Karyopharm Therapeutics (KPTI) 2025 Earnings Call Presentation

Myelofibrosis Opportunity - Karyopharm is focused on the transformative myelofibrosis opportunity in 2025, with a peak annual revenue opportunity of up to approximately $1 billion[4, 12] - The company expects top-line data from the Phase 3 SENTRY trial of selinexor in myelofibrosis in late 2025/early 2026[4] - Data from a Phase 1 trial showed that 78.6% of intent-to-treat patients achieved SVR35 at week 24 with selinexor 60 mg + ruxolitinib[24] - In the Phase 1 trial, the average improvement in Absolute TSS (Abs-TSS) was 18.5, suggesting a positive outcome with Abs-TSS can be achieved in the SENTRY Phase 3 trial[33] Clinical Trial Data and Development - The Phase 2 XPORT-MF-035 trial showed that spleen volume reduction rates more than doubled with selinexor compared to physician's choice, with 33% achieving SVR35 compared to 13%[48, 49] - In the XPORT-MF-035 trial, patients who progressed on physician's choice (Rux) and received selinexor showed a meaningful spleen volume reduction[51] - Selinexor meaningfully decreased transfusion burden by approximately 50% in the XPORT-MF-035 trial[59] - The SENTRY (XPORT-MF-034) Phase 3 trial of selinexor in combination with ruxolitinib in JAKi naïve myelofibrosis has passed futility analysis and continues as planned, with top-line data expected in late 2025/early 2026[39, 41] Endometrial Cancer - Karyopharm is focusing the Phase 3 XPORT-EC-042 trial on patients with TP53wt EC that are pMMR or dMMR and medically ineligible to receive a checkpoint inhibitor[75] - Data from the SIENDO trial (ASCO 2024) showed an encouraging signal of long-term median PFS benefit of 39.5 months in the TP53 Wild-Type/pMMR subgroup[82]