In short
AZD2962 is an investigational drug being studied in blood cancers, specifically myelodysplastic syndromes (MDS) and dysplastic chronic myelomonocytic leukaemia (CMML). It belongs to a class of medicines called IRAK4 inhibitors, which target a protein involved in inflammation. One completed Phase 1/2 clinical trial enrolled 77 participants to assess its safety and tolerability.
Key points
- AZD2962 is an investigational IRAK4 inhibitor with no marketing authorisation.
- It is being studied in myelodysplastic syndromes and dysplastic chronic myelomonocytic leukaemia.
- The single registered trial is Phase 1/2 and is now completed.
- The trial enrolled 77 participants across multiple dose levels.
- It is given as a film-coated tablet taken orally.
- The trial measured dose-limiting toxicities and safety as primary outcomes.
What is AZD2962?
AZD2962 is a chemically synthesised substance that acts as an IRAK4 inhibitor. IRAK4 stands for interleukin-1 receptor-associated kinase 4, a protein involved in immune signalling and inflammation. AZD2962 is an investigational substance, meaning it has no marketing authorisation in the product records and is not yet approved as a medicine. The product is supplied by AstraZeneca AB. It is also referred to simply as AZD2962, which is both its registry name and product name.
How does AZD2962 work?
AZD2962 works by blocking the activity of IRAK4, a protein that plays a central role in inflammatory signalling within cells. In blood cancers such as myelodysplastic syndromes, inflammation in the bone marrow can help abnormal blood cells survive and grow. By inhibiting IRAK4, researchers hope to reduce this inflammation and potentially interfere with the survival of abnormal blood cells.
Which conditions is AZD2962 studied for?
- Myelodysplastic syndromes (MDS) — blood disorders where the bone marrow does not produce enough healthy blood cells.
- Dysplastic chronic myelomonocytic leukaemia (CMML) — a related blood cancer with features of both myelodysplastic and myeloproliferative disorders.
How is AZD2962 given?
AZD2962 is administered as a film-coated tablet taken by mouth (oral route). The trial tested multiple dose levels, starting with a small dose and increasing stepwise once each dose level was shown to be safe.
What do the clinical trials show?
One completed Phase 1/2 clinical trial (EU CT number 2025-520786-44-00) evaluated AZD2962 in 77 participants with haematologic neoplasms, specifically myelodysplastic syndromes and dysplastic chronic myelomonocytic leukaemia. The trial was open-label and multicentre, meaning both participants and researchers knew which treatment was being given, and the study took place at multiple sites.
The primary outcomes focused on safety and tolerability. These included the incidence of dose-limiting toxicities (DLTs) during the observation period, the incidence and severity of adverse events and serious adverse events, changes in laboratory values, vital signs, physical examinations and ECG results, and the duration of exposure and relative dose intensity. The study aimed to identify the optimal biological dose, which is the dose level that provides the intended biological effect while remaining tolerable.
The trial began with a first dose level given to a limited number of participants. The dose was increased to the next level only once the current level was considered safe. This process continued until either the highest planned dose level was reached or the dose was not tolerated. Additional participants could be added to a dose level to gather more information.
What side effects are known?
The registered trial measured the incidence, severity and relationship to the investigational medicinal product of adverse events and serious adverse events as primary outcomes. However, the available sources do not report specific side effects that occurred during the study, so no list of known side effects can be provided.