Ono Pharmaceutical Co., Ltd. · TSE:4528

Ono Pharmaceutical — Investor Day (June 2026)

61 slides from Ono Pharmaceutical's investor day presentation, Jun 8, 2026

  1. Slide 1: R&D Day

    R&D Day June 8, 2026 ond ONO PHARMA

  2. Slide 2: Forward-Looking Statements

    Forward-Looking Statements Forecasts and other forward-looking statements included in this document are based on information currently available and certain assumptions that the Company deems reasonable. Actual performance and other results may differ significantly due to various factors. Such facto

  3. Slide 3: Today's Agenda

    Today’s Agenda Opening Toichi Takino Representative Director, President and COO The data of POC Study Tatsuya Okamoto • ONO-4578 (ASCO2026) Corporate Officer / • ONO-2808 (7th World Parkinson Congress) Executive Vice President, Clinical Development Drug discovery activities in the Seishi Katsumata C

  4. Slide 4: Commitment to Three Priority Areas

    Commitment to Three Priority Areas As of May 8, 2026 ・Initiation of three global Phase 3 and data readouts from seven POC studies in FY2026 ・In the priority areas of oncology, immunology & inflammation, and neurology, four new pipelines have initiated phase 1 study. 4578 7429 Oncology Sapablursen Op

  5. Slide 5: Upcoming Product Launches

    Upcoming Product Launches As of June 8, 2026 FY2024 FY2025 FY2026 FY2027 FY2028+ Global Expansion Including Japan Tirabrutinib Sapablursen ONO-4685 QINLOCK® ROMVIMZA® QINLOCK® Tenosynovial Giant Cell ONO-4059 GIST 2L ONO-0530 T-cell Lymphoma/ GIST 4L Polycythemia Vera Autoimmune Diseases Tumor Prima

  6. Slide 6: Prospect for the Future

    Prospect for the Future Growth Drivers Peak Sales Pipeline (JPY bn) Global QINLOCK (GIST) 50 – 70 Global Products ROMVIMZA (TGCT) 50 – 70 Japanese Products Tirabrutinib (PCNSL) 20 – 30 (other than OPDIVO) Sapablursen (PV) 50 – 100 ONO-4578 (GC) 100 - Japanese Products (OPDIVO) ONO-2808 (MSA) 100 - J

  7. Slide 7: Key Event Schedule for FY2026

    Key Event Schedule for FY2026 FY2026 ONO-4059 (VELEXBRU) PCNSL Approval ONO-2017, Cenobamate QINLOCK Partial-onset Seizures GIST 4L ONO-4578 Gastric Cancer 1L ONO-0530, Sapablursen ONO-2808 Phase3 Polycythemia Vera Multiple System Atrophy QINLOCK GIST 2L ONO-1110 ONO-2020 Postherpetic Neuralgia Alzh

  8. Slide 8: Today's Agenda

    Today’s Agenda Opening Toichi Takino Representative Director, President and COO The data of POC Study Tatsuya Okamoto • ONO-4578 (ASCO2026) Corporate Officer / • ONO-2808 (7th World Parkinson Congress) Executive Vice President, Clinical Development Drug discovery activities in the Seishi Katsumata C

  9. Slide 9: Today's Contents

    Today’s Contents ONO-4578 Latest Information ONO-2808 Latest information 8 /60

  10. Slide 10: ONO-4578-08 Study

    ONO-4578-08 Study EP4 Receptor Antagonist 1L Treatment for HER2-negative Gastric Cancer 9 / 60

  11. Slide 11: ONO-4578 with nivolumab and chemotherapy as 1L gastric cancer treatment

    ONO-4578 combined with nivolumab (NIVO) and chemotherapy (chemo) as First-line (1L) treatment for patients with HER2- negative unresectable advanced or recurrent (adv/rec) gastric/gastroesophageal junction cancer (G/GEJ): A randomized, double-blind, phase 2 trial (ONO-4578-08) Sung Hee Lim1*, Izuma

  12. Slide 12: ONO-4578-08 Trial Design

    ONO-4578-08 Trial Design ONO-4578-08: Asian (Japan/Korea/Taiwan), randomized, double-blinded, Phase 2 trial (NCT06256328) ・ONO-4578 40mg, QD Primary Endpoint Key Eligibility Criteria ・Nivolumab 360mg, Q3W • PFS (per investigator) • Unresectable adv/rec HER2 (-) ・Chemotherapy, Q3W G/GEJ cancer （SOXa

