Ono Pharmaceutical Co., Ltd. · TSE:4528
Ono Pharmaceutical — Investor Day (May 2025)
39 slides from Ono Pharmaceutical's investor day presentation, May 20, 2025
Slide 1: R&D Day – ROMVIMZA –
R&D Day – ROMVIMZA – May 20, 2025
Slide 2: Agenda
Agenda Opening（9:00-9:10） 代表取締役 社長 COO Representative Director, President and Chief Operating Officer 滝野 十一 Toichi Takino ROMVIMZA（9:10-10:05） Matthew L. Sherman, M.D. Margarida Duarte Michelle DiNapoli Executive Vice President, Executive Vice President, Senior Vice President, Chief Medical Officer,
Slide 3: 注意事項
注意事項 この資料に記載されている業績見通し等の将来に関する記述は、当社が現在入手している情報及び合 理的であると判断する一定の前提に基づいており、実際の業績等は様々な要因により、大きく異なる 可能性があります。 以下に、事業展開上のリスク要因となる可能性があると考えられる主な事項を挙げますが、これらに 限定されるものではありません。 ( ⅰ ) 新製品開発の失敗 ( ⅱ ) 医療保険制度の改革による事業環境の変化 ( ⅲ ) 競合品や後発品の影響により、期待した成果を得られない可能性 ( ⅳ ) 第三者による知的財産の侵害等 ( ⅴ ) 自然災害や火災などで、生産の停滞･遅延発生による製品供
Slide 4: Deciphera社の業績動向
Deciphera社の業績動向 • 2024年6月に買収完了し2024年7月よりP/L連結開始 • 上市済の医薬品「QINLOCK」の販売は堅調に進捗しており、2025年3月期売上は255億円であった。 2026年3月期売上は340億円を見込む。 • 2025年2月には腱滑膜巨細胞腫治療薬「ROMVIMZA」の米国での販売を開始。 2025年7月を目途にOno Pharma USとDecipheraの機能を統合。2027年度の単年単体黒字化を見込む。 2025年3月期 製品売上：261億円（計画比102.4%） 実績 費用：423億円 (2024.7-2025.3) -研究開発費 242億円、
Slide 5: 開発パイプライン（オプジーボを除く）
開発パイプライン（オプジーボを除く） 2025年4月24日現在 Phase 1 Phase 1/2 Phase 2 ピボタル試験 申請・承認 ONO-7475 DCC-3116 ONO-4578 QINLOCK® 膵がん、EGFR変異陽性NSCLC／ QINLOCK® 固形がん,悪性腫瘍／ P1/P2 胃がん、結腸・直腸がん ／P2 GIST2L KIT Exon 11+17/18／P3 GIST4L／40か国以上で承認 P1 ONO-8250 ONO-7427 TM HER2陽性固形がん／P1 固形がん／ P1/P2 ROMVIMZA TGCT／米国上市 ONO-7428 ONO-4482
Slide 6: Decipheraとのシナジー効果
Decipheraとのシナジー効果 2025年7月米欧の開発・販売事業をDecipheraに統合して、 グローバル展開の販売を推進・加速する Decipheraを中心に グローバル展開 2025年7月米欧の開発・販売 事業をDecipheraに統合 米国小野薬品 英国小野薬品 （OPUS） (OPUK) 米国：Tirabrutinibの開発 ケンブリッジ ロンドン グローバル：Sapablursenの開発（P3~） • 青塗はDeciphera社の拠点施設国 （米国、スイス、ドイツ、フランス、イタリア、スペイン、オランダ） • 国旗はQINLOCKの上市国 （EU4 ドイツ、フランス、イタリア
Slide 7: 今後10年の成長イメージ
今後10年の成長イメージ + グローバル製品（QINLOCK、ROMVIMZA、VELEXBRU、Sapablursen）の売上拡大 + オプジーボの皮下注製剤及び合剤のロイヤルティは静注製剤の特許満了後も継続 + ONO-2017、Gel-Oneの国内上市 + 自社創製品の上市 － 2025-2026年にかけて、糖尿病関連製品（フォシーガ、グラクティブ）の特許満了 － オプジーボの特許満了（2028年米国、2030年欧州、2031年日本） 開発早期品の上市＆ 更なるライセンス品の獲得 グローバル売上 ロイヤルティ 国内売上（OPD以外） 国内売上（OPD） 2025 2030 2035 イメ
Slide 8: High Unmet Need and MOTION Results
HIGH UNMET NEED AND MOTION RESULTS Matthew L. Sherman, M.D.
