2023 ASH annual meeting

Ibrahim Aldoss (City of Hope National Medical Center, Duarte, CA, USA) and colleagues presented results from the AUGMENT-101 trial (NCT04065399) testing revumenib, an inhibitor of the menin–KMT2A interaction. 94 patients aged 30 days or older with relapsed or refractory KMT2A-rearranged (KMT2Ar) acute leukaemia received revumenib at 163 mg (or 95 mg/m2 if bodyweight was <40 kg), orally every 12 h in 28-day cycles. Median age was 37·0 years (range 1·3–75·0); 78 (83%) had acute myeloid leukaemia (AML) and 16 (17%) had acute lymphocytic leukaemia (ALL) or mixed phenotype acute leukaemia. After a median follow-up of 6·1 months, the planned interim analysis showed that 13 (22·8%, 95% CI 12·7–35·8) of 57 patients had a complete response (CR) or CR with partial haematological recovery surpassing the planned cutoff for positivity of the primary endpoint. Grade 3 or worse treatment-related adverse events (AEs) were observed in 51 (54%) of 94 patients, the most common being differentiation syndrome (16%), febrile neutropenia (14%), and QTc prolongation (14%). 2023 ASH Annual MeetingDaniel Wolff (University Hospital Regensburg, Regensburg, Germany) and colleagues presented results from the phase 2 AGAVE-201 trial ( NCT04710576 ), testing three doses of the anti-CSF-1R monoclonal antibody axatilimab (0·3 mg/kg every 2 weeks, 1 mg/kg every 2 weeks, or 3 mg/kg every 4 weeks) to treat chronic graft-versus-host disease (GVHD). 241 patients with relapsed or refractory chronic GVHD were randomly allocated (1:1:1) to the three dose cohorts; the primary endpoint of overall response rate in the first six cycles (24 weeks) was 74% (95% CI 63–83) at 0·3 mg/kg, 67% (55–77) at 1 mg/kg, and 50% (39–61) at 3 mg/kg. Full-Text PDF