ASH 2025 | Fedratinib plus CC-486, an oral HMA, in accelerated-phase myelofibrosis: the FAMy trial

The prognosis of patients with myelofibrosis who progress to the accelerated phase usually is very bad because the accelerated phase is then followed by the blast phase and we don’t have really a standard therapy for these patients. Allogeneic stem cell transplantation remains the only option, but this is only eligible for a small group of patients because of the age, because of comorbidities, and that’s why we don’t have a standard of therapy for the majority of our patients. And the combination of a JAK inhibitor plus the hypomethylating agent, for example, azacitidine, in combination with ruxolitinib, which we all know, has been used in phase 1, phase 2 trials in patients with accelerated and blast phase MPN, and they seem to have the best results. 

And in this trial, we chose fedratinib because it has additional function in addition to being a JAK inhibitor, and we use the combination with an oral hypomethylating agent that is already approved for AML maintenance therapy after induction chemotherapy to allow patients to have oral therapies they can take at home. And this was an academic-initiated trial in Germany, multi-center. And we have included several patients in a dose-level escalation. And we present the data. The tolerability is quite good. And we still have patients after two years going on with the trial. But at the end of the day, we had to stop enrollment because we had too few, thank God, patients with accelerated phase. So this seems that with the available therapies, the JAK inhibitors, we are having, and this is what we want, fewer cases of very advanced MPN.

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