Slide 9: Tenosynovial Giant Cell Tumor: A Locally Aggressive Tumor Associated with Substantial Morbidity
Tenosynovial Giant Cell Tumor: A Locally Aggressive Tumor Associated with Substantial Morbidity DIAGNOSIS AND PATIENT BURDEN UNMET NEED • Long path to diagnosis • Existing Product: Black box warning and Risk Evaluation and Mitigation • High disease burden Strategy (REMS) program due to • Severe pain
Slide 10: Significant Unmet Medical Need
ROMVIMZA | TENOSYNOVIAL GIANT CELL TUMOR Significant Unmet Medical Need TGCT is a locally aggressive tumor with “ • substantial morbidity including severe pain, limited function, swelling, and stiffness The impact is huge. You get really limited by the • Many patients are not amenable to surgery tum
Slide 11: A Multicenter, Randomized, Placebo-Controlled, Double-Blind Study
ROMVIMZA | PHASE 3 MOTION STUDY OF PATIENTS WITH TGCT A Multicenter, Randomized, Placebo-Controlled, Double-Blind Study Phase 3 MOTION Study DOUBLE-BLIND PERIOD OPEN-LABEL PERIOD Assessed the efficacy and safety of vimseltinib for the treatment of patients with TGCT not amenable Part 1: Eligible stu
Slide 12: Study Met Primary and All Six Secondary Endpoints
ROMVIMZA | PHASE 3 MOTION STUDY OF PATIENTS WITH TGCT Study Met Primary and All Six Secondary Endpoints Primary Endpoint Key Secondary Endpoint ORR at Week 25 by RECIST v1.1 ORR at Week 25 by Tumor Volume Score (TVS) 50% 80% 40% 70% 67% 40% Response Rate by RECIST (%) 60% Response Rate by TVS (%) OR
Slide 13: Key Secondary Endpoint: Active Range of Motion
ROMVIMZA | PHASE 3 MOTION STUDY OF PATIENTS WITH TGCT Key Secondary Endpoint: Active Range of Motion Improvement in Active Range of Motion (ROM) 25% 20% 18.4% Improvement in ROM at Week 25 (%) 15% ~5X Improvement difference of 14.6% IMPROVEMENT IN between vimseltinib and placebo ROM (95% CI: 4.0%, 2
Slide 14: ROMVIMZA Provided Statistically Significant and Clinically Meaningful Improvements Versus Placebo
ROMVIMZA Provided Statistically Significant and Clinically Meaningful Improvements Versus Placebo Vimseltinib Placebo Statistically Clinically At week 25 P-value n = 83 n = 40 significant meaningful Active Range of Motion % Mean change from baseline (SE) 18.4 (6.5) 3.8 (7.2) % Difference (95% CI
Slide 15: ROMVIMZA Provided Statistically Significant and Clinically Meaningful Improvements Versus Placebo
ROMVIMZA Provided Statistically Significant and Clinically Meaningful Improvements Versus Placebo Active Range of Motion Physical Function†1 Health Status‡ 8 20 30 Vimseltinib Vimseltinib Vimseltinib * ** Improvement Placebo Improvement * * Improvement Placebo 6 *** ** ** 15 Placebo ** * LS mean (SE
Slide 16: ROMVIMZA Was Generally Well Tolerated with Few Discontinuations
ROMVIMZA Was Generally Well Tolerated with Few 16 Discontinuations Due to Treatment Emergent Adverse Events TEAEs in ≥15% of Vimseltinib Placebo participants in either n = 83 n = 39a treatment arm • Most TEAEs were Grade 1/2 Preferred term, n (%) All grades Grade 3/4 All grades Grade 3/4 Periorbital
Slide 17: MOTION Primary Results Demonstrated the Clinical and Functional Benefits of ROMVIMZA
MOTION Primary Results Demonstrated the Clinical and Functional Benefits of ROMVIMZA in Participants with TGCT PIVOTAL PHASE 3 MOTION STUDY Met its primary and all key secondary endpoints and demonstrated a well-tolerated safety profile Primary Endpoint ORR at Week 25 • 40% for ROMVIMZA vs. 0% for p
