Lurbinectedin (PM01183), an active compound in platinum-resistant/refractory ovarian cancer (PRROC) patients: Results of a two-stage, controlled phase II study.

5505 Background: PM01183 is a new anticancer agent that blocks the trans-activated transcription and induces the formation of double-strand breaks in a wide range of cancer cell lines, including platinum-resistant (Pt-res). Methods: PRROC patients (pts) with less than 3 prior chemotherapy (CT) containing lines, adequate organ function and performance status (PS) 0-2 were included. The primary endpoint was overall response rate (ORR) (by RECIST v1.1 and/or Rustin criteria). Secondary endpoints were progression free survival (PFS), overall survival (OS) and safety. Pts were treated with i.v. PM01183 (P1), 7 mg flat dose, q3wk in the first stage. In the second stage, pts were randomized (1:1) to PM01183 (P2) or topotecan (T) (standard or weekly regimen). Cross-over to the P2 arm was allowed after progression to T. Results: 81 pts were included (P1/P2/T: 22/30/29). Global median characteristics of pts were balanced: age 61 years; PS 1; Pt-res (P1/P2/T: 16/17/16 pts); prior bevacizumab: 18.5% of pts, median pr...