ASH 2022 | Long-term safety of momelotinib in myelofibrosis: pooled data from three Phase III randomized trials

Momelotinib is the JAK1/JAK2, and ACVR1 inhibitor. And through the inhibition of these three markers, it provides a benefit to the patients in three different ways. Through JAK1 and JAK2 improve the spleen and symptoms and through ACVR1 inhibition, which would lead to a decrease in hepcidin, it appears to be able to improve the anemia. So it’s pretty valuable because we don’t have the drugs or available therapies, approved therapies that will be able to provide all this together. It has been in clinical studies, momelotinib has been for quite some time to try to provide to regulatory bodies proof that it should be out there for using in a regular community setting. So we needed to do several Phase III studies to get it finally in a situation where it’s under review by the FDA United States for possible approval by next summer.

And so SIMPLIFY studies, SIMPLIFY-1 and SIMPLIFY-2 studies focused primarily on the control of spleen and symptoms. And SIMPLIFY-1 was a frontline study comparing it to ruxolitinib. And the SIMPLIFY-2 was a Phase III study in patients that were broadly intolerant to ruxolitinib. And in all these studies and now in the Phase III randomized study called MOMENTUM, which is the full-fledged second-line study after ruxolitinib comparing the momelotinib to danazol anabolic steroid, we see the same trend. And that is that there is, regardless of the phase of the disease, a good control of the spleen symptoms and anemia. And anemia benefit is unique. And that coupled with improving quality of life, anemia and quality of life may actually lead to its approval. Now having that experience with so many patients over many years, it was important to show and that’s what the presentation is, that it’s safe.

And the dose intensity by the end large stays very high. 95 or 96% of the patients stay on the assigned dose without need for much those adjustments, at least from the last MOMENTUM to study, which is very good sign. Because not only that we want to have a patient that have a benefit from new therapy, we want that therapy to be safe and simple. And so safer means no dose adjustments necessary because there are no safety concerns and that is equal then to simplicity because you don’t need to worry much about it. So the characteristics of this drug, if it gets approved, will certainly enhance its utility in management of the patients in community setting. Providing simplicity, safety, and efficacy over a much longer period of time as this presentation tells us.