Slide 18: New Growth Opportunity in Chronic Graft vs. Host Disease
ROMVIMZA | CGVHD New Growth Opportunity in Chronic Graft vs. Host Disease • As an oral agent, ROMVIMZA may offer best- in-class CSF1R option as single agent or in combination with other oral cGVHD therapies • Chronic GVHD affects 30-50% of allogeneic hematopoietic cell transplant recipients (14,000
Slide 19: TGCT Market Overview & Opportunity
TGCT MARKET OVERVIEW & OPPORTUNITY Margarida Duarte
Slide 20: Deciphera Is at a Major Inflection Point
Deciphera Is at a Major Inflection Point Becoming a multiproduct company QINLOCK approved in >40 countries for GIST and ROMVIMZA recently approved in the US for TGCT High unmet need in TCGT Locally aggressive tumor with substantial morbidity and an unaddressed market ROMVIMZA efficacy and safety pro
Slide 21: Building on Our Success with QINLOCK
Building on Our Success with QINLOCK Complementary Commercial Opportunities • Established relationships with physicians who treat GIST and Pipeline TGCT • 70%-80% overlap in US Future prescribing physicians for GIST and TGCT • MOTION study deployed in major 2025+ sarcoma sites across the globe 2020+
Slide 22: Significant Opportunity to Benefit Patients with TGCT
Significant Opportunity to Benefit Patients with TGCT U.S. Opportunity E.U. Opportunity 1,400 Incident Patients1 9,000 1,300 Comparable incidence $700MM+ U.S. TAM2 Prevalent Incident and recurrence rates in Patients1 Patients1 Europe3 Seen by Oncologists Seen by Surgeons No approved therapies No
Slide 23: Untapped Opportunity – Market Expansion Expected
Untapped Opportunity – Market Expansion Expected New product launches alongside increased disease awareness and referrals to oncologists Large and untapped opportunity will expand the TGCT TKI market Patients Prevalent patients lost to follow up receiving a TKI Patients not reaching an oncologist Ma
Slide 24: US Launch
US LAUNCH Michelle DiNapoli
Slide 25: Just Launched in the US
Just Launched in the US Launched February 14, 2025 INDICATIONS AND USAGE ROMVIMZA is indicated for treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. 25 / 40
Slide 26: ROMVIMZA Added to the NCCN Guidelines as Preferred Regimen
ROMVIMZA Added to the NCCN Guidelines as Preferred Regimen Category 1 Preferred Rating Regimen 27 / 40
Slide 27: Two FDA-Approved Treatments for TGCT in the US
Two FDA-Approved Treatments for TGCT in the US: ROMVIMZA and Existing Product ROMVIMZA Existing Product Indication Indicated for treatment of adult patients with Indicated for the treatment of adult patients with symptomatic tenosynovial symptomatic tenosynovial giant cell tumor giant cell tumor (TG
Slide 28: Driving Uptake Through Focus on Oncologists and Patients
At Launch, Deciphera Will Drive Uptake of ROMVIMZA Through Focus on Two Critical Stakeholders: Oncologists and Patients Oncologists -------------------------- TGCT Patients -------------------------- Objective: Drive awareness, Objective: Engage, empower, differentiation and uptake with medical & ac
Slide 29: Driving Awareness of ROMVIMZA Approval With Oncologists
