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.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

UP­DAT­ED: J&J's Xarel­to, Amar­in's Vas­cepa are cost-ef­fec­tive, not bud­get friend­ly — ICER

ICER, an increasingly influential cost-effectiveness watchdog in the United States, has concluded in its review of treatments for cardiovascular disease that while the cost of J&J’s Xarelto and Amarin’s Vascepa meet its benchmark for value pricing — the two treatments will not likely treat as many patients as hoped without surpassing the annual budget threshold calculated by ICER for each therapy.