Major R&D Pipeline. As of October Phase 1 Phase 2 Phase 3 Application. Red: new or update. Oncology. Therapeutic area

Transcription

1 Major R&D Pipeline As of October 2017 Therapeutic area Phase 1 Phase 2 Phase 3 Application Oncology DS3032 (US/JP) (MDM2 inhibitor) PLX7486 (US) (FMS / TRK inhibitor) PLX8394 (US) (BRAF inhibitor) PLX9486 (US) (KIT inhibitor) DS3201 (JP/US) (EZH1/2 inhibitor) PLX73086 (US) (CSF1R inhibitor) PLX51107 (US) (BRD4 inhibitor) DS8273 (US) (AntiDR5 antibody) DS1123 (JP) (AntiFGFR2 antibody) U31402 (JP/US) (AntiHER3 ADC) DS1001 (JP) (IDH1m inhibitor) DS1205 (US) (AXL inhbitor) PLX2853 (US) (BRD4 inhibitor) Patritumab (EU) (U31287 / AntiHER3 antibody) Pexidartinib (US) (PLX3397 / Glioblastoma / CSF1R/ KIT/FLT3ITD inhibitor) DS1647 (JP) (Glioblastoma / G47Δ virus) Quizartinib (JP) (AC220 / AML2 nd / FLT3ITD inhibitor) DS8201 (JP/US/EU) (Breast cancer/antiher2 ADC) DS8201 (JP/Asia) (Gastric cancer/antiher2 ADC) Denosumab (JP) (AMG 162 / Breast cancer adjuvant/ AntiRANKL antibody) Nimotuzumab (JP) (DE766 / Gastric cancer / AntiEGFR antibody) Quizartinib (US/EU/Asia) (AC220 / AML2 nd / FLT3ITD inhibitor) Quizartinib (US/EU/Asia) (AC220 / AML1 st / FLT3ITD inhibitor) Pexidartinib (US/EU) (PLX3397 / TGCT / CSF1R/KIT/FLT3 ITD inhibitor) Cardiovascular Metabolic DS1040 (US/EU/JP) (Acute ischemic stroke / TAFIa inhibitor) DS2330 (Hyperphosphatemia) DS9231/TS23 (Thrombosis / α2pi inactivating antibody) Edoxaban (JP) (DU176b / AF / FXa inhibitor) Prasugrel (JP) (CS747 / Ischemic stroke / Antiplatelet agent) Esaxerenone (JP) (CS3150/Hypertension/ MR antagonist) Esaxerenone (JP) (CS3150 / DM nephropathy / MR antagonist) Edoxaban (ASCA etc.) (DU176b / AF / FXa inhibitor) Edoxaban (ASCA etc.) (DU176b / VTE / FXa inhibitor) Others DS1971 (Chronic pain) DS1501 (US) (Osteoporosis / AntiSiglec15 antibody) DS7080 (US) (AMD / Angiogenesis inhibitor) DS5141 (JP) (DMD / ENA oligonucleotide) VN0102/JVC001 (JP) (MMR vaccine) DS1211 (US) (TNAP inhibitor) Mirogabalin (US/EU) (DS5565 / FM / α2δ ligand) Mirogabalin (JP/Asia) (DS5565 / DPNP/ α2δ ligand) Mirogabalin (JP/Asia) (DS5565 / PHN / α2δ ligand) VN0105 (JP) (DPTIPV / Hib vaccine) Laninamivir (JP) (CS8958 / Antiinfluenza / nebulizer) Hydromorphone (JP) (DS7113 / Cancer pain / Opioid μ receptor agonist) <Injection> Intradermal Seasonal Influenza Vaccine (JP) (VN100 / prefilled i.d. vaccine for seasonal flu) VN0107/MEDI3250 (JP) (Nasal spray flu vaccine) Red: new or update

