Development
Pipeline

R&D pipeline

Code Name
Generic name

Indication

Phase 1 2 3

-
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

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

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

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※ 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
Preparation underway for clinical trial in JP
Product Name in US: Lenmeldy
Product Name in Europe: Libmeldy

• ※
  For detailed information on ziftomenib(Product Name in US: KOMZIFTI)'s development status, please refer to Kura Oncology's website. https://kuraoncology.com/
• ※
  On January 30, 2026, Kyowa Kirin announced that termination of the current KHK4083/AMG 451（rocatinlimab） collaboration with Amgen and Kyowa Kirin will regain control of KHK4083/AMG 451 development and commercialization program.

Major Applications and Approvals