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News
A new weapon against leukemia enters the clinic. Amphista Therapeutics has dosed the first patient in its BRAMLIE Phase 1 trial of AMX-883, a first-in-class BRD9 protein degrader targeting acute myeloid leukemia (AML) and high-risk MDS. Unlike existing targeted therapies, AMX-883 shows activity across diverse AML subtypes regardless of mutation status, potentially addressing a major gap in treatment.
A new weapon against leukemia enters the clinic
Amphista Therapeutics has dosed the first patient in its BRAMLIE Phase 1 trial of AMX-883, a first-in-class oral BRD9 protein degrader for acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). The trial, authorized by the FDA in June 2026, will evaluate the safety, pharmacokinetics, and preliminary efficacy of AMX-883 monotherapy, with a subsequent combination study planned in relapsed/refractory AML.
What makes AMX-883 stand out is its mutation-agnostic activity. Current targeted AML therapies only work in patients with specific genetic mutations, leaving many without effective options. AMX-883 uses a novel "Targeted Glue™" mechanism to recruit the E3 ligase DCAF16, rapidly degrading BRD9 — a protein that sustains leukemic stem cells and blocks normal myeloid differentiation — across diverse AML subtypes.
By the Numbers
Why it matters: With resistance to standard-of-care agents like venetoclax on the rise, AMX-883 represents a potentially broad-acting new treatment pathway for one of the most aggressive and deadly blood cancers.
