
Verastem wins breakthrough nod for combo therapy in ovarian cancer; EMA picks up speedy review of BioMarin's gene therapy
Verastem has earned the FDA’s breakthrough therapy designation for its novel RAF/MEK inhibitor VS-6766 as a combo therapy with in-house FAK inhibitor defactinib to treat second-line, recurrent low-grade serous ovarian cancer regardless of KRAS mutant status, the company said Monday.
The combination is being tested in the investigator-initiated Phase I/II FRAME trial, where early results showed an ORR of 52% with KRAS mutant ORR at 70%, KRAS wild-type ORR at 44%, and KRAS status undetermined ORR at 0%. The most common side effects seen in the study were rash, creatine kinase elevation, nausea, hyperbilirubinemia and diarrhea.
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