Mer­ck­'s Keytru­da makes the cut as a sec­ond-line monother­a­py for cer­tain esophageal can­cer pa­tients

Mer­ck’s cor­ner­stone check­point in­hibitor Keytru­da has added an­oth­er jew­el to its crown.

The US drug­mak­er on Wednes­day dis­closed that the PD-1 drug had won ap­proval as a monother­a­py for pa­tients with re­cur­rent lo­cal­ly ad­vanced or metasta­t­ic squa­mous cell car­ci­no­ma of the esoph­a­gus, whose tu­mors ex­press PD-L1, and whose dis­ease has pro­gressed de­spite one or more pri­or lines of sys­temic ther­a­py.

About 17,650 new esophageal can­cer cas­es will be di­ag­nosed this year in the Unit­ed States, the Amer­i­can Can­cer So­ci­ety es­ti­mates. Typ­i­cal­ly, pa­tients with the ad­vanced form of the dis­ease have lim­it­ed treat­ment op­tions.

The ap­proval was based on the KEYNOTE-181 study, which test­ed the drug ver­sus chemother­a­py in a broad group of 628 pre­vi­ous­ly-treat­ed pa­tients with re­cur­rent lo­cal­ly ad­vanced or metasta­t­ic esophageal can­cer. The main goal of the study — over­all sur­vival — was met on­ly in a sub­group of pa­tients, in pa­tients whose tu­mors ex­press PD-L1.

In its Jan­u­ary up­date, the com­pa­ny said that Keytru­da re­duced the risk of death by 31% in pa­tients with squa­mous cell car­ci­no­ma or ade­no­car­ci­no­ma in the study. The sub­group with PD-L1-pos­i­tive pa­tients — which con­sti­tut­ed 222 pa­tients — achieved a me­di­an OS of 9.3 months ver­sus 6.7 months for the chemother­a­py group. They al­so had a 12-month OS rate of 43% while the chemother­a­py arm ex­pe­ri­enced 20%.

How­ev­er, for the in­ten­tion-to-treat pop­u­la­tion, the dif­fer­ence in OS be­tween the two arms was not sta­tis­ti­cal­ly sig­nif­i­cant. In the 401 pa­tient sub­group of squa­mous cell car­ci­no­ma pa­tients, the Keytru­da group ex­pe­ri­enced a me­di­an OS of 8.2 months, com­pared to 7.1 months for the chemother­a­py arm — an­oth­er non-sta­tis­ti­cal­ly sig­nif­i­cant dif­fer­ence.

Da­ta from the KEYNOTE-180 study, which in­clud­ed 121 pa­tients with lo­cal­ly ad­vanced or metasta­t­ic esophageal can­cer who pro­gressed on or af­ter at least two pri­or sys­temic treat­ments for ad­vanced dis­ease, al­so con­tributed to the ap­proval, Mer­ck said.

Mer­ck $MRK is al­so run­ning a sep­