Karyopharm Therapeutics Inc. (NASDAQ: KPTI) has doses the first patient in its new second phase trial assessing oral selinexor as a monotherapy relative to physicians’ choice in myelofibrosis patients that have previously received JAK 1/2 inhibitor.
Study to evaluate Selinexor monotherapy
This multicenter, randomized, open-label Phase 2 trial aims to assess the efficacy and safety of selinexor monotherapy. Up to 112 patients will be randomized 1:1 to receive either low-dose, once-weekly oral selinexor or their physician’s conventional treatment (according to clinical practice). The study’s primary objective is the percentage of patients with a 35 percent spleen volume reduction (SVR) from baseline, as determined by an Independent Review Committee. The first patient was dosed by the Lead Principal Investigator, Dr. Alessandro Lucchesi, Sr. Consultant and Research Hematologist, at the Instituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST) Meldola, Italy, under the supervision of Professor Giovanni Martinelli, Chief Scientific Director.
The start of this Phase 2 study comes on the heels of promising preliminary results from the Phase 2 ESSENTIAL study, which will be discussed in an oral presentation by Srinivas Tantravahi, MBBS, MRCP, University of Utah Hospital, at the American Society of Hematology (ASH) 2021 Annual and Exposition on Saturday, December 11, 2021.
Karyopharm committed to addressing myelofibrosis
Chief Scientific Officer and co-founder of Karyopharm Sharon Shacham said, “As part of our strategic imperatives and pipeline priorities, we remain focused on diseases with the highest unmet needs and greatest potential to make an impact in patient outcomes. Myelofibrosis is a serious and life–threatening blood cancer defined by stem cell–derived clonal myeloproliferation, bone marrow fibrosis, anemia, enlarged spleen, low blood counts and short survival. With JAK inhibitors being the only class of drugs approved for this disease, there remains limited therapeutic options for patients who either progress following treatment with a JAK inhibitor or are intolerant.”
Shacham added, “We believe selinexor has the potential to hold an important place in the myelofibrosis treatment paradigm and with dosing of the first patient underway, we plan to provide updates as the study progresses.”