Slides
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Oct 30, 2025 FY2025 Q2 Financial Results
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/46 Today’s Attendees 伊藤 雅樹 Masaki Itoh 執行役員 開発本部長 Corporate Officer / Executive Director, Clinical Development 岡本 達也 Tatsuya Okamoto 代表取締役 社長 COO Representative Director, President and Chief Operating Officer 滝野 十一 Toichi Takino 執行役員 営業本部長 Corporate Officer / Executive Director, Sales and Marketing 北田 浩一 Hirokazu Kitada オンコロジー統括部長 Director of Oncology Business Division 高橋 宏幸 Hiroyuki Takahashi 1 常務執行役員 経営戦略本部 経営管理統括部長 Corporate Executive Officer / Division Director, Corporate Strategy & Planning, Business Management Division,
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/46 Agenda 2026年3月期第2四半期 決算概要について Financial Results FY2025 Q2 （14:00-14:20） 開発品の進捗状況 Development Pipeline Progress Status（14:20-14:40） 執行役員 開発本部長 Corporate Officer / Executive Director, Clinical Development 岡本 達也 Tatsuya Okamoto 質疑応答 Q&A Session（14:55-15:15） オプジーボの動向 Trend of OPDIVO（14:40-14:55） 代表取締役 社長 COO Representative Director, President and Chief Operating Officer 滝野 十一 Toichi Takino 執行役員 営業本部長 Corporate Officer / Executive Director, Sales and Marketing 北田 浩一 Hirokazu Kitada 2
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/46 Forward-Looking Statements 3 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 factors include, but are not limited to: Information about pharmaceutical products (including products currently in development) included in this document is not intended to constitute an advertisement of medical advice. Forward-looking statements: This presentation contains forward-looking statements regarding the Company’s future plans, strategies, and performance. Current assumptions: These statements are based on current expectations, assumptions, and information available to management at this time. Risks and uncertainties: Forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially. No guarantee of outcomes: Forecasts, targets, and projections are not guarantees of future performance or achievement of stated goals. Official guidance: Official financial guidance should be referred to in accordance with relevant regulatory requirements and disclosures. Product/market risks: Risks include, but are not limited to, product development challenges, regulatory approvals, market acceptance, and competition. Economic/industry risks: Additional risks may arise from changes in economic conditions, currency fluctuations, and healthcare policy reforms. No obligation to update: The Company undertakes no obligation to update or revise any forward-looking statements as a result of new information or future events. Prevailing language: In the event of any inconsistency between language versions, the original Japanese language version shall prevail.
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/46 FY2025 Q2 Financial Results 4//46
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/46 Profit and Loss Recognition Period for Deciphera 5 Regarding the profit and loss recognition for Deciphera Pharmaceuticals, Inc., three months were recorded in the same period last year, while six months have been recorded this year. Three months included June, 30 September, 30April, 1 FY2024Q2 FY2025Q2 Six months included
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/46 FY2025Q2 Sales Revenue Revenue increased by ¥16.8 billion (7.0%) year on year to ¥257.1 billion, Domestic Sales : While sales of FORXIGA expanded, overall sales slightly decreased mainly due to a decline in OPDIVO sales. Overseas Sales : Sales of QINLOCK increased by ¥10.0 billion to ¥18.1 billion. Sales of ROMVIMZA were ¥2.8 billion mainly due to the higher-than-expected acquisition of new prescriptions. FY2025Q2 Core Profit for the Period Core profit for the period increased by ¥2.8 billion (5.5%) to ¥53.8 billion. Although expenses increased due to the inclusion of three additional months of R&D and SG&A expenses for Deciphera compared to the previous year, the increase in sales exceeded these expenses, resulting in a profit increase. FY2025 Financial Result Forecast Sales and profit for the year is expected to increase compared to the previous fiscal year. Although a decrease in sales is expected due to the entry of generic versions of FORXIGA tablets, an increase in sales and profits is anticipated as this will be offset by the growth in sales of QINLOCK and ROMVIMZA, as well as an increase in overseas royalty revenue. Highlights of Financial Results for FY2025Q2 (Core Basis) Status of Development Pipeline Cenobamate (ONO-2017) : Approval application filed (Japan) ROMVIMZA: Approved (EU), Announced 2-year efficacy and safety results in the Phase 3 trial (MOTION) ONO-4578: Achieved primary endpoint in Phase 2 trial ONO-2808: Confirmed efficacy signals and tolerability in Phase 2 trial For the FY2025Q2 ending March 2026, we recorded increased revenue and profit. 6
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/46 7 Royalty and Others ¥82.2 billion YoY +5.1 billion (+6.7%) Goods and Products Sales ¥175.0 billion YoY +11.7 billion (+7.1%) Revenue ¥257.1billion YoY +16.8 billion (+7.0%) FY2025Q2 : Sales Revenue
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/46 ・OPDIVO -4.1 ・FORXIGA +5.1 ・GLACTIV -2.7 （¥ Billion） Revenue 240.3 ＋16.8 (+7.0%) FY2024Q2 Revenue FY2025Q2 Revenue Increase Decrease Goods and products (except Deciphera) -1.1 Royalty revenue Higher overseas sales +6.5 Royalty revenue Foreign exchange rate -1.8 Deciphera Sales revenue +12.9 Revenue 257.1 Domestic sales decreased due to intensified competition affecting OPDIVO, despite the increase in sales of FORXIGA Tablet. However, overall sales increased by ¥16.8 billion year on year, driven by the revenue from Deciphera. 8 FY2025Q2 : Sales Revenue (Breakdown) Others +0.3 ・QINLOCK +10 ・ROMVIMZA +2.8
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/46 9 ¥ in Billion FY2024Q2 FY2025Q2 YoY FY2025 Forecast*Change Change(%) Revenue 240.3 257.1 16.8 7.0％ 490.0 Goods and products 163.3 175.0 11.7 7.1％ 330.0 Royalty and others 77.0 82.2 5.1 6.7％ 160.0 Goods and Products (Domestic) FY2024Q2 FY2025Q2 YoY FY2025 Forecast*Change Change(%) OPDIVO Intravenous Infusion 62.6 58.5 -4.1 -6.5% 125.0 FORXIGA Tablets 43.7 48.8 5.1 11.6% 80.0 ORENCIA for Subcutaneous Injection 13.5 13.8 0.3 2.1% 28.0 GLACTIV Tablets 9.6 6.9 -2.7 -28.2% 12.0 VELEXBRU Tablets 5.2 6.0 0.8 15.8% 11.0 ONGENTYS Tablets 3.8 4.5 0.7 18.6% 9.0 PARSABIV Intravenous Injection 4.2 4.5 0.3 7.4% 9.0 KYPROLIS for Intravenous Infusion 4.6 4.0 -0.5 -12.1% 9.0 * The consolidated financial forecast for the fiscal year ending March 2026, announced on May 8, 2025, is provided. ・Sales revenue of domestic products is shown in a gross sales basis (shipment price), and sales revenue of overseas products is shown in a net sales basis. FY2025Q2 : Sales Revenue by Product (Domestic)
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/46 10 ¥ in Billion FY2024Q2 FY2025Q2 YoY FY2025 Forecast*Change Change(%) Revenue 240.3 257.1 16.8 7.0％ 490.0 Goods and products 163.3 175.0 11.7 7.1％ 330.0 Royalty and others 77.0 82.2 5.1 6.7％ 160.0 Goods and Products (Overseas) FY2024Q2 FY2025Q2 YoY FY2025 Forecast*Change Change(%) OPDIVO 6.5 7.2 0.7 11.5% 13.5 QINLOCK® 8.1 18.1 10.0 123.3% 34.0 ROMVIMZATM － 2.8 － － 5.0 Royalty and others FY2024Q2 FY2025Q2 YoY Change Change(%) OPDIVO 56.4 59.4 3.0 5.3% KEYTRUDA® 12.8 13.8 1.0 7.5% FY2025Q2 : Sales Revenue by Product (Overseas) / Royalty * The consolidated financial forecast for the fiscal year ending March 2026, announced on May 8, 2025, is provided. ・Sales revenue of domestic products is shown in a gross sales basis (shipment price), and sales revenue of overseas products is shown in a net sales basis.
