Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

Bris­tol My­er­s' Op­di­vo notch­es a dou­ble win against chemo in esophageal can­cer, po­ten­tial­ly set­ting up 1st-line nod

Bris­tol My­ers Squibb is one of a group of drug­mak­ers prep­ping for a tense FDA ad­comm lat­er this month to re­view a host of ac­cel­er­at­ed ap­provals. Look­ing to start the month on the right note, the drug­mak­er rolled out new da­ta for its PD-(L)1 check­point in­hibitor Op­di­vo show­ing more promise in esophageal can­cer.

Op­di­vo in com­bi­na­tion with chemother­a­py and a com­bi­na­tion of Op­di­vo and Yer­voy beat out chemo alone in ex­tend­ing the lives of first-line esophageal squa­mous cell car­ci­no­ma pa­tients whose tu­mors are metasta­t­ic or can’t be sur­gi­cal­ly re­moved, ac­cord­ing to in­ter­im re­sults re­leased Thurs­day.

A com­bi­na­tion of Op­di­vo and chemo hit its pri­ma­ry OS and PFS end­points over chemo alone in PD-(L)1 ex­press­ing tu­mors at a pre­spec­i­fied check-in on the Phase III CHECK­MATE-648 study. Mean­while, Op­di­vo plus Yer­voy showed more mixed re­sults, hit­ting its OS pri­ma­ry end­point but miss­ing the PFS co-pri­ma­ry end­point, Bris­tol My­ers said.

Bris­tol My­ers is tout­ing the re­sults as the first time a PD-(L)1 check­point in­hibitor has shown first-line ef­fi­ca­cy across all up­per GI tu­mor lo­ca­tions, in­clud­ing in the stom­ach, gas­troe­sophageal junc­tion, and esoph­a­gus. The study backed up re­sults from CHECK­MATE-649, which test­ed Op­di­vo plus chemo in first-line pa­tients with ad­vanced or metasta­t­ic gas­tric can­cer, gas­troe­sophageal junc­tion can­cer or esophageal ade­no­car­ci­no­ma.

The FDA agreed to re­view re­sults from that study as part of a pri­or­i­ty re­view grant­ed in Feb­ru­ary, with an ac­tion date set for May 25. Earn­ing a nod in those ear­ly pa­tients would be a clear leg up on Mer­ck’s su­per­star check­point in­hibitor Keytru­da, which sports an ap­proval in third-line pa­tients with the PD-(L)1 bio­mark­er dat­ing back to Sep­tem­ber 2017 but has since flopped con­fir­ma­to­ry tri­als in first- and sec­ond-line use.

CHECK­MATE-648 is an­oth­er win un­der Bris­tol My­ers’ belt as it pre­pares for a three-day ad­comm in April to re­view a broad range of im­mune check­point in­di­ca­tions with ac­cel­er­at­ed ap­provals that flopped con­fir­ma­to­ry stud­ies, in­clud­ing a sin­gle Op­di­vo in­di­ca­tion.

In De­cem­ber, Bris­tol walked on Op­di­vo’s third-line-or-lat­er FDA ap­proval for small-cell lung can­cer af­ter con­fir­ma­to­ry tri­als for an ac­cel­er­at­ed nod in that in­di­ca­tion back in 2018 failed to show ben­e­fit in ex­tend­ing pa­tients’ lives. Based on find­ings from its Check­mate-451 and -331 tri­als, which found Op­di­vo failed to hit its OS pri­ma­ry end­points as both a monother­a­py and com­bo treat­ment with Yer­voy, Bris­tol de­cid­ed to pull its ap­proval based on the FDA’s rec­om­men­da­tions to fol­low post-mar­ket­ing sci­ence.

Just a week be­fore that, Bris­tol opt­ed to for­go its chances in brain can­cer glioblas­toma, an­oth­er chron­i­cal­ly dif­fi­cult-to-treat in­di­ca­tion, fol­low­ing mul­ti­ple clin­i­cal fails. The drug­mak­er ad­mit­ted a com­bo of Op­di­vo on top of stan­dard-of-care temo­zolo­mide plus ra­di­a­tion couldn’t beat place­bo in ex­tend­ing over­all sur­vival in pa­tients with new­ly di­ag­nosed MGMT-pos­i­tive glioblas­toma mul­ti­forme who pre­vi­ous­ly had their tu­mor sur­gi­cal­ly re­moved.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Emma Walmsley, GlaxoSmithKline CEO (Kevin Dietsch/Pool via CNP/Alamy)

Glax­o­SmithK­line hus­tles the 7th PD-1 past the fin­ish line with Jem­per­li. But how big will up­take be?

Everything came up sevens for GlaxoSmithKline on Thursday as the pharma notched the seventh PD-1 approval seven years after the first such drugs were OK’ed in Keytruda and Opdivo. But will it bring GSK good fortune?

The FDA granted accelerated approval to dostarlimab, to be branded Jemperli, to treat recurrent or advanced endometrial cancer in a specific subset of patients following platinum-based chemo. It’s a drug that came to GSK through its buyout of Tesaro, which it snapped up for $5.1 billion back in December 2018.

Bio­phar­ma ramps up lob­by­ing spend as drug pric­ing leg­is­la­tion nears

The top biopharma companies in the world collectively spent more than $40 million in just the first quarter of 2021 on lobbying Congress as part of preparations to stave off major drug pricing legislation that’s expected later this year.

Although the numbers are not dramatically higher than what the companies collectively spent in the first quarter of 2020, some like GlaxoSmithKline, Teva, Merck and Johnson & Johnson have already increased their quarterly lobbying spend in 2021 by about $1 million more each when compared to recent quarters in 2020.

House Committee on Oversight and Reform Chairwoman Carolyn Maloney (Getty Images)

House De­moc­rats call on Emer­gent ex­ecs to tes­ti­fy on qual­i­ty is­sues next month

The House Oversight Committee is investigating Covid-19 vaccine producer Emergent BioSolutions, which secured a $628 million US government contract to make AstraZeneca and J&J vaccines despite “a long, documented history” of quality control issues, Democrats said in a letter to the contract manufacturer’s executives.

Emergent’s Baltimore plant, which was shuttered on Monday by FDA, has been embroiled in controversy after being forced to destroy millions of AstraZeneca and J&J doses due to an ingredient mix-up and possible contamination.

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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