Karyopharm Therapeutics (KPTI) investor relations material

Key program updates

• Lead compound selinexor (XPOVIO) is approved in multiple myeloma in over 50 countries and is now being advanced in myelofibrosis and endometrial cancer.

• Phase III SENTRY trial in myelofibrosis showed significant improvement in spleen volume reduction (SVR35) and a promising early overall survival (OS) signal.

• Enrollment completed for the EC-042 phase III endometrial cancer trial, with data readout expected mid-year.

• Regulatory engagement ongoing with FDA and EMA, with more clarity on the path forward expected in the next 1-2 quarters.

• Cash runway extends into late Q3, covering major upcoming milestones.

Myelofibrosis SENTRY trial insights

• SVR35 rate nearly doubled with selinexor/ruxolitinib combo vs. ruxolitinib alone; 50% achieved SVR35 at week 24.

• OS hazard ratio of 0.43 (p=0.0222) with early and sustained separation of Kaplan-Meier curves.

• Consistent benefit across all patient subgroups and ruxolitinib dose levels.

• SVR35 predicted OS, suggesting it as a surrogate marker for long-term outcomes.

• Manageable safety profile with fewer deaths due to progression and toxicity in the combination arm.

Disease modification and subgroup analysis

• Rapid and substantial reduction in variant allele frequency (VAF) observed, supporting disease modification.

• Consistent efficacy across prognostic subgroups, spleen sizes, and early treatment emphasized for best outcomes.