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/46 11 R&D Expense ¥71.0 billion YoY +5.7 billion (+8.8％) Revenue ¥257.1 billion YoY +16.8 billion (+7.0％) Core Operating Profit ¥70.1 billion YoY +4.7 billion (+7.2％) SG&A Expense ¥61.1 billion YoY +5.6 billion (+10.2％) FY2025Q2 : Core Operating Profit
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/46 ・Sales +12.9 ・EXP-16.2 12 While R&D and SG&A expenses have been recorded by Deciphera, which were not recorded in the first quarter of the previous fiscal year, core operating profit increased by ¥4.7 billion year on year to ¥70.1 billion due to an increase in royalty revenue and a promotion of cost efficiency. YoY Breakdown Main reason Royalty and others : +4.9 Goods and product : -1.1 Sales revenue (excluding Deciphera) Main reason - Decrease in research costs and clinical trial costs R&D expenses (excluding Deciphera) Main reason - Increase in co-promotion fee for FORXIGA tablet - Promotion of cost efficiency SG&A expenses (excluding Deciphera) Excluding Deciphera’s Revenue & Expenses ＋8.0 Revenue +3.9 Core operating profit 70.1 SG&A expenses +0.2Cost of sales -0.3 Deciphera -3.3R&D expenses ＋4.1 Core operating profit 65.4 +4.7(+7.2%) FY2024Q2 Core operating profit FY2025Q2 Core operating profit Increase Decrease (¥ Billion) FY2025Q2 : Core Operating Profit (Breakdown) Other income and expenses +0.1
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/46 13 ¥ in Billion FY2024Q2 FY2025Q2 YoY FY2025 Forecast*Change Change(%) Revenue 240.3 257.1 16.8 7.0％ 490.0 Cost of sales 53.9 54.8 0.9 1.7％ 103.5 R&D expenses 65.3 71.0 5.7 8.8％ 150.0 SG&A expenses 55.4 61.1 5.6 10.2％ 120.0 Other income 0.6 0.6 -0.0 -2.6％ 0.5 Other expenses 0.9 0.8 -0.1 -15.8％ 3.0 Core operating profit 65.4 70.1 4.7 7.2％ 114.0 Core profit before tax 65.2 70.7 5.5 8.4％ 114.0 Core profit for the period (attributable to owners of the Company) 51.0 53.8 2.8 5.5％ 91.0 YoY Breakdown R&D ratio：27.6％ Main reason - R&D expenses from Deciphera - Milestone payment to LigaChem Bioscience, Inc. R&D expenses +¥5.7 billion (+8.8%) Main reason - SG&A expenses from Deciphera - Increase in co-promotion fee for FORXIGA tablet SG&A expenses +¥5.6 billion (+10.2%) FY2025Q2 : Financial Overview (Core) COGS ratio : 21.3% Main reason - Increase in cost of goods sold Cost of sales +¥0.9 billion (+1.7%) * The consolidated financial forecast for the fiscal year ending March 2026, announced on March 8, 2025, is provided.
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/46 14 ¥ in Billion FY2024Q2 FY2025Q2 YoY FY2025 Forecast*Change Change(%) Revenue 240.3 257.1 16.8 7.0％ 490.0 Cost of sales 64.0 72.0 8.0 12.5％ 135.0 R&D expenses 68.8 71.0 2.2 3.2％ 150.0 SG&A expenses 58.4 61.2 2.7 4.7％ 120.0 Operating profit 48.8 52.1 3.3 6.7％ 85.0 Profit before tax 47.5 52.2 4.6 9.7％ 85.0 Profit for the period (attributable to owners of the Company) 37.4 40.1 2.7 7.1％ 67.0 Main reason - Amortization expenses related to intangible assets acquired through acquisitions Cost of sales +¥8.0 billion R&D ratio : 27.6％ Main reason - R&D expenses from Deciphera - Absence of impairment loss related to development compounds R&D expenses +¥2.2 billion Main reasons - SG&A expenses from Deciphera - Increase in co-promotion fee for FORXIGA tablet - Absence of expenses associated with the acquisition of Deciphera SG&A expenses +¥2.7 billion (Ref) FY2025Q2 : Financial Overview (Full Basis) * The consolidated financial forecast for the fiscal year ending March 2026, announced on March 8, 2025, is provided. YoY Breakdown
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/46 15 ¥ in Billion IFRS (Full) basis Adjustment Core basis Amortization Impairment loss Others Total Sales revenue 257.1 － 257.1 Cost of sales 72.0 -12.5 -4.7 -17.2 54.8 Gross profit 185.2 +12.5 － +4.7 +17.2 202.4 R&D expenses 71.0 － 71.0 SG&A expenses 61.2 -0.1 -0.1 61.1 Other income /expenses -0.9 -0.7 -0.7 -0.2 Operating profit 52.1 +12.5 － +5.5 +18.0 70.1 Operating profit ratio 20.2% － 27.2% Finance income / Finance cost 0.1 -0.5 -0.5 0.6 Profit before tax 52.2 +12.5 － +6.0 +18.5 70.7 Income tax expense 12.2 +3.3 +1.5 +4.8 17.0 Profit for the year 40.1 +9.2 － +4.5 +13.7 53.8 Main reasons - Amortization expenses related to intangible assets acquired through acquisitions or in-licensing - Amortization expenses related to inventories from PPA Cost of sales -¥17.2 billion No Adjustment R&D expenses Main reason - Termination Fee for lease contract cancellation SG&A expenses and Other income&expense (Ref) FY2025Q2 : Reconciliation from Full to Core Basis Breakdown
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/46 ¥ in Billion FY2024 Actual FY2025 Forecast Change Change (%) Revenue 486.9 490.0 3.1 0.6% Cost of sales 106.9 103.5 -3.4 -3.1% R&D expenses 143.3 150.0 6.7 4.7% SG&A expenses 122.2 120.0 -2.2 -1.8% Core operating profit 112.7 114.0 1.3 1.2% Core profit before tax 113.9 114.0 0.1 0.1% Income tax expense 23.4 23.0 -0.4 -1.8% Core profit for the year 90.4 91.0 0.6 0.7% FY2025 : Financial Forecast (Core/Compared to the Previous Year) * The exchange rate assumed for the second half of the fiscal year is ¥145 per US dollar. Breakdown Main reason - Decrease in sales related to FORXIGA tablets and long-term listed products Cost of sales -¥3.4 billion Main reasons - Costs related to Deciphera Pharmaceuticals (from 9 months to 12 months) - Costs associated with sapablursen in-licensed from Ionis Pharmaceuticals, Inc. - Promotion of cost efficiency measures R&D expenses +¥6.7 billion Main reasons - Costs related to Deciphera Pharmaceuticals (from 9 months to 12 months) - Promotion of cost efficiency measures SG&A expenses -¥2.2 billion There is no change from the consolidated financial forecasts, announced on May 8th, 2025. 16
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/46 FY2025 : Financial Forecast (Sales Revenue by Product) 17 Goods and Products (Domestic) FY2024 Actual FY2025 Previous forecast Revision from previous forecast FY2025 Revised forecast YoY Change Change(%) OPDIVO Intravenous Infusion 120.3 125.0 -5.0 120.0 -0.3 -0.3% FORXIGA Tablets 89.6 80.0 80.0 -9.6 -10.7% ORENCIA for Subcutaneous Injection 26.6 28.0 28.0 1.4 5.2% GLACTIV Tablets 18.3 12.0 12.0 -6.3 -34.6% VELEXBRU Tablets 10.5 11.0 11.0 0.5 4.4% ONGENTYS Tablets 7.6 9.0 9.0 1.4 17.8% KYPROLIS for Intravenous Infusion 8.6 9.0 9.0 0.4 4.6% PARSABIV Intravenous Injection 8.4 9.0 9.0 0.6 6.7% Goods and Products (Overseas) FY2024 Actual FY2025 Previous forecast Revision from previous forecast FY2025 Revised forecast YoY Change Change(%) OPDIVO 13.1 13.5 13.5 0.4 2.9％ QINLOCK® 25.5 34.0 2.0 36.0 10.5 41.2% ROMVIMZATM － 5.0 3.0 8.0 － ・Sales revenue of domestic products is shown in a gross sales basis (shipment price), and sales revenue of overseas products is shown in a net sales basis.
