Vonjo

(Pacritinib)

ApprovedSmall molecule

Vonjo is a small molecule that works as Activin Receptor Type-1 Inhibitor; Tyrosine-Protein Kinase JAK2 Inhibitor; Tyrosine-Protein Kinase Receptor FLT3 Inhibitor; Tyrosine-Protein Kinase Receptor FLT3 Inhibitor; Interleukin-1 Receptor-Associated Kinase 1 Inhibitor; Tyrosine-Protein Kinase JAK2 Inhibitor.[2] It is being used across 23 indications, with its most advanced program in Approved.[1]

Vonjo is one of 432 small molecule programs targeting Macroglobulinemia. It is currently in Approved.

Indications

Total Trials

Active Trials

431

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

MacroglobulinemiaRecruiting

ThrombocytopeniaTerminated

Primary MyelofibrosisApproved

LeukemiaApproved

MyelofibrosisTerminated

Lung AdenocarcinomaRecruiting

Chronic Myelomonocytic LeukemiaRecruiting

Liver DiseaseApproved

Breast CancerApproved

Prostate CancerTerminated

MacroglobulinemiaPhase 2

Recruiting

ThrombocytopeniaPhase 3

Terminated

Primary MyelofibrosisApproved

Approved

LeukemiaApproved

Approved

MyelofibrosisPhase 3

Terminated

Lung AdenocarcinomaPhase 2

Recruiting

Chronic Myelomonocytic LeukemiaPhase 2

Recruiting

Liver DiseaseApproved

Approved

Breast CancerApproved

Approved

Prostate CancerPhase 2

Terminated

Showing 10 of 23 indications

Competition

Competitive Landscape

706 competitors in Breast Cancer

#DrugCompanyPhaseTrials

Vonjo

SSobi, Inc.

Approved

46 2

Adenocard

Astellas

Preclinical

2 3

Lexiscan

Astellas

Preclinical

1 4

Methylthioninium Chloride

PProvepharm SAS

Preclinical

3 5

Duragesic

CCephalon

Preclinical

1 6

Sporanox

SSebela Pharmaceuticals Development LLC

Preclinical

1 7

Vfend

PPrism Pharmaceuticals

Preclinical

—8

Ablavar

LLantheus Medical Imaging

Preclinical

2 9

Tc99m Sestamibi

MM.D. Anderson Cancer Center

Preclinical

2 10

Naltrexone/bupropion

Novo Nordisk

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 1Not yet recruiting

Myelofibrosis

NCT06414681

Q1 2027

Phase 2Active, not recruiting

Myelofibrosis

NCT03645824

Q3 2027

PreclinicalRecruiting

Graft Vs Host Disease

NCT05531786

Phase 3Recruiting

Essential Thrombocythemia

NCT03165734

Phase 1/2Recruiting

Lung Adenocarcinoma

NCT06516887

Q4 2027

Phase 2Recruiting

Acute Myeloid Leukemia

NCT04282187

Clinical

Trials by Indication[1]

Myelofibrosis5 trials

Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

Terminated·CCTI BioPharma·Phase 3·NCT01773187·327 enrolled

Myelofibrosis Treated With Pacritinib Before aSCT. (HOVON134MF)

Active, not recruiting·SStichting Hemato-Oncologie voor Volwassenen Nederland·Phase 2·NCT03645824·61 enrolled

Combination of Tagraxofusp with Pacritinib in Patients with Intermediate-1 or Higher Myelofibrosis, Who Have Had Prior Therapy with the Approved JAK Inhibitors or in Which Therapy with the Approved JAK Inhibitors is Not Appropriate, Contraindicated or Declined

Not yet recruiting·UUniversity of Kansas Medical Center·Phase 1·NCT06414681·20 enrolled

Show 2 more trials

A Phase II Non-Controlled, Open-Label, Efficacy, Safety, Pharmacokinetic, and Pharmacodynamic Study of Pacritinib in Myelofibrosis

