Nipent

(Pentostatin)

ApprovedSmall molecule

Nipent is a small molecule that works as Adenosine Deaminase Inhibitor.[2] It is being used across 7 indications, with its most advanced program in Approved.[1]

Nipent is one of 184 small molecule programs targeting Graft Versus Host Disease. It is currently in Approved.

Indications

Total Trials

Active Trials

183

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Graft Versus Host DiseaseApproved

LeukemiaApproved

AnemiaActive, not recruiting

Sickle Cell DiseaseActive, not recruiting

Hairy Cell LeukemiaActive, not recruiting

Severe Aplastic AnemiaRecruiting

Allogeneic Hematopoietic Cell TransplantActive, not recruiting

Graft Versus Host DiseaseApproved

Approved

LeukemiaApproved

Approved

AnemiaApproved

Active, not recruiting

Sickle Cell DiseasePhase 2

Active, not recruiting

Hairy Cell LeukemiaPhase 2

Active, not recruiting

Severe Aplastic AnemiaPhase 1

Recruiting

Allogeneic Hematopoietic Cell TransplantPhase 2

Active, not recruiting

Competition

Competitive Landscape

119 competitors in Leukemia

#DrugCompanyPhaseTrials

Nipent

Pfizer

Approved

39 2

Cytosar

Teva

Phase 3

52 3

Trexall

Jazz Pharmaceuticals

Phase 3

30 4

Fludara

Sanofi

Phase 3

21 5

Cytoxan

BBaxter Healthcare Corporation

Phase 3

57 6

Rituxan

Roche

Phase 3

16 7

Adriamycin

Pfizer

Phase 3

11 8

Oncovin

Eli Lilly

Phase 3

10 9

Decadron

AAspen Global Incorporated

Vepesid

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

Timeline

Expected Milestones

Q1 2027

Phase 1Active, not recruiting

Sickle Cell Disease

NCT03249831

Q2 2027

Phase 1Recruiting

Severe Aplastic Anemia

NCT05757310

Q4 2027

Phase 2Active, not recruiting

Sickle Cell Disease

NCT02105766

Q2 2030

Phase 2Active, not recruiting

Allogeneic Hematopoietic Cell Transplant

NCT03663933

Clinical

Trials by Indication[1]

Chronic Lymphocytic Leukemia13 trials

Pentostatin, Cyclophosphamide, and Rituximab in Treating Patients With Previously Untreated Chronic Lymphocytic Leukemia

Completed·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT00541034·49 enrolled

Pentostatin, and Rituximab With and Without Cyclophosphamide for Previously Untreated B-Chronic Lymphocytic Leukemia

Completed·OOhio State University Comprehensive Cancer Center·Phase 2·NCT00201721·28 enrolled

Pentostatin, Cyclophosphamide, and Rituximab in Treating Patients With Chronic Lymphocytic Leukemia or Other B-cell Cancers

Completed·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT00003658·60 enrolled

Show 10 more trials

Pentostatin, Cyclophosphamide, Rituximab, and Mitoxantrone in Treating Patients With Chronic Lymphocytic Leukemia or Other Low-Grade B-Cell Cancer

Completed·MMemorial Sloan Kettering Cancer Center·Phase 1/2·NCT00546377·50 enrolled

Pentostatin and Rituximab in Treating Patients With Low-Grade Non-Hodgkin's Lymphoma or Chronic Lymphocytic Leukemia

Completed·PPharmatech Oncology·Phase 2·NCT00026351

Pentostatin, Alemtuzumab, and Rituximab in Treating Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Completed·MMayo Clinic·Phase 2·NCT00669318·41 enrolled

Ofatumumab, Pentostatin, and Cyclophosphamide in Treating Patients With Untreated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Completed·MMayo Clinic·Phase 2·NCT01024010·82 enrolled

Combination of Pentostatin, Bendamustine and Ofatumumab for Treatment of Chronic Lymphocytic Leukemia and Lymphoma

Terminated·SSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University·Phase 1·NCT01352312·10 enrolled