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/46 18 ¥ in Billion FY2024 Actual FY2025 Forecast Change Change (%) Revenue 486.9 490.0 3.1 0.6% Cost of sales 147.9 135.0 -12.9 -8.8% R&D expenses 149.9 150.0 0.1 0.1% SG&A expenses 125.7 120.0 -5.7 -4.5% Operating profit 59.7 85.0 25.3 42.3% Profit before tax 59.3 85.0 25.7 43.3% Income tax expense 9.2 18.0 8.8 96.5% Profit for the year 50.0 67.0 16.9 33.8% FY2025 : Financial Forecast (Full / Compared to the Previous Year) * The exchange rate assumed for the second half of the fiscal year is ¥145 per US dollar. For the second half of the fiscal year, the sensitivity to exchange rates is assumed to be an increase of ¥0.7 billion in revenue and an increase of ¥0.2 billion in operating profit for every ¥1 depreciation of the yen. Breakdown Main reasons - Decrease in sales related to FORXIGA tablets and long-term listed products - Absence of sales milestone on FORXIGA recorded in the previous fiscal year Cost of sales -¥12.9 billion Main reasons - Costs related to Deciphera Pharmaceuticals (from 9 months to 12 months) - Costs associated with sapablursen in- licensed from Ionis Pharmaceuticals, Inc. - Absence of impairment losses on development compounds in the previous fiscal year R&D expenses +¥0.1 billion Main reasons - Costs related to Deciphera Pharmaceuticals (from 9 months to 12 months) - Promotion of cost efficiency measures SG&A expenses -¥5.7 billion There is no change from the consolidated financial forecasts, announced on May 8th, 2025.
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/46 Application for Approval of Cenobamate in Japan 19
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/46 Cenobamate（ONO-2017） 20 • An antiepileptic drug with two action - Inhibition voltage-dependent sodium currents and positive modulation of γ-aminobutyric acid type A (GABAA) ion channels1) - • Application for approval was submitted at the end of September based on the results of the Phase 3 clinical trial in patients with partial-onset seizures in Japan, South Korea, and China. Source：Chen Z et al : JAMA Neurol. 2018 Mar 1;75(3):279-286 【Primary Endpoint of Phase 3 Clinical trial】 Median percentage change in 28-day focal seizure frequency (MITT-M population） Source：Sunita N Misra, Louis Ferrari, Zhen Hong, et.al., A Randomized, Double－Blind, Placebo－Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Adjunctive CENOBAMATE in Asian Patients with Focal Seizures. AES 2024 1) Ono pharmaceutical press release in Oct., 2020 2) Japanese Society of Epilepsy. Guidebook for Board-Certified Epileptologists, Revised 2nd Edition. Shindan to Chiryo Sha; 2020. 3) Chen Z et al : JAMA Neurol. 2018 Mar 1;75(3):279-286 【Cenobamate】 • U.S. approved: 2020 / Europe approved : 2021 • The cumulative number of prescriptions worldwide is approximately 220,000 (June 2025 data). • In Phase 3 clinical trial, patients with uncontrolled partial-onset seizures despite treatment with 1 to 3 ASMs, once-daily cenobamate therapy for 12 weeks at all dose levels resulted in a significant reduction in the median percent change in seizure frequency. • Compared to existing treatments, the combination therapy showed a favorable safety profile and was well tolerated. Seizure freedom is achieved in 50% of patients with the first antiepileptic drug, after adding more drugs, the maximum rate reaches approximately 70%. 【Epilepsy】 • Epilepsy is a chronic brain disorder occurring at any age in which seizures are triggered by abnormal excitability of nerve cells in the brain. • In Japan, approximately 1 million people with epilepsy, and approximately 86,000 new cases are diagnosed each year. 2) • Approximately 30% of patients with epilepsy are drug-resistant, meaning that even with combination therapy using existent anti-seizure medications, they are unable to achieve seizure freedom. 3) Number of Concomitant Antiepileptic Drugs and Rate of Seizure Freedom
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/46 ROMVIMZA : Phase 3 Trial: 2-year data - European Society For Medical Oncology 2025 presentation data - 21
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/46 MOTION Phase 3 Trial: Study Design and Methods1 22 Key eligibility criteria Patients ≥18 years old with a confirmed diagnosis of symptomatic TGCT for which surgical resection would potentially cause worsening of functional limitation or severe morbidity Previous treatment with imatinib or nilotinib was allowed Randomization was stratified by geographical region and tumor location Clinicaltrials.gov identifier: NCT05059262 Open-label periodDouble-blind period Part 1: Eligible patients were assigned to receive either vimseltinib or matching placebo for 24 weeks Part 2: Long-term treatment phase in which all patients may receive open- label vimseltinib to week 49 Patients may continue treatment after week 49 in the extension period Extension period Data cutoff: February 22, 2025. aReported reason due to “fall.” 1. Gelderblom H, et al. Lancet. 2024;403(10445):2709-19. IRR, independent radiological review; TGCT, tenosynovial giant cell tumor. • In total, 118 patients received vimseltinib • In the vimseltinib arm, 73/83 continued to receive treatment in part 2 • In the placebo arm, 35/40 crossed over to vimseltinib in part 2 • Median (range) treatment duration was 23.6 months (2–36) for patients randomized to vimseltinib and 19.1 months (1–30) for those who crossed over to vimseltinib • At data cutoff, 51% (60/118) remain on treatment, and reasons for treatment discontinuation are: • Withdrawal by patient (n = 29) • Adverse event (n = 14) • Physician decision (n = 3) • Progressive disease by IRR (n = 2) • Noncompliance with study drug (n = 2) • Unrelated death (n = 1) a • Other (n = 7)Primary and key secondary endpoints assessed at the end of part 1, the beginning of week 25 Vimseltinib 83 patients 30 mg twice weekly Continued on vimseltinib 73 patients 30 mg twice weekly Placebo 40 patients Crossed over to vimseltinib 35 patients 30 mg twice weekly Randomized 2:1 Continued on vimseltinib 30 mg twice weekly Continued on vimseltinib 30 mg twice weekly
