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Pipeline Novartis Annual Report 2014 | 49 Pipeline

Pipeline Novartis Annual Report 2014 | 49 Pipeline

48 | Novartis Annual Report 2014 innovation innovation | pipeline Novartis Annual Report 2014 | 49 pipeline

Novartis is consistently rated as MAJOR DEVELOPMENT PROJECTS having one of the industry’s most PHASE l phaSE ll phaSE lll Submission respected development pipelines, with Planned more than 200 projects in clinical Project/product Division Common name Mechanism of action Potential indication/disease area Route of administration filing dates 1,2 development, including 135 in the Oncology Pharmaceuticals Division, as of BYL719 Pharmaceuticals alpelisib PI3K3 inhibitor Solid tumors Oral ≥2019 Dec. 31, 2014. LJM716 Pharmaceuticals – HER3 inhibitor Solid tumors Intravenous ≥2019 EGF816 Pharmaceuticals – Epidermal growth factor receptor Solid tumors Oral ≥2019

Many of these projects, which include new BGJ398 Pharmaceuticals – Pan-FGF receptor kinase inhibitor Solid tumors Oral ≥2019 molecular entities as well as additional indica- INC280 Pharmaceuticals capmatinib cMET inhibitor Non-small cell Oral 2018 tions and different formulations for marketed Tasigna Pharmaceuticals nilotinib BCR-ABL inhibitor Chronic myeloid leukemia treatment-free remission Oral 2016 products, are for potentially best-in-class or LGX818 Pharmaceuticals encorafenib RAF inhibitor Solid tumors Oral ≥2019 Afinitor/Votubia Pharmaceuticals everolimus mTOR4 inhibitor Non-functioning GI and lung neuroendocrine tumors, Oral 2015 first-in-class medicines that could significantly (RAD001) tuberous sclerosis complex seizures, DLBCL5 advance treatment standards for patients LCI699 Pharmaceuticals osilodrostat Aldosterone synthase inhibitor Cushing’s disease Oral 2017 worldwide. This table provides an overview of BKM120 Pharmaceuticals buparlisib PI3K3 inhibitor Metastatic , hormone receptor-positive, aromatase Oral 2015 inhibitor resistant, mTOR4 inhibitor naive [lead indication]; selected projects in confirmatory development. metastatic breast cancer, hormone receptor-positive, aromatase We use the traditional pipeline model as a inhibitor and mTOR4 inhibitor resistant solid tumors LEE011 Pharmaceuticals ribociclib CDK4/66 inhibitor HR+, HER2 negative advanced breast cancer (postmenopausal Oral 2016 platform (e.g. Phase I-III). However, we have women)[lead indication]; HR+, HER2 negative advanced breast tailored the process to be simpler, more cancer (premenopausal women); solid tumors flexible and efficient. Our development para- MEK1627 Pharmaceuticals binimetinib MEK8 inhibitor NRAS mutant [lead indication], LGSOC,9 solid tumors Oral 2016 digm consists of two parts: Exploratory MEK1627 + LGX818 Pharmaceuticals binimetinib, encorafenib MEK8 inhibitor + RAF inhibitor RAF mutant melanoma Oral 2016 Development and Confirmatory Development. PKC412 Pharmaceuticals midostaurin Signal transduction inhibitor [lead indication], Oral 2015 aggressive systemic mastocytosis

Signifor LAR (SOM230) Pharmaceuticals pasireotide analogue Cushing’s disease Long-acting release, 2016 Exploratory development Intramuscular injection This consists of clinical proof-of-concept studies, Zykadia (LDK378) Pharmaceuticals ceritinib ALK10 inhibitor ALK10+ advanced non-small cell (post chemo- Oral US approved which are small clinical trials that combine therapy and post crizotinib)[lead indication], ALK10+ advanced EU registration non-small cell lung cancer (chemotherapy naive, crizotinib naive) elements of traditional Phase I/II testing. These Jakavi Pharmaceuticals ruxolitinib Janus kinase inhibitor Polycythemia vera Oral EU registration customized trials are designed to give early LBH589 Pharmaceuticals panobinostat Pan-deacetylase inhibitor Relapsed or relapsed-and-refractory multiple myeloma Oral US registration insights into issues such as safety, efficacy and (pan-DACi) EU registration toxicity for a drug in a given indication. Once a Exjade Pharmaceuticals deferasirox Iron chelator Iron overload Oral FCT US registration film-coated tablet (FCT) positive proof of concept has been established, the drug moves to the Confirmatory Develop- Cardiovascular and Metabolism ment stage. BGS649 Pharmaceuticals – Aromatase inhibitor Obese hypogonadotropic hypogonadism Oral ≥2019

