Ono Pharmaceutical Co., Ltd. · TSE:4528
Ono Pharmaceutical — Investor Day (June 2025)
15 slides from Ono Pharmaceutical's investor day presentation, Jun 4, 2025
Slide 1: R&D Day – PROSPECT Study Data Presentation
R&D Day – PROSPECT Study Data Presentation – June 4, 2025
Slide 2: Agenda
Agenda PROSPECT Study（10:30-10:45） Vice President, Medical Affairs, ONO PHARMA USA Thomas Lechner, MSc. Ph.D. Closing（10:45-10:55） Corporate Officer / Executive Director, Clinical Development Tatsuya Okamoto Q&A Session（10:55-11:15） 2 / 14
Slide 3: Cautionary Notes
Cautionary Notes 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
Slide 4: Tirabrutinib for relapsed or refractory PCNSL: efficacy and safety from the phase II PROSPECT study
Tirabrutinib for the treatment of relapsed or refractory primary central nervous system lymphoma: efficacy and safety from the phase II PROSPECT study
Slide 5: Key Takeaway Points
PROSPECT: Key Takeaway Points The PROSPECT study was a phase II, open-label, multicenter, US-based study of tirabrutinib in patients with r/r PCNSL The first efficacy and safety findings from the PROSPECT study support tirabrutinib monotherapy as a potentially effective treatment option for patients
Slide 6: Background
PROSPECT: Background • Primary central nervous system lymphoma (PCNSL) is a rare, aggressive form of non-Hodgkin lymphoma localized to the central nervous system1,2 • In the relapsed/refractory setting, treatment options are limited, standard of care is not well established, and prognosis is poor1,2
Slide 7: Study Design and Methods
PROSPECT: Study Design and Methods Eligibility • Age ≥18 years Endpoints • ECOG PS 0-2 Relapsed/refractory PCNSL Primary • Measurable brain lesion with EOTb • ORR per IPCG criteria, a minimum diameter >1.0 cm Tirabrutinib assessed by IRC Follow-up 480 mg QDa Secondary • Disease r/r status • At least
Slide 8: Patient Disposition and Characteristics
PROSPECT: Patient Disposition and Characteristics Characteristic Tirabrutinib (N=48) Patients screened Age, median years (range) 65.5 (34-87) for eligibility (N=66) Sex, male, n (%) 21 (44) Excluded (n=18) Did not meet eligibility ECOG PS, n (%) criteria (n=13) 0 9 (19) Patient withdrawal (n=3) 1 30
Slide 9: Overall Response Rate and Duration of Response
PROSPECT: Overall Response Rate and Duration of Response Primary Endpoint: ORR by IRCa Duration of Response by IRC ORR by IRC 1.0 n (%) 95% CI 0.8 Probability of Patients ORR (CR+CRu+PR) 32 (67) 52, 80 Continuing Response CRR (CR+CRu) 21 (44) 29, 59 0.6 CR 13 (27) 15, 42 0.4 CRu 8 (17) 7, 30 PR 0.2
Slide 10: Progression-Free Survival and Overall Survival
PROSPECT: Progression-Free Survival and Overall Survival Progression-Free Survival by IRC Overall Survival 1.0 1.0 Progression-Free Survival 0.8 Probability of Survival 0.8 Probability of 0.6 0.6 0.4 0.4 0.2 0.2 Tirabrutinib Tirabrutinib 0.0 0.0 0 3 6 9 12 15 18 21 24 27 30 0 3 6 9 12 15 18 21 24 27
Slide 11: Adverse Events
PROSPECT: Adverse Events Tirabrutinib (N=48) TEAEs in ≥15% of Patients Any grade, Grade ≥3, Fall TEAEs n (%) n (%) Fatigue Anemia Patients with ≥1 TEAE 47 (98) 27 (56) Lymphopenia Headache Diarrhea Patients with ≥1 treatment-related TEAE 36 (75) 13 (27) Pruritus Rash Patients with TEAEs leading to d
Slide 12: Conclusions
PROSPECT: Conclusions • PROSPECT was a phase II, open-label, multicenter, US-based study of tirabrutinib in patients with relapsed or refractory PCNSL • Tirabrutinib demonstrated a high ORR, prolonged DOR, and reasonable PFS with a well- tolerated side effect profile • Expanding on experience in Jap
Slide 13: Acknowledgments
Acknowledgments We thank the patients and their families for making the PROSPECT study possible We also thank the investigators and clinical trial teams who participated in the study This study was funded by Ono Pharmaceutical Co. Ltd 13 / 14
Slide 14: Lay Summary
PROSPECT: Lay Summary • Primary central nervous system lymphoma (PCNSL) is a rare tumor that occurs in the brain, spinal cord, and other parts of the central nervous system • This kind of cancer can be treated with chemotherapy, but the cancer commonly comes back • The PROSPECT study tested tirabrut
Slide 15: ONO PHARMA
ono ONO PHARMA