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/46 MOTION Phase 3 Trial: Efficacy 23 + denotes response was ongoing at the last assessment. Dark blue and patterned shading represents the DOR. Baseline for all patients (including those who crossed over from placebo to vimseltinib) was defined as the last assessment prior to treatment with vimseltinib. aData cutoff: August 22, 2023, from Gelderblom H, et al. Lancet. 2024;403(10445):2709-19. bData cutoff: February 22, 2025. cTVS response corresponds to ≥50% reduction in estimated tumor volume.1 1. Peterfy C, et al. Future Oncol. 2022;18(12):1449-59. CI, confidence interval; CR, complete response; DOR, duration of response; IRR, independent radiological review; N/A, not applicable; NE, not evaluable; NR, not reached; ORR, objective response rate; PD, progressive disease; PR, partial response; RECIST v1.1, Response Evaluation Criteria in Solid Tumors version 1.1; SD, stable disease; TVS, Tumor Volume Score. Week 25 ≥2 years on studyb Vimseltinib n = 83 Placebo n = 40 Vimseltinib n = 83 Crossover n = 35 RECIST v1.1 ORR, n (%) (95% CI) 33 (40)a (29 to 51) 0 (0 to 9) 40 (48) (37 to 59) 19 (54) (37 to 71) Complete response 4 (5) 0 19 (23) 4 (11) Partial response 29 (35) 0 21 (25) 15 (43) DOR, median (range), months NR b (2.5+ to 30.9+) N/A NR (0.03+ to 30.9+) NR (0.03+ to 25.4+) TVSc ORR, n (%) (95% CI) 56 (67) a (56 to 77) 0 (0 to 9) 67 (81) (71 to 89) 25 (71) (54 to 85) Complete response 4 (5) 0 20 (24) 4 (11) Partial response 52 (63) 0 47 (57) 21 (60) DOR, median (range), months NR b (2.5+ to 33.1+) N/A NR (2.4+ to 33.1+) NR (1.9+ to 25.4+) Response assessed by IRR per RECIST v1.1 and TVS 2-Year Results: Vimseltinib Shows Robust and Durable Antitumor Efficacy. ORR by RECIST v1.1 • 48% (40/83) in the randomized vimseltinib group (where patients continued to receive vimseltinib in part 2) • 54% (19/35) in the crossover group (where patients randomized to placebo crossed over to vimseltinib in part 2) ORR by Tumor Volume Score (TVS) • 81% (67/83) in the randomized vimseltinib group (where patients continued to receive vimseltinib in part 2) • 71% (25/35) in the crossover group (where patients randomized to placebo crossed over to vimseltinib in part 2) The median DOR per RECIST v1.1 and TVS was still not reached for both groups after ≥2 years on study
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/46 MOTION Phase 3 Trial: Safety 24 Preferred term, n (%) Vimseltinib n = 83 Crossover n = 35 Vimseltinib total n = 118 All Grades Grade 3/4 All Grades Grade 3/4 All Grades Grade 3/4 Periorbital edemaa 40 (48) 4 (5) 17 (49) 1 (3) 57 (48) 5 (4) Pruritusa 31 (37) 3 (4) 11 (31) 2 (6) 42 (36) 5 (4) Face edemaa 28 (34) 1 (1) 9 (26) 0 37 (31) 1 (1) Arthralgia 27 (33) 0 9 (26) 0 36 (31) 0 Blood CPK increased 26 (31) 12 (14) 10 (29) 7 (20) 36 (31) 19 (16) Astheniaa 27 (33) 1 (1) 8 (23) 1 (3) 35 (30) 2 (2) Fatigue 30 (36) 1 (1) 5 (14) 0 35 (30) 1 (1) AST increased 23 (28) 1 (1) 11 (31) 0 34 (29) 1 (1) Headachea 25 (30) 1 (1) 9 (26) 1 (3) 34 (29) 2 (2) Rash 27 (33) 0 6 (17) 0 33 (28) 0 Hypertension 18 (22) 6 (7) 11 (31) 4 (11) 29 (25) 10 (8) Edema peripheral 21 (25) 0 8 (23) 0 29 (25) 0 Nausea 22 (27) 0 6 (17) 0 28 (24) 0 Rash maculopapulara 20 (24) 2 (2) 6 (17) 0 26 (22) 2 (2) Diarrhea 15 (18) 1 (1) 8 (23) 0 23 (19) 1 (1) ALT increased 13 (16) 0 8 (23) 0 21 (18) 0 COVID-19 16 (19) 1 (1) 3 (9) 0 19 (16) 1 (1) Generalized edema 15 (18) 1 (1) 4 (11) 0 19 (16) 1 (1) Data cutoff: February 22, 2025. aDenotes AEs without grade 4 criteria per Common Terminology Criteria for AEs version 5.0. AE, adverse event; ALT, alanine aminotransferase; AST, aspartate aminotransferase; COVID-19, coronavirus disease-2019; CPK, creatine phosphokinase; SAE, serious AE; TEAE, treatment-emergent AE. • Most TEAEs were grade 1/2, and grade 3/4 TEAEs were similar between randomized vimseltinib and crossover groups • There were no new TEAEs (preferred terms) in ≥15% of patients receiving vimseltinib and no new SAEs in >1 patient • There was no evidence of cholestatic hepatotoxicity or drug-induced liver injury • TEAEs led to dose interruption in 63% (74/118) and dose reduction in 58% (68/118) of patients, and 12% (14/118) of patients discontinued due to TEAEs • TEAEs that led to treatment discontinuation in >1 patient were periorbital edema (n = 3), pruritus (n = 3), and rash (n = 2)
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/46 Tenosynovial giant cell tumor(TGCT) Market Potential in the U.S. 25
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/46 U.S. TGCT: Potential Market and Growth Opportunities 1 Deciphera internal analysis of U.S. claims data; eligible patients defined as diagnosed, Rx-treated, and recently engaged with a medical oncologist (or a surgeon); claims data span 2012-2022; estimates shown are for 2022; prevalent estimate includes incident patients; estimates are inherently uncertain TGCT Patients: Approximately 15,000/year¹ (epidemiological estimate) Step 1 Step 2 Step 3 Seen by Oncologist TKI Treatment Seen by Oncologist Non TKI Treatment Seen by Surgeons Approximately 700 patients/year Approximately 700 patients /year Approximately 1,300 patients/year 26 ＊TKI: Tyrosine kinase inhibitors
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/46 Development Pipeline Progress Status 27
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/46 Status of regulatory filing for approval in Japan, US and Europe 28 FY2024 (results) FY2025 FY2026 BRAFTOVI 〔1L-BRAF-mutant Colorectal cancer〕 with Cetuximab and FOLFOX 2024/12 〔1L- Colorectal cancer (MSI-H)〕 with YERVOY CheckMate-8HW 2024/09⇒2025/08 〔1L-Hepatocellular carcinoma〕 with YERVOY CheckMate-9DW 2024/08⇒2025/06 As of October 30, 2025 Filed Approved Met PE OPDIVO Other than OPDIVO PE : Primary endpoint DCC-3014（ROMVIMZA） 〔TGCT〕 2024/08⇒2025/02 DCC-3014（ROMVIMZA） 〔TGCT〕 2024/07⇒2025/09 〔 Neoadjuvant, Adjuvant - NSCLC〕 with Chemo CheckMate-77T ONO-2017 〔 Partial-onset seizures 〕 2025/09 〔Adjuvant Hepatocellular carcinoma〕 CheckMate-9DX 〔Neoadjuvant, Adjuvant - Bladder cancer〕 with Chemo ONO-4538-86 ONO-4059（VELEXBRU） 〔2L-PCNSL〕
