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.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.