#AACR20: Ear­ly da­ta sug­gest No­var­tis' In­cyte-dis­cov­ered MET in­hibitor helps lung can­cer pa­tients with brain le­sions

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.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

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.

Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

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.

Thank you, next: Take­da hands Ovid $196M cash to rein back in Phase III-ready seizure drug, re­viv­ing bat­tered stock

Soticlestat made it.

Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones.

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Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

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.