LIK066 Pharmaceuticals – SGLT 1/2 inhibitor Type 2 diabetes Oral ≥2019 Confirmatory development ACZ885 Pharmaceuticals canakinumab Anti-interleukin-1ß Secondary prevention of cardiovascular events Subcutaneous 2017 These are projects for which a positive proof monoclonal antibody injection of concept has been established. They have LCQ908 Pharmaceuticals pradigastat Diacylglycerol acyl Familial chylomicronemia syndrome Oral 2015 -1 inhibitor elements of traditional Phase II/III testing and RLX030 Pharmaceuticals serelaxin Recombinant form of human Acute Intravenous infusion 2016 include trials aimed at confirming the safety relaxin-2 hormone and efficacy of the drug in the given indication Tekturna Pharmaceuticals Direct Reduction of cardiovascular death/hospitalizations Oral 2016 in chronic heart failure leading up to submission of a dossier to LCZ696 Pharmaceuticals , Angiotensin receptor, Chronic heart failure with reduced ejection fraction Oral US registration health authorities for approval. Like traditional (as sodium salt complex) neprilysin inhibitor [lead indication], chronic heart failure with preserved EU registration ejection fraction Phase III testing, this stage can also include trials that compare the drug to the current Respiratory standard of care for the disease, in order to BCT197 Pharmaceuticals – Anti-inflammatory agent Chronic obstructive pulmonary disease Oral ≥2019 evaluate the drug’s overall risk/benefit profile. QAW039 Pharmaceuticals fevipiprant CRTH2 antagonist Asthma Oral ≥2019 The pipeline table provides an overview of QAX576 Pharmaceuticals – Anti-interleukin-13 Allergic diseases Subcutaneous ≥2019 selected projects in Confirmatory Develop- monoclonal antibody injection ment. See the glossary on pages 50 and 52 QGE031 Pharmaceuticals – High-affinity anti-IgE Asthma Subcutaneous ≥2019 monoclonal antibody injection for further explanation of the terms used. Seebri (NVA237) Pharmaceuticals glycopyrronium bromide Long-acting muscarinic antagonist Chronic obstructive pulmonary disease Inhalation EU approved US registration11

Ultibro (QVA149) Pharmaceuticals indacaterol, Long-acting beta2 adrenergic agonist Chronic obstructive pulmonary disease Inhalation EU approved glycopyrronium bromide and long-acting muscarinic antagonist US registration11

1 Filings that have received approval in one of the markets (US or EU) but are awaiting approval in the other market 6 Cyclin-dependent kinase 4/6 are included in the table. 7 Conditional on completion of the previously announced transactions with GSK, we expect to return our rights in MEK162 to Array BioPharma Inc. 2 Refers to lead indication only 8 Combination of mitogen-activated kinase and extracellular signal-regulated kinase 3 Phosphoinositide 3-kinase inhibitor 9 Low-grade serous 4 Mammalian target of rapamycin 10 Anaplastic kinase 5 Diffuse large B-cell lymphoma 11 Submission pending acceptance by FDA 50 | Novartis Annual Report 2014 innovation innovation | pipeline Novartis Annual Report 2014 | 51 pipeline continued glossAry MAJOR DEVELOPMENT PROJECTS PHASE l phaSE ll phaSE lll Submission Project/product Project refers to the Novartis Planned 1,2 reference code (combination of three letters Project/product Division Common name Mechanism of action Potential indication/disease area Route of administration filing dates and three numbers) used for projects in Immunology and Dermatology development. Product refers to the brand name QAW039 Pharmaceuticals fevipiprant CRTH2 antagonist Atopic dermatitis Oral ≥2019 ACZ885 Pharmaceuticals canakinumab Anti-interleukin-1ß Hereditary periodic fevers Subcutaneous 2016 for a marketed product. monoclonal antibody injection

