Novartis AG Investor Relations. Q Results. Investor Presentation July 18, 2018

Transcription

1 Novartis AG Investor Relations Q Results Investor Presentation July 18, 2018

2 Advancing our pipeline of potential blockbuster launches Expected launches 3 Aimovig 2 Migraine Kymriah DLBCL Lutathera NET 4 CV risk reduction BAF312 SPMS namd AVXS-101 SMA 6 nraxspa Entresto HFpEF OMB157 Relapsing MS QAW039 QVM149 SEG101 Sickle cell disease External recognition 3 #1 #1 in value creation from pipeline products in value creation from advanced therapies 1. Exact launches and timing depends on filing date, HAs decisions and timelines 2. Aimovig is in collaboration with Amgen. Companies co-commercialize in the US (Amgen books sales to third parties), Novartis has Aimovig exclusive rights in rest of world excluding Japan 3. Source: EvaluatePharma World Preview Novartis Q Results July 18, 2018 Novartis Investor Presentation

3 AVXS-101: US regulatory submission anticipated in H as planned Pre-BLA meeting Commercial product comparable to the product used in the Phase 1 trial Phase 1 data in SMA Type 1 will form the primary basis for the BLA submission (H2 2018); all available clinical data will be submitted (incl. some preliminary Phase 3 STR1VE data) An integrated overview of the safety findings from the development program 1 will be provided Newborn screening for SMA is now officially recommended by US HHS for inclusion in the Recommended Uniform Screen Panel (RUSP) 1. Includes CL-101, LT-001, CL-102. CL-303, CL-304 trials 15 Novartis Q Results July 18, 2018 Novartis investor presentation

4 New data 1 confirm AVXS-101 could be foundational gene replacement therapy (GRT) for SMA Type 1 CHOP-INTEND Scores Age 1 (months) Natural History 1 Rapid onset; sustained efficacy regardless of severity at treatment 1 Most patients achieving CHOP-INTEND >50, above natural history benchmarks All patients alive and without need for permanent ventilation 2 years post-grt Patients observed in long-term follow-up continue to achieve new milestones Only 4 of 12 patients able to swallow safely at enrollment, reflecting disease severity; 11 of 12 swallowing safely for oral feeding at month 24 follow up Black dashed line denotes SMA Type 1 natural history (Finkel et al., 2014) 1. Presented at AAN 2018; text bullets reflect Cohort 2 (proposed therapeutic dose), n=12 16 Novartis Q Results July 18, 2018 Novartis Investor Presentation

5 AVXS-101 clinical development plan to cover all pediatric patients and SMA types SMA Type Q Q Q Q Q Completion 3 START (CL-101) 1 (n=15; Open Label, Dose Comparison, Single i.v. dose) START Long-term follow-up (LT-001 / NCT ) 12 patients enrolled SMA Type 2 Pre-symptomatic SMA Type 1,2,3 Pediatric SMA Type 1,2,3 STR1VE (CL-303 / NCT ) Single i.v. dose Initiated in Q4 2017, patient enrollment complete STR1VE EU (CL-302 / NCT ) Single i.v. dose Initiated Q2 STRONG (CL-102 / NCT ) Dose Comparison, Single i.t. dose Started in Q1 2018; dose escalation enrolled; expansion starting in Q3 SPR1NT (CL-304 / NCT ) Single i.v. dose Initiated Q2 REACH Single i.t. dose TBC i.v. Intravenous; i.t. Intrathecal See appendix slide 34 for references 17 Novartis Q Results July 18, 2018 Novartis Investor Presentation

