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NPM1m Acute Myeloid Leukemia

NPM1-mutant (NPM1m) AML, which is distinguished by mutations in the NPM1 gene that drive the leukemic phenotype, is the most common type of cytogenetically normal adult acute myeloid leukemia (AML).

Similar to KMT2A-rearranged acute leukemia, NPM1m AML is highly dependent on the expression of specific developmental genes shown to be negatively impacted by inhibitors of the menin-KMT2A interaction. NPM1m AML is routinely diagnosed through currently available screening techniques. There are currently no approved therapies indicated for NPM1m AML.

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<em>NPM1</em>-mutant (<em>NPM1m</em>) AML, which is distinguished by mutations in the <em>NPM1</em> gene that drive the leukemic phenotype, is the most common type of cytogenetically normal adult acute myeloid leukemia (AML).

NPM1 mutations are found in ~30% of patients with AML1,2

<50% 5-year overall survival3

Relapsed/refractory NPM1m AML is an acute leukemia with a critical unmet need
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My whole world changed when I received my diagnosis for acute leukemia. I had myeloblasts in 96% of my bone marrow.  I needed to get treatment right away. However, after two months of two different treatments with little response, I felt myself losing hope.

 

I asked my doctor what would happen if I failed my third treatment. He looked at me and said that people receive poor prognoses every day.  I sat there, dumbstruck— and I was only 25.  So, I left the hospital that very day and ultimately began my clinical trial experience.”

Connor, Patient 

References:

  1. Ranieri R, et al. Leukemia. 2022;36(10):2351-2367.
  2. Arber DA, et al. Arch Pathol Lab Med . 2017;141:1342-1393.
  3. Stein E, Aldoss I, DiPersio J, et al. Slides presented at the American Society of Hematology Annual Meeting, Atlanta, GA (2021, December).