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/46 Development status of OPDIVO 29 Target disease Treatment Line Treatment Phase Japan Korea Taiwan US EU Non-small cell lung cancer Neo-adjuvant・Adjuvant with Chemo Ⅲ Ⅲ Ⅲ Approved Approved Colorectal cancer MSI-H／dMMR (1st) with Ipi Approved － － Approved Approved Hepatocellular carcinoma Adjuvant Monotherapy Ⅲ Ⅲ Ⅲ Ⅲ Ⅲ 1st with Ipi Approved Approved Approved Approved Approved Urothelial cancer / Bladder cancer Neo-adjuvant ・Adjuvant with Chemo Ⅲ Ⅲ Ⅲ Ⅲ Ⅲ Rhabdoid tumor 2nd Monotherapy Ⅱ － － － － Richter transformation 2nd Monotherapy Ⅱ － － － － Solid tumor － ONO-4538HSC (Comibination with vorhyaluronidase alfa) Ⅰ － － Approved Approved ※Red: Update after announcement of FY 2024 financial result in May 2025 ※Red: Update after Q1 FY2025 in August ・Approval or filed/awaiting approval in the past year ・Ongoing key clinical trials for approval As of October 30, 2025
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/46 30 Development pipeline (Oncology) ① Code (Generic name)MOA, Modality Target Indication PI PI/II PII PIII F A Status Area ID BRAFTOVI Capsule (Encorafenib) BRAF inhibitor BRAF-mutant thyroid cancer FY2024.12 Filing accepted JP, US, EU, KR, TW and others★ NCT04607421 QINLOCK (ripretinib) KIT inhibitor Gastrointestinal Stromal Tumor 2L KIT Exon 11+17/18 (GIST) FY2025 Primary Completion US, EU, KR, TW and others NCT05734105 ONO-4059 (tirabrutinib) BTK inhibitor Primary central nervous system lymphoma (PCNSL) ≥2L FY2027 Primary Completion US NCT07104032 Primary central nervous system lymphoma (PCNSL) 1L, ≥2L FY2025 Primary Completion (Part A) (Actual） US NCT04947319 ONO-4578 PG receptor (EP4) antagonist Gastric cancer＊ FY2025 Primary Completion (Actual） JP, KR, TW NCT06256328 Colorectal cancer＊ FY2027 Primary Completion JP, US, EU and others NCT06948448 Non-small cell lung cancer＊ FY2026 Primary Completion JP NCT06542731 Hormone receptor-positive, HER2-negative breast cancer FY2026 Primary Completion JP NCT06570031 ONO-0530（sapablursen） Antisense oligonucleotide targeting TMPRSS6 Polycythemia Vera FY2025 Primary Completion US, EU and others NCT05143957 ONO-4482 (relatlimab) Anti-LAG-3 antibody Melanoma＊ FY2024 Primary Completion (Actual） JP, US, EU and others NCT01968109 ONO-7427 Anti-CCR8 antibody Solid tumor＊ FY2025 Primary Completion JP, US, EU and others NCT04895709 DCC-3116 (inlexisertib) ULK inhibitor Advanced Malignancies (with ripretinib) FY2026 Primary Completion US NCT05957367 ※Red: Update after announcement of FY 2024 financial result in May 2025 ※Red: Update after Q1 FY2025 in August ＊： Combination with OPDIVO, ★ : Development rights countries: JP, KR Estimated study completion date shown in jRCT or ClinicaiTrials.gov F : Filed, A : Approval EU : European countries MOA : Mode of Action As of October 30, 2025
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/46 31 Development pipeline (Oncology) ② Code (Generic name)MOA, Modality Target Indication PI PI/II PII PIII F A Status Area ID DCC-3009 Pan-KIT inhibitor Gastrointestinal Stromal Tumor FY2028 Primary Completion US NCT06630234 ONO-7913 (magrolimab) Anti CD47 antibody Pancreatic cancer＊ FY2026 Primary Completion JP NCT06532344 Colorectal cancer＊ FY2027 Primary Completion JP NCT06540261 ONO-4685 PD-1 x CD3 bispecific antibody T-cell lymphoma FY2025 Primary Completion US NCT05079282 FY2028 Primary Completion JP NCT06547528 ONO-8250 iPSC-derived HER2 CAR T-cell therapy HER2-expressing Solid tumor FY2029 Primary Completion US NCT06241456 ONO-7428 Anti-ONCOKINE-1 antibody Solid tumor FY2029 Primary Completion JP NCT06816108 DCC-2812 GCN2 Activator Renal Cell Carcinoma, Urothelial Cancer, Castration-Resistant Prostate Cancer FY2028 Primary Completion US NCT06966024 ＊： Combination with OPDIVO Estimated study completion date shown in jRCT or ClinicaiTrials.gov As of October 30, 2025 ※Red: Update after announcement of FY 2024 financial result in May 2025 ※Red: Update after Q1 FY2025 in AugustF : Filed, A : ApprovalMOA : Mode of Action
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/46 32 Development pipeline (Non-oncology) ① Code (Generic name)MOA, Modality Target Indication PI PI/II PII PIII F A Status Area ID ROMVIMZA DCC-3014 (vimseltinib) CSF-1R inhibitor Tenosynovial Giant Cell Tumor FY2024 US: Approval FY2025 EU: Approval US, EU and others NCT05059262 chronic Graft Versus Host Disease FY2029 Primary Completion US NCT06619561 ONO-2017(cenobamate)Inhibition of voltage-gated sodium currents/positive allosteric modulator of GABAA ion channel Partial-onset seizures FY2025 JP : Filed JP, KR and others★1 NCT04557085 Primary generalized tonic-clonic seizures FY2026 Primary Completion JP NCT06579573 VELEXBRU Tablet (ONO-4059：tirabrutinib) BTK inhibitor Pemphigus FY2027 Primary Completion JP NCT06696716 ONO-8531（povetacicept） BAFF/APRIL dual antagonist IgA Nephropathy FY2028 Primary Completion JP, US, EU, KR, TW and others★2 NCT06564142 ONO-5532（Gel-One） Cross-linked hyaluronate Knee osteoarthritis FY2027 Completion JP jRCT2031240621 Hip osteoarthritis FY2027 Completion JP jRCT2061240110 ONO-2808 S1P5 receptor agonist Multiple System Atrophy FY2025 Primary Completion (Actual) JP, US NCT05923866 As of October 30, 2025 ※Red: Update after announcement of FY 2024 financial result in May 2025 ※Red: Update after Q1 FY2025 in August ★1 : Development rights country: JP, ★2 : Development rights countries: JP, KR Estimated study completion date shown in jRCT or ClinicaiTrials.gov F : Filed, A : Approval EU : European countries MOA : Mode of Action
