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.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,600+ biopharma pros reading Endpoints daily — and it's free.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,600+ biopharma pros reading Endpoints daily — and it's free.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,600+ biopharma pros reading Endpoints daily — and it's free.

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,600+ biopharma pros reading Endpoints daily — and it's free.

Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,600+ biopharma pros reading Endpoints daily — and it's free.

Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,600+ biopharma pros reading Endpoints daily — and it's free.