MOMELOTINIB’S SPLEEN, SYMPTOM AND ANEMIA EFFICACY IS MAINTAINED
POSTER 12
IN INTERMEDIATE/HIGH RISK MYELOFIBROSIS PATIENTS WITH
THROMBOCYTOPENIA
Jean-Jacques Kiladjian1, Uwe Platzbecker2, Jiri Mayer3, Árpád Illés4, Witold Prejzner5, Tomasz Wozny6,
Doroteya Todorieva7, Alessandro M. Vannucchi8, Ilya Kirgner9, Zsolt Nagy10, Sebastian Grosicki11, Åsa
Derolf12, Mihaela Cornelia Lazaroiu13, Sung-Soo Yoon14, Yeow Tee Goh15, Nikolas von Bubnoff16, Srdan
Verstovsek17, Barbara Klencke18, Rafe Donahue18 and Ruben Mesa19
(1)Hôpital Saint-Louis & Université de Paris, Paris, France
(2)Leipzig University Hospital, Leipzig, Germany
(3)Central European Institute of Technology (CEITEC), Masaryk University, Brno, Czech Republic,
(4)Doctoral School of Clinical Medicine, University of Debrecen, Debrecen, Hungary
(5)Department of Hematology and Transplantology Medical University of Gdansk, Gdansk, Poland,
(6)Department of Hematology Szpital MSWiA w Poznaniu, Poland, Poznań, Poland
(7)UMHAT Pleven, Pleven, Bulgaria
(8)CRIMM, Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi,
Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
(9)Division of Hematology Sackler Faculty of Medicine Tel Aviv University, Tel Aviv, Israel
(10)1st Department of Internal Medicine, Semmelweis University, Budapest, Hungary
(11)Department of Cancer Prevention, School of Public Health, Silesian Medical University, Katowice,
Poland
(12)Department of Medicine, Division of Hematology, Karolinska University Hospital Solna, Karolinska
Institutet, Stockholm, Sweden
(13)Policlinica de Diagnostic Rapid Brasov, Brasov, Romania
(14)Seoul National University Hospital, Seoul, Korea, Republic of (South)
(15)Department of Haematology, Singapore General Hospital, Singapore, Singapore
(16)Department of Hematology and Oncology, Medical Center, University of Schleswig Holstein,
Lübeck, Germany
(17)Department of Leukemia, MD Anderson Cancer Center, Houston, TX
(18)Sierra Oncology Inc., Vancouver, Canada
(19)UT Health San Antonio Cancer Center, San Antonio, TX
Momelotinib (MMB), is a JAK1, JAK2 and ACVR1 inhibitor, which has shown activity in
myelofibrosis (MF), a condition marked by splenomegaly, constitutional symptoms and
progressive anemia and thrombocytopenia. MMB restores iron homeostasis and red blood
cell production, reducing transfusions and can also improve or maintain platelet (PLT) counts.
Consistent with MMB’s biological profile, low myelosuppressive potential and favorable
hematological tolerability, sustained high dose intensity of MMB was maintained in >85% of
patients treated in the previously completed SIMPLIFY studies. Here we report post-hoc
comparative efficacy analyses for spleen, symptom and transfusion independence (TI)
response by baseline PLT strata in patients from these global Phase 3 studies. SIMPLIFY-1 (S1)
was double-blind in intermediate/high risk JAKi-naïve patients with MF randomized 1:1 to
MMB or ruxolitinib (RUX) over a 24-week treatment period. SIMPLIFY-2 (S2) compared MMB
to best available therapy (BAT; 88% of which was RUX) in previously RUX-exposed patients
with MF. Baseline PLTs ≥50 × 109/L were required in S1, while there was no lower PLT limit for
S2.