How do you re­place a rock star like Scott Got­tlieb at the FDA? Maybe you can't

Any­one look­ing for a con­ve­nient weath­er vane to de­ter­mine the re­ac­tion to Scott Got­tlieb’s abrupt de­par­ture from the FDA need go no fur­ther than the $XBI. The S&P Biotech ETF took a 4% hit. And it was down an­oth­er 1.8% ahead of the bell on Thurs­day.


The mar­ket and the in­dus­try loved Got­tlieb, as we un­der­scored on sev­er­al oc­ca­sions with in­dus­try sur­veys high­light­ing the in­tense en­thu­si­asm for some­one who ad­vo­cat­ed for col­lab­o­rat­ing with the drug in­dus­try. Bio­phar­ma had a part­ner in Got­tlieb, and they all knew it.

Va­p­ing and the opi­oid epi­dem­ic — while big pub­lic pol­i­cy is­sues — meant ab­solute­ly noth­ing to the in­dus­try. An agency com­mit­ted to push­ing ap­provals while em­brac­ing in­no­va­tion at a time some land­mark ther­a­pies are be­ing ap­proved at a more rapid pace: That was pure gold. And it wasn’t just lip ser­vice. In this brave new world J&J could slip around the gold stan­dard on de­pres­sion drug da­ta and score an OK this week for es­ke­t­a­mine, a drug they plan to make a block­buster. A few years ago, that like­ly wouldn’t have hap­pened.

The one thing that could set­tle in­vestors and ex­ecs back down would be a new head of the FDA that could walk the line be­tween main­tain­ing the some­what weak­ened gold stan­dard on piv­otal da­ta and a free-for-all that would clear­ly aban­don a com­mit­ment to ap­prov­ing safe and ef­fec­tive drugs that met the risk/ben­e­fit analy­sis. 

Got­tlieb emerged ear­ly as a fa­vorite for the FDA job. He had ex­pe­ri­ence at the agency, he fit in­to the Trump ad­min­is­tra­tion’s orb with vows to speed things up, and an amaz­ing num­ber of in­dus­try in­sid­ers knew him and liked him.

He quick­ly be­came a rock star in bio­phar­ma, tweet­ing his way through every day about pol­i­cy.

So how do you re­place him and main­tain the same lev­el of en­thu­si­asm in bio­phar­ma? The usu­al sus­pects don’t cut it. 

We’re not even sure right now who will be the in­ter­im chief. Stephen Os­troff has tak­en the helm on a strict­ly in­ter­im ba­sis be­fore, but he re­tired last fall. Amy Aber­nethy was named prin­ci­pal deputy com­mis­sion­er just 2 months ago, com­ing to the FDA from a se­nior sci­en­tif­ic po­si­tion at Flat­iron af­ter a stint at Duke. She’s been tapped as the like­ly in­ter­im chief, and will def­i­nite­ly make the ru­mor mill as pos­si­bly the next head of the FDA. But she’s still large­ly an un­known in the in­dus­try, so don’t ex­pect much en­thu­si­asm right off the bat.

On the oth­er hand, if she gets the in­ter­im job, she’ll have a chance to shine. And Pres­i­dent Trump doesn’t seem pressed to get big jobs filled fast in Wash­ing­ton DC, so the in­ter­im role could last awhile. 

Na­tion­al Can­cer In­sti­tutes di­rec­tor Ned Sharp­less has emerged as one of the most fre­quent­ly cit­ed can­di­dates for the FDA job, with an ear­ly push from the Wall Street Jour­nal, which al­so quick­ly named Brett Giroir, as­sis­tant sec­re­tary at HHS, as an­oth­er pos­si­ble suc­ces­sor.

Jim O’Neill ev­i­dent­ly had a shot at the job a cou­ple of years ago, but the in­dus­try was shocked by the fact that Pe­ter Thiel’s can­di­date was even in the run­ning. He’s been as­so­ci­at­ed with some loony en­deav­ors, like seast­eading and the hunt for im­mor­tal­i­ty, and the in­dus­try sees him as a dis­as­ter wait­ing to hap­pen. In this en­vi­ron­ment, that makes O’Neill a cred­i­ble threat again, though it seems far fetched.

But he’s still go­ing to make every­one’s list. Fun­ny how that works.

Richard Paz­dur

How about Richard Paz­dur, the on­col­o­gy czar at the FDA whose com­mit­ment to rapid drug re­views has played a ma­te­r­i­al role in rev­o­lu­tion­iz­ing on­col­o­gy R&D over the last 5 years? It’s been sug­gest­ed. But when it comes to agency in­sid­ers, though, no one out­shines Janet Wood­cock at CDER. She not on­ly knows where the bod­ies are buried at the FDA, she of­ten helped put them there. Maybe she could cap a decades-long run as FDA chief? 

Janet Wood­cock

Stranger things have hap­pened.