6 AVXS-101 ready for launch in 2019 Regulatory status US: On track for H BLA submission (Breakthrough Therapy designation) EU: Submission anticipated in H (PRIME designation) Japan: Pre-submission discussions planned Q (Sakigake designation) Clinical readouts WMS 2018: Anticipate updated pivotal data from STR1VE in SMA Type 1 AAN 2019: Anticipate data from SPR1NT, the Phase 3 pre-symptomatic study STRONG: Phase 1 study in patients with SMA Type 2 expected at an upcoming medical meeting, once the data are sufficiently robust as to be meaningful Manufacturing Commercial scale-up underway: Manufacturing facility in the Chicago area fully operational, product inventory currently in production 170,000-square foot facility in Durham, NC: Expected to be fully operational in Novartis Q Results July 18, 2018 Novartis Investor Presentation

7 2018 pipeline milestones Regulatory decisions and opinions H H Kymriah DLBCL (US) Aimovig 2 Migraine (EU) = 6 Tafinlar + Mekinist Adjuvant melanoma (US) Kymriah Pediatric and young adult r/r ALL (EU) = 6 Lutathera NET (US) Kymriah DLBCL (EU) = 6 Gx Advair 1, COPD (US) Tafinlar + Mekinist Adjuvant melanoma (EU) + ATC (US) ( 7 ) Aimovig 2 Migraine (US) Gilenya Pediatric MS (US) Glatopa 40mg Relapsing MS (US) GP2017 Adalimumab BS (EU) = LA-EP2006 Peg-filgrastim BS (EU) = GP1111 Infliximab BS (EU) GP Rituximab BS (US) Submissions CV risk reduction (US/EU) BAF312 MS (EU) = BAF312 MS (US) namd (US/EU) = Kisqali Advanced BC (US/EU) 3 BYL719 HR+ BC (US/EU) = AS (JP) AVXS-101 SMA 9 (US) = CTL019 Pediatric ALL + DLBCL (JP) Major trial readouts Achieved Missed = On track Promacta 1st line SAA (US/EU) Kisqali Advanced BC (MONALEESA-3) 4 LIK Obesity LJN452 NASH 5 BYL719 HR+ BC = INC280 ALK- cmet amplified NSCLC = Entresto HFpEF (interim analysis) = 1. Complete Response Letter received from FDA; Advair is a registered trademark of Glaxo Group Ltd. 2. Aimovig is in collaboration with Amgen. 3. Indication expansion based on MONALEESA-3 & 7 results 4. Data presented at ASCO 5. IA readout achieved as planned in H1, data to be presented in H2 6. Positive CHMP opinion receive 7. US approval in ATC received; EU approval in adj. melanoma still outstanding 8. Complete Response Letter received from FDA 9. i.v. formulation in type 1 SMA 10. Program discontinued 28 Novartis Q Results July 18, 2018 Novartis Investor Presentation

8 Clinical Trials Update Includes selected ongoing or recently concluded global trials of Novartis development programs/products which are in confirmatory development or marketed (typically Phase 2 or later). For further information on all Novartis clinical trials, please visit:

