Grafapex

(Treosulfan)

ApprovedSmall molecule

MMedexus Pharma, Inc.

Grafapex is one of 279 small molecule programs targeting Non-Hodgkins Lymphoma. It is currently in Approved.

Indications

Total Trials

Active Trials

278

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Non-Hodgkins LymphomaApproved

Lymphoid NeoplasmApproved

Hodgkins LymphomaApproved

Multiple MyelomaApproved

MelanomaActive, not recruiting

Clinical TrialRecruiting

AML - Acute Myeloid LeukemiaRecruiting

Acute Lymphoblastic LeukaemiaRecruiting

Allogeneic Hematopoietic Stem Cell TransplantationNot yet recruiting

Acute Myeloid LeukemiaTerminated

Non-Hodgkins LymphomaApproved

Approved

Lymphoid NeoplasmApproved

Approved

Hodgkins LymphomaApproved

Approved

Multiple MyelomaApproved

Approved

MelanomaApproved

Active, not recruiting

Clinical TrialPhase 2

Recruiting

AML - Acute Myeloid LeukemiaPhase 2

Recruiting

Acute Lymphoblastic LeukaemiaPreclinical

Recruiting

Allogeneic Hematopoietic Stem Cell TransplantationPhase 2

Not yet recruiting

Acute Myeloid LeukemiaApproved

Terminated

Showing 10 of 13 indications

Competition

Competitive Landscape

282 competitors in Multiple Myeloma

#DrugCompanyPhaseTrials

Grafapex

MMedexus Pharma, Inc.

Approved

34 2

Empliciti

Bristol-Myers Squibb

Phase 3

45 3

Fludara

Sanofi

Phase 3

47 4

Keytruda

Merck & Co.

Phase 3

17 5

Sarclisa

AAzienda U.S.L. 1 di Massa e Carrara

Phase 3

42 6

Biaxin

AbbVie

Phase 3

15 7

Opdivo

Bristol-Myers Squibb

Phase 3

11 8

Azatiq

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Phase 3

23 9

Treanda

AAzurity Pharmaceuticals

Neulasta

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

Timeline

Expected Milestones

Q4 2026

Phase 1Recruiting

Renal Impairment

NCT05534620

Q2 2028

Phase 2Recruiting

Clinical Trial

NCT05636787

Q3 2028

Phase 2Recruiting

Lymphoid

NCT03980769

Q1 2029

Phase 2Recruiting

Nijmegen Breakage Syndrome

NCT06198842

Phase 2Recruiting

Hematological Malignancies

NCT04195633

Clinical

Trials by Indication[1]

Nijmegen breakage syndrome3 trials

Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome

Not yet recruiting·FFederal Research Institute of Pediatric Hematology, Oncology and Immunology·Phase 2·NCT07316595·24 enrolled

Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome

Recruiting·FFederal Research Institute of Pediatric Hematology, Oncology and Immunology·Phase 2·NCT06198842·10 enrolled

Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage Syndrome

Unknown status·FFederal Research Institute of Pediatric Hematology, Oncology and Immunology·Phase 2·NCT04400045·10 enrolled

Acute Lymphoblastic Leukemia2 trials

Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00860574·96 enrolled

Treosulfan and Fludarabine in Treating Younger Patients Who Are Undergoing a Donor Stem Cell Transplant for Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndrome

Completed·OOHSU Knight Cancer Institute·Phase 1/2·NCT00253513·60 enrolled

Children2 trials

Treosulfan/Fludarabine/Low Dose TBI as a Preparative Regimen for Children With AML/MDS Undergoing Allo HCT

Completed·CCenter for International Blood and Marrow Transplant Research·Phase 2·NCT01772953·40 enrolled

Treosulfan Pharmacokinetics in Children Undergoing Allogeneic HSCT

Completed·GGreat Ormond Street Hospital for Children NHS Foundation Trust··NCT02048800·61 enrolled

Hematological Malignancies2 trials

Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies

Recruiting·FFred Hutchinson Cancer Center·Phase 2·NCT04195633·60 enrolled

Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Advanced Hematological Malignancies After Treosulfan-based Conditioning Therapy - A Clinical Phase II Study

Completed·Mmedac GmbH·Preclinical·NCT01063647·56 enrolled

Myelodysplastic Syndrome2 trials

Treo/Flu/TBI With Donor Stem Cell Transplant for Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia

Unknown status·FFred Hutchinson Cancer Center·Phase 2·NCT01894477·102 enrolled

Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)

Completed·Mmedac GmbH·Phase 2·NCT01062490·45 enrolled

23 more indications

Stem Cell Transplant1 trial

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00919503·98 enrolled

Acute Myeloid Leukaemia (AML)1 trial

Treosulfan Based Conditioning Acute Myeloid Leukaemia (AML)

Completed·Mmedac GmbH·Phase 2·NCT01063660·75 enrolled

Malignant Disease1 trial

Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases

Completed·Mmedac GmbH·Phase 2·NCT02349906·106 enrolled

Allogeneic Stem Cell Transplantation1 trial

MiniMUD Study - Unrelated Reduced Intensity Conditioning With Treosulfan® for Allogeneic Stem Cell Transplantation in Patients With Hematological Malignancies

Unknown status·HHospices Civils de Lyon·Phase 2·NCT00129155·30 enrolled

Clinical Trial1 trial

Clinical Trial Investigating the Chemotherapeutic Compound Treosulfan (Trecondi® Ideogen) in Myeloma Patients