2 11. Major R&D Pipeline (Innovative pharmaceuticals) As of October 2017 Oncology (Latestage pipeline products) Denosumab/AMG 162 Ranmark (JP) Quizartinib/AC220 Pexidartinib/PLX3397 AntiRANKL antibody Breast cancer adjuvant JP P3 Injection Amgen 2020 The fully human monoclonal antibody to target RANK Ligand, an essential mediator of osteoclast formation. US/EU/Asia P FLT3ITD inhibitor Acute Myeloid Leukemia (AML) US/EU/Asia P3 Oral 2021 JP P Kinase inhibitor against a receptortype tyrosine kinase, FLT3. Therapeutic effect for patients with acute myeloid leukemia harboring FLT3ITD mutation is expected. Tenosynovial Giant Cell Tumor (TGCT) US/EU P Solid tumors Asia P1 CSF1R/KIT/FLT3ITD inhibitor Glioblastoma US P2 Oral ckit Melanoma Asia P1/2 Melanoma, Solid tumors US P1/2 Merck & Co., Inc. The moleculartargeted agent to inhibit CSF1R, KIT and FLT3ITD. This agent is expected to reduce tumor cell proliferation and expansion of metastases. Additional indication Relapsed and refractory AML patients Newly diagnosed AML patients Relapsed and refractory AML patients Including pigmented villonodular synovitis Including TGCT Combination with pembrolizumab in collaboration with Merck Nimotuzumab/DE766 Patritumab/U31287 DS1647(G47 ) AntiEGFR antibody Gastric cancer JP P3 Injection InnoClMAb Pte Ltd 2020 The humanized monoclonal antibody to target Epidermal Growth Factor Receptor(EGFR). This antibody is expected to be a best in class EGFR, safety against the skin toxicity and the efficacy comparable to the other antibodies. AntiHER3 antibody Head & neck cancer EU P2 Injection The fully human monoclonal antibody to target HER3, one of the Epidermal Growth Factor Receptor (EGFR) family of proteins. HER3 is overexpressed in many tumors of epithelial origin and HER2/HER3 dimers and EGFR/HER3 dimers are expected more potent to induce tumor cell proliferation than homodimers of HER2 or EGFR. Granted SAKIGAKE designation from MHLW. Investigator Oncolytic HSV1 Glioblastoma JP P2 Injection ActiVec Inc. Initiated Study is ongoing Orphan drug designation from MHLW in July The third generation oncolytic herpes simplex virus type 1(HSV1), geneticallyengineered to restrict virus replication to tumor cells. This oncolytic virus therapy is expected equal or better safety and better efficacy profile compare to existing oncolytic virus. DS8201 Breast cancer JP/US/EU P AntiHER2 antibody drug conjugate Injection Gastric cancer JP/Asia P Breast cancer, Urothelial (bladder) cancer US/EU P1 prep BristolMyers Squibb Granted Fast Track designation for HER2 positive metastatic breast cancer by U.S. FDA Granted Breakthrough Therapy designation for HER2 positive locally advanced or metastatic breast cancer who have been treated with trastuzumab and pertuzumab and have disease progression after adotrastuzumab emtansine (TDM1) by U.S. FDA Combination with nivolumab The drug (payload) conjugated through linker to the fully human monoclonal antibody to target HER2, one of the Epidermal Growth Factor Receptor (EGFR) family of proteins. Payload is potent topoisomerase I inhibitor with high membrane permeability. With bystander effect it provides neighbor cell death. Drugto antibody ratio is 78.

3 Oncology (Earlystage pipeline products) Solid tumors US/JP P1 DS3032 MDM2 inhibitor Leukemia US P1 PLX7486 FMS/TRK inhibitor Solid tumors US P1 DS8273 AntiDR5 antibody Solid tumors US P1 Solid tumors US P1 PLX8394 BRAF inhibitor Leukemia US P1 PLX9486 KIT inhibitor Solid tumors US P1 DS1123 AntiFGFR2 antibody Solid tumors JP P1 NonHodgkin's lymphoma JP P1 DS3201 EZH1/2 inhibitor Acute Myeloid Leukemia (AML) US P1 PLX73086/AC708 CSF1R inhibitor Tenosynovial Giant Cell Tumor (TGCT) US P1 PLX51107 BRD4 inhibitor Hematologic malignancies US P1 Breast cancer JP P1 U31402 AntiHER3 antibody drug conjugate NonSmall Cell Lung Cancer (NSCLC) US P1 prep DS1001 IDH1 mutant inhibitor Glioma JP P1 DS1205 AXL inhibitor NonSmall Cell Lung Cancer (NSCLC) US P1 prep PLX2853 BRD4 inhibitor Solid tumors US P1 Combination with osimertinib

4 CardiovascularMetabolics (Latestage pipeline products) Edoxaban/DU176b Lixiana (JP) LIXIANA (EU, Asia) SAVAYSA (US) Prasugrel/CS747 Effient (US, Asia) Efient (JP, EU) Esaxerenone/CS3150 Atrial fibrillation (AF) ASCA etc. Submitted Antiplatelet agent Ischemic stroke JP P3 Oral Ube Industries 2019 Hypertension JP P Oral Exelixis, Inc. Diabetic nephropathy JP P BR (14/6*), CN(15/8) submitted * means June 2014, ditto Factor Xa inhibitor Venous thromboembolism (VTE) ASCA etc. Submitted Oral BR (14/6), CN(15/8) submitted Elderly patients with nonvalvular atrial fibrillation (AF) JP P Additional dosage and formulation for elderly (80 years and older) AF patients The once daily oral anti coagulant (FXa inhibitor) discovered by Daiichi Sankyo. Edoxaban specifically, reversibly and directly inhibits the enzyme, Factor Xa, a clotting factor in the blood. The oral antiplatelet agent. Prasugrel helps to keep blood platelets from clumping together and developing a blockage in an artery. MR antagonist Additional indication, P3 studies completed The agent inhibits aldosterone binding to Mineralocorticoid Receptor (MR) which stimulate the sodium absorption into kidney. This agent is expected to exhibit antihypertensive and organprotective effect. CardiovascularMetabolics (Earlystage pipeline products) DS1040 TAFIa inhibitor Acute ischemic stroke, Acute pulmonary embolism US/EU/JP P1 DS2330 DS9231/TS23 Hyperphosphatemia treatment 2 plasmin inhibitorinactivating antibody Hyperphosphatemia in chronic kidney disease (CKD) Thrombosis (Acute pulmonary embolism, ischemic stroke) P1 P1 Translational Sciences, Inc. Inlisencing from Translational Sciences, Inc.