9 Planned filings 2018 to 2022 AVXS-101 Spinal Muscular Atrophy 22 BAF312 (EU) SPMS 21 BYL719 + fulv HR+, HER2 (-) postmenopausal adv. BC 9 2 nd line LCI699 Cushing s disease namd LAM320 MDR 8 tuberculosis Kisqali + fulv HR+, HER2 (-) postmenopausal adv. or metastatic BC 9 1 st /2 nd line Kisqali + tmx + gsn/or NSAI + gsn HR+, HER2 (-) premenopausal adv. or metastatic BC 9 1 st line Lucentis ROP 10 Lucentis Diabetic retinopathy 1. Secondary prevention of cardiovascular events 2. Diffuse large B-cell lymphoma 3. Severe aplastic anemia 4. Chronic myeloid leukemia 5. Long-acting release 6. Non-small cell lung cancer 7. Neovascular age-related macular degeneration 8. Multi-drug resistant 9. Breast cancer 10. Retinopathy of prematurity 11. Indolent non-hodgkin s lymphoma 12. Non-radiographic axial spondyloarthritis 13. Preserved ejection fraction 14. Graft-versus-host disease 15. Neuroendocrine tumors 16. Chronic spontaneous urticaria / chronic idiopathic urticaria 17. Psoriatic arthritis head-to-head study versus adalimumab 18. Ankylosing spondylitis head-to-head study versus adalimumab 19. Acute myeloid leukemia 20. Chronic obstructive pulmonary disease 21. Chronic lymphocytic leukemia 22. IV formulation type 1 SMA st line colorectal cancer / 1 st line renal cell carcinoma INC280 NSCLC 6 PDR001 + Tafinlar +Mekinist Metastatic BRAF V600+ melanoma SEG101 Sickle cell disease nraxspa 12 Entresto Heart failure (PEF) 13 OMB157 Relapsing multiple sclerosis QMF149 QVM149 LA-EP2006 (pegfilgrastim, US) Chemotherapy-induced neutropenia and others (same as originator) Combination abbreviations: fulv fulvestrant tmx tamoxifen gsn goserelin NSAI Non-steroidal aromatase inhibitor Taf Tafinlar (dabrafenib) Mek Mekinist (trametinib) ABL001 CML 4 3rd line QAW039 Entresto Post-acute myocardial infarction PsA H2H 17 CTL019 (Kymriah US) r/r Follicular Lymphoma Jakavi Chronic GVHD 14 Jakavi Acute GVHD 14 Diabetic macular edema Xolair Nasal Polyps UNR844 Presbyopia QGE031 CSU/CIU 16 ZPL389 Atopic dermatitis 1 st Line NSCLC 2 nd Line NSCLC CLL 21 r/r DLBCL in 1st relapse New molecule New indication New formulation Biosimilars BYM338 Hip fracture recovery CAD106 Alzheimer s disease CFZ533 Solid Organ Transplant CNP520 Alzheimer s disease CSJ117 Severe ECF843 Dry eye EMA401 Peripheral neuropathic pain HDM201 Acute myeloid leukemia KAE609 Malaria KAF156 Malaria LHW090 Resistant hypertension LJN452 Non-alcoholic steatohepatitis LMI070 Spinal muscular atrophy LOU064 Chronic spontaneous urticaria MTV273 Multiple myeloma 2022 QBW251 COPD 20 VAY736 Autoimmune Hepatitis VAY785 Non-alcoholic steatohepatitis VPM087 CRC 1L/RCC 1L 23 ABL001 CML 4 1st line Adjuvant NSCLC BYM338 Sarcopenia AS H2H 18 + pembrolizumab - r/r DLBCL INC280 NSCLC 6 (EGFRm) Kisqali HR+, HER2 (-) BC 9 (adjuvant) PDR001 Metastatic Melanoma Rydapt AML 19 (FLT3 wild type) Retinal vein occlusion VAY736 Primary Sjoegren s syndrome 36 Novartis Q Results July 18, 2018 Novartis Investor Presentation

