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.
Verastem’s drug takes a novel dual-target approach to the RAS pathway, which is implicated in cancer recurrence with about 30% of all cancer tumors expressing mutant RAS.
Verastem is also testing the VS-6766 alone and combination with defactinib as part of the phase II registration RAMP 201 study. — Kyle Blankenship
EMA grants BioMarin’s request for a speedy review of gene therapy
The European Medicines Agency will speedily review BioMarin’s valoctocogene roxaparvovec, a gene therapy to treat severe hemophilia A, with an opinion expected in the first half of next year, the company said.
With the EMA agreeing to give the therapy a closer look, BioMarin said it plans to submit an associated marketing application by June, based on pivotal Phase III data that includes one-year follow-up results as well as four and three years of follow-up from an ongoing Phase I/II dose escalation study.
Meanwhile, BioMarin also expects to submit two-year follow-up safety and efficacy data from that Phase III test to the FDA, with a potential resubmission of its application scheduled for Q2 2022.
The FDA shot down BioMarin’s application for the therapy back in August, citing the need to see that two-year follow-up data. — Kyle Blankenship
Francesco De Rubertis’ legacy play Centessa sets terms for $285M IPO
Ahead of an expected IPO later this week, Centessa is setting terms for what it hopes is a massive raise.
The company expects to see $285 million come in with the raise, offering 15 million shares at a price range of $18 to $20. At the midpoint of that range, Centessa would have a market value of about $1.8 billion, according to Renaissance Capital.
Centessa comes from the mind of Francesco De Rubertis, making a legacy play for a 10-in-1 holding company. His vision revolves around taking the asset-centric mindset that he’s been preaching at Medicxi over the years and rolling up a bunch of biotech upstarts into one outfit.
They’ll be going forward without Moncef Slaoui, the former GSK exec and Operation Warp Speed chief following a “substantiated” sexual harassment case for which he subsequently apologized. — Max Gelman