In short
CHM-029 is an investigational small-molecule menin inhibitor being developed for acute myeloid leukaemia (AML). One clinical trial is currently authorised in the United Kingdom, a phase 1/2 study that has not yet started recruiting, with a planned enrolment of 6 participants. The trial will test CHM-029 alone and in combination with standard AML treatments to assess safety and determine the optimal dose.
Key points
- CHM-029 is an investigational menin inhibitor being studied in acute myeloid leukaemia (AML).
- The only registered trial is a phase 1/2 study currently authorised in the United Kingdom.
- The trial plans to enrol 6 participants with newly diagnosed or relapsed/refractory AML.
- The main goal is to assess safety and tolerability and find the maximum tolerated dose (MTD) or optimal biological dose (OBD).
- CHM-029 will be tested as monotherapy and in combination with azacitidine, cytarabine, venetoclax, daunorubicin or posaconazole.
- The substance is classified as an antineoplastic and a new molecular entity.
What is CHM-029?
CHM-029 is an investigational small-molecule drug classified as an antineoplastic, meaning it is being developed to treat cancer. It is a new molecular entity, which means it has not previously been approved as a medicine in any form. The substance is also known by the alternative name CHM-029, and its originator is CHARM Therapeutics. CHM-029 has not received orphan drug designation and currently has no marketing authorisation; it remains an investigational agent in early clinical development.
How does CHM-029 work?
CHM-029 belongs to a class of drugs called menin inhibitors. Menin is a protein that interacts with other proteins involved in the development of certain leukaemias, particularly acute myeloid leukaemia. By blocking menin, CHM-029 is designed to interfere with the molecular processes that drive the growth and survival of leukaemia cells. The precise impact of this inhibition in patients is what the ongoing clinical trial aims to establish.
Which conditions is CHM-029 studied for?
- Acute myeloid leukaemia (AML) — a cancer of the blood and bone marrow in which abnormal white blood cells grow quickly and crowd out normal blood cells. The registered trial includes patients who are newly diagnosed as well as those whose disease has come back (relapsed) or has not responded to previous treatment (treatment-refractory).
How is CHM-029 given?
CHM-029 is given by mouth as an oral formulation. In the registered trial, the oral form of CHM-029 is being studied both on its own and in combination with other cancer medicines that are given by mouth or by infusion into a vein.
What do the clinical trials show?
One clinical trial of CHM-029 is registered in the EU Clinical Trials Information System and on ClinicalTrials.gov. The study, with the identifier NCT07751991, is a phase 1/2 trial currently authorised in the United Kingdom. It plans to enrol 6 participants with acute myeloid leukaemia. The trial is interventional, meaning participants receive the study drug as part of the research.
The primary goal of the trial is to assess the safety and tolerability of CHM-029. Researchers will record the incidence and severity of adverse events using the National Cancer Institute Common Terminology Criteria for Adverse Events, version 6.0, as well as the incidence of dose-limiting toxicities. They will also measure changes in clinical laboratory parameters and record any dose interruptions, dose modifications or discontinuations due to adverse events. A further aim is to determine the optimal biological dose in the first cohort of participants.
The trial is designed to determine the maximum tolerated dose and/or the optimal biological dose of CHM-029 when given as monotherapy and when combined with standard-of-care therapies for AML. The combination partners listed in the trial record include azacitidine, cytarabine, venetoclax, daunorubicin and posaconazole. Participants may have newly diagnosed AML or relapsed or treatment-refractory AML.
What side effects are known?
The AdisInsight drug profile lists an "Adverse events" section for CHM-029, but the available source excerpt does not provide specific side-effect data. Because CHM-029 is still in early clinical development, the safety information is being collected in the ongoing phase 1/2 trial. No detailed side-effect profile can yet be reported.