  13. Slide 13: Demographics and Baseline Characteristics

    Demographics and Baseline Characteristics 12 ONO-4578 group Placebo group ONO-4578 group Placebo group (n = 150) (n = 76) (n = 150) (n = 76) Age, median (range), years 66.0 (27–84) 67.5 (36–86) Number of organs with metastases Male sex 113 (75.3) 64 (84.2) ≤1 63 (42.0) 29 (38.2) Country >1 87 (58.0)

  14. Slide 14: PFS per investigator: Primary Endpoint

    PFS per investigator : Primary Endpoint median PFS follow-up of 8.5 months ONO-4578 Placebo group group Event n (%) 67/150 (44.7) 47/76 (61.8) Median PFS (95% CI) 9.0 (7.1–11.1) 6.9 (4.4–8.4) HR (90% CI) 0.67 (0.48–0.92) P-value 0.040 No. at risk ONO-4578 150 106 77 37 0 0 Placebo 76 57 35 10 1 0 •

  15. Slide 15: PFS by PD-L1 CPS (≥1 vs <1)

    PFS by PD-L1 CPS (≥1 vs <1) 14 median PFS follow-up of 8.5 months CPS≥1 CPS<1 or Indeterminate ONO-4578 Placebo ONO-4578 Placebo group group group group Event n (%) 49/117 (41.9) 38/57 (66.7) Event n (%) 18/33 (54.5) 9/19 (47.4) Median PFS (95% CI) 9.9 (8.5–NA) 5.7 (4.1–8.3) Median PFS (95% CI) 5.6

  16. Slide 16: OS: Secondary Endpoint

    OS : Secondary Endpoint 15 ONO-4578 Placebo group group Event n (%) 42/150 (28.0) 30/76 (39.5) Median OS (95% CI) NA (NA–NA) 12.7 (10.4–NA) HR (95% CI) 0.60 (0.37–0.96) No. at risk ONO-4578 150 139 127 87 24 0 Placebo 76 72 59 34 9 0 • Although OS data are immature (minimum FU: 7.4 months) and shoul

  17. Slide 17: Post-hoc extended OS

    Post-hoc extended OS 16 minimum OS follow-up of 16.1 months Nakayama I, Ryu M-H, Lim S H, et al. Journal of Clinical Oncology10.1200/JCO-26-01072R1 Epub 2026 June 1. 16 /60

  18. Slide 18: Overall Survival by PD-L1 CPS (≥1 vs <1)

    Overall Survival by PD-L1 CPS (≥1 vs <1) 17 CPS≥1 CPS<1 or Indeterminate ONO-4578 Placebo ONO-4578 Placebo group group group group Event n (%) 26/117 (22.2) 25/57 (43.9) Event n (%) 16/33 (48.5) 5/19 (26.3) Median OS (95% CI) NA (NA–NA) 12.7 (7.8–NA) Median OS (95% CI) 9.6 (7.1–NA) NA (6.1–NA) HR (9

  19. Slide 19: Summary of Anti-tumor Response

    Summary of Anti-tumor Response 18 ONO-4578 group Placebo group (n = 150) (n = 76) ORR (95% CI), % 62.0 (53.7–69.8) 48.7 (37.0–60.4) Odds ratio (95% CI) 1.72 (0.98–3.00) BOR, n (%) CR 4 (2.7) 0 PR 89 (59.3) 37 (48.7) SD 31 (20.7) 26 (34.2) PD 12 (8.0) 9 (11.8) NE 14 (9.3) 4 (5.3) DOR, median (95% CI)

  20. Slide 20: Overall Safety Summary

    Overall Safety Summary 19 ONO-4578 group Placebo group All treated (n = 149) (n = 75) Any grade Grade3–5 Any grade Grade3–5 Any AEs 149 (100.0) 118 (79.2) 75 (100.0) 52 (69.3) Serious AEs 80 (53.7) 72 (48.3) 32 (42.7) 26 (34.7) AEs leading to death 12 (8.1) 3 (4.0) Any TRAEs 146 (98.0) 89 (59.7) 74

  21. Slide 21: Discussion on the Results of ONO-4578-08 Study

    Discussion on the Results of ONO-4578-08 Study 20/60 /60

  22. Slide 22: Comparison with Checkmate-649 study

    [Prognostic Factors] Comparison with Checkmate-649 study (CM649)  The proportion of patients aged ≥65, with diffuse type and peritoneal metastasis was approximately 10–30% higher than in CM649.  The proportion of patients with PS 1 and ≥2 metastatic organs was approximately 10–20% lower than in CM

