This page provides the latest information on Kyowa Kirin’s pharmaceutical development pipeline. Regarding clinical trial information for each pharmaceutical product, we are promoting information disclosure through public clinical trial registries on a separate page.
R&D pipeline
Modalities
Indication
-
ziftomenib※
Frontline Acute Myeloid Leukemia (AML)
(Combination)
- Formulation:
- Oral
- Mechanism of Action:
- Menin Inhibitor
- In-House or Licensed:
- Kura Oncology
- Remarks:
- Clinical study is being conducted as a global product
NPM1-mutant AML/KMT2A-rearranged AML
Combinations with venetoclax + azacitidine, and cytarabine + daunorubicin
KOMET-017
KHK4083/AMG 451
rocatinlimab
Moderate to Severe Atopic Dermatitis
- Formulation:
- Injection
- Mechanism of Action:
- Anti-OX40 Antibody
- In-House or Licensed:
- In-House
- Remarks:
- POTELLIGENT
Human monoclonal antibody production technology
Collaboration agreement with Amgen for the development of KHK4083/AMG 451
in all the countries except for Japan.
Clinical study is being conducted in JP, NA, EU, UK, Middle East, Asia, Oceania,
and other regions as a global product. - Clinical Trial Information:
- NCT05398445,NCT05651711,NCT05724199,NCT05704738,
NCT05882877,NCT05899816,NCT05633355,NCT06224192
KHK4083/AMG 451
rocatinlimab
Prurigo Nodularis
- Formulation:
- Injection
- Mechanism of Action:
- Anti-OX40 Antibody
- In-House or Licensed:
- In-House
- Remarks:
- POTELLIGENT
Human monoclonal antibody production technology
Collaboration agreement with Amgen for the development of KHK4083/AMG 451
in all the countries except for Japan.
Clinical study is being conducted in JP, NA, EU, Asia, and Oceania as a global product. - Clinical Trial Information:
- NCT06527404
OTL-203
-
MPS-IH (Hurler Syndrome)
- Mechanism of Action:
- Hematopoietic Stem Cell (HSC) Gene Therapy
- In-House or Licensed:
- In-House
- Remarks:
- Rare Pediatric Disease (RPD) and Fast Track designations (FDA)
Priority Medicines (PRIME) designation (EMA)
Area of clinical study: NA and EU
-
ziftomenib※
R/R Acute Myeloid Leukemia (AML)
(Monotherapy)
- Formulation:
- Oral
- Mechanism of Action:
- Menin Inhibitor
- In-House or Licensed:
- Kura Oncology
- Remarks:
- The detailed results presented at ASCO in June 2025 and published on JCO in September 2025
Adult Relapsed or Refractory AML with a NPM1 Mutation
KOMET-001
KHK4083/AMG 451
rocatinlimab
Moderate to Severe Asthma
- Formulation:
- Injection
- Mechanism of Action:
- Anti-OX40 Antibody
- In-House or Licensed:
- In-House
- Remarks:
- POTELLIGENT
Human monoclonal antibody production technology
Collaboration agreement with Amgen for the development of rocatinlimab
in all the countries except for Japan.
Clinical study is being conducted in JP, NA, EU, Asia, and Oceania as a global product. - Clinical Trial Information:
- NCT06376045
KHK4951
tivozanib
Diabetic Macular Edema
- Formulation:
- Ophthalmic
- Mechanism of Action:
- VEGF Receptor Tyrosine Kinase Inhibitor
- In-House or Licensed:
- In-House
- Remarks:
- Clinical study is being conducted in JP, NA, Asia, and Oceania as a global product.
- Clinical Trial Information:
- NCT06116916
KHK4951
tivozanib
Neovascular Age-Related Macular Degeneration
- Formulation:
- Ophthalmic
- Mechanism of Action:
- VEGF Receptor Tyrosine Kinase Inhibitor
- In-House or Licensed:
- In-House
- Remarks:
- Clinical study is being conducted in JP, NA, Asia, and Oceania as a global product.
- Clinical Trial Information:
- NCT06116890
OTL-201
-
MPS-IIIA (Sanfilippo Syndrome type A)
- Mechanism of Action:
- Hematopoietic Stem Cell (HSC) Gene Therapy
- In-House or Licensed:
- In-House
- Remarks:
- Rare Pediatric Disease (RPD) designation (FDA)
Preparation underway for registrational study (equivalent to PhⅢ study)
-
ziftomenib※
R/R Acute Lymphoblastic Leukemia (ALL)
(Monotherapy)
- Formulation:
- Oral
- Mechanism of Action:
- Menin Inhibitor
- In-House or Licensed:
- Kura Oncology
- Remarks:
- Clinical study is being conducted in NA and EU as a global product.
