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Karyopharm Initiates Pivotal Phase 3 Study of XPO1 Inhibitor Selinexor and Ruxolitinib in JAK Inhibitor (JAKi) Naïve Myelofibrosis
Phase 3 Study is Supported by Previously Presented Phase 1 Study Results, Including a 78.6% SVR35 and 58.3% TSS50 in Intent to Treat Patients at Week 24 at
About this update from Karyopharm Therapeutics Inc.
[{"type":"text","content":"Phase 3 Study is Supported by Previously Presented Phase 1 Study Results, Including a 78.6% SVR35 and 58.3% TSS50 in Intent to Treat Patients at Week 24 at the 60mg Dose\nNEWTON, Mass., June 28, 2023 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced the initiation of a pivotal Phase 3 clinical trial (XPORT-MF-034) (NCT04562389) to assess the efficacy and safety of once-weekly selinexor 60mg in combination with ruxolitinib in JAKi-naïve patients with myelofibrosis.\n\n \n \n \n \n \n \n\n \nThe randomized, double-blind, placebo-controlled study is expected to enroll 306 JAKi-naive patients with intermediate or high-risk myelofibrosis. Patients will be randomized 2:1 to ruxolitinib plus selinexor 60mg or ruxolitinib plus placebo in 28-day cycles. Ruxolitinib dose will be determined by the investigators based on the patients' baseline platelet count per the drug's prescribing information. The co-primary endpoints of the study are spleen volume response rate of ≥ 35% (SVR35) and symptom improvement of ≥ 50% (TSS50) at week 24, with a key secondary endpoint of anemia response at week 24.\n\"Selinexor and ruxolitinib appear to work synergistically, resulting in meaningful improvements in spleen response and total symptom score for patients with myelofibrosis,\" said Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. \"We believe that an opportunity exists to expand upon the initial response, depth, and duration of JAK inhibitors to ultimately improve patient outcomes. This combination has the potential to become a cornerstone treatment in front-line myelofibrosis and we are excited to start this pivotal trial to deliver on our goal of bringing forward an innovative new approach for the treatment of myelofibrosis that can benefit MF patients.\"\n\"The substantial degree of spleen volume reduction observed across all subgroups with selinexor 60mg in combination with ruxolitinib is very encouraging. There is a significant unmet need in the treatment of patients with myelofibrosis, and these data demonstrate that the addition of XPO1 inhibition with selinexor with standard-of-care ruxolitinib has the potential to significantly improve outcomes for first-line myelofibrosis patients,\" said Dr. John Mascarenhas, Professor of M...