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

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

The National Institutes of Health has again declined to use so-called “march-in” rights to lower the price of Astellas and Pfizer’s prostate cancer drug Xtandi despite being invented at UCLA with grants from the US Army and NIH.

“Given the remaining patent life and the lengthy administrative process involved for a march-in proceeding, NIH does not believe that use of the march-in authority would be an effective means of lowering the price of the drug,” NIH told prostate cancer patients Robert Sachs and Clare Love, in a letter shared with Endpoints News. The institutes’ analyses found Xtandi “to be widely available to the public,” an indication that there was not a pressing need for the US to act.