VIDEO: 'Robust' pipeline of developing treatments for AML

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Despite the approval of, at this point, nine or 10 new therapies for acute myeloid leukemia since 2017, we continue to have an incredibly robust pipeline of developing agents. There is the advent of a potentially new class of targeted therapies for acute myeloid leukemia and those are the menin inhibitors for treatment of two biological subsets, patients with KMT2A or MLL rearranged acute leukemias and AML patients with NMP1 mutations which represent about a third of newly diagnosed cases.

In early trials, multiple menin inhibitors have demonstrated clinical efficacy with response rate about 20% to 30% in heavily pretreated patients. And clinical trials are embarking with the incorporation of menin inhibitors into the upfront setting.

Although we haven't seen as much development of immunotherapy as we would like for the myeloid malignancies, we are starting to see glimmers of hope with the development of bispecific antibodies targeting antigens on AML cells as well as activating CD3 T cells. And we're also seeing development of CAR T cells targeting specific antigens on those AML cells. Although we haven't seen any breakthrough technologies in the bispecifics or CAR T, right now for myeloid malignancy is we're optimistic that some of these more novel approaches might finally bear fruit in the next couple of years.

Lastly, we've seen antibody drug conjugates make a resurgence. We obviously have gemtuzumab ozogamicin [Mylotarg, Pfizer], which is a CD33 antibody drug conjugate approved for combination upfront therapy with intensive chemotherapy. We are now seeing newer agents such as CD123 antibody drug conjugates starting to make an impact potentially in combination regimens.