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/46 33 Development pipeline (Non-oncology) ② Code (Generic name)MOA, Modality Target Indication PI PI/II PII PIII F A Status Area ID ONO-1110 Endocannabinoid regulation Postherpetic Neuralgia FY2026 Primary Completion JP NCT06708416 Fibromyalgia FY2026 Primary Completion JP NCT06752590 Hunner Type Interstitial Cystitis FY2026 Primary Completion JP NCT06752603 Major Depressive Disorder FY2026 Primary Completion JP NCT06792136 Social Anxiety Disorder FY2026 Primary Completion JP NCT06805565 ONO-2020 Epigenetic regulation Alzheimer’s Disease FY2026 Primary Completion JP, US NCT06881836 Agitation Associated with Dementia Due to Alzheimer's Disease FY2026 Primary Completion JP NCT06803823 ONO-4685 PD-1 x CD3 bispecific antibody Autoimmune disease FY2024 Completion (jRCT) JP jRCT2071220081 FY2024 Primary Completion(Actual) EU NCT05332704 ONO-4915 PD-1 x CD19 bispecific antibody Autoimmune disease FY2026 Completion (jRCT) JP jRCT2071240056 As of October 30, 2025 ※Red: Update after announcement of FY 2024 financial result in May 2025 ※Red: Update after Q1 FY2025 in August Estimated study completion date shown in jRCT or ClinicaiTrials.gov Shaded boxes indicate studies on healthy volunteers. F : Filed, A : Approval EU : European countries MOA : Mode of Action
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/4634 ONO-4578-08 study EP4 antagonist / Gastric Cancer, 1L
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/46 ONO-4578 overview Compound information Compound ONO-4578 Originator Ono Pharmaceutical Co., Ltd. Mechanism Prostaglandin receptor (EP4) antagonist Formulation Tablet Target indication Solid tumor Development status Phase II : gastric cancer 1L (JP, KR, TW) : colorectal cancer 1L (JP, US, EU, etc) Phase I : non-small cell lung cancer (JP) : hormone receptor-positive, HER2-negative breast cancer (JP) Mechanism of Action Prostaglandin E2 (PGE2) is a bioactive lipid produced by the cyclooxygenase (COX) pathway and exerts various actions via its receptors (EP1-EP4). COX-2 is overexpressed in solid tumor1). PGE2 has been reported to induce myeloid-derived suppressor cells (MDSC) and M2 macrophages in the tumor microenvironment through one of its receptors, EP4, and suppress activation of cytotoxic T cells 2). ONO-4578, a novel selective EP4 antagonist, is expected to have an antitumor effect by abolishing the tumor immunosuppressive mechanism that PGE2 constructs via EP4. 1) Bing L, et al. Cancer Cell Int; 2015:15:106 2) Yukinori T, et al. Front Immunol. 2020;11:324 Fig 2．Effect of ONO-4578 on intra-tumoral immune cells in syngeneic mouse colorectal cancer MC38 tumor-bearing model In syngeneic mouse tumor-bearing model, ONO-4578 improved immunosuppressive tumor microenvironment and showed antitumor effects (Figs. 1 and 2). Furthermore, ONO-4578 enhanced its antitumor effect by co-administration with anti-mouse PD-1 antibody (αPD- 1) (Fig. 1). AACR 2020: Poster #4443 35 Non-clinical data Fig 1．Time course of median tumor volume in syngeneic mouse colorectal cancer MC38 tumor-bearing model mMDSC M2 macrophage dendritic cells CD8-positive T cells
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/46 Phase I results 36 OPDIVO Non-PD in best response ONO-4578 + OPDIVO One prior therapy Study treatment PD progressive disease3rd line 4th line Efficacy in patients pre-treated with OPDIVO ESMO 2023: Poster #1546 Signature score was calculated as mean of log-transformed expression value BOR, best overall response; PD, progressive disease; PR, partial response; SD, stable disease Scr, Screening period; C2D15, Cycle2 Day15 (1 Cycle=4 Weeks) After administration, increases both in the M1/M2 macrophage ratio and in the T cell signature score were confirmed. Tumor shrinkage was observed in more than half of the patients who had previously achieved clinical benefit with OPDIVO and then worsened. ORR % of patients whose tumors shrank treated untreated treated untreated pre post pre post pre post pre post
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/46 ONO-4578-08 Study Design 37 Objective This clinical trial was conducted in patients with previously untreated, HER2-negative unresectable advanced or recurrent gastric cancer or gastroesophageal junction cancer. ONO-4578 in combination with nivolumab and fluoropyrimidine-based and platinum-based chemotherapy which is one of the standard treatments in this setting was compared with placebo in combination with nivolumab and chemotherapy. The primary endpoint of this trial is progression-free survival (PFS). Target population Previously untreated, HER2-negative unresectable advanced or recurrent gastric cancer (including gastroesophageal junction cancer) Study design A multicenter, double-blind, randomized controlled Phase 2 clinical trial Usage・ Dosage ONO-4578 group：ONO-4578 40mg QD／nivolumab 360mg Q3W／Chemotherapy (SOX*1 or CapeOX*2) Q3W Placebo group：Placebo QD／nivolumab 360mg Q3W／Chemotherapy (SOX*1 or CapeOX*2) Q3W Endpoint Primary endpoint：progression-free survival（PFS） Secondary endpoint：overall survival（OS）, objective response rate（ORR）, duration of response（DOR）, safety, etc Target enrollment 210 patients［Japan, South Korea and Taiwan］ • Untreated, unresectable advanced or recurrent gastric cancer (including gastroesophageal junction cancer) • HER2-negative • ECOG PS 0-1 • Neo-adjuvant or adjuvant chemotherapy allowed if completed ≧180 days prior to recurrence R 2:1 Stratification factors • PD-L1 CPS • PS • Peritoneal metastasis • ONO-4578 40mg QD PO • Nivolumab 360mg, Q3W • Chemotherapy, Q3W （SOX*1 or CapeOX*2） • Placebo QD PO • Nivolumab 360mg, Q3W • Chemotherapy, Q3W （ SOX*1 or CapeOX*2 ） *1SOX：S-1 40mg/m2 orally twice daily (days 1-14) and oxaliplatin 130mg/m2 IV (day1), Q3W *2CapeOX： capecitabine 1000mg/m2 orally twice daily (day1-14) and oxaliplatin 130mg/m2 IV (day1), Q3W N=210 N=140 N=70 Continue Until • Disease Progression • Unacceptable toxicity • Withdrawal consent Follow up / Outcome survey
