FDA gives Mer­ck ‘re­al-time’ OK for Keytru­da, of­fer­ing a peek at the lat­est ex­ten­sion of the agen­cy's da­ta ex­press high­way

The FDA doesn’t al­ways wait for a mar­ket­ing ap­pli­ca­tion to come in be­fore it be­gins its re­view these days. And that suits Mer­ck just fine.

The fed­er­al agency has stamped a full ap­proval — fol­low­ing last year’s con­di­tion­al OK — on Mer­ck’s mega-block­buster Keytru­da for front­line use in a chemo com­bo for fight­ing non-squa­mous non-small cell lung can­cer. Reg­u­la­tors based their de­ci­sion on the da­ta set from KEYNOTE-189, put on dis­play last April, one in a se­ries of late-stage tri­als that Mer­ck has been rolling out to grab the lead on the PD-1/L1 mar­ket from a group of tough com­peti­tors at Bris­tol-My­ers Squibb.

The green light is any­thing but un­ex­pect­ed. But it is note­wor­thy for rea­sons that the en­tire in­dus­try should be pay­ing at­ten­tion to.

Roger Perl­mut­ter

What dis­tin­guish­es this par­tic­u­lar OK is the tim­ing. FDA com­mis­sion­er Scott Got­tlieb has been push­ing a new pi­lot pro­gram called re­al-time on­col­o­gy re­views, where reg­u­la­tors start their work ahead of a for­mal ap­pli­ca­tion. The first re­al-time OK went to No­var­tis a few weeks ago for two com­bi­na­tions us­ing Kisqali against breast can­cer. And top-tier drug de­vel­op­ers are find­ing that in­stead of just be­ing greet­ed with an open door, some­times the door is be­ing tak­en off the hinges en­tire­ly.

In this case they were deal­ing with a haz­ard ra­tio of 0.49, mark­ing a 51% drop in the risk of death for pa­tients tak­ing the com­bo. There’s still no me­di­an sur­vival da­ta for the com­bo avail­able, but the matchup of the Keytru­da/chemo ap­proach ver­sus chemo alone pro­vid­ed a clear set of ad­van­tages for Mer­ck.

  • The me­di­an pro­gres­sion-free sur­vival rate was 8.8 months for the com­bo, 4.9 months for chemo alone.
  • The over­all re­sponse rate was 48% com­pared to 19% in the con­trol.
  • Me­di­an re­sponse du­ra­tion hit 11.2 months for the Keytru­da arm and 7.8 months for con­trol.

These re­al-time re­views now on of­fer at the FDA look to ex­tend the da­ta ex­press high­way that’s been built for the agency’s break­through drug des­ig­na­tion and oth­er hur­ry-up cam­paigns that have trans­formed the speed and de­sign of on­col­o­gy stud­ies over the past few years. In this case the FDA wants to re­ly en­tire­ly on US stud­ies, ex­clud­ing any­thing out­side the bor­ders, while pre­fer­ring drugs that are de­liv­er­ing clear ben­e­fits and are be­ing test­ed on some ob­vi­ous end­points. And they don’t want any for­mu­la­tion changes to pon­der when mak­ing a snap call.

The FDA is tak­ing no chances with Keytru­da, a land­mark drug by any de­f­i­n­i­tion that is be­ing pushed through hun­dreds of com­bi­na­tion stud­ies and new ap­proach­es. Got­tlieb is like­ly to earn even more de­vo­tion from the in­dus­try’s lead­ers for fol­low­ing through on im­prov­ing the FDA’s re­spon­sive­ness. And Mer­ck is hap­py to have the fresh set of brag­ging rights to­day.

“Keytru­da is rapid­ly be­com­ing a foun­da­tion for the treat­ment of ap­pro­pri­ate pa­tients with metasta­t­ic non-small cell lung can­cer,” said Mer­ck R&D chief Roger Perl­mut­ter. “To­day’s ap­proval of the ex­pand­ed la­bel for Keytru­da based on da­ta from the KEYNOTE-189 tri­al is an im­por­tant mile­stone.”

 

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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