5 Internal Medicine (Latestage pipeline products) Hydromorphone/DS7113 Submitted by Daiichi Sankyo Propharma in February 2017 Opioid μreceptor agonist Cancer pain JP Submitted Injection 2017 Drug with new route of administration Narcotic analgesics. Immediate and extendedrelease tablets were launced in Japan in June Mirogabalin/DS5565 Laninamivir/CS8958 Inavir (JP) Fibromyalgia US/EU P α2δ ligand Diabetic peripheral neuropathic pain JP/Asia P3 Oral 2018 Postherpetic neuralgia JP/Asia P The pain therapy agent to reduce the neurotransmitter release from nerve terminals. This agent is expected to show the good balanced efficacy and safety profile. Neuraminidase inhibitor Influenza JP P3 Inhalation 2019 The longacting neuraminidase inhibitor and acts directly on the pulmonary and tracheal sites of infection. Additional dosage form, nebulizer formulation, announced topline result of P3 study Internal Medicine (Earlystage pipeline products) DS1971 Analgesic agent Chronic pain P1 DS1501 AntiSiglec15 antibody Osteoporosis US P1 DS7080 Angiogenesis inhibitor Neovascular agerelated macular degeneration (AMD), Diabetic Macular Edema (DME) US P1 DS5141 ENA oligonucleotide Duchenne muscular dystrophy (DMD) JP P1/2 Granted SAKIGAKE designation from MHLW in April 2017 DS1211 TNAP inhibitor Prevention of ectopic cacification diseases US P1 Research collaboration with Sanford Burnham Prebys Medical Discovery Institute

6 Vaccines VN100 VN0107/MEDI3250 VN0105 Intradermal seasonal influenza vaccine Prevention of seasonal influenza JP Submitted Injection* Terumo Corporation Live attenuated influenza vaccine Prevention of seasonal influenza JP Submitted DPTIPV/Hib vaccine Prevention of pertussis, diphtheria, tetanus, poliomyelitis and Hib Nasal spray AstraZeneca/ MedImmune JP P3 Injection Sanofi Pasteur 2019 Submitted by Japan Vaccine in April 2015 * prefilled syringe The Vaccine is a prefilled syringe type, intradermal influenza HA vaccine codeveloped by four companies [Daiichi Sankyo, Terumo Corporation, Japan Vaccine and Kitasato Daiichi Sankyo Vaccine Co., Ltd.]. The intradermal injection device for this vaccine is developed by Terumo Corpotation. This device, which offers a more easytouse, surefire method to administer the vaccine than current methods. The device is also expected to ease patient hesitation to be injected and lower the risk of damaging peripheral blood vessels and nerves within the subcutaneous tissue. The US brand name of this vaccine is FluMist Quadrivalent that is a live attenuated influenza vaccine which is administered as a nasal spray and contains four protective strains. Submitted by Daiichi Sankyo in June 2016 Codevelop with Sanofi K.K.and KDSV A combination vaccine reconstituting Hib with precipitated and purified pertussisdiphtheriatetanusinactivated polio vaccine (Salk vaccine) vaccine launched in Japan, as 1st in the class of pentavalent vaccine (DPTIPV/Hib). VN0102/JVC001 MeaslesMumpsRubella vaccine Prevention of Measles, Mumps and Rubella JP P1/2 Injection Japan vaccine company is conducting the P1/2 study

7 Stageup (major changes from the FY2017 Q1 Financial Announcement in July 2017) Class Target indication Current region/stage DS8201 AntiHER2 antibody drug conjugate Breast cancer JP/US/EU P2 DS8201 AntiHER2 antibody drug conjugate Gastric cancer JP/Asia P2 DS8201 AntiHER2 antibody drug conjugate Breast cancer, urothelial (bladder) cancer US/EU P1 prep Combination with nivolumab U31402 AntiHER3 antibody drug conjugate Nonsmall Cell Lung Cancer (NSCLC) US P1 prep DS1205 AXL inhibitor Nonsmall Cell Lung Cancer (NSCLC) US P1 prep Combination with osimertinib PLX2853 BRD4 inhibitor Solid tumors US P1 Esaxelenone/CS3150 MR antagonist Diabetic nephropathy JP P3 DS1211 TNAP inhibitor Prevention of ectopic cacification diseases US P1 Discontinue (major changes from the FY2017 Q1 Financial Announcement in July 2017) Class CL108 Vemurafenib/PLX4032 Zelboraf Opioid μreceptor agonist combination of antiemetic Target indication Acute pain Region / stage when discontinued US Received CRL Reason Strategic decision to discontinue with a recent portfolio and U.S. market review BRAF inhibitor Melanoma adjuvant US P3 Decided to discontinue the development after reviewing its study result