Date: 2014-10-10
Type of information: Presentation of results at a congress
phase: 1
Announcement: presentation of results at the 56th annual meeting of the American Society of Hematology (ASH), to be held December 6-9 in San Francisco, Calif.
Company: Epizyme (USA - MA)
Product: EPZ-5676
Action
mechanism: EPZ-5676 is a small molecule inhibitor of DOT1L created with Epizyme\'s proprietary product platform, for the treatment of patients with acute leukemia in which the MLL gene is rearranged due to a chromosomal translocation (MLL-r) or a partial tandem duplication (MLL-PTD). Due to these rearrangements, DOT1L is misregulated, resulting in the increased expression of genes causing leukemia.
Disease: acute lymphoblastic leukemia (ALL) acute myeloid leukemia (AML)
Therapeutic area: Cancer - Oncology
Country: Germany, The Netherlands, USA
Trial
details: This phase 1, open-label, dose-escalation & expanded cohort, continuous IV infusion, multi-center study of the safety, tolerability,PK & PD of EPZ-5676 will determine the safe dose of EPZ-5676, to evaluate the safety of EPZ-5676 in patients with advanced hematologic malignancies, and to conduct a preliminary assessment of the anti-leukemia activity of EPZ-5676 in patients with acute leukemias bearing rearrangements of the MLL gene. Currently this study is in the MLL-r restricted/expansion phase and is only enrolling patients with rearrangements involving the MLL gene, including 11q23 or partial tandem duplications (PTD). (NCT01684150)
Latest
news: * On October 10, 2014, Epizyme, a clinical stage biopharmaceutical company creating innovative personalized therapeutics for patients with genetically defined cancers, announced that clinical and pre-clinical data on its first-in-class histone methyltransferase (HMT) inhibitors will be highlighted in oral and poster presentations at the 56th annual meeting of the American Society of Hematology (ASH), to be held December 6-9 in San Francisco, Calif. The DOT1L Inhibitor EPZ-5676: Safety and Activity in Relapsed/Refractory Patients with MLL-Rearranged Leukemia: Identification of a First-in-Class PRMT5 Inhibitor with Potent In Vitro and In Vivo Activity in Pre-clinical Models of Mantle Cell Lymphoma. Elayne Penebre , Ph.D., Senior Scientist, Epizyme Pediatric Dose Determinations for the Phase 1 Study of the DOT1L Inhibitor, EPZ-5676, in MLL-r Acute Leukemia: Leveraging Early Clinical Data in Adults through Physiologically-Based Pharmacokinetic Modeling. Nigel Waters , Ph.D., Director, Drug Metabolism and Pharmacokinetics, Epizyme. Epizyme believes that EPZ-5676 was the first HMT inhibitor to enter human clinical development. Epizyme is currently conducting a two-stage Phase 1 study in adult MLL-r and MLL-PTD patients and in May 2014 , initiated a Phase 1b study of EPZ-5676 in pediatric patients with rearrangements of the MLL gene. The adult dose escalation stage has completed enrollment, and the adult MLL-r and MLL-PTD expansion stage is now enrolling patients.
EPZ-5676 has been granted orphan drug designation for the treatment of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) by the FDA and by the European Commission in Europe .
Epizyme retains all U.S. rights to EPZ-5676 and has granted Celgene an exclusive license to EPZ-5676 outside of the U.S.