Cosentyx (AIN457) Pharmaceuticals secukinumab Anti-interleukin-17 Psoriasis [lead indication], ankylosing spondylitis, Subcutaneous US registration Common name Official international non- monoclonal antibody psoriatic arthritis injection EU registration LDE225 Pharmaceuticals sonidegib Smoothened receptor/ Advanced basal cell Oral US registration proprietary name or generic name for an hedgehog signaling inhibitor EU registration individual molecular entity as designated by the World Health Organization Neuroscience CJM112 Pharmaceuticals – Anti-interleukin-17 Immune disorders Subcutaneous ≥2019 Mechanism of action Specific biochemical monoclonal antibody injection interaction with a molecular target such as CAD106 Pharmaceuticals – Beta-amyloid-protein therapy Alzheimer’s disease Intramuscular ≥2019 injection a receptor or , through which a drug BAF312 Pharmaceuticals siponimod Sphingosine-1-phosphate Secondary progressive multiple sclerosis Oral ≥2019 substance produces its pharmacological effect receptor modulator Gilenya Pharmaceuticals fingolimod Sphingosine-1-phosphate Chronic inflammatory demyelinating polyradiculoneuropathy Oral 2017 receptor modulator Potential indication/indications Disease or BYM338 Pharmaceuticals bimagrumab Inhibitor of activin receptor type II Sporadic inclusion body myositis [lead indication], Intravenous infusion 2016 condition for which a compound or marketed hip fracture, sarcopenia product is in development and is being studied as a potential therapy Cell and Gene Therapy CTL019 Pharmaceuticals tisagenlecleucel-T CD19-targeted chimeric antigen Adult and pediatric acute lymphoblastic leukemia Intravenous 2016 receptor T-cell immunotherapy [lead indication], diffuse large B-cell lymphoma Route of administration Path by which a FCR001 Pharmaceuticals – Inducing stable donor chimerism Renal transplant Infusion ≥2019 medicinal preparation is administered into and immunological tolerance the body, such as oral, subcutaneous or HSC835 Pharmaceuticals – Stem cell regeneration Stem cell transplantation Infusion ≥2019 intravenous

Infectious Diseases Phase I First clinical trials of a new compound, H7N912 Vaccines H7N9 vaccine Pandemic influenza Prevention of H7N9 influenza Intramuscular ≥2015 generally performed in a small number of Acellular pertussis Vaccines Tdap vaccine Pediatric Prevention of tetanus, diphtheria and pertussis Intramuscular ≥2015 combination healthy human volunteers, to assess the clinical C. difficile13 Vaccines C. difficile vaccine Hospital infections Prevention of C. difficile disease Intramuscular ≥2015 safety and tolerability, as well as metabolic and Human Vaccines HIV vaccine HIV Prevention of HIV disease Intramuscular ≥2015 pharmacologic properties of the compound immunodeficiency virus (HIV)14

S. aureus Vaccines S. aureus vaccine Hospital infections Prevention of S. aureus disease Intramuscular ≥2015 Phase II Clinical studies with patients who have KAF156 Pharmaceuticals – Malaria Oral ≥2019 the target disease, with the aim of continuing KAE609 Pharmaceuticals cipargamin PfATP4 inhibitor Malaria Oral 2017 the Phase I safety assessment in a larger group, Group B streptococcus Vaccines Group B streptococcus Maternal Prevention of group B streptococcus Intramuscular ≥2015 assessing the efficacy of the drug in the patient vaccine population, and determining the appropriate H5N1 influenza Vaccines Pandemic influenza vaccine Pandemic Prevention of H5N1 influenza Intramuscular ≥2015 cell culture vaccine12 doses for further evaluation MenABCWY Vaccines Meningococcal A, B, C, W Meningitis Prevention of meningococcal A, B, C, W and Y disease Intramuscular ≥2015 and Y vaccine

P. aeruginosa13 Vaccines P. aeruginosa vaccine Hospital infections Prevention of P. aeruginosa disease Intramuscular ≥2015

Cell culture QIV Vaccines Seasonal influenza vaccine Seasonal influenza Prevention of seasonal influenza Intramuscular ≥2015

aQIV pediatric Vaccines Seasonal influenza vaccine Seasonal influenza Prevention of seasonal influenza Intramuscular ≥2015

Fluad US Vaccines Seasonal influenza vaccine Seasonal influenza Prevention of seasonal influenza Intramuscular 201411

Flucelvax Vaccines Seasonal influenza vaccine Seasonal influenza Prevention of seasonal influenza Intramuscular 201411 age 4+ US

Bexsero US Vaccines Meningococcal B vaccine Meningitis Prevention of meningococcal B disease Intramuscular 2014

1 Filings that have received approval in one of the markets (US or EU) but are awaiting approval in the other market are included in the table. 2 Refers to lead indication only 11 Submission pending acceptance by FDA 12 Collaboration with United States Department of Health and Human Services 13 Collaboration with Valneva 14 Collaboration with United States National Institutes of Health 52 | Novartis Annual Report 2014 innovation innovation | pipeline Novartis Annual Report 2014 | 53 pipeline continued