Fludarabine, Cyclophosphamide, and Rituximab Versus Pentostatin, Cyclophosphamide, and Rituximab in Previously Untreated or Treated B-Cell Chronic Lymphocytic Leukemia Patients

Completed·UUS Oncology Research·Phase 3·NCT00254163·184 enrolled

Pentostatin, Cyclophosphamide, and Rituximab With or Without Bevacizumab in Treating Patients With B-Cell Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Completed·MMayo Clinic·Phase 2·NCT00816595·68 enrolled

Trial of Pentostatin Plus Cyclophosphamide With Ofatumumab (PCO) in Older Patients With Chronic Lymphocytic Leukemia

Completed·NNiguarda Hospital·Phase 2·NCT01681563·47 enrolled

Combination Chemotherapy and Rituximab in Treating Patients With Chronic Lymphocytic Leukemia or Lymphocytic Lymphoma

Completed·HHoag Memorial Hospital Presbyterian·Phase 2·NCT00049413

Rituximab, Pentostatin, Cyclophosphamide, and Lenalidomide in Treating Patients With Previously Untreated B-Cell Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Completed·MMayo Clinic·Phase 2·NCT00602836·45 enrolled

Sickle Cell Disease3 trials

A Blood Stem Cell Transplant for Sickle Cell Disease

Active, not recruiting·—·Phase 1·NCT03249831·3 enrolled

Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT03077542·57 enrolled

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia in Individuals With Higher Risk of Transplant Failure

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT02105766·56 enrolled

Stem Cell Transplant3 trials

Donor Stem Cell Transplant, Pentostatin, and Total-Body Irradiation in Treating Patients With Hematological Cancer

Withdrawn·UUniversity of Nebraska·Phase 1/2·NCT00816413

Pentostatin and Lymphocyte Infusion in Preventing Graft Rejection in Patients Who Have Undergone Donor Stem Cell Transplant

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00096161·36 enrolled

Safety and Efficacy of Pentostatin and Low Dose TBI With Allogenic Peripheral Blood Stem Cell Transplant

Completed·UUniversity of Nebraska·Phase 2·NCT00571662·76 enrolled

Kidney Cancer2 trials

Low-Intensity Stem Cell Transplantation With Multiple Lymphocyte Infusions to Treat Advanced Kidney Cancer

Completed·NNational Cancer Institute (NCI)·Phase 2·NCT00923845·25 enrolled

Pentostatin Followed by Peripheral Stem Cell Transplantation in Treating Patients With Advanced Kidney Cancer

Completed·JJonsson Comprehensive Cancer Center·Phase 1/2·NCT00006968·4 enrolled

Acute Graft Versus Host Disease2 trials

Phase II Trial of Pentostatin in Steroid Refractory Acute Graft Versus Host Disease

Completed·OOhio State University Comprehensive Cancer Center·Phase 2·NCT00201786·8 enrolled

Treatment for Acute Graft-Versus-Host Disease (BMT CTN 0302)

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT00224874·180 enrolled

14 more indications

Chronic Graft Versus Host Disease2 trials

Pentostatin for High Risk and Refractory Chronic Graft Versus Host Disease in Children

Completed·—·Phase 2·NCT00144430·60 enrolled

Pentostatin in Treating Patients With Refractory Chronic Graft-Versus-Host Disease

Completed·AAlliance for Clinical Trials in Oncology·Phase 2·NCT00074035·39 enrolled

Bone Marrow Transplantation2 trials

A Study of Ruxolitinib vs Best Available Therapy (BAT) in Patients With Steroid-refractory Chronic Graft vs. Host Disease (GvHD) After Bone Marrow Transplantation (REACH3)

Completed· Incyte logo

Incyte·Phase 3·NCT03112603·330 enrolled

Methotrexate or Pentostatin for Graft-versus-host Disease Prophylaxis in Risk-adapted Allogeneic Bone Marrow Transplantation for Hematologic Malignancies

