Amphista doses first patient in AML trial of protein degrader

pharmafile | October 20, 2026 | News story | |  AML, FDA, Oncology, clinical trial, drug development, pharma 

Amphista Therapeutics has dosed the first patient in a phase 1 trial of AMX-883, an experimental oral therapy targeting acute myeloid leukaemia (AML) and high-risk myelodysplastic syndromes (MDS).

The BRAMLIE trial will assess the safety, pharmacokinetics and preliminary efficacy of AMX-883 as a monotherapy in approximately 54 participants. The US Food and Drug Administration authorised the study in June 2026.

AMX-883 is a targeted protein degrader designed to remove BRD9, a component of a chromatin-remodelling complex involved in maintaining leukaemic stem cells and preventing myeloid cell differentiation. The drug recruits the E3 ligase DCAF16 to trigger BRD9 degradation.

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Amphista said preclinical studies showed activity across diverse AML subtypes, independently of mutation status. The company plans to investigate the candidate in combination with existing treatments in patients with relapsed or refractory AML, followed by potential evaluation in earlier treatment settings.

Dr Eytan Stein, Lead Investigator for the trial and Director of the Program for Drug Development in Leukemia at Memorial Sloan Kettering Cancer Center, said there was an urgent need for new treatments, particularly as resistance to established therapies such as venetoclax emerges.

AMX-883 is an early-stage candidate, and the phase 1 study will provide the first clinical assessment of its safety and pharmacological profile.

Pic: RDNE stock project

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