Phase III Large-scale clinical studies with MAJOR DEVELOPMENT PROJECTS ­several hundred to several thousand patients, PHASE l phaSE ll phaSE lll Submission which are conducted to establish the safety Planned 1,2 and efficacy of the drug-specific indications Project/product Division Common name Mechanism of action Potential indication/disease area Route of administration filing dates for regulatory approval. Phase III trials also Ophthalmology RTH258 Alcon – Anti-VEGF15 single-chain Wet age-related macular degeneration Intravitreal injection ≥2017 may be used to compare a new drug against antibody fragment a current standard of care to evaluate the over- Lucentis Pharmaceuticals ranibizumab Anti-VEGF15 monoclonal Choroidal neovascularization and macular edema,16 Intravitreal injection 2016 all benefit-risk relationship of the new medicine. antibody fragment retinopathy of prematurity (ROP) OAP030 (Fovista) Pharmaceuticals – Aptamer anti-PDGF17 Wet age-related macular degeneration Solution 2016

AcrySof IQ ReSTOR Alcon – Multifocal, aspheric and cylinder Cataractous lens replacement with or without presbyopia, Surgical 2016 Advanced development Advanced development Medical device ­project Toric 3.0D diopter correcting intraocular lens and with astigmatism US and Japan for which a positive proof of concept has been range expansion IOL established and studies are being conducted EXE844b Alcon finafloxacin Anti-infective Otitis media-tympanostomy tube surgery Topical 2016 US EXZ829 Alcon olopatadine hydrochloride Antihistamine and Allergic conjunctivitis Topical Submitted US to establish the safety, efficacy or performance mast cell stabilization to address regulatory requirements for obtain- AOSept Plus/ Alcon – Disinfection and cleaning Contact lens care Lens care Submitted US Advanced development US Clear Care Plus 2016 Japan ing marketing authorization with HydraGlyde

AcrySof IQ ReSTOR Alcon – Multifocal, aspheric and cylinder Cataractous lens replacement with or without presbyopia, Surgical Submitted Japan Advanced development Japan Submission An application for marketing Toric 2.5D IOL correcting intraocular lens and with astigmatism 2015 US AcrySof IQ Alcon – Multifocal aspheric intraocular lens Cataractous lens replacement with or without presbyopia Surgical Submitted US approval has already been filed with one or ReSTOR 2.5D IOL both of the following regulatory agencies: AcrySof IQ Alcon – Multifocal, aspheric and cylinder Cataractous lens replacement with or without presbyopia, Surgical Submitted US FDA (United States), EMA (European Union). ReSTOR Toric 3.0D IOL correcting intraocular lens and with astigmatism Novartis has not yet received marketing authorization from both regulatory agencies.1 Biosimilars The application contains comprehensive GP2013 Sandoz rituximab Anti-CD20 antibody Non-Hodgkin lymphoma, chronic lymphocytic leukemia, Intravenous rheumatoid arthritis, granulomatosis with polyangiitis data and information gathered during (also known as Wegener’s granulomatosis), and microscopic polyangiitis and others (same as originator) human clinical trials and animal studies GP2015 Sandoz etanercept TNF-α inhibitor Arthritidies (rheumatoid arthritis, ankylosing spondylitis, psoriatic Subcutaneous conducted through the various phases of drug arthritis), plaque psoriasis and others (same as originator) development. GP2017 Sandoz adalimumab TNF-α inhibitor Arthritidies (rheumatoid arthritis, ankylosing spondylitis, psoriatic Subcutaneous arthritis), plaque psoriasis and others (same as originator)

HX575 Sandoz epoetin alfa Erythropoiesis-stimulating agent Chronic disease, chemotherapy-induced anemia Subcutaneous and US 1 Filings that have received approval in one of the markets and others (same as originator) intravenous (either US or EU ) but are awaiting approval in the other market HX575 s.c. Sandoz epoetin alfa Erythropoiesis-stimulating agent Chronic kidney disease Subcutaneous EU (extension are included in the table. nephrology, appproved as Binocrit since 2007)

LA-EP2006 Sandoz pegfilgrastim Pegylated granulocyte Chemotherapy-induced neutropenia and others Subcutaneous colony-stimulating factor (same as originator)

EP2006 Sandoz filgrastim Granulocyte colony-stimulating factor Chemotherapy-induced neutropenia, mobilization of peripheral Subcutaneous and US blood progenitor cells and others (same as originator) intravenous

1 Filings that have received approval in one of the markets (US or EU) but are awaiting approval in the other market are included in the table. 2 Refers to lead indication only 15 Vascular endothelial growth factor 16 Choroidal neovascularization and macular edema secondary to conditions other than age-related macular degeneration, diabetic macular edema, retinal vein occlusion and pathologic myopia 17 Anti-platelet-derived growth factor

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