Recruiting·IInsel Gruppe AG, University Hospital Bern·Phase 2·NCT05636787·120 enrolled

Ocular Melanoma1 trial

Study of Metastatic Ocular Melanoma

Unknown status·—·Phase 2·NCT00168870·48 enrolled

Bone Marrow Transplant1 trial

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT04965597·40 enrolled

Treatment1 trial

A Trial With Metronomic Low-dose Treosulfan, Pioglitazone and Clarithromycin Versus Standard Treatment in NSCLC

Unknown status·UUniversity Hospital Regensburg·Phase 2·NCT02852083·86 enrolled

Allogeneic Hematopoietic Stem Cell Transplantation1 trial

MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases

Not yet recruiting·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT07493538·132 enrolled

Renal Impairment1 trial

Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics

Recruiting·Mmedac GmbH·Phase 1·NCT05534620·36 enrolled

Conditioning1 trial

Clinical Phase III Trial Treosulfan-based Conditioning Versus Reduced-intensity Conditioning (RIC)

Completed·Mmedac GmbH·Phase 3·NCT00822393·570 enrolled

Ewing sarcoma1 trial

Study in Localized and Disseminated Ewing Sarcoma

Completed·UUniversity Hospital Muenster·Phase 3·NCT00987636·907 enrolled

Ovarian Cancer1 trial

Preference Study With Elderly Patients Recurrent Ovarian Cancer

Completed·NNorth Eastern German Society of Gynaecological Oncology·Phase 3·NCT00170690·123 enrolled

Lymphoid Malignancies1 trial

Treosulfan-based Conditioning for Allogeneic Stem-cell Transplantation (SCT) in Lymphoid Malignancies

Unknown status·SSheba Medical Center·Phase 2·NCT01079013·40 enrolled

Elderly Patients1 trial

Phase II Trial in Elderly Patients With AML or MDS in Complete Remission Not Eligible for Allogenic Transplant

Completed·CCiceri Fabio·Phase 2·NCT03961919·15 enrolled

Cancer1 trial

Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients With Hematological Cancer Who Are Undergoing Umbilical Cord Blood Transplant

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00796068·130 enrolled

Malignancies1 trial

Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies

Completed·Mmedac GmbH·Phase 2·NCT02333058·70 enrolled

Transplantation1 trial

Treosulfan-based Conditioning for Transplantation in AML/MDS

Completed·—·Phase 2·NCT00491634·24 enrolled

Lymphoid1 trial

Allogeneic Hematopoietic Cell Transplantation for Patients With Non-Malignant Disorders Using Treosulfan, Fludarabine, and Thiotepa

Recruiting·FFred Hutchinson Cancer Center·Phase 2·NCT03980769·40 enrolled

Acute Lymphoblastic Leukaemia1 trial

Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia

Recruiting·SSt. Anna Kinderkrebsforschung·Preclinical·NCT01949129·1,800 enrolled

AML - Acute Myeloid Leukemia1 trial

Randomized Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation

Recruiting·TTechnische Universität Dresden·Phase 2·NCT07025824·220 enrolled

Acute Myeloid Leukemia1 trial

A Phase II Randomized Study to Assess Outcomes With Treosulfan-Based Versus Clofarabine-Based Conditioning in Patients With Myelodysplastic Syndromes With Excess Blasts (MDS-EB), or Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Transplantation (HCT)

Terminated·FFred Hutchinson Cancer Center·Phase 2·NCT04994808·23 enrolled

Unspecified1 trial

T&B Depletion Non Malignant

Unknown status·FFranco Locatelli·Phase 2·NCT01810926·130 enrolled

Regulatory

FDA Approval Timeline[4]

Jan 21, 2025

GRAFAPEX

NDA NDA214759

Sources

References

[1]
ClinicalTrials.gov — 34 trial records for Treosulfan. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Treosulfan compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Treosulfan. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA214759). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Grafapex. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Medexus Pharma, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Approved[1]
Company: MMedexus Pharma, Inc.

Patent Landscape[5]

Patent Cliff

Oct 12, 2026

Earliest patent expiration — generic entry possible after this date

Patent #Expires

7199162Oct 12, 2026

Grafapex

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q2 2028

Q3 2028

Q1 2029

Trials by Indication[1]

Nijmegen breakage syndrome3 trials

Acute Lymphoblastic Leukemia2 trials

Children2 trials

Hematological Malignancies2 trials

Myelodysplastic Syndrome2 trials

Stem Cell Transplant1 trial

Acute Myeloid Leukaemia (AML)1 trial

Malignant Disease1 trial

Allogeneic Stem Cell Transplantation1 trial

Clinical Trial1 trial

Ocular Melanoma1 trial

Bone Marrow Transplant1 trial

Treatment1 trial

Allogeneic Hematopoietic Stem Cell Transplantation1 trial

Renal Impairment1 trial

Conditioning1 trial

Ewing sarcoma1 trial

Ovarian Cancer1 trial

Lymphoid Malignancies1 trial

Elderly Patients1 trial

Cancer1 trial

Malignancies1 trial

Transplantation1 trial

Lymphoid1 trial

Acute Lymphoblastic Leukaemia1 trial

AML - Acute Myeloid Leukemia1 trial

Acute Myeloid Leukemia1 trial

Unspecified1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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