Withdrawn·BBaxalta now part of Shire·Phase 2·NCT02584777

Investigator-Initiated, Open-Label, Phase II Trial of Selinexor in Combination With Pacritinib in Patients With Myelofibrosis Who Are JAK Inhibitor-Naïve and Have Cytopenias (ILLUMINATE)

Recruiting·JJohn Mascarenhas·Phase 2·NCT07447817·26 enrolled

Myelodysplastic Syndromes3 trials

Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes (MDS)

Terminated·MM.D. Anderson Cancer Center·Phase 2·NCT02469415·3 enrolled

Phase I/II Trial of Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

Recruiting·NNational Cancer Institute (NCI)·Preclinical·NCT06303193·160 enrolled

A Phase 1/2 Study of Pacritinib in Combination With Azacitidine for the Treatment of IPSS-M Moderate Low to Very High Risk Myelodysplastic Syndrome

Withdrawn·TThomas Jefferson University·Preclinical·NCT07387354

Myeloproliferative Neoplasms2 trials

Pacritinib w/ Talazoparib in Pts w/ Myeloproliferative Neoplasms Unresponsive to JAK2 Inhibition

Recruiting·FFox Chase Cancer Center·Phase 1·NCT06218628·24 enrolled

Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)

Terminated·MM.D. Anderson Cancer Center·Phase 2·NCT02410551·4 enrolled

Prostate Cancer2 trials

Pacritinib for Biochemical Relapse After Definitive Treatment for Prostate Cancer

Terminated·MMedical College of Wisconsin·Phase 2·NCT04635059·6 enrolled

A Single-Arm, Open-label, Phase II Study Evaluating Pacritinib in Participants With Metastatic Castrate-Resistant Prostate Cancer That Progressed on or After Prior Treatment With Androgen Receptor Signaling Inhibitors - (POSTPONE)

Recruiting·MMedical College of Wisconsin·Phase 2·NCT07226713·32 enrolled

Essential Thrombocythemia2 trials

Phase 2 Study: An Open-Label, Randomized, Phase 2 Dose-Finding Study of Pacritinib in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post- Essential Thrombocythemia Myelofibrosis Previously Treated With Ruxolitinib

Completed·CCTI BioPharma·Phase 2·NCT04884191·165 enrolled

A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

Recruiting·SSwedish Orphan Biovitrum·Phase 3·NCT03165734·399 enrolled

30 more indications

Acute Myeloid Leukemia2 trials

Pacritinib and Chemotherapy in Treating Patients With Acute Myeloid Leukemia and FLT3 Mutations

Completed·BBhavana Bhatnagar·Phase 1·NCT02323607·13 enrolled

A Phase 2 Trial Investigating Decitabine in Combination With a JAK-Inhibitor as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant in Patients With Accelerated/Blast Phase Myeloproliferative Neoplasms

Recruiting·UUniversity of Washington·Phase 2·NCT04282187·25 enrolled

Thrombocytopenia2 trials

A Study of Selinexor Monotherapy in Subjects With JAK Inhibitor-naïve Myelofibrosis and Moderate Thrombocytopenia

Recruiting·KKaryopharm Therapeutics Inc·Phase 2·NCT05980806·58 enrolled

Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia

Terminated·CCTI BioPharma·Phase 3·NCT02055781·311 enrolled

Older Patients1 trial

Pacritinib Combined With Decitabine or Cytarabine in Older Patients With AML

Terminated·WWeill Medical College of Cornell University·Phase 2·NCT02532010·13 enrolled

Macroglobulinemia1 trial

A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia

Recruiting·—·Phase 2·NCT06986174·30 enrolled

Solid Tumors1 trial

A Single Arm, Phase Ib/II Trial of Single Agent Pacritinib in Patients With 1q21.3 Amplified Solid Tumors Enriching for Interleukin-1 Receptor-associated Kinase 1 Pathway Activation (PAIR)

Active, not recruiting·NNational University Hospital, Singapore·Phase 1/2·NCT04520269·74 enrolled

