Karyopharm Therapeutics (KPTI) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Company and pipeline overview

• Focuses on nuclear export inhibition to develop therapies for cancer, with lead asset selinexor (XPOVIO) approved for multiple myeloma in over 50 countries.

• Projected $130–$150 million in revenue this year from multiple myeloma.

• Expanding into myelofibrosis and endometrial cancer, both areas of high unmet need.

Myelofibrosis program and SENTRY trial

• SENTRY phase III trial evaluated selinexor plus ruxolitinib vs. ruxolitinib alone in newly diagnosed myelofibrosis patients.

• Achieved approximately double the SVR35 rate (spleen volume reduction ≥35%) for the combination, with rapid and sustained response.

• Showed a promising overall survival signal (hazard ratio 0.43, nominal P value 0.0222) at 12 months median follow-up.

• Noted deep reduction in clonal burden (VAF) and maintained a favorable safety profile with no new safety signals.

• SENTRY data to be presented as a late-breaking abstract at ASCO, including efficacy, disease modification, and safety.

Regulatory and market outlook for myelofibrosis

• FDA has shown flexibility in approvals based on SVR35 even when TSS is not met, as seen with pacritinib and momelotinib.

• Early overall survival benefit strengthens the case for approval.

• Near-term compendia (NCCN) listing could enable reimbursement and access, with potential to reach 50% of peak revenue before full label approval.

• Myelofibrosis market estimated as multi-billion dollar, with selinexor's peak potential up to $1 billion.

• Engaging with EU regulators, with updates expected in the future.