  23. Slide 23: Mechanism of ONO-4578

    Mechanism of ONO-4578  Prostaglandin E2 (PGE2) is derived from arachidonic acid via cyclooxygenase-2 (COX-2).  COX-2 is overexpressed in solid tumor.1 PGE2PGE2 has been reported to induce myeloid-derived suppressor cells (MDSC) and M2 macrophages in the tumor microenvironment through one of its re

  24. Slide 24: ONO-4578 Development Status

    ONO-4578 Development Status Develop Indication ment Status Regions Study ID 2023 2024 2025 2026 2027 2028 2029 2030 phase First-line gastric PIII In preparation P3: Gastric cancer cancer＊ Key data Japan, P2: Gastric First-line PⅡ obtained in Korea, NCT06256328 cance colorectal cancer＊ FY2025 Taiwan

  25. Slide 25: ONO-4578 with Nivolumab and Chemotherapy in mCRC

    Safety, Efficacy, And Biomarkers Of ONO-4578, An EP4 Antagonist, In Combination With Nivolumab And Chemotherapy In Treatment-naive And Proficient Mismatch Repair (pMMR)/Microsatellite Stable (MSS) Metastatic Colorectal Cancer (mCRC) Yoshinori Kagawa1, Takeshi Kato2, Manabu Shiozawa3, Kensei Yamaguch

  26. Slide 26: Study Design & Patient Characteristics

    Study Design & Patient Characteristics 25 /60

  27. Slide 27: Results: Efficacy (Overall Population)

    Results: Efficacy (Overall Population) n (%) [95% CI] Objective response ratea 25 (73.5) [55.6, 87.1] Disease control ratea 31 (91.2) [76.3, 98.1] Best overall responsea Complete Response 0 [0.0, 10.3] Partial Response 25 (73.5) [55.6, 87.1] Stable Disease 6 (17.6) [6.8, 34.5] Progressive Disease 2

  28. Slide 28: Results: Efficacy & BM Analysis (CPS-Positive/Negative)

    Results: Efficacy & BM Analysis (CPS-Positive/Negative) PD-L1 CPS ≥1 (n=17) PD-L1 CPS <1 (n=14) Objective response rate, n (%) [95% CI]a 15 (88.2) [63.6, 98.5] 9 (64.3) [35.1, 87.2] Progression free survival in monthsb Median [95% CI] 12.3 [6.9, NA] 7.4 [6.9, NA] Progression free survival rate At 6

  29. Slide 29: ONO-4578 Development Status (Phase 2 and beyond)

    ONO-4578 Development Status (Phase 2 and beyond) Develop Indication ment Status Regions Study ID 2023 2024 2025 2026 2027 2028 2029 2030 phase First-line gastric PIII In preparation P3: Gastric cancer cancer＊ Key data Japan, P2: Gastric First-line PⅡ obtained in Korea, NCT06256328 cance colorectal c

  30. Slide 30: ONO-2808-03 study

    ONO-2808-03 study S1P5 Receptor Agonist Multiple System Atrophy (MSA) 29 /60

  31. Slide 31: ONO-2808 and MSA

    ONO-2808 and MSA Compound information  Progressive neurodegenerative disease with cerebellar atrophy  Average onset age: 55–60 years  Severe and rapidly progressive Compound ONO-2808 <Features>  Currently symptomatic treatment with limited efficacy Originator ONO Pharma  Estimated patients (203

  32. Slide 32: Poster: LBP37.09

    Poster: LBP37.09 Safety, Tolerability, and Preliminary Efficacy of ONO-2808, a Sphingosine-1-Phosphate Receptor 5 Agonist, in Multiple System Atrophy Anne-Marie Wills’, U Schur! Atsushi Takeda’, Susan Perlman’, Tomoko eda”. Carlos Singer, Daniel D Truong’, Pravin Khemans", Praveen Dayal” Pinky Agarw

  33. Slide 33: ONO-2808-03 study design

    ONO-2808-03 study design (NCT05923866) Extension part Double-blinded Screening core Blinded Open-label Follow-up rollover 24 weeks To week 32 To week 80 To week 92 Patients diagnosed with MSA with symptom onset within 5 years (N = 92) ONO-2808 Placebo low dose Stratified by MSA subtype ONO-2808 ONO-

  34. Slide 34: Baseline demographics and clinical characteristics

    Baseline demographics and clinical characteristics Placebo ONO-2808 ONO-2808 ONO-2808 ONO-2808 low medium high total Characteristic n = 23 n = 23 n = 23 n = 23 n = 69 Age, years Mean (SD) 59.6 (6.7) 63.2 (7.8) 63.6 (4.5) 60.4 (7.7) 62.4 (6.9) Median (range) 58 (51–76) 63 (46–80) 65 (54–74) 60 (49–73