Absorption1 trial

Investigate Absorption, Metabolism, Excretion, and Mass Balance of Pacritinib

Completed·CCTI BioPharma·Phase 1·NCT02803762·6 enrolled

Relapsed/Refractory1 trial

Pacritinib in Relapsed/Refractory T-cell Lymphoproliferative Neoplasms

Recruiting·UUniversity of Michigan Rogel Cancer Center·Phase 2·NCT04858256·100 enrolled

Lymphoproliferative Disorders1 trial

Pacritinib in Relapsed/Refractory Lymphoproliferative Disorders

Terminated·UUniversity of Michigan Rogel Cancer Center·Phase 1·NCT03601819·4 enrolled

Acute Lymphoblastic Leukemia1 trial

Targeted Therapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia

Terminated·OOHSU Knight Cancer Institute·Phase 2·NCT01620216·12 enrolled

Colorectal Cancer1 trial

Pacritinib to Inhibit JAK/STAT Signaling in Refractory Colorectal Cancer

Terminated·WWashington University School of Medicine·Phase 2·NCT02277093·11 enrolled

Completed·CCTI BioPharma·Phase 1·NCT02807116·18 enrolled

Renal Impairment1 trial

PK of Pacritinib in Patients With Mild, Moderate, Severe Renal Impairment and ESRD Compared to Healthy Subjects

Completed·CCTI BioPharma·Phase 1·NCT02807077·40 enrolled

Myelodysplastic Syndrome1 trial

Pacritinib in Combination With Low Dose Decitabine in Intermediate-High Risk Myelofibrosis or Myeloproliferative Neoplasm (MPN)/Myelodysplastic Syndrome (MDS)

Withdrawn·WWashington University School of Medicine·Phase 1·NCT02564536

Non-Small Cell Lung Cancer1 trial

Pacritinib in Patients With Endothelial Growth Factor (EGFR) Mutant Non-small Cell Lung Cancer (NSCLC) After EGFR Tyrosine Kinase Inhibitor (TKI)

Terminated·WWashington University School of Medicine·Phase 1·NCT02342353·8 enrolled

Tics1 trial

Determine Pacritinib Pharmacokinetics in Impaired Hepatic Patients and Healthy Subjects

Completed·CCTI BioPharma·Phase 1·NCT02765724·28 enrolled

Systemic1 trial

To Evaluate the Effect of Clarithromycin on the Systemic Exposure of Pacritinib in Healthy Subjects

Completed·CCTI BioPharma·Phase 1·NCT02807051·20 enrolled

COVID-191 trial

PRE-VENT Study in Hospitalized Patients With Severe COVID-19 With or Without Cancer

Terminated·CCTI BioPharma·Phase 2·NCT04404361·200 enrolled

Myeloproliferative Neoplasm1 trial

Phase I Trial of Pacritinib in Combination With Venetoclax and Azacitidine for the Treatment of Accelerated and Blast Phase Myeloproliferative Neoplasms

Not yet recruiting·RRoswell Park Cancer Institute·Phase 1·NCT07679334·20 enrolled

VEXAS1 trial

PAXIS: A Phase 2a and 2b Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Pacritinib in Patients With VEXAS Syndrome

Recruiting·SSwedish Orphan Biovitrum·Phase 2·NCT06782373·156 enrolled

KSHV Inflammatory Cytokine Syndrome (KICS)1 trial

Phase II Study of Pacritinib in Kaposi Sarcoma Herpesvirus (KSHV)-Associated Multicentric Castleman Disease and KSHV-Associated Inflammatory Cytokine Syndrome (KICS)

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT06052618·75 enrolled

Recurrent Mantle Cell Lymphoma1 trial

A Pilot Study of Pacritinib Combined With a BTK Inhibitor in Patients With Relapsed/Refractory Mantle Cell Lymphoma

Recruiting·CCity of Hope Medical Center·Phase 1·NCT06675123·10 enrolled

Myelofibrosis，MF1 trial

Pacritinib For The Reduction Of Bone Marrow Fibrosis In Patients With Myelofibrosis Who Have Thrombocytopenia; A Multicenter, Open-Label, Single Arm, Phase II Exploratory Study

