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.

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Amgen VP of R&D David Reese

Am­gen rolls out da­ta for KRAS in­hibitor com­bo study in col­orec­tal can­cer, hop­ing to move on from ug­ly ear­ly re­sults

With the first win for its KRAS inhibitor sotorasib in hand, Amgen is pushing ahead with an aggressive clinical plan to capitalize on its first-to-market standing. The drugmaker thinks combinations — in-house or otherwise — could offer a path forward, and one early readout from that strategy is bearing fruit.

A combination of Amgen’s sotorasib and its EGFR inhibitor Vectibix posted an overall response rate of 27% in 26 patients with advanced colorectal cancer (CRC) with the KRAS-G12C mutation, according to data from the larger Phase Ib/II CODEBREAK 101 study set to present at this weekend’s virtual ESMO Congress.

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As­traZeneca touts Imfinzi im­munother­a­py com­bos for lung can­cer in push to dri­ve PD-L1 drug up­take

Facing the big dogs in the PD-(L)1 space, AstraZeneca has taken its own contender Imfinzi into blockbuster territory in its four years on the market but sees even bigger things for the drug. Combinations could be the key, and early results from a mid-stage test are adding some fuel to that strategy.

Imfinzi combined with one of two investigational immunotherapies — a CD73 antibody dubbed oleclumab or an Innate’s anti-NGK2a named monalizumab — topped Imfinzi alone in terms of overall response and progression-free survival in patients with stage III non-small cell lung cancer whose tumors had not worsened during concurrent chemoradiation, according to interim data from the Phase II COAST trial set to be presented at #ESMO21.

Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Eu­ro­pean study finds that Gilead­'s Covid-19 an­tivi­ral remde­sivir shows no clin­i­cal ben­e­fit

Gilead’s remdesivir — or Veklury, as it’s marketed in the US — raked in around $2.8 billion last year as the only FDA-approved antiviral to treat Covid-19. But new data from a European study suggest the drug, which has been given to about half of hospitalized Covid patients in the country, has no actual benefit.

The open-label DisCoVeRy trial enrolled Covid-19 patients across 48 sites in Europe to test a handful of treatments, including remdesivir, lopinavir–ritonavir, lopinavir–ritonavir and interferon beta-1a, and hydroxychloroquine. To participate, patients had to show symptoms for seven days and require oxygen support. A total of 429 patients were randomized to receive remdesivir plus standard of care, while 428 were assigned to standard of care alone.

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Gri­fols drops $1B on Ger­man hold­ing com­pa­ny in con­tin­ued plas­ma push

One Spanish biotech is beefing up its plasma therapy operations, and on Friday, it announced that it’s doing so in a billion-dollar deal.

Grifols is now the largest shareholder of Biotest, a company valued at more than $1.8 billion. By teaming up, the two will try to increase the number of plasma therapies available and increase patient access around the world, Grifols said in a press release.

The company did so by acquiring holding company Tiancheng Pharmaceutical, the Germany-based owner of nearly 90% of Biotest shares, for nearly $1.27 billion. Grifols now owns nearly 90% of Biotest voting rights and almost 45% of the total share capital of Biotest.

Covid-19 roundup: FDA re­veals boost­er ad­comm ques­tion; Eli Lil­ly's an­ti­body cock­tail cleared for pre­ven­tion

The FDA released briefing documents this week from the agency and Pfizer each outlining their arguments for today’s Covid-19 booster shot adcomm, but one thing conspicuously missing was the question on which panel members would be voting. But late Thursday night, regulators published that question.

Adcomm members will be asked whether or not the safety and efficacy data from Pfizer/BioNTech’s original Phase III study “support approval” of a booster shot at least six months after the second dose in individuals older than 16. The question notably excludes the real-world data from Israel and other analyses that Pfizer and the Biden administration had said would be a centerpiece of their arguments for boosters.

A Pfiz­er part­ner wel­comes ex-ADC Ther­a­peu­tics CMO Jay Fein­gold to the team; Amid tough sled­ding, Im­muno­vant choos­es Eli Lil­ly alum as CFO

→ Last week we told you about the CMO revolving door at ADC Therapeutics, as Joseph Camardo replaced the departing Jay Feingold. The next opportunity for Feingold in the CMO slot has opened up at antibody-drug conjugate and mAb developer Pyxis Oncology, which has added several new execs and scientific advisory board members in recent months, including ex-Immunovant CFO Pamela Yanchik Connealy. Before his tenure at ADC, Feingold was Daiichi Sankyo’s VP of US medical affairs and chairman of the Global Medical Affairs Oversight Committee. Within weeks in March, Pyxis struck a licensing deal with Pfizer for two of its ADCs and raked in $152 million from a Series B round.

Multiple antibiotic resistant Pseudomonas aeruginosa bacterium

A new way to in­fil­trate (and de­stroy) some of the dead­liest drug-re­sis­tant bugs

About four years ago, Ruben Tommasi, the gregarious scientific chief of antibiotics startup Entasis, walked into a meeting with his top chemist and top biologist to chew over another batch of unchanging results.

“It felt like we were running the same experiment over and over,” Tommasi told Endpoints News. “We had all sort of come to that point in time where we felt like we were banging our heads against the wall.”

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