Karyopharm Therapeutics (KPTI) Piper Sandler 36th Annual Healthcare Conference summary

Commercial performance and outlook

• Guided 2023 net revenue of $145–$155 million, with three consecutive quarters of net revenue growth in the multiple myeloma franchise and double-digit demand growth from Q2 to Q3.

• Focus remains on maintaining a profitable multiple myeloma franchise while leveraging commercialization capabilities for pipeline expansion.

• The next 12–18 months are expected to be highly active, with significant pipeline and commercial milestones anticipated.

Pipeline development and clinical progress

• Advancing three main programs: multiple myeloma (Phase 3 selinexor + PomDex), myelofibrosis (selinexor + ruxolitinib), and endometrial cancer (EC-042, currently under FDA discussion).

• Myelofibrosis program has evolved endpoints to SVR35 and absolute TSS, aligning with regulatory and clinical trends to improve probability of success.

• Enrollment for the myelofibrosis Phase 3 trial is expected to complete in the first half of 2025, with data readout in the second half of 2025.

Myelofibrosis clinical data and trial design

• Phase 1 data for selinexor + ruxolitinib in JAK inhibitor-naive patients showed 79% achieved SVR35 at week 24, with 100% achieving it at any time; TSS 50 at week 24 was 58%.

• Responses were durable, with no progression among responders as of August 2023, and safety profile was manageable, especially at lower selinexor doses with antiemetic support.

• Phase 3 trial endpoints updated from TSS 50 to absolute TSS, reflecting a more sensitive and comprehensive measure of symptom improvement.

• Trial size increased from 306 to 350 to enhance statistical power without affecting timelines.