Blueprint stumbles in PhIII GIST trial, sending stock sliding, but analysts preach caution
When the FDA approved Blueprint’s first drug three months ago, they split their decision. Ayvakit would be approved for gastrointestinal stromal tumors (GIST) with a rare mutation called PDGFRa. The other half of their application for plain old fourth line GIST would have to wait. Regulators wanted to know how the drug performed in a Phase III trial.
Results are now in from that Phase III trial and they spelled a death-knell. In a study of 476 patients with third or fourth line GIST, Ayvakit (avapritinib) performed worse on the primary endpoint than the already-approved Bayer GIST drug, regorafenib. Progression-free survival was 4.2 months, compared with 5.6 months for Ayvakit.
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