Rain Oncology falls 81% after Phase 3 setback for lead asset

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- Rain Oncology (NASDAQ:RAIN) fell ~81% in the pre-market Monday after announcing that its lead product candidate, milademetan, did not reach the primary endpoint in a Phase 3 trial for patients with liposarcoma, a rare type of cancer.
- The trial, known as MANTRA, was designed to evaluate milademetan compared to trabectedin, a current standard of care as a late-line option for dedifferentiated (DD) liposarcoma.
- According to its topline data, the 175-patient trial did not reach the primary endpoint of progression-free survival (PFS) as median PFS for milademetan and trabectedin arms stood at 3.6 months and 2.2 months, respectively (hazard ratio = 0.89, p=0.53)
- Regarding safety, ~12% and 19% of patients in milademetan and trabectedin arms discontinued the trial due to adverse events, respectively. Nausea, thrombocytopenia, anemia, vomiting, and neutropenia were among the most common treatment-emergent AEs in the milademetan arm.
- In the wake of the setback, Rain (RAIN) has decided to halt further development of milademetan for DD LPS.