Potential Myelofibrosis Treatment Met Phase 3 Study Endpoints
California-based Sierra Oncology, Inc. today announced positive topline data from the pivotal Phase 3 MOMENTUM study evaluating momelotinib (MMB) in myelofibrosis patients who are symptomatic and anemic and previously treated with an approved JAK inhibitor.
The trial met all of its primary and key secondary endpoints. The Topline data announced based on 195 patients (MMB n = 130; DAN n = 65) include:
- Primary Endpoint of Total Symptom Score (TSS) of >50%: 25% in the MMB arm vs. 9% in the control arm (p=0.0095)
- Secondary Endpoint of Transfusion Independence (TI): 31% in the MMB arm vs. 20% in the control arm (one-sided p=0.0064; non-inferiority)
- Secondary Endpoint of Splenic Response Rate (SRR) >35%: 23% in the MMB arm vs. 3% in the control arm (p=0.0006)
- The rate of Grade 3 or worse adverse events in the randomized treatment period was 54% in the MMB arm and 65% in the control arm. Serious treatment-emergent adverse events were 35% in the MMB arm and 40% in the control arm.
- Mean baseline characteristics for all patients were TSS of 27, Hemoglobin (Hgb) of 8 g/dL, and platelet count of 145 x 10 9/L
"As a clinician, I am thrilled to see data that confirm the potential of momelotinib as a treatment option for myelofibrosis patients who are anemic or at risk of becoming anemic," said Ruben Mesa, MD, FACP, Executive Director of the Mays Cancer Center, home to UT Health San Antonio, MD Anderson Cancer Center, and co-Principal Investigator of the study, in a press statement.
"Anemia of myelofibrosis is strongly correlated with reduced quality of life and a decrease in overall survival."
"Half of all myelofibrosis patients present with anemia at diagnosis, and virtually all become anemic over time."
"With currently approved therapies being myelosuppressive, it's wonderful to know that we may soon have such an effective treatment option for these patients."
Myelofibrosis is a rare blood cancer that results from dysregulated JAK-STAT signaling and is characterized by constitutional symptoms, splenomegaly (enlarged spleen), and progressive anemia.
The full data set will be presented at an upcoming medical meeting, says the Company.
Sierra Oncology is a late-stage biopharmaceutical company located in San Mateo, CA, on a mission to deliver targeted therapies that treat rare forms of cancer.