Completed·SSt. Jude Children's Research Hospital·Phase 3·NCT01188798·6 enrolled

Lymphoma1 trial

Reduced-Intensity Regimen Before Allogeneic Transplant for Patients With Relapsed Non-Hodgkin's or Hodgkin's Lymphoma

Terminated·EEastern Cooperative Oncology Group·Phase 2·NCT00057954·6 enrolled

Allogeneic Stem Cell Transplantation1 trial

A Trial of Extracorporeal Photopheresis, Pentostatin, and Total Body Irradiation in Patients Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation for the Treatment of Malignancies

Completed·YYale University·Phase 2·NCT00402714·14 enrolled

Allogeneic Hematopoietic Cell Transplant1 trial

Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT03663933·71 enrolled

Relapsed or Refractory1 trial

Pentostatin, Cyclophosphamide, and Rituximab Followed By Campath-1H in Patients With Relapsed or Refractory B-Cell CLL

Completed·EEastern Cooperative Oncology Group·Phase 2·NCT00074282·102 enrolled

Phase II1 trial

Phase II Trial of Pentostatin and Targeted Busulfan

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT00496340·42 enrolled

Severe Aplastic Anemia1 trial

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

Recruiting·—·Phase 1·NCT05757310·6 enrolled

Peripheral Blood Stem Cell Transplantation1 trial

Pentostatin and Alemtuzumab as a Preparative Regimen for Allogeneic Peripheral Blood Stem Cell Transplantation

Terminated·UUniversity of Arizona·Phase 2·NCT00698685·14 enrolled

Methotrexate1 trial

Phase I/II Study of Pentostatin Combined With Tacrolimus and Mini-Methotrexate for GVHD Prevention After MUD BMT

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT00506922·150 enrolled

Mesothelioma1 trial

SS1P and Pentostatin Plus Cyclophosphamide for Mesothelioma

Completed·NNational Cancer Institute (NCI)·Phase 1/2·NCT01362790·55 enrolled

T-Cell Neoplasm1 trial

Alemtuzumab and Pentostatin In T-cell Neoplasms

Terminated·MM.D. Anderson Cancer Center·Phase 2·NCT00453193·26 enrolled

Myelodysplastic Syndromes1 trial

Reduced-Intensity Regimen Before Donor Bone Marrow Transplant in Treating Patients With Myelodysplastic Syndromes

Completed·EEastern Cooperative Oncology Group·Phase 2·NCT00045305·17 enrolled

Hairy Cell Leukemia1 trial

Randomized Phase II Trial of Rituximab With Either Pentostatin or Bendamustine for Multiply Relapsed or Refractory Hairy Cell Leukemia

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT01059786·69 enrolled

Regulatory

FDA Approval Timeline[4]

Oct 11, 1991

NIPENT

NDA NDA020122

Sources

References

[1]
ClinicalTrials.gov — 39 trial records for Pentostatin. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Pentostatin compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Pentostatin. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA020122). U.S. Food & Drug Administration.
[5]
SEC EDGAR — Pfizer company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Adenosine Deaminase Inhibitor[2]
Modality: Small molecule[2]
Highest Phase: Approved[1]
Company: Pfizer

Nipent

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q2 2027

Q4 2027

Q2 2030

Trials by Indication[1]

Chronic Lymphocytic Leukemia13 trials

Sickle Cell Disease3 trials

Stem Cell Transplant3 trials

Kidney Cancer2 trials

Acute Graft Versus Host Disease2 trials

Chronic Graft Versus Host Disease2 trials

Bone Marrow Transplantation2 trials

Lymphoma1 trial

Allogeneic Stem Cell Transplantation1 trial

Allogeneic Hematopoietic Cell Transplant1 trial

Relapsed or Refractory1 trial

Phase II1 trial

Severe Aplastic Anemia1 trial

Peripheral Blood Stem Cell Transplantation1 trial

Methotrexate1 trial

Mesothelioma1 trial

T-Cell Neoplasm1 trial

Myelodysplastic Syndromes1 trial

Hairy Cell Leukemia1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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