ONO-4059

Ono Pharmaceutical Co. Ltd · Phase 3 active Small molecule

ONO-4059 is a PI3K inhibitor Small molecule drug developed by Ono Pharmaceutical Co. Ltd. It is currently in Phase 3 development for Relapsed or refractory lymphoma, Other hematologic malignancies. Also known as: Tirabrutinib hydrochloride, Tirabrutinib Hydrochloride.

ONO-4059 is a small-molecule inhibitor of phosphoinositide 3-kinase (PI3K) that blocks PI3K signaling to suppress tumor cell proliferation and survival.

ONO-4059 is a small-molecule inhibitor of phosphoinositide 3-kinase (PI3K) that blocks PI3K signaling to suppress tumor cell proliferation and survival. Used for Relapsed or refractory lymphoma, Other hematologic malignancies.

Likelihood of approval

61.3% vs 58.3% industry baseline

If approved by FDA: likely 2028–2030

Steps remaining: NDA/BLA submission

Confidence: High

Why this estimate

• Baseline phase 3 → approval rate +58.3pp
  Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
• Oncology Phase 3 boost +3.0pp
  Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.

Predicted approval windows by jurisdiction (conditional on FDA approval)

Regulator	Country	Likely year	Lag vs FDA
FDA	US	2028–2030	—
EMA	EU	2029–2031	+0.7 yr
MHRA	GB	2029–2031	+0.7 yr
Health Canada	CA	2029–2032	+0.9 yr
TGA	AU	2029–2032	+1.2 yr
PMDA	JP	2029–2032	+1.5 yr
NMPA	CN	2030–2033	+2.3 yr
MFDS	KR	2029–2032	+1.4 yr
CDSCO	IN	2029–2033	+1.8 yr
ANVISA	BR	2030–2033	+2.3 yr

Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).

Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.

At a glance

Generic name	ONO-4059
Also known as	Tirabrutinib hydrochloride, Tirabrutinib Hydrochloride
Sponsor	Ono Pharmaceutical Co. Ltd
Drug class	PI3K inhibitor
Target	PI3K (phosphoinositide 3-kinase)
Modality	Small molecule
Therapeutic area	Oncology
Phase	Phase 3

Mechanism of action

PI3K is a key enzyme in cell survival and proliferation pathways frequently dysregulated in cancer. By inhibiting PI3K, ONO-4059 reduces downstream signaling through AKT and mTOR, leading to reduced cell survival and growth. This mechanism is particularly relevant in hematologic malignancies and solid tumors with PI3K pathway activation.

Approved indications

• Relapsed or refractory lymphoma
• Other hematologic malignancies

Common side effects

• Diarrhea
• Neutropenia
• Thrombocytopenia
• Anemia
• Hyperglycemia

Key clinical trials

• IGNITE: Study of Tirabrutinib vs Rituximab/Temozolomide for Relapsed/Refractory Primary Central Nervous System Lymphoma (PCNSL) (PHASE3)
• A Study of Oral LOXO-305 in Patients With Previously Treated CLL/SLL or NHL (PHASE1, PHASE2)
• ONO-4059 Study in Patients With Steroid-resistant Pemphigus (PHASE3)
• Study of Tirabrutinib (ONO-4059) in Patients With Primary Central Nervous System Lymphoma (PROSPECT Study) (PHASE2)
• A Study of Nemtabrutinib (MK-1026) in Participants With Relapsed or Refractory Hematologic Malignancies (ARQ 531-101/MK-1026-001) (PHASE1, PHASE2)
• Dose Escalation and Dose Expansion Study of Tirabrutinib in Combination With Other Targeted Anti-cancer Therapies in Adults With B-cell Malignancies (PHASE1)
• Phase I Study Evaluating Tolerability, Safety, Pharmacokinetics, and Efficacy of Combined ONO-4059 and R-MPV Therapy for PCNSL (PHASE1)
• Study to Assess the Long-term Safety and Efficacy of Tirabrutinib in Adults With Relapsed/Refractory B-cell Malignancies (PHASE1)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• ONO-4059 CI brief — competitive landscape report
• ONO-4059 updates RSS · CI watch RSS
• Ono Pharmaceutical Co. Ltd portfolio CI

Frequently asked questions about ONO-4059

What is ONO-4059?

ONO-4059 is a PI3K inhibitor drug developed by Ono Pharmaceutical Co. Ltd, indicated for Relapsed or refractory lymphoma, Other hematologic malignancies.

How does ONO-4059 work?

ONO-4059 is a small-molecule inhibitor of phosphoinositide 3-kinase (PI3K) that blocks PI3K signaling to suppress tumor cell proliferation and survival.

What is ONO-4059 used for?

ONO-4059 is indicated for Relapsed or refractory lymphoma, Other hematologic malignancies.

Who makes ONO-4059?

ONO-4059 is developed by Ono Pharmaceutical Co. Ltd (see full Ono Pharmaceutical Co. Ltd pipeline at /company/ono-pharmaceutical-co-ltd).

Is ONO-4059 also known as anything else?

ONO-4059 is also known as Tirabrutinib hydrochloride, Tirabrutinib Hydrochloride.

What drug class is ONO-4059 in?

ONO-4059 belongs to the PI3K inhibitor class. See all PI3K inhibitor drugs at /class/pi3k-inhibitor.

What development phase is ONO-4059 in?

ONO-4059 is in Phase 3.

What are the side effects of ONO-4059?

Common side effects of ONO-4059 include Diarrhea, Neutropenia, Thrombocytopenia, Anemia, Hyperglycemia.

What does ONO-4059 target?

ONO-4059 targets PI3K (phosphoinositide 3-kinase) and is a PI3K inhibitor.

Related

• Drug class: All PI3K inhibitor drugs
• Target: All drugs targeting PI3K (phosphoinositide 3-kinase)
• Manufacturer: Ono Pharmaceutical Co. Ltd — full pipeline
• Therapeutic area: All drugs in Oncology
• Indication: Drugs for Relapsed or refractory lymphoma
• Indication: Drugs for Other hematologic malignancies
• Also known as: Tirabrutinib hydrochloride, Tirabrutinib Hydrochloride