Deciphera Is Swiftly Driving Awareness Of ROMVIMZA Approval With Oncologists POSITIVE FEEDBACK FROM HCPS1 Reach to Sarcoma Centers of Excellence A better drug than pexidartinib, especially in terms of toxicity. Third Party Educational Webinars – Medical Oncologist Increased website traffic Sid
Slide 30: Driving Awareness of ROMVIMZA and TGCT Education with Patients
Likewise, Deciphera is Driving Awareness of ROMVIMZA and TGCT Education with Patients Patient Media Highlights EXCITEMENT FROM • Strong Patient Interest PATIENT COMMUNITY >11K visits to ROMVIMZA.com • Intent to Request ROMVIMZA from HCP Patients are making appointments to ask about ROMVIMZA! Doctor
Slide 31: ROMVIMZA Has Demonstrated Positive Market Receptivity
ROMVIMZA Has Demonstrated Positive Market Receptivity Positive Product Impressions Broad Prescriber Base Fast Patient Access • Strong positive reaction to no • ROMVIMZA Rx coming from • Positive Feedback on product REMS and no black box warning Sarcoma Centers of Excellence, profile from Payers • Mi
Slide 32: ROMVIMZA Usage Across Various Patient Profiles
ROMVIMZA Usage Across Various Patient Profiles Aligns with Oncologists’ Positive Perceptions Existing Product Patients switching Patients Pre- and TKI naive patients2 Failures due from other TKIs, Post- Surgery2 to Toxicity2 including imatinib2 ~90% of oncologists >80% of oncologists >80% of oncolog
Slide 33: Significant Growth Opportunities in TGCT and cGVHD
Significant Growth Opportunities in TGCT and cGVHD ROMVIMZA just approved in the US with encouraging early launch indicators Significant commercial opportunity in TGCT that is highly synergistic with GIST Potential for market expansion with increased awareness and referrals Under review by the Europ
Slide 34: Early-Stage Pipeline
Early-Stage Pipeline Matthew L. Sherman, M.D.
Slide 35: Solving Limitations of Classical Kinase Inhibitors
Solving Limitations of Classical Kinase Inhibitors We take advantage of variation in the switch-control amino acid environment to design superior, drug-like molecules Higher kinase Increased potency specificity • Insensitive to • Enhanced kinome cellular [ATP] selectivity • Extended • Stabilize inac
Slide 36: Switch Control Platform Has Delivered Multiple Approved Agents and Clinical Stage Compounds
Switch Control Platform has Delivered Multiple Approved Agents and Clinical Stage Compounds QINLOCK VIMSELTINIB DCC-3116 DCC-3084 DCC-3009 DCC-2812 KIT Inhibitor CSF1R Inhibitor ULK Inhibitor Pan-RAF Inhibitor Pan-KIT Inhibitor GCN2 Activator Notes: CSF1R=colony-stimulating factor 1 receptor; GCN2=g
Slide 37: Driving Innovation Through Our Proven Discovery Engine
Driving Innovation Through Our Proven Discovery Engine Fueled by our proprietary drug discovery platform, we intend to advance multiple drug candidates to treat cancer DCC-3116 (ULK) DCC-3084 (RAF) DCC-3009 (KIT) DCC-2812 (GCN2) • Potential first-in-class • Potential best-in-class • Potential best-i
Slide 38: Deciphera Clinical Development – Completed, Ongoing and Planned Studies
Deciphera Clinical Development – Completed, Ongoing and Planned Studies 6 Active Development Programs Ripretinib Vimseltinib >500 DCC-3116, DCC-3084, DCC-3009 >3,000 Global Sites DCC-2812 (pre-IND) Participants >25 Countries US, Canada, Europe, SE Asia, Australia, South America >25 Clinical Protocol
Slide 39: ONO PHARMA
ono ONO PHARMA