Turn on cookies to sign in
Signing in saves your progress to your Curie account. We can only do that with cookies on — turn them on to continue.
The FDA has granted breakthrough therapy designation to enzomenib (DSP-5336), an oral menin inhibitor, for patients with relapsed/refractory acute leukemia carrying a KMT2A rearrangement. This designation enables expedited FDA review for a patient population with very limited treatment options and poor outcomes. The drug is currently being evaluated in the phase 1/2 Horizen-1 trial.
The FDA has granted breakthrough therapy designation to enzomenib (DSP-5336), an investigational oral drug developed by Sumitomo Pharma America, for patients with relapsed/refractory (R/R) acute leukemia harboring a KMT2A rearrangement — a genetic alteration associated with particularly poor outcomes. The designation is designed to speed up the drug's development and review process, potentially getting it to patients faster.
Enzomenib works by blocking the interaction between two proteins — menin and KMT2A — that drive leukemic cell growth. In preclinical studies, it selectively inhibited the growth of leukemia cell lines with KMT2A rearrangements or NPM1 mutations, while also promoting cell differentiation. The drug is now being evaluated as monotherapy in the registrational phase 2 portion of the Horizen-1 trial (NCT04988555), which spans sites across North America, Europe, and Asia.
Key Takeaways:
Why it matters: Patients with R/R KMT2A-rearranged acute leukemia have very few effective treatment options and face grim prognoses. Breakthrough therapy designation signals the FDA's recognition of enzomenib's potential and could meaningfully shorten the timeline to approval.
