Acute myeloid leukemia (AML) is a highly heterogenous blood cancer that has a diverse array of underlying causes. Owing to this, intensive chemotherapy has historically been the default approach, which has serious limitations resulting from toxicities. To address this serious unmet medical need, drug developers continue to pursue a myriad of innovative approaches to treat AML, including bispecific antibodies, cell therapies and cancer vaccines. There are several innovative therapies in development that we believe may show promise.
The relatively recent approvals of new classes of targeted therapies have evolved the treatment landscape for AML; BCL-2, FLT3 and ID-H1/2 inhibitors have had an important impact on patient survival, and their use is now commonplace in AML treatment. Accordingly, worldwide sales of these classes of drugs are estimated to grow considerably over the coming years (Exhibit 1), with the total AML treatment market growing at a c 32% CAGR from 2022 to 2028 to US$10.1bnEvaluatePharma). This highlights the opportunity for novel therapies in the space. Despite this success, however, the diverse nature of AML biology means large portions of AML patients are left underserved.
Exhibit 1: Forecasted global AML drug sales
Source: Edison Investment Research. Note: Forecasts are attributable to consensus estimates compiled by EvaluatePharma
The development pipeline for AML continues to be an innovative space, driven by the heterogeneity of the disease and the limited success of certain treatment modalities commonly used in other oncology indications, for example checkpoint inhibitors. The heterogeneity of AML is such that recently approved drugs, like BCL2s, FLT3s and IDH-1/2 inhibitors are not suitable or effective for many AML patients, leaving the room for other innovative approaches. Therefore, a range of different approaches are in development for the treatment of AML, including cutting-edge therapies like bispecific antibodies, CAR T-cell therapies, epigenetic drugs and cancer vaccines. Furthermore, the identification of at-risk patient populations and the need for effective maintenance therapies to combat relapse have led to a more holistic view of AML therapy, beyond first-line intensive chemotherapy.