Recruiting·GGrupo Español de Enfermedades Mieloproliferativas Crónicas PH Negativas·Phase 2·NCT07394153·30 enrolled

Graft Vs Host Disease1 trial

Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Recruiting·NNational Cancer Institute (NCI)·Preclinical·NCT05531786·50 enrolled

Accelerated Phase Myeloproliferative Neoplasm1 trial

A Phase 2 Trial Investigating the Addition of Pacritinib to a Hypomethylating Agent as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant in Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms

Recruiting·UUniversity of Washington·Phase 2·NCT07148947·27 enrolled

Leukemia, Myelomonocytic, Chronic1 trial

A Randomized Phase 2 Study of Pacritinib vs. Hydroxyurea in Patients With Advanced Proliferative Chronic Myelomonocytic Leukemia

Recruiting·TTheradex·Phase 2·NCT07033598·66 enrolled

Chronic Myelomonocytic Leukemia1 trial

Pacritinib in Combination With Azacitidine in Patients With Chronic Myelomonocytic Leukemia

Recruiting·UUnknown Sponsor·Preclinical·NCT06159491·26 enrolled

E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome1 trial

A Phase 1 Study of Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome

Recruiting·WWashington University School of Medicine·Phase 1·NCT06538181·15 enrolled

Regulatory

FDA Approval Timeline[4]

Feb 28, 2022

VONJO

NDA NDA208712

Aug 15, 2023

VONJO

NDA NDA208712

Nov 12, 2024

VONJO

NDA NDA208712

Aug 22, 2025

VONJO

NDA NDA208712

Oct 1, 2025

VONJO

NDA NDA208712

Mar 18, 2026

VONJO

NDA NDA208712

Jul 20, 2026

VONJO

NDA NDA208712

Sources

References

[1]
ClinicalTrials.gov — 46 trial records for Pacritinib. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Pacritinib compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Pacritinib. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA208712). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Vonjo. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Sobi, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Activin Receptor Type-1 Inhibitor; Tyrosine-Protein Kinase JAK2 Inhibitor; Tyrosine-Protein Kinase Receptor FLT3 Inhibitor; Tyrosine-Protein Kinase Receptor FLT3 Inhibitor; Interleukin-1 Receptor-Associated Kinase 1 Inhibitor; Tyrosine-Protein Kinase JAK2 Inhibitor[2]
Modality: Small molecule[2]
Highest Phase: Approved[1]
Company: SSobi, Inc.

Patent Landscape[5]

Patent Cliff

May 5, 2028

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9573964May 5, 2028

8153632Jan 17, 2029

8980873Mar 25, 2030

Vonjo

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2027

Q3 2027

Q4 2027

Trials by Indication[1]

Myelofibrosis5 trials

Myelodysplastic Syndromes3 trials

Myeloproliferative Neoplasms2 trials

Prostate Cancer2 trials

Essential Thrombocythemia2 trials

Acute Myeloid Leukemia2 trials

Thrombocytopenia2 trials

Older Patients1 trial

Macroglobulinemia1 trial

Solid Tumors1 trial

Absorption1 trial

Relapsed/Refractory1 trial

Lymphoproliferative Disorders1 trial

Acute Lymphoblastic Leukemia1 trial

Colorectal Cancer1 trial

Immune Suppression1 trial

Chronic Lymphocytic Leukemia/small Lymphocytic Lymphoma1 trial

Lung Adenocarcinoma1 trial

Interaction1 trial

Renal Impairment1 trial

Myelodysplastic Syndrome1 trial

Non-Small Cell Lung Cancer1 trial

Tics1 trial

Systemic1 trial

COVID-191 trial

Myeloproliferative Neoplasm1 trial

VEXAS1 trial

KSHV Inflammatory Cytokine Syndrome (KICS)1 trial

Recurrent Mantle Cell Lymphoma1 trial

Myelofibrosis，MF1 trial

Graft Vs Host Disease1 trial

Accelerated Phase Myeloproliferative Neoplasm1 trial

Leukemia, Myelomonocytic, Chronic1 trial

Chronic Myelomonocytic Leukemia1 trial

E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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