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Karyopharm's XPO1 plus JAK inhibitor regimen both hits and misses in Phase III myelofibrosis trial

Karyopharm's XPO1 plus JAK inhibitor regimen both hits and misses in Phase III myelofibrosis trial

Newton, Massachusetts-based Karyopharm Therapeutics (Nasdaq: KPTI) reported that selinexor (Xpovio) combined with ruxolitinib nearly doubled the spleen volume response rate versus ruxolitinib alone in JAK inhibitor-naïve myelofibrosis, with Phase III data simultaneously published in the Journal of Clinical Oncology at ASCO 2026. The trial achieved onme of its co-primary endpoints in relation to spleen volume reduction, but fell short in the second co-primary endpoint average change in total symptom score.

The SENTRY trial (NCT04562389) is a randomized, placebo-controlled Phase III study enrolling 353 JAKi-naïve myelofibrosis patients with platelet counts above 100 × 10⁹/L, randomized 2:1 to selinexor 60 mg once weekly plus ruxolitinib or placebo plus ruxolitinib. On the co-primary endpoint of spleen volume reduction ≥ 35% (SVR35) at week 24, 49.8% of patients in the combination arm responded versus 28.0% in the control arm (odds ratio 2.58; 95% CI 1.60–4.17; p < 0.05).

The second co-primary endpoint — average change in absolute total symptom score over 24 weeks — did not reach statistical significance; the adjusted mean difference was 0.97 points (95% CI −1.07 to 3.02; p=0.825), with similar symptom improvement observed across both arms. A pre-specified secondary analysis of overall survival showed a hazard ratio of 0.43 (95% CI 0.19–1.00; nominal one-sided p=0.022), with Kaplan-Meier curve separation emerging around month 9 and 95.3% of combination-arm patients alive at the February 2026 data cut versus 89.8% in the control arm. Grade 3+ treatment-emergent adverse events occurred in 70% of the combination arm versus 50% of controls, driven primarily by hematologic toxicity; thrombocytopenia (59% vs 43%) and nausea (57% vs 17%) were the most differentiated all-grade events.

Selinexor inhibits exportin 1 (XPO1), a nuclear export protein, retaining tumor suppressor proteins in the nucleus — a mechanism distinct from JAK inhibition and theoretically complementary to ruxolitinib's pathway. The myelofibrosis combination landscape also includes Syndax's axatilimab and Bristol Myers Squibb's Reblozyl (luspatercept) in related settings, though cross-trial comparisons are limited by differing populations and endpoints.

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The trial's mixed outcome leaves Karyopharm in an unusual position. While the combination delivered one of the strongest spleen response improvements seen in a randomized frontline MF study, the failure to improve symptom burden could complicate regulatory discussions. The emerging survival signal may ultimately prove more important than either co-primary endpoint if it strengthens with longer follow-up, although the current analysis remains immature.


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