  35. Slide 35: Efficacy-mUMSARS / MSA-P group

    Efficacy-mUMSARS / MSA-P group Progression Suppression 34 /60

  36. Slide 36: Efficacy-mUMSARS / MSA-C group

    Efficacy-mUMSARS / MSA-C group Progression Suppression 35 /60

  37. Slide 37: Efficacy-vMRI measurements

    Efficacy-vMRI measurements 36 /60

  38. Slide 38: Efficacy-vMRI measurements / MSA-P group

    Efficacy-vMRI measurements / MSA-P group 37 /60

  39. Slide 39: Efficacy-vMRI measurements / MSA-C group

    Efficacy-vMRI measurements / MSA-C group 38 /60

  40. Slide 40: TEAEs in ≥10% of patients in any treatment group

    TEAEs in ≥10% of patients in any treatment group during the double-blinded core part Placebo ONO-2808 ONO-2808 ONO-2808 ONO-2808 low medium high total Preferred term, n (%) n = 23 n = 23 n = 23 n = 23 n = 69 8 (35) 9 (39) 2 (9) 4 (17) 15 (22) Urinary tract infection Headache 0 3 (13) 2 (9) 6 (26) 11

  41. Slide 41: Transaminase elevations adjudicated by the IDMC

    Transaminase elevations adjudicated by the IDMC at week 24 Placebo ONO-2808 ONO-2808 ONO-2808 ONO-2808 low medium high total Category, n (%) n = 23 n = 23 n = 23 n = 23 n = 69 Any transaminase elevation confirmed by IDMC 0 1 (4) 3 (13) 4 (17) 8 (12) Any treatment-related transaminase elevation by hi

  42. Slide 42: Discussion on the Results of ONO-2808-03 Study

    Discussion on the Results of ONO-2808-03 Study 41/60

  43. Slide 43: Modified UMSARS for ONO-2808

    Modified UMSARS for ONO-2808 Used in the ONO-2808 Phase 2 study (maximum score: 27) Part I: Historical Review (Activities of Daily Living) Part II: Motor Examination Scale (Motor - Assesses average function over the past 2 weeks based on an interview Function Assessment) Rated on the more affected s

  44. Slide 44: Modified UMSARS for Amlenetug (Lu AF82422)

    Modified UMSARS for Amlenetug (Lu AF82422) Used in Phase 2 study of Lundbeck’s investigational drug (Amlenetug, Lu AF82422; maximum score, 48) Part I: Historical Review (Activities of Daily Living) Part II: Motor Examination Scale (Motor - Assesses average function over the past 2 weeks based on an

  45. Slide 45: ONO-2808 Summary

    ONO-2808 Summary MSA-P 2 Double-blinded Blinded rollover Open-label core extension extension g 0 # a 8 ro & 6 5 go4 E 3 g 2 3 zo 2 ‘Number of patients we Piacebo/ONOZIORIOw 13 2 9 8 7 ‘ =A ONO2208100 " " 9 6 5 ‘ “a 0N0 2008 medium 2 0 9 ‘ —#- ONO.2E08 high 2 R 8 ‘Data cxf. Februry 24,2026. CFB in mU

  46. Slide 46: ONO-2808 Development Status

    ONO-2808 Development Status Develo Indication(s) pment Status Regions Study ID 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 phase Planned in Japan, P3: MSA (planned) MSA In P3 the US, TBD （MSA-P） preparation and EU ONO-2808-03-001 study (TBD) Japan P2: MSA Key data NCT059238 MSA P2 obtained in

  47. Slide 47: Today's Agenda

    Today’s Agenda Opening Toichi Takino Representative Director, President and COO The data of POC Study Tatsuya Okamoto • ONO-4578 (ASCO2026) Corporate Officer / • ONO-2808 (7th World Parkinson Congress) Executive Vice President, Clinical Development Drug discovery activities in the Seishi Katsumata C

  48. Slide 48: ONO's Drug Discovery

    ONO’s Drug Discovery Focus Area Immunology & Oncology Inflammation Neurology Cambridge, MA since 1998 London since 1998 ◆ Discovery Research Alliance Activities ◆ Discovery Research Alliance Activities Deciphera Pharmaceuticals, founded in 2003 Lawrence, KS since 2024 Japan ◆ Research Institute  Re

  49. Slide 49: Drug Discovery Strategy in Neurology

    Drug Discovery Strategy in Neurology  Elucidate disease biology to address unmet medical needs  Develop disease-modifying therapies, not only symptomatic treatments  Target glial cells, not only neurons  Leverage clinical insights to improve translatability to humans Deliver new drugs through cu