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/46 Annual Number of Gastric Cancer Patients (Japan) 38 1) Globocan 2022: Stomach Cancer, Japan, World Health Organization Available at: https://gco.iarc.who.int/media/globocan/factsheets/populations/392-japan-fact-sheet.pdf 2) Ono internal estimates • Approximately 126,000 patients per year¹ Gastric Cancer Prevalence • Approximately 27,000 patients per year² • HER2-negative: Approximately 22,000 patients per year² Unresectable, Advanced, or Recurrent Gastric Cancer (Eligible for Chemotherapy) US : Estimated 11,000 patients (HER2-negative: 9,000) EU : Estimated 26,000 patients (HER2-negative: 22,000) anti-PD-1 antibody + chemotherapy (HER2-negative, Claudin-negative) : approx. 50% Reference²
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/4639 ONO-2808-03 study S1P5 Receptor Agonist MSA；Multiple System Atrophy
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/46 ONO-2808 and MSA Compound ONO-2808 Originator ONO Pharmaceutical Co., Ltd. Mechanism S1P5 receptor agonist Formulation Oral Target indication MSA (Multiple System Atrophy ) Development status Phase II (US, Japan) Multiple System Atrophy years0 3 6 9 Gregor K. Wenning, N Engl J Med 2015; 372(3) Movement disorder Autonomic dysfunction Aid-requiring walking Wheelchair mobility Bedridden Death Progressive neurodegenerative disease with cerebellar atrophy Average onset age: 55–60 years Severe and rapidly progressive Currently symptomatic treatment with limited efficacy Estimated patients US 1), 2): 15,000～50,000, Japan 3)：10,000 <Features> 40 ＊EU5：France, Germany, Italy, Spain, UK Ref) In-house material Blue：Nucleus Green：α-Syn Red：Neuron S1P5 agonist suppressed α-Syn accumulation in neuronal axons WT Vehicle S1P5 agonist α-Syn-expressing mice Primary whole-brain cultures from oligodendrocyte-specific human α-Syn-expressing mice Neuron Oligodendrocyte （ Myelin sheath ） Microglia AstrocyteNormal MSA ：Monomer α-Syn ：Oligomer α-Syn ：GCI* ：Cytokine Aberrant α-Syn α-Syn : α-Synuclein GCI : Glial intracytoplasmic inclusion body Compound information 1) National Institutes of Health: https://www.ninds.nih.gov/health-information/disorders/multiple-system-atrophy, 2) Kaplan S, et al. Parkinsonism Relat Disord. 2023;117:105920. 3) https://www.nanbyou.or.jp/entry/59
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/46 Outline of the global phase II ONO-2808-03 study 41 ONO-2808-03 study Objective To investigate the safety, tolerability, and pharmacokinetics of repeated-dose oral administration of ONO-2808 in patients with MSA. Study design Placebo-controlled, double-blind, randomized, parallel-group study Target criteria • Patients 30 to 80 years of age diagnosed with MSA • Patients defined as a maximum of 5 years since the onset of symptoms such as parkinsonism, ataxia, orthostatic hypotension and urinary dysfunction • Patients with an anticipated survival of at least 3 years Endpoint Primary endpoint • Safety, tolerability Secondary endpoint • PK、concentration in cerebrospinal fluid (CSF) Exploratory Endpoints • UMSARS (Historical Review, Motor Examination) • MSA-QoL、MoCA(Montreal Cognitive Assessment), COMPASS-31* • Brain volume(MRI：Brain stem, Cerebellum), White matter(Diffusion MRI) • Plasma BM(Neurofilament Light Chain, α-Syn, proteomics, etc.) Dosage Double-blind phase：Placebo, low dose, medium dose, high dose Extension phase：Maintain dose level(P group switches to low dose after completion of the transition period) Duration Double-blind phase：6 months Extension phase: 14months(Transition period for 2 months, extension phase for 12 months) Enrollment 20 patients per group, 80 in total Trial countries US, Japan COMPASS-31* : Autonomic Examination onset Image of efficacy More than ●● % reduction in the increase in UMSARS from baseline compared to the placebo group Progression 5years 9years
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/46 Trend of OPDIVO 42
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/46 Sales Trend of OPDIVO by Each Cancer Source: Estimation from external and internal data 43 FY2024 (Result) FY2025 (Forecast) ¥ 120.3 Bil ¥ 120.0 Bil GC ESC NSCLC RCC UC Others Preserve our competitiveness; Retain share No.1 new patient Renew growth: focus on key indications & recognition
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/46 The result of Clinical Trial - NSCLC 1L（PD-L1 negative）- Term OS Rate （％） (Month） CheckMate 227 Trial ― OPDIVO + YERVOY ― OPDIVO + Chemo ― Chemo therapy Term 22％ 20％ OS Rate 100 0 80 90 70 60 40 30 10 50 20 （％） （Month）8478726660544842363024181260 8％ 7％ CheckMate 9LA Trial ― OPTIVO + YERVOY + Chemo ― Chemo therapy OPDIVO + YERVOY + Chemo Five-year overall survival rates Six-year overall survival rates 22% 20% OPDIVO + YERVOY Five-year overall survival rates Six-year overall survival rates 19% 16% Carbone DP, et al. ESMO Open. 2025 Jun;10(6):105123 Suresh S. Ramalingam, et al. WCLC2023#OA14.03 44
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/46 5% 13% 17% 25% 31% 27% 30% 26%27% 25% 24% 20% 17% 16%17% 17% 16% 23% 18% 20% 17% 20% 30% 74% 73% 69% 62% 51% 46% 46% 47% 48% 56% 59% 58% 59% 59% 59% 60% 55% 59% 58% 57% 62% 66% 61% 7% 10% 7% 6% 10% 8% 10% 10% 10% 9% 7% 7% 9% 10% 7% 7% 9% 9% 6% 8% 6% 6% 4% 8% 3% 5% 6% 9% 8% 10% 7% 6% 10% 7% 7% 8% 19% 12% 11% 16% 15% 15% 16% 14% 16% 8% 10% 7% 8% 8% 8% 7% 9% 8% 7% 7% 5% 4% 6% 3% Nov 2020 Dec 2020 Feb 2021 May 2021 Aug 2021 Nov 2021 Feb 2022 May 2022 Aug 2022 Nov 2022 Mar 2023 May 2023 Jul 2023 Sep 2023 Nov 2023 Jan 2024 Mar 2024 May 2024 Jul 2024 Sep 2024 Nov 2024 Jan 2025 Apr 2025 Jul 2025 Mar 2026 Prescription Ratio in Patients Newly Treated※ for 1L NSCLC ※Patients starting 1L treatment within the last 1 month (Except Driver Mutation) Source: Primary research results （Nov 2020~Jul 2025: n=167~245) (Before approval) 45 OPDIVO Product B Product A Product C Others JCOG study discontinued 63% 17%
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/46 The Clinical Trial Result of HCC 1L 46 * Percentage of high-dose steroid use CheckMate 9DW Study OPDIVO＋YERVOY Control Group （molecular-targeted drug） OS 23.7 months 20.6 months PFS 9.1 months 9.2 months ORR 36% 13% DOR 30.4 months 12.9 months Three-years overall survival rates （follow-up data） 38% 24% Steroid 29%* - Treatment-related death 3.6% 0.9% Lancet. 2025 May 24;405(10492):1851-1864.
