On June 11, 2021 Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company on a quest to deliver targeted therapies that treat rare forms of cancer, reported data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting highlighting transfusion independence with momelotinib is associated with improved overall survival, including in patients with anemia at baseline (Press release, Sierra Oncology, JUN 11, 2021, View Source [SID1234583900]). In addition, transfusion independence is seen irrespective of baseline degree of anemia, platelet count or transfusion status.

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"Momelotinib has previously been shown to provide an anemia benefit for myelofibrosis patients, resulting in higher rates of transfusion independence. The data presented at EHA (Free EHA Whitepaper) are particularly exciting because they suggest myelofibrosis patients randomized to momelotinib who achieve or maintain transfusion independence at Week 24 achieve a clinically relevant improvement in overall survival, the gold standard measurement of oncology treatment.," said Barbara Klencke, MD, Chief Medical Officer at Sierra Oncology. "The correlation between Week 24 transfusion independence response and overall survival observed with momelotinib is unique among JAK inhibitors. This suggests that the likelihood of achieving or maintaining transfusion independence may become an important consideration for physicians when recommending myelofibrosis treatment options. With this in mind, we wanted to understand which patients were most likely to maintain or achieve transfusion independence by Week 24. In other data presented at EHA (Free EHA Whitepaper), we report observing higher rates of transfusion independence with momelotinib relative to ruxolitinib, regardless of a patient’s baseline degree of anemia, platelet count or transfusion status."

Transfusion Independence is Associated with Improved Overall Survival in Myelofibrosis Patients Receiving Momelotinib

Efficacy data examining the association between transfusion independence and overall survival for momelotinib patients from SIMPLIFY-1 (JAKi-naïve) and SIMPLIFY-2 (JAKi-exposed) were presented in an oral presentation by Ruben Mesa, MD, Director of the Mays Cancer Center, UT Health San Antonio, MD Anderson Cancer Center. Previously published data demonstrated higher transfusion independence response rates in the momelotinib arms of SIMPLIFY-1 (67% vs. 49%) and SIMPLIFY-2 (43% vs. 21%) compared with ruxolitinib or best available therapy, respectively. Additionally, robust overall survival was observed with extended momelotinib treatment in both JAKi-naïve and JAKi-exposed patients (median not yet reached in SIMPLIFY-1 and 34.3 months in SIMPLIFY-2) as reported at ASH (Free ASH Whitepaper) 2020. These new analyses being presented at EHA (Free EHA Whitepaper) 2021 suggest JAKi-naïve patients receiving momelotinib achieve or maintain transfusion independence (TI) at Week 24 have favorable overall survival compared to non-responders, with a similar trend observed in SIMPLIFY-2.

Key Data Presented

In SIMPLIFY-1 (JAKi-naïve patients)
Patients randomized to momelotinib who were TI responders show a three-year overall survival of 80% compared to 50% for non-responders (HR=0.30; p<0.0001)
Patients with anemia at baseline (RBC transfusion <12 weeks before baseline, or baseline Hgb <10 g/dL) show a similar survival advantage for momelotinib patients who achieve a TI response at Week 24 compared to TI non-responders (HR=0.32; p=0.0006)
In SIMPLIFY-2 (JAKi-exposed patients)
Patients randomized to momelotinib who were TI responders at Week 24 show a trend toward better OS compared to TI non-responders (HR=0.52; p=0.0652)
Improved Transfusion Independence Rates for Momelotinib vs. Ruxolitinib in Anemic JAKi Naïve Myelofibrosis Patients Independent of Baseline Platelet or Transfusion Status

Progressive anemia is a common occurrence in myelofibrosis, with nearly all patients requiring transfusions as their disease advances. As noted in the EHA (Free EHA Whitepaper) oral presentation, JAKi-naïve patients receiving momelotinib who maintain or achieve transfusion independence at Week 24 have favorable overall survival compared to non-responders, with a similar trend observed in SIMPLIFY-2.

Data presented in a poster presentation by Jean-Jacques Kiladjian, MD, PhD, Professor of Clinical Pharmacology, Paris Diderot University and Consultant Hematologist and Head of the Clinical Investigation Center, Saint Louis Hospital, show that the prognostically important Week 24 TI rates in JAKi-naïve myelofibrosis patients were consistently higher in anemic patients receiving momelotinib compared to ruxolitinib in all patient subsets with baseline hemoglobin <14 g/dL and all subsets defined by baseline platelet count and baseline transfusion status. Combined with data from the oral presentation, these data suggest the goal of achieving TI should become an important driver of treatment decisions in myelofibrosis.