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Street View: Karyopharm's cancer drug trial results limit regulatory odds

ReutersMar 25, 2026 11:49 AM

Karyopharm Therapeutics KPTI.O said on Tuesday its therapy led to significant reduction in spleen size in a late-stage study of patients with myelofibrosis, a rare blood cancer, meeting only one of its two main goals

The drug combination failed to meet the study's second main goal of statistically significant symptom improvement

At least two analysts lowered PT on the company

MIXED RESULTS DIM CHANCE OF APPROVAL

Cantor Fitzgerald ("overweight") says the likelihood of FDA approval is minimal, given the regulatory precedent of placing significant importance on showing clear symptom improvement

H.C. Wainwright ("buy," PT: $8) says missing the symptom goal weakens KPTI's path with regulators, noting other myelofibrosis drugs with similar results were not taken forward, though early survival data could still provide support

RBC Capital Markets ("outperform," PT: $23) says "the data is clearly imperfect, but between spleen, survival, and hints of disease modification, we believe there is enough here to warrant regulatory discussions"

Jefferies ("buy," PT: $14) says there is low chance of FDA approval, based on the current results, even though the early survival signal could still provide support

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