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※
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
KK8398
infigratinib
Achondroplasia
- Formulation:
- Oral
- Mechanism of Action:
- FGFR3 Inhibitor
- In-House or Licensed:
- QED Therapeutics
- Remarks:
- Clinical study is being conducted in JP
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
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※
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
FLT3/NPM1 co-mutated AML
Combinations with cytarabine + daunorubicin, and quizartinib
KOMET-007
-
ziftomenib※
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※
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※
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※
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
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
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
-
ziftomenib※
Acute Myeloid Leukemia (AML)
(Monotherapy)
- Formulation:
- Oral
- Mechanism of Action:
- Menin Inhibitor
- In-House or Licensed:
- Kura Oncology
- Remarks:
- Adult Relapsed or Refractory AML with a NPM1 Mutation
Preparation underway for Ph Ⅱ clinical trial in JP
KK8398
infigratinib
Hypochondroplasia
- Formulation:
- Oral
- Mechanism of Action:
- FGFR3 Inhibitor
- In-House or Licensed:
- QED Therapeutics
- Remarks:
- Preparation underway for Ph Ⅲ clinical trial in JP
OTL-200
atidarsagene autotemcel
Early-onset Metachromatic Leukodystrophy (MLD)
- Mechanism of Action:
- Hematopoietic Stem Cell (HSC) Gene Therapy
- In-House or Licensed:
- In-House
- Remarks:
- Orphan Regenerative Medicine Product
Designation in JP
A Ph.3 clinical study in JP is under preparation
Product Name in US: Libmeldy
Product Name in Europe: Lenmeldy
KK2223
-
Cutaneous T cell lymphoma
Peripheral T cell lymphoma
- In-House or Licensed:
- In-House
- Remarks:
- Clinical study is prepared under way for PhⅠ
-
※For detailed information on ziftomenib(Product Name in US: KOMZIFTI)'s development status, please refer to Kura Oncology's website. https://kuraoncology.com/
-
Note:Since clinical studies of KHK4084/AMG 451(rocatinlimab) for moderate to severe atopic dermatitis, prurigo nodularis, and moderate to severe asthma were discontinued, the relevant information was deleted from this table.
-
Note:On April 24, 2026, the Company announced that the first patient has been dosed in the phase II clinical trial in Japan evaluating ziftomenib for the treatment of relapsed or refractory NPM1-mutated AML.