10 Pipeline of key projects in confirmatory development BYM338 Hip fracture recovery CAD106 Alzheimer s disease CFZ533 Solid Organ Transplant CNP520 Alzheimer s disease CSJ117 Severe ECF843 Dry eye EMA401 Peripheral neuropathic pain HDM201 Acute myeloid leukemia KAE609 Malaria KAF156 Malaria 1. Chronic myeloid leukemia 2. Non-small cell lung cancer 3. Chronic spontaneous urticaria / chronic idiopathic urticaria 4. Neuroendocrine tumors 5. Breast cancer 6. Neovascular age-related macular degeneration 7. Secondary prevention of cardiovascular events 8. Indolent non-hodgkin s lymphoma 9. Non-radiographic axial spondyloarthritis 10. Psoriatic arthritis head-to-head study versus adalimumab 11. Ankylosing spondylitis head-to-head study versus adalimumab Combination abbreviations: fulv fulvestrant NSAI Non-steroidal aromatase inhibitor ltz letrozole Taf Tafinlar (dabrafenib) tmx tamoxifen Mek Mekinist (trametinib) gsn goserelin Early Clinical Trials Registration Trials Phase III / Pivotal In Registration LHW090 Resistant hypertension LMI070 Spinal muscular atrophy LJN452 Non-alcoholic steatohepatitis LOU064 Chronic spontaneous urticaria MTV273 Multiple myeloma QBW251 COPD UNR844 Presbyopia VAY736 Autoimmune Hepatitis VAY785 Non-alcoholic steatohepatitis VPM087 CRC 1L/RCC 1L 24 ZPL389 Atopic dermatitis ABL001 CML1 1st line BYM338 Sarcopenia + pembrolizumab - r/r DLBCL INC280 NSCLC 2 (EGFRm) PDR001 Metastatic Melanoma VAY736 Primary Sjoegren s syndrome 12. Diffuse large B-cell lymphoma 13. Preserved ejection fraction 14. Graft-versus-host disease 15. Multi-drug resistant 16. Retinopathy of prematurity 17. Severe aplastic anemia 18. Acute myeloid leukemia 19. Acute lymphoblastic leukemia 20. Secondary progressive multiple sclerosis 21. Long-acting release 22. Chronic lymphocytic leukemia 23. IV formulation type 1 SMA st line colorectal cancer / 1 st line renal cell carcinoma 37 Novartis Q Results July 18, 2018 Novartis Investor Presentation ABL001 CML 1 3rd line AVXS-101 Spinal Muscular Atrophy 23 BAF312 (EU) SPMS 20 BYL719 + fulv HR+, HER2 (-) postmenopausal adv. BC 5 2 nd line INC280 NSCLC 2 LCI699 Cushing s disease PDR001 + Tafinlar +Mekinist Metastatic BRAF V600+ melanoma QAW039 namd 6 SEG101 Sickle cell disease QGE031 CSU/CIU 3 Adjuvant NSCLC 2 1 st Line NSCLC 2 2 nd Line NSCLC 2 nraxspa 9 PsA H2H 10 AS H2H 11 r/r DLBCL in 1st relapse CLL 22 Entresto Heart failure (PEF) 13 Entresto Post-acute myocardial infarction Jakavi Acute GVHD 14 Jakavi Chronic GVHD 14 LAM320 MDR 15 tuberculosis Lucentis Diabetic retinopathy Kisqali + fulv HR+, HER2(-) postmenopausal adv. or metastatic BC 5 1st/2nd line a) In collaboration with Amgen; companies to co-commercialize in the US, Novartis to have AMG 334 exclusive rights in rest of world excluding Japan b) Approved in US, submitted in EU r/r Follicular Lymphoma Kisqali + tmx + gsn/or NSAI + gsn HR+, HER2(-) premenopausal adv. or metastatic BC 5 1st line Kisqali HR+, HER2(-) BC 5 (adjuvant) Lucentis ROP 16 OMB157 Relapsing multiple sclerosis QMF149 QVM149 Diabetic macular edema Retinal vein occlusion Rydapt AML 18 (FLT3 wild type) Xolair Nasal Polyps LA-EP2006 (pegfilgrastim, US) Chemotherapy-induced neutropenia and others (same as originator) Aimovig a Migraine prophylaxis BAF312 (US) SPMS 20 CTL019 (Kymriah b US) Pediatric/young adult ALL 19 LTW888 b Retinopathy Sec. prev. CV events 1 CTL019 (Kymriah b US) r/r DLBCL 12 Promacta /Revolade SAA 17 1st line FTY720 Pediatric multiple sclerosis Tafinlar + Mekinist b BRAF V600+ melanoma (adjuvant) GP2017 (adalimumab) Arthritides, plaque psoriasis and others (same as originator) GP2013 (rituximab, US) Follicular lymphoma, DLBCL 12 and others (same as originator) LA-EP2006 (pegfilgrastim, EU) Chemotherapy-induced neutropenia and others (same as originator) New molecule New indication New formulation Biosimilars