Scott Got­tlieb gave a savvy re­view of what we can ex­pect at the FDA; When times got rough, a biotech CEO demon­strat­ed re­al in­tegri­ty

Smart, rea­son­able and ex­treme­ly well pre­pared: What did we learn from Scott Got­tlieb’s con­fir­ma­tion hear­ing?

On Wednes­day, when Scott Got­tlieb showed up for his con­fir­ma­tion hear­ing at the Sen­ate, it be­came quick­ly ap­par­ent that the prospec­tive FDA com­mis­sion­er was the best pre­pared per­son talk­ing that day. What the Dems had billed as a sharp at­tack on Got­tlieb’s in­dus­try ties quick­ly dis­in­te­grat­ed in­to a se­ries of ran­dom, dis­joint­ed com­ments di­rect­ed against large­ly un­re­lat­ed is­sues.

Com­mis­sion­er-des­ig­nate Scott Got­tlieb at his hear­ing Zach Gib­son/Get­ty Im­ages

The Re­pub­li­cans were there to pat him on the back, which they man­aged to do just fine. A few used the oc­ca­sion to preen to their vot­ers. It’s what law­mak­ers do. No mat­ter.

It was a non-event if you were look­ing for fire­works and con­fronta­tion. If you lis­tened care­ful­ly, though, there was some re­al meat to this ex­change as well, and some vi­tal­ly im­por­tant in­sights in­to how he sees his role in man­ag­ing re­form from the in­side rather than es­tab­lish­ing him­self as the agency’s biggest crit­ic.

It was a huge­ly im­por­tant mo­ment for the bio­phar­ma in­dus­try, which has had to fear the worst over the past three months.

Got­tlieb looked like he had been prep­ping for this mo­ment for the past 20 years, or more. He has a back­ground in the FDA and deep ties to the in­dus­try. Over the past decade he has amassed a line­up of con­nec­tions to in­vest­ment groups and bio­phar­ma com­pa­nies. He will not set him­self in op­po­si­tion to the in­dus­try, nor should he. More im­por­tant­ly, he’s pre­pared to cham­pi­on the R&D gold stan­dard on safe­ty and ef­fi­ca­cy.

Three cheers. But in ad­di­tion to all those jobs he pur­sued un­der the Oba­ma ad­min­is­tra­tion, there was one more po­si­tion he vol­un­teered for: FDA com­mis­sion­er-in-wait­ing.

Back be­fore his nom­i­na­tion, I took a close look at Got­tlieb. Based on the pub­lic record and peo­ple close to him, it was clear he had a spe­cif­ic agen­da in mind. And he ticked off most of those box­es to the sen­a­tors.

He was go­ing to go af­ter com­plex gener­ics, clear­ing the way for new com­pe­ti­tion to some very ex­ten­sive ther­a­pies. He would cel­e­brate Richard Paz­dur’s clear achieve­ments in ac­cel­er­at­ing can­cer drug re­views — a role that has over­hauled and speed­ed a mas­sive amount of prod­uct de­vel­op­ment in the field — and promised to get some lag­gards at the agency up to speed.

He likes adap­tive tri­al de­signs, look­ing for new and bet­ter ways to in­tel­li­gent­ly as­sess safe­ty/ef­fi­ca­cy.

Drugs for rare dis­eases al­so look like they’ll be a pri­or­i­ty. There’s a way for skilled FDA staff to over­see small, fo­cused stud­ies for new drugs that can avoid the mess that Sarep­ta cre­at­ed and still suf­fers from.

I would have liked to have heard more specifics, such as pub­lish­ing CRLs (or trans­paren­cy in gen­er­al), how he will over­haul the or­phan drug process and do­ing away with the need for some out­comes stud­ies ahead of ap­provals, when they can be done af­ter­wards.

(Though can you al­so hear pay­ers sharp­en­ing their knives for those drugs? When reg­u­la­to­ry risk de­clines, pay­er risk ris­es dis­pro­por­tion­ate­ly these days. And af­ter Gilead’s So­val­di primed the pump, it’s a brand new day in the pay­er uni­verse.)

Maybe we can get some of that ex­tra de­tail in the fi­nal Q&A round to come. Pub­lish­ing CRLs won’t over­ly dis­turb the re­spon­si­ble play­ers in the in­dus­try and Got­tlieb could add some much-need­ed trans­paren­cy as a way to dis­tin­guish his stint as an in­de­pen­dent com­mis­sion­er. It’s not all go­ing to be cozy and fun.

What I was par­tic­u­lar­ly pleased to hear is that Got­tlieb views re­cruit­ing tal­ent as his biggest chal­lenge, while look­ing for a “bot­tom up” ap­proach from the cen­ters to dri­ve re­form. Got­tlieb’s stint as FDA com­mis­sion­er would be long and fruit­less if he took a con­fronta­tion­al po­si­tion with staff.

So what do we get?

We get a com­mis­sion­er who’s pledged to speed things up some more, re­spon­si­bly. He’s go­ing to bring in new, bad­ly need­ed tal­ent. And we hope he clear­ly choos­es trans­paren­cy over the long and un­ac­cept­able black box that the agency has long been re­quired to work in — though that has yet to be seen.

I’ll take it.


In­tegri­ty mat­ters more than ever in bio­phar­ma

I’ve been writ­ing about bio­phar­ma on a dai­ly ba­sis for close to 14 years now. And I’m more than fa­mil­iar with the re­struc­tur­ing drill.

Com­pa­ny X runs in­to some trou­ble. The biotech boat be­comes a life raft, or a log, and staffers have to go. There’s a press re­lease, a canned quote from the CEO say­ing how grate­ful they were to the new­ly job­less, and on to the next thing.

So I was more than a lit­tle shocked when CEO Ron Co­hen reached out to talk to me about the lay­offs and down­siz­ing at Acor­da this week fol­low­ing a set­back on his patent front for Ampyra. That nev­er hap­pens. But he spelled out what the cuts meant, where the ax would fall and how they could mon­e­tize as­sets.

Co­hen has a Plan B. He hasn’t been stand­ing on the tracks, wait­ing for the train to come. It’s ex­tra­or­di­nar­i­ly high risk, and Plan B hasn’t al­ways gone ac­cord­ing to plan over the years — which is par for this unique course. And now fail­ure is not an op­tion.

That’s not a great place to be. No one en­vies his po­si­tion. But in­stead of duck­ing and tak­ing the easy way out, Co­hen demon­strat­ed re­al in­tegri­ty in stand­ing up and tak­ing it all on, di­rect­ly.

Sure, you could throw a cheap shot or two in his di­rec­tion. But in this in­dus­try in­tegri­ty mat­ters more than ever. And I re­spect it more than ever. We all should.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources. 

As­traZeneca's di­a­betes drug Farx­i­ga helps pa­tients with heart dis­ease and with­out di­a­betes in land­mark tri­al

Months ago, data on J&J’s $JNJ Invokana indicated the diabetes drug conferred cardiovascular (CV) benefit in patients who do and do not have preexisting CV disease. On Tuesday, AstraZeneca’s $AZN rival treatment, Farxiga, was shown to cut the risk of CV death or the worsening of heart failure in patients with heart disease, in a landmark trial.

The treatments, in addition to Jardiance from Eli Lilly $LLY, belong to a class of diabetes drugs called sodium-glucose co-transporter 2 (SGLT2) inhibitors, which work by curbing the absorption of glucose via the kidneys so that surplus glucose is excreted through urination.

Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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