Apatorsen is an antisense inhibitor of heat shock protein 27 (Hsp27), a chaperone protein that regulates cell survival via androgen receptor and other signaling pathways. A Phase 1 study was conducted in patients with CRPC and other cancers known to overexpress Hsp27. Apatorsen had an acceptable safety profile and a MTD was not defined. Clinical activity at doses ≥400 mg was suggested by effects on stable measurable disease, declines in PSA, other tumor markers, and CTCs.
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