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Novel Agent Slows High-Risk Myelofibrosis in Initial Trial

Published: Monday, Dec 19, 2016

Naveen Pemmaraju, MD

Naveen Pemmaraju, MD

A third of patients with previously treated, high- and intermediate-risk myelofibrosis had objective responses to the investigational Second mitochondria-derived activator of caspases (SMAC) mimetic LCL161, results of a small phase II trial showed.

Median overall survival has yet to be reached, Pemmaraju and colleagues reported.


  1. Infante JR, Dees EC, Olszanski AJ, et al. Phase I dose-escalation study of LCL161, an oral inhibitor of apoptosis proteins inhibitor, in patients with advanced solid tumors. J Clin Oncol. 2014;32(28):3103-3110. doi: 10.1200/JCO.2013.52.3993.
  2. Fleischman AG, Aichberger KJ, Luty SB, et al. TNFα facilitates cloncal expansion of JAK2V617F positive cells in myeloproliferative neoplasms. Blood. 2011;118(24):6392-6398. doi: 10.1182/blood-2011-04-348144.
  3. Pemmaraju N, Carter BZ, Kantarjian H, et al. Results for phase II clinical trial of LCL161, a SMAC mimetic, in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF) or post-essential thrombocytosis myelofibrosis (post-ET MF). Paper presented at: 58th Annual ASH Meeting; December 3-6, 2016; San Diego, CA. Abstract 3105.

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