Two years ago I called Got­tlieb the Re­pub­li­can shad­ow com­mis­sion­er, out front of­fer­ing po­si­tions on re­form­ing the FDA. We don’t seem to have one of those now, by virtue of the fact that no one ex­pect­ed Got­tlieb to stand down so soon. And with a na­tion­al elec­tion loom­ing next year with a wild­ly con­tro­ver­sial pres­i­dent, any­one who does get the job may ul­ti­mate­ly prove an in­ter­im chief in any case.

Bio­phar­ma hates reg­u­la­to­ry un­cer­tain­ty of any kind. This year we’ve had a gov­ern­ment shut­down fol­lowed by an abrupt res­ig­na­tion by Got­tlieb. Un­cer­tain­ty at the FDA ap­pears to now be the rule. 

In a note to in­vestors, Jef­feries’ Michael Yee not­ed that who­ev­er steps in will find an agency that has es­tab­lished a “pro-in­no­va­tion” ap­proach that won’t sud­den­ly end now, re­gard­less of who gets the job.

That sounds rea­son­able, but it won’t stop the fret­ting that’s go­ing on now. Ap­proach­es are one thing, ex­e­cu­tion is an­oth­er.

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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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).

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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Takeda's trans­la­tion­al cell ther­a­py group revs up for a race to the clin­ic with off-the-shelf CAR-T

Four years after Takeda launched a wide-ranging induced pluripotent stem cell project with the researchers at Nobel prize-winning Shinya Yamanaka’s lab at the University of Kyoto, the pharma company is taking delivery of the first of what it hopes will be a whole pipeline of iPS cell-derived therapies that can deliver on the promise of off-the-shelf CAR-T therapies.

From here, Stefan Wildt — the head of pharma sciences and translational cell therapy at Takeda — and his group of 100-plus scientists will be charged with steering their way to the clinic as they build out the manufacturing and support work for this pipeline-in-the-making. 

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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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John McHutchison in 2012. Getty Images

The $1.1M good­bye: Gilead CSO John McHutchi­son is out as Daniel O’Day shakes up the se­nior team

Just a little more than a year after John McHutchison grabbed a promotion to become CSO at Gilead in the wake of Norbert Bischofberger’s exit, he’s out amid a shakeup of the senior team that is also triggering the departure of two other top execs.

Gilead stated that McHutchison “has decided to step down” from the job as of August 2nd. And their SEC filing notes that he’ll be getting a $1.1 million check to settle up on his contract.

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Thomas Gajewski, David Steinberg. (CRI, Pyxis)

Bay­er, Long­wood back star re­searcher's deep dive in­to the tu­mor mi­croen­vi­ron­ment for new I/O tar­gets

From PD-1 targeting to the RAS pathway to the STING complex, Thomas Gajewski has spent the past two decades of his career decoding the various ways the immune system can be unleashed to defend against cancer. So when the University of Chicago professor comes around to putting all his findings into a new platform for finding new targets, VCs and pharma groups alike pay attention.

“He’s been studying T cells for 20 years, plus he’s one of the world’s leaders if not the world leader in the space,” David Steinberg, partner at Longwood Fund, said. “Furthermore, let me add he did a lot of the foundational research and also some of the seminal clinical trials in the existing set of I/O agents. He understands the space really well, he understands the current strengths, and I think he understood really well what was missing, so he knew where to look.”

Kamala Harris speaking yesterday at the Des Moines Register Iowa Presidential Candidate Forum [via Getty]

Who’s the tough­est on drug prices? A game of po­lit­i­cal one-up­man­ship is dri­ving the pol­i­cy de­bate in Wash­ing­ton

Earlier this week we got a look at Senator Kamala Harris’ position on drug prices. She’s proposing that HHS take an average price from single-payer systems like the UK, Germany and Canada — which leverage market access for lower prices — and use that to set the US price. Anything drug companies collect above that would be taxed at a rate of 100%.

And the rhetoric is scathing:
While families struggle to make it to the end of the month, pharmaceutical companies are turning record profits. They’re spending nearly as much on advertising as R&D. They’re manipulating their market power to hike prices on lifesaving generic drugs. They’re making twice the profit of the average industry in America and still increased drug prices by 10.5% over the past six months alone. Meanwhile, they are charging dramatically higher prices to American consumers.
That’s an escalation on Joe Biden’s plan, which includes drug importation from those cheaper markets as well as allowing Medicare to negotiate prices — something that virtually all Dems agree on now.

SJ Lee [File photo]

Go­ing in­side cells, Sung Joo Lee has sketched some big goals for his small — but glob­al — team of drug hunters

For a small biotech based in South Korea with a research arm in Cambridge, MA, Orum Therapeutics has sketched out some big goals aimed at developing antibodies for intracellular targets. And now they have a new $30 million round to push the work forward, aiming at a slate of currently undruggable quests.

Orum has been working on a platform tech out of Ajou University that relies on endocytosis to smuggle antibodies and their cargo inside a cell. They’ve published work in Nature that illustrates its preclinical potential in RAS mutations, and KRAS is on their list of targets. 

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