In short
Enzomenib (DSP-5336) is an investigational oral medication being studied in acute leukemia. It targets specific genetic changes in leukemia cells, namely MLL rearrangements and NPM1 mutations. One registered trial, a Phase 1/2 study with 498 participants, is currently authorised to evaluate its safety and clinical activity.
Key points
- Enzomenib (DSP-5336) is an investigational oral medication for acute leukemia.
- It is being studied in Phase 1/2 trials for relapsed or refractory acute leukemia with MLL rearrangement or NPM1 mutation.
- The registered trial plans to enroll 498 participants.
- The trial status is Authorised, meaning it has received regulatory approval to proceed.
- Phase 1 focuses on safety and tolerability, while Phase 2 evaluates clinical activity.
- Enzomenib is given as a tablet taken by mouth.
What is enzomenib?
Enzomenib, also known by its development code DSP-5336, is a chemically synthesised substance classified under the therapeutic area of neoplasms. It is an investigational medicine, meaning it has no marketing authorisation in the product records and is not yet approved for routine use. The product is supplied by Sumitomo Pharma Oncology, Inc. In the clinical trial records, enzomenib appears as the test substance in two trials.
How does enzomenib work?
The available registry data does not provide a detailed description of enzomenib's mechanism of action. However, the clinical trial focuses on patients with acute leukemia carrying specific genetic alterations: mixed lineage leukemia (MLL) rearrangements or nucleophosmin 1 (NPM1) mutations. These genetic changes are known to drive the growth of leukemia cells, and the trial is designed to test whether enzomenib can target leukemia cells harbouring these alterations.
Which conditions is enzomenib used or studied for?
- Acute myeloid leukemia (AML) with NPM1 mutation — a type of blood cancer where a specific gene mutation drives the disease; the Phase 2 part of the trial includes patients with relapsed or refractory AML with an NPM1 mutation.
- Acute leukemia with MLL rearrangement — a group of acute leukemias, including AML, acute lymphoblastic leukemia (ALL), and acute leukemia of ambiguous lineage, that carry a rearrangement of the MLL gene; the Phase 2 part includes patients with relapsed or refractory acute leukemia with an MLL rearrangement.
How is enzomenib given?
Enzomenib is formulated as a tablet and is given by mouth (oral administration). The trial interventions list enzomenib as an oral drug.
What do the clinical trials show?
One trial of enzomenib is registered in the EU Clinical Trials Information System (CTIS) with the identifier 2022-502741-10-00. It is a Phase 1/2 interventional study with a planned enrollment of 498 participants. The trial is currently authorised, meaning it has received regulatory approval to proceed.
The study is divided into two parts. Phase 1 aims to assess the safety and tolerability of enzomenib monotherapy in patients with relapsed or refractory AML, ALL, or acute leukemia of ambiguous lineage, and to determine the recommended Phase 2 dose (RP2D). Phase 2 aims to evaluate the clinical activity of enzomenib monotherapy in patients with relapsed or refractory acute leukemia with an MLL rearrangement or relapsed/refractory AML with an NPM1 mutation.
What is measured in the trials?
In Phase 1, the primary outcomes include safety assessments such as dose-limiting toxicities (DLTs) assessed by the NCI CTCAE version 5.0, treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), changes in vital signs, clinical laboratory values, electrocardiogram (ECG) parameters, and echocardiogram (ECHO) parameters. Tolerability is measured through dose interruptions, dose reductions, and dose discontinuations. In Phase 2, the primary outcome is complete remission (CR) plus complete remission with partial hematologic recovery (CRh).
Who can take part in these trials?
The trial includes patients with relapsed or refractory acute leukemia. Phase 1 includes patients with AML, ALL, or acute leukemia of ambiguous lineage. Phase 2 focuses specifically on patients with relapsed or refractory acute leukemia with an MLL rearrangement or relapsed/refractory AML with an NPM1 mutation.
What side effects are known?
The available sources do not provide a list of known side effects for enzomenib. The Phase 1 part of the registered trial is specifically designed to assess safety and tolerability, including dose-limiting toxicities and treatment-emergent adverse events.