation By FDA And Accepted For A Late Breaking Oral Presentation At ASCO

19 Sources: WHO, Globocan 2014; EvaluatePharma March 12, 2015 & Decision Resources Future Potential Pacritinib Targets Include Cancers With High Unmet Needs Cancer Type Est. # Patients Globally 2020 Market Opportunity Chronic Lymphocytic Leukemia (CLL) Most common type of leukemia in adults, affects B cell lymphocytes which originate in the bone marrow, crowding out healthy blood cells 105,000 $4.8B Myelodysplastic Syndromes (MDS & CMML) Bone marrow malignancy affecting red and white cells and platelets; causes anemia, clotting disorders, immune deficiency and increased risk of developing leukemia 80,000 $1.7B Polycythemia Vera (PV) Disorder where the bone marrow makes too many red blood cells, but can also result in over production of white blood cells and platelets 200,000 $0.5B Our Assets Target Patients Who Have Limited Treatment Options

20 Sources: Decision Resources 2014 Imalumab: Targeting Multiple Pathways Controlling Tumor Microenvironment Imalumab value proposition Opportunity (# Of Patients) First-in-class mab targets oxidized macrophage migration inhibitory factor (oxmif) in cancer cells Reduces inflammation and cell proliferation, promotes cancer cell death In combination with other therapies, potential to: Reduce tumor growth, spread and recurrence Increase progression free survival Manageable side effects with combination therapy for advanced cancers # In Thousands # In Thousands Metastatic Colorectal (mcrc) 3 rd Line: ~40,000 patients EU5 U.S. Japan Malignant Ascites/Ovarian: ~10,000 patients EU5 U.S. Japan Imalumab Inhibition Of oxmif Has The Potential To Improve Progression Free Survival In Patients With Late Stage Cancers

21 *Excludes recently announced Oncaspar acquisition Building A Third Business In Oncology Key takeaways Strategically focusing on orphan diseases and targeted populations with significant unmet need and limited treatment options Continuing to bolster oncology portfolio of effective, differentiated and personalized treatment options in collaboration with leading R&D partners Leveraging existing global commercial capabilities and footprint, with heritage of expertise in patient services Accelerating momentum in building a targeted Oncology business Acquiring portfolio including Oncaspar, market-leading therapy for ALL, as well as next-generation formulations, new chemical entities and new indications; expected to close 2H 2015 Filed nal-iri (MM-398) in EU for pancreatic cancer post-gemcitabine Announced positive top-line Phase III data for Pacritinib for myelofibrosis Establishing A Focused Oncology Business With $500M+ In Sales By 2020* ($700M+ With Oncaspar)

22 Baxalta Incorporated 2015 Investor Conference