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Appendix
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OPDIVO Approval Track Record(1) Appendix -1 Target disease Treatment Line Treatment Phase Japan Korea Taiwan US EU Melanoma Adjuvant・1st・2nd Monotherapy, with Ipi (1st only) Approved Approved Approved Approved Approved 1st Combination drug† (relatlimab) － － － Approved Approved Non-small cell lung cancer Neo-adjuvant with Chemo Approved Approved Approved Approved Approved 1st with Ipi Approved Approved Approved Approved － with Ipi/Chemo Approved Approved Approved Approved Approved with Chemo Approved － － － － with Chemo (NSQ) Revision of labeling Approved Approved － － 2nd Monotherapy Approved Approved Approved Approved Approved Hodgkin’s lymphoma Relapsed /Refractory Monotherapy Approved Approved Approved Approved Approved Head and neck cancer 2nd Monotherapy Approved Approved Approved Approved Approved Malignant pleural mesothelioma 1st with Ipi Approved Approved Approved Approved Approved 2nd Monotherapy Approved － － － － Malignant mesothelioma (Excluding Pleura) 1st Monotherapy Approved － － － － As of October 30, 2025 †Combination drug (Relatlimab) : ONO-7121(Opdivo+Relatlimab (ONO-4482))
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OPDIVO Approval Track Record(2) Target disease Treatment Line Treatment Phase Japan Korea Taiwan US EU Gastric cancer 1st with Chemo Approved Approved Approved Approved Approved 3rd Monotherapy Approved Approved Approved － － Esophageal cancer Adjuvant Monotherapy Approved Approved Approved Approved Approved 1st with Ipi, with Chemo Approved Approved Approved Approved Approved 2nd Monotherapy Approved Approved Approved Approved Approved Colorectal cancer MSI-H／dMMR (3rd) Monotherapy Approved － Approved Approved － with Ipi Approved Approved Approved Approved Approved★ ★ Hepatocellular carcinoma 2nd with Ipi － － Approved Approved － As of October 30, 2025 ★★2nd Line Appendix -2
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OPDIVO Approval Track Record(3) Target disease Treatment Line Treatment Phase Japan Korea Taiwan US EU Renal cell carcinoma 1st with Ipi Approved Approved Approved Approved Approved with TKI Approved Approved Approved Approved Approved 2nd Monotherapy Approved Approved Approved Approved Approved Urothelial cancer / Bladder cancer Adjuvant Monotherapy Approved Approved Approved Approved Approved 1st with Chemo Approved Approved Approved Approved Approved 2nd Monotherapy － Approved Approved Approved Approved Cancerof unknownprimary 1st Monotherapy Approved － － － － Epithelialskin malignancies 1st Monotherapy Approved － － － － Flat dose 240 mg (every 2 weeks) Approved Approved Approved Approved Approved 360 mg (every 3 weeks) Approved Approved Approved Approved Approved 480 mg (every 4 weeks) Approved Approved Approved Approved Approved As of October 30, 2025 Appendix -3
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Key milestones in FY2025 Q1 (FY ending March 2026) (Development pipeline) Product/ Code(Generic name) Target indication/Study name Progress Product to be approved ROMVIMZA (vimseltinib) Tenosynovial Giant Cell Tumor (TGCT) Approved in EU (Sep.2025) OPDIVO Hepatocellular carcinoma(1st with Ipi) / CheckMate-9DW Approved in KR, TW (Jul.2025) MSI-high Colorectal cancer(1st with Ipi) / CheckMate-8HW Approved in JP (Aug.2025) Gastric cancer (1st with Ipi, chemo) / ONO-4538-113 Discontinued (Oct.2025) ONO-2017(cenobamate) Partial-onset seizures Filed in JP (Sep.2025) P3 ONO-4059(tirabrutinib) Primary central nervous system lymphoma(PCNSL) Started in US(Aug.2025) P2 ONO-4578 Gastric cancer (with OPDIVO) Met the primary endpoint in JP, KR, TW (Oct.2025) ONO-2808 Multiple System Atropy Safety and efficacy signals were observed in JP, US (Oct.2025) Events from July 2025 to October 30 Appendix -4 As of October 30, 2025
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Key milestones in FY2025 Q1 (FY ending March 2026) (Drug discovery partnerships & Research collaborations/Licensing & Co-promotion) Title Progress Ono Enters into a Basic Agreement with Seikagaku for Co-development and Marketing Collaboration on Gel-One for the treatment of Osteoarthritis in Japan Definitive Agreement (Aug.2025) P3 ongoing in JP ONO Enters into an Option and Collaboration Agreement with Numab to Develop Multi-specific Antibody NM49 (2024.2~) Discontinued Ono Expands Drug Discovery Collaboration with Neurimmune AG in the Field of Neurodegenerative Diseases (2022.1~) Discontinued Appendix -5 As of October 30, 2025 (Development pipeline) Product/ Code(Generic name) Target indication/Study name Progress P1/2 DCC-3116 （inlexisertib） Solid tumor (with sotorasib) Discontinued in US(Sep.2025) DCC-3084 Advanced Malignancies Discontinued in US(Sep.2025) P1 DCC-2812 Renal Cell Carcinoma, Urothelial Cancer, Castration-Resistant Prostate Cancer Started in US(Aug.2025) ONO-7475 （tamnorzatinib） EGFR-mutated non-small cell lung cancer Discontinued in JP(Jul.2025) Events from July 2025 to October 30
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38% 42% 43% 42% 43% 42% 45% 49% 52% 49% 48% 53% 54% 60% 34% 30% 25% 20% 21% 21% 25% 22% 17% 16% 19% 15% 14% 11% 26% 12% 12% 14% 10% 12% 10% 12% 8% 9% 4% 8% 9% 7% 31% 16% 19% 19% 23% 21% 20% 19% 23% 21% 24% 25% 24% 24% 6% 1% 2% 1% 2% 2% 1% 1% 2% 1% 3% 3% 1% 2%4% 2% 0% 2% 1% 1% 2% 0% 1% 1% 1% 1% 0% 2% Mar-May 2022 Jun-Aug 2022 Sep-Nov 2022 Dec-Feb 2022 Mar-May 2023 Jun-Aug 2023 Aug-Oct 2023 Nov-Jan 2023 Feb-Apr 2024 May-Jul 2024 Aug-Oct 2024 Nov-Jan 2024 Feb-Apr 2025 May-Jul 2025 Mar 2026 OPDIVO Product H Product K Product I Product J Others Prescription Ratio in Patients Newly Treated※ for 1L ESC(Squamous Cell Carcinoma) Source: Primary research results （May 2022~Jul 2025: n=150~155)※Patients starting treatment within the last 3 month (Before approval) Appendix -6