Syndax Pharmaceuticals (SNDX) and Incyte (INCY) announced that results from the Phase 1/2 trial of axatilimab, Syndax’s anti-CSF-1R antibody, in patients with recurrent or refractory chronic graft-versus-host disease following two or more prior lines of therapy, were published in the Journal of Clinical Oncology. A total of 40 patients with refractory disease who received a median of four prior systemic therapies, some of which included ibrutinib, ruxolitinib and belumosudil, were treated in the Phase 1/2 dose escalation trial. Thirty-nine patients were evaluable for response as of the data cutoff. All study participants were treated for a median duration of 29 weeks, with all responding patients treated for a median duration of 38 weeks at the time of data cut-off. Results showed: Overall response rate by cycle 7 day 1 was 82% in the Phase 2 cohort and 67% among all evaluable patients treated in the dose escalation study. Best ORR observed at any point during the study in all patients treated was 69%. Median duration of response for Phase 2 responding patients was not reached; 33% of patients experiencing sustained response lasting 20 weeks or longer. A decrease in glucocorticoids doses was observed in 52% of responding patients. Responses were observed across a range of organ systems with difficult to treat manifestations such as lung, skin, and joints and fascia. A clinically meaningful disease improvement using the Lee Symptom Score of at least 7 points was seen in 58% of all evaluable patients. Axatilimab was well tolerated with a favorable safety profile in this refractory population. The most common adverse events were consistent with on-target effects of CSF-1R inhibition. In the Phase 1 cohort, two dose limiting toxicities were reported, both at the 3 mg/kg every two weeks dose. There were no greater than or equal toGrade 3 on-target toxicities of CSF-1R blockade seen in the Phase 2 cohort. There was no incidence of cytomegalovirus or other viral reactivation, and no apparent increases in risk for infection. Serious adverse events occurred in 40% of patients, with seven patients discontinuing the study intervention due to adverse events, four of which were deemed treatment-related.
Published first on TheFly
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