  50. Slide 50: Pipeline Overview (Neurology)

    AD : Alzheimer’s Disease DPN : Diabetic Peripheral Neuropathy Pipeline Overview (Neurology) ALS : Amyotrophic Lateral Sclerosis IBS : Irritable Bowel Syndrome ACI : Acute Cerebral Infarction PD : Parkinson’s Disease CH : Cerebral Hemorrhage MS : Multiple Sclerosis CIPN : Chemotherapy-Induced Periphe

  51. Slide 51: Glial Cell-targeted Drug Discovery

    AD : Alzheimer’s Disease Glial Cell-targeted Drug Discovery CIPN : Chemotherapy-Induced Peripheral Neuropathy DPN : Diabetic Peripheral Neuropathy IBS : Irritable Bowel Syndrome MSA : Multiple System Atrophy Pioneering Glial Drug Discovery ONO-2506 ONO-2952 ONO-2506（Arocyte, injection） Research and

  52. Slide 52: Lipid-targeted Drug Discovery (S1P Receptor Modulation)

    Lipid-targeted Drug Discovery (S1P Receptor Modulation) ONO-4641 ONO-1266 ONO-2808 S1P1＆5 agonist S1P antagonist Selective S1P5 agonist S1P1 S1P2 S1P3 S1P4 S1P5 Expression Whole body Whole body Neural cells, vascular Lymphocytes Neural cells, profile （lymphocytes, neural cells, endothelial cells vas

  53. Slide 53: Ion Channel-targeted Drug Discovery

    Ion Channel-targeted Drug Discovery • BioFocus (now Charles River) Utilizing Platform & Technology • Evotec Through Open Innovation • Vanderbilt University GABAAα5 • Xention (now Metrion Biosciences) negative allosteric modulator Experience in ONO-8590580 Multiple Ion Channel Drug Discovery Neuron 

  54. Slide 54: Challenges and Strategies in Neurological Drug Discovery

    Challenges and Strategies in Neurological Drug Discovery Challenges Strategies Target Validation  Research collaboration for target discovery  Understanding disease pathology  Utilizing disease models and evaluation systems from  Access to brain tissue and cerebrospinal fluid academia that recap

  55. Slide 55: Challenges and Strategies in Neurological Drug Discovery

    Challenges and Strategies in Neurological Drug Discovery The Tohoku University Tohoku Medical Megabank Collaboration with UK Dementia Research Institute Organization (ToMMo) ・ Conducting community-based cohort study and three-generation cohort study launched in 2013 ・ Performing large-scale whole ge

  56. Slide 56: Challenges and Strategies in Neurological Drug Discovery

    Challenges and Strategies in Neurological Drug Discovery Introduction of Mahoro robots Brain organoid model using iPS cells Organoid 10-cent coin 1-yen coin Reproducing six human brain cell types Stably culturing iPS cells to support drug discovery projects Pericytes Astrocytes Endothelial Neurons O

  57. Slide 57: Challenges and Strategies in Neurological Drug Discovery

    Challenges and Strategies in Neurological Drug Discovery AI-driven drug discovery with Tokyo-1 Drug discovery partnership with Vanderbilt University December 10, 2015 Binding Site Analysis https://news.vumc.org/2015/12/10/vanderbilt -ono-pharmaceutical-sign-drug-discovery- agreement/ https://lab.van

  58. Slide 58: Challenges and Strategies in Neurological Drug Discovery

    Challenges and Strategies in Neurological Drug Discovery Co-development of brain PET ligands (CNS drug discovery alliance) Longitudinal clinical study of the disease (investigator-initiated observational study) Co-development of α-synuclein pathology PET Identification of clinical Successful imaging

  59. Slide 59: Development Pipeline (Neurology)

    Development Pipeline (Neurology) Code Target Indication PⅠ PⅡ PⅢ Filed Approval Partial-onset seizures ONO-2017 Partial-onset seizures (pediatric) Primary generalized tonic-clonic seizures ONO-2808 Mutiple system atrophy Postherpetic neuralgia Fibromyalgia ONO-1110 Hunner-type interstitial cystitis

  60. Slide 60: Summary

    Summary  Ono is strengthening our pipeline by integrating internal and external expertise across the stage of the drug discovery process.  Ono leverages cutting-edge technologies and open innovation to deliver innovative new drugs as quickly as possible to patients worldwide, addressing unmet medi

  61. Slide 61: Dedicated to the Fight against Disease and Pain

    Dedicated to the Fight against Disease and Pain

Ono Pharmaceutical — Investor Day (June 2026) on 6ix