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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