Development
Pipeline

Creating a Future of Pharmaceuticals: Kyowa Kirin’s Frontline Development

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.

Click here for “Public Clinical Trial Registries”

As of Sep. 30, 2026

R&D pipeline

Modalities

Indication

small molecule -
ziftomenib※ Acute Myeloid Leukemia (AML)
(Combination) phase 3
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 (for NPM1-mutant only), and cytarabine + daunorubicin
KOMET-017
small molecule KK8398
infigratinib Achondroplasia phase 3
Formulation:
Oral
Mechanism of Action:
FGFR3 Inhibitor
In-House or Licensed:
QED Therapeutics
Remarks:
Clinical study is being conducted in JP
Orphan Medicine Product Designation in JP
HSC-GT OTL-203
- MPS-IH (Hurler Syndrome) phase 3
Mechanism of Action:
Hematopoietic Stem Cell (HSC) Gene Therapy
In-House or Licensed:
In-House
Remarks:
Rare Pediatric Disease (RPD), Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations (FDA)
Priority Medicines (PRIME) designation (EMA)
Area of clinical study: NA and EU
small molecule KHK4951
tivozanib Diabetic Macular Edema phase 2
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
small molecule KHK4951
tivozanib Neovascular Age-Related Macular Degeneration phase 2
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
HSC-GT OTL-201
- MPS-IIIA (Sanfilippo Syndrome type A) phase 2
Mechanism of Action:
Hematopoietic Stem Cell (HSC) Gene Therapy
In-House or Licensed:
In-House
Remarks:
Rare Pediatric Disease (RPD) designation (FDA)
Orphan Drug Designation (FDA, EMA)
Innovation Passport Designation (MHRA)
small molecule -
ziftomenib※ Acute Myeloid Leukemia (AML)
(Monotherapy) phase 2
Formulation:
Oral
Mechanism of Action:
Menin Inhibitor
In-House or Licensed:
Kura Oncology
Remarks:
Adult Relapsed or Refractory AML with a NPM1 Mutation
PhⅡ clinical study is being conducted in JP
Orphan Drug Designation in JP
small molecule -
ziftomenib※ Acute Myeloid Leukemia (AML)
(Combination) phase 1
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
small molecule -
ziftomenib※ Acute Lymphoblastic Leukemia (ALL)
(Monotherapy) phase 1
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
small molecule -
ziftomenib※ Acute Myeloid Leukemia (AML)
(Combination) phase 1
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
small molecule -
ziftomenib※ Acute Myeloid Leukemia (AML)
(Combination) phase 1
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/FLT3-mutant AML
Combinations with gilteritinib, FLAG-IDA, LDAC
KOMET-008
antibody KK2260
- Advanced or Metastatic Solid Tumors phase 1
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
antibody KK2269
- Advanced or Metastatic Solid Tumors phase 1
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
antibody KK2845
- Acute Myeloid Leukemia (AML) and other Hematologic Malignancies phase 1
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
antibody KK3910
- Essential Hypertension phase 1
Formulation:
Injection
In-House or Licensed:
In-House
Remarks:
Clinical study is being conducted in JP as a global product
Clinical Trial Information:
NCT06823947
antibody KK4277
- Systemic Lupus Erythematosus/
Cutaneous Lupus Erythematosus phase 1
Formulation:
Injection
Mechanism of Action:
Anti-PTPRS Humanized Antibody
In-House or Licensed:
SBI Biotech
Remarks:
POTELLIGENT
Clinical study is completed in JP and Asia
Clinical Trial Information:
NCT05411016
antibody KK8123
- X-linked Hypophosphatemia phase 1
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
small molecule -
ziftomenib※ Acute Myeloid Leukemia (AML)
(Monotherapy) phase 1
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
antibody KK2223
- Cutaneous T cell lymphoma
Peripheral T cell lymphoma phase 1
In-House or Licensed:
In-House
Remarks:
Antibody-Drug Conjugate
Clinical study is being conducted for PhⅠin NA
Clinical study is preparing for PhⅠin EU
antibody KK8005
- X-linked Hypophosphatemia phase 1
In-House or Licensed:
In-House
Remarks:
Clinical study is being conducted for PhⅠ/Ⅱin JP as a global product
antibody KK4408
- Autoimmune Disease phase 0
In-House or Licensed:
In-House
Remarks:
REGULGENT
Clinical study is preparing for PhⅠ/Ⅱ
small molecule KK8398
infigratinib Hypochondroplasia phase 0
Formulation:
Oral
Mechanism of Action:
FGFR3 Inhibitor
In-House or Licensed:
QED Therapeutics
Remarks:
Preparation underway for Ph Ⅲ clinical trial in JP
Orphan Drug Designation in JP
HSC-GT OTL-200
atidarsagene autotemcel Early-onset Metachromatic Leukodystrophy (MLD) phase 0
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: Lenmeldy
Product Name in EU: Libmeldy
  • ※
    For detailed information on ziftomenib(Product Name in US: KOMZIFTI)'s development status, please refer to Kura Oncology's website. https://kuraoncology.com/Open in new window
  • Note:
    Our main progress from September 30, 2026 is as follows.
    On October 6, 2026, the first patient has been dosed in the phase Ⅰ/ II clinical trial in Japan evaluating KK4408 for the treatment of autoimmune disease

Major Applications and Approvals

Code Name, Generic Name, Product Name OTL-200(atidarsagene autotemcel, Product Name in EU/US:Libmeldy/Lenmeldy)
Indication Metachromatic Leukodystrophy(MLD)
Application/Under Review filed in Japan
Countries/Regions Received Approval in 2026 ─
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