Months af­ter cap­ma­tinib — the MET in­hibitor de­signed to cross the blood-brain bar­ri­er dis­cov­ered by In­cyte and li­censed by No­var­tis — se­cured the FDA’s pri­or­i­ty re­view in pa­tients with a form of lung can­cer, the Swiss drug­mak­er has re­port­ed en­cour­ag­ing, al­beit ear­ly, da­ta in pa­tients whose can­cer had spread to the brain.

Da­ta from 13 pa­tients with metasta­t­ic MET ex­on 14 skip­ping non-small cell lung can­cer with brain metas­tases, were un­veiled at the vir­tu­al­ly-held Amer­i­can As­so­ci­a­tion for Can­cer Re­search con­fer­ence on Mon­day.

Da­ta from the mid-stage GEOM­E­TRY mono-1 study showed that 7 of the 13 ex­pe­ri­enced an in­tracra­nial re­sponse, of which 4 had a com­plete res­o­lu­tion of all brain le­sions.

The tri­al en­rolled 97 heav­i­ly pre-treat­ed (co­hort 4) and treat­ment-naive (co­hort 5b) pa­tients al­to­geth­er at the April 15 cut­off.

The over­all re­sponse rate — the study’s main goal — was 40.6% in pre-treat­ed pa­tients ver­sus 67.9% in the treat­ment-naive group. The me­di­an du­ra­tion of re­sponse, a key sec­ondary end­point, was 9.72 months in pre-treat­ed pa­tients com­pared to 11.14 months in the treat­ment-naive arm. An­oth­er sec­ondary goal, me­di­an pro­gres­sion-free sur­vival, al­so fa­vored the treat­ment-naive arm with 9.69 months, against 5.42 months in the pre­vi­ous­ly treat­ed pa­tients.

From a safe­ty stand­point, cap­ma­tinib was man­age­able. The most com­mon tox­i­c­i­ty-re­lat­ed ad­verse events across all co­horts were pe­riph­er­al ede­ma, nau­sea, in­creased blood cre­a­ti­nine and vom­it­ing.

There are cur­rent­ly no ap­proved ther­a­pies that specif­i­cal­ly tar­get MET ex­on 14 skip­ping non-small cell lung can­cer (NSCLC). NSCLC ac­counts for ap­prox­i­mate­ly 85% of lung can­cer di­ag­noses, while MET ex­on 14 skip­ping mu­ta­tions oc­cur in 3-4% of new­ly di­ag­nosed ad­vanced NSCLC cas­es.

Vas Narasimhan No­var­tis

No­var­tis chief Vas Narasimhan has tout­ed the ther­a­py as one of sev­en po­ten­tial block­busters the com­pa­ny is hop­ing to bring on to the mar­ket in 2020 — if cap­ma­tinib is launched, orig­i­nal dis­cov­er­er In­cyte stands to earn over half a bil­lion dol­lars in mile­stone pay­ments and roy­al­ties. The drug has al­so been grant­ed break­through sta­tus and pri­or­i­ty re­view for MET ex­on 14 skip­ping lung can­cer, with an FDA de­ci­sion ex­pect­ed by Au­gust.

Pfiz­er’s ri­val MET and ALK in­hibit­ing ri­val drug, Xalko­ri, was grant­ed break­through ther­a­py sta­tus in pa­tients with metasta­t­ic NSCLC with MET ex­on 14 mu­ta­tions who had re­ceived pri­or ther­a­py in 2018. In Jan­u­ary, the US drug­mak­er dis­closed da­ta on 65 evalu­able pa­tients. Da­ta showed the ther­a­py-in­duced me­di­an du­ra­tion of re­sponse was 9.1 months and me­di­an pro­gres­sion-free sur­vival of 7.3 months.

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepreneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film “Fantastic Voyage,” the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his and his co-founders’ own accounts, along with some seed cash from friends and family.