KMT2A-rearranged ALL
KOMET-001
-
ziftomenib※
R/R Acute Myeloid Leukemia (AML)
(Monotherapy)
- Formulation:
- Oral
- Mechanism of Action:
- Menin Inhibitor
- In-House or Licensed:
- Kura Oncology
- Remarks:
- Clinical study is being conducted in NA and EU as a global product.
Non-NPM1-mutant AML/Non-KMT2A-rearranged AML
KOMET-001
-
ziftomenib※
Frontline Acute Myeloid Leukemia (AML)
(Combination)
- Formulation:
- Oral
- Mechanism of Action:
- Menin Inhibitor
- In-House or Licensed:
- Kura Oncology
- Remarks:
- Clinical study is being conducted in NA as a global product.
NPM1-mutant AML/KMT2A-rearranged AML
Combinations with venetoclax + azacitidine, and cytarabine + daunorubicin
KOMET-007
-
ziftomenib※
Frontline Acute Myeloid Leukemia (AML)
(Combination)
- Formulation:
- Oral
- Mechanism of Action:
- Menin Inhibitor
- In-House or Licensed:
- Kura Oncology
- Remarks:
- Clinical study is being conducted in NA and EU as a global product.
NPM1-mutant AML/KMT2A-rearranged AML
Combinations with gilteritinib, FLAG-IDA, LDAC
KOMET-008
KK2260
-
Advanced or Metastatic Solid Tumors
- Formulation:
- Injection
- Mechanism of Action:
- EGFR-TfR1Bispecific Antibody
- In-House or Licensed:
- In-House
- Remarks:
- REGULGENT
Fully human antibody production technology
Clinical study is being conducted in JP, and a clinical study is prepared
under way for PhI in NA as a global product. - Clinical Trial Information:
- NCT06248411
KK2269
-
Advanced or Metastatic Solid Tumors
- Formulation:
- Injection
- Mechanism of Action:
- EpCAM-CD40Bispecific Antibody
- In-House or Licensed:
- In-House
- Remarks:
- REGULGENT
Fully human antibody production technology
Clinical study is being conducted in JP and NA as a global product. - Clinical Trial Information:
- jRCT2031230372,NCT06266299
KK2845
-
Acute Myeloid Leukemia (AML)
- Mechanism of Action:
- Anti-TIM-3 ADC
- In-House or Licensed:
- In-House
- Remarks:
- Antibody-Drug Conjugate
Clinical study is being conducted in JP as a global product. - Clinical Trial Information:
- NCT06812104
KK4277
-
Systemic Lupus Erythematosus/
Cutaneous Lupus Erythematosus
- Formulation:
- Injection
- Mechanism of Action:
- Anti-PTPRS Humanized Antibody
- In-House or Licensed:
- SBI Biotech
- Remarks:
- POTELLIGENT
Clinical study is being conducted in JP and Asia. - Clinical Trial Information:
- NCT05411016
KK8123
-
X-linked Hypophosphatemia
- Formulation:
- Injection
- Mechanism of Action:
- Anti-FGF23 Fully Human Antibody
- In-House or Licensed:
- In-House
- Remarks:
- Clinical study is being conducted in NA and EU as a global product.
- Clinical Trial Information:
- NCT06525636
KK3910
-
Essential Hypertension
- Formulation:
- Injection
- In-House or Licensed:
- In-House
- Remarks:
- Clinical study is being conducted in JP as a global product
- Clinical Trial Information:
- NCT06823947
KK8398
infigratinib
Achondroplasia
- Formulation:
- Oral
- Mechanism of Action:
- FGFR3 Inhibitor
- In-House or Licensed:
- QED Therapeutics
- Remarks:
- Preparation underway for Ph Ⅲ in JP
-
※For detailed information on ziftomenib's development status, please refer to Kura Oncology's website. https://kuraoncology.com/
-
※Launch of clinical trial of ziftomenib evaluating dual inhibition of NPM1 and FLT3 mutations in patients with newly diagnosed AML （KOMET-007） was announced in October, 2025.
-
※OTL-200 was granted orphan regenerative medicine product designation for early-onset MLD in Japan in October 2025
