Roy Baynes, Merck Research Laboratories CMO (file photo)

Look­ing to re­bound from re­jec­tion in TNBC, Mer­ck­'s Keytru­da pass­es a new kid­ney can­cer test

Keytru­da has long been a dom­i­nant force in bio­phar­ma, rack­ing up bil­lions in sales for Mer­ck and con­tin­u­ing to ex­pand in­to more and more types of can­cer. And com­ing on the heels of a rare CRL in triple-neg­a­tive breast can­cer, the drug took an­oth­er step to­ward a po­ten­tial next in­di­ca­tion.

Mer­ck’s block­buster met its pri­ma­ry end­point in a Phase III study look­ing at Keytru­da as an ad­ju­vant ther­a­py for pa­tients with re­nal cell car­ci­no­ma fol­low­ing sur­gi­cal re­moval of a kid­ney, the Big Phar­ma an­nounced. Com­pared with place­bo, Keytru­da demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in dis­ease-free sur­vival.

“These new da­ta are the re­sult of our re­search to eval­u­ate the role of Keytru­da in help­ing pa­tients with ear­li­er stages of dis­ease,” Mer­ck Re­search Lab­o­ra­to­ries CMO Roy Baynes said in a state­ment, “and are the first pos­i­tive re­sults for an an­ti-PD-1 ther­a­py in the ad­ju­vant treat­ment of pa­tients with re­nal cell car­ci­no­ma.”

Mer­ck did not re­lease spe­cif­ic da­ta points or p-val­ues in their an­nounce­ment, but not­ed that the study en­rolled 950 who were ran­dom­ized in­to re­ceiv­ing ei­ther Keytru­da IV in­fu­sions once every three weeks up to 17 times, or place­bo.

The study is con­tin­u­ing to be eval­u­at­ed for sec­ondary end­points, which in­clude over­all sur­vival and safe­ty, Mer­ck said. Re­searchers did re­veal that safe­ty in this tri­al was con­sis­tent with pre­vi­ous stud­ies of the drug.

Re­nal cell car­ci­no­ma al­ready sits among Keytru­da’s swath of ap­provals, with the drug notch­ing an OK two years ago in com­bi­na­tion with ax­i­tinib for the first-line treat­ment in pa­tients with ad­vanced stages of the can­cer.

Thurs­day’s news comes a lit­tle over a week af­ter the FDA slapped down Mer­ck’s pitch to ex­pand the drug in­to triple-neg­a­tive breast can­cer as both a pre-op­er­a­tive (with chemo) and post-op (with­out chemo) treat­ment with a CRL. Mer­ck had ex­am­ined Keytru­da as a post-sur­gi­cal monother­a­py fol­low­ing a com­bo of Keytru­da and plat­inum-based chemother­a­py, but their da­ta were panned at an ODAC hear­ing in Feb­ru­ary.

The com­mit­tee unan­i­mous­ly rec­om­mend­ed wait­ing for fur­ther da­ta for Mer­ck’s I/O su­per­star, say­ing the phar­ma should wait for more de­fin­i­tive safe­ty da­ta from its Phase III study. At is­sue was that re­searchers hadn’t watched pa­tients long enough af­ter treat­ment to de­ter­mine EFS and OS, hav­ing seen just 53% of tar­get­ed EFS events and 32% of OS events.

But Mer­ck went ahead any­way, de­spite FDA con­cerns over the ma­tu­ri­ty of the dataset af­ter mul­ti­ple meet­ings. The CRL didn’t, how­ev­er, af­fect Keytru­da’s ap­proval as a com­bi­na­tion ther­a­py with chemo in lo­cal­ly ad­vanced, PD-1 ex­press­ing TNBC tu­mors that are metasta­t­ic or can­not be sur­gi­cal­ly re­moved.

The new RCC da­ta al­so come at a time when reg­u­la­tors are get­ting tougher on drug­mak­ers that miss their mark in con­fir­ma­to­ry stud­ies fol­low­ing ac­cel­er­at­ed ap­provals. Al­so last month, Mer­ck pulled a Keytru­da in­di­ca­tion for metasta­t­ic small cell lung can­cer, part of what it de­scribed as an “in­dus­try-wide eval­u­a­tion” by the FDA of such miss­es.

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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