Momelotinib is a potent, selective and orally-bioavailable JAK1, JAK2 & ACVR1 inhibitor with a differentiated therapeutic profile in myelofibrosis encompassing a range of meaningful anemia benefits, including eliminating or reducing the need for frequent blood transfusions, as well as achieving substantive spleen and constitutional symptom control.
Momelotinib has been investigated in two completed Phase 3 trials for the treatment of myelofibrosis. More than 1,200 subjects have received momelotinib since clinical studies began in 2009. Momelotinib is covered by patents anticipated to provide exclusivity to 2035 in the U.S. and 2033 in the EU.
Myelofibrosis: A Chronic Myeloproliferative Neoplasm (MPN)
Momelotinib Potentially Addresses the Key Needs in Myelofibrosis
The majority of myelofibrosis patients have anemia at diagnosis or develop it during treatment with other therapies. Anemia is the most significant negative prognostic indicator in myelofibrosis patients and, as a result, one of the most important disease consequences to address.
The therapeutic focus in myelofibrosis has traditionally been on treating the enlarged spleen and constitutional symptoms common to the disease; however, optimal drug therapy would also address disease-related cytopenias, including anemia and transfusion dependency, while also improving splenomegaly and symptoms.
No other JAKi has demonstrated a broader ability to address the needs of myelofibrosis patients. Only momelotinib has robust spleen, symptom and anemia benefits.
Anemia in Myelofibrosis: Momelotinib’s Unique Mechanism of Action
Momelotinib has repeatedly demonstrated a broad range of clinically relevant anemia-related effects:
- High rates of transfusion independence, either via conversion from transfusion dependency, or maintenance of baseline independence.
- Absolute Hgb increase and decreased supportive Hgb requirements.
- Low rates and severity of hematologic adverse events.
The Phase 2 and Phase 3 data to date demonstrate that momelotinib consistently improves the anemia that frequently occurs in advanced myelofibrosis, postulated via ACVR1 inhibition. ACVR1 is a member of the TGFβ superfamily of receptors that regulate the iron metabolism pathway. ACVR1 activates the transcription of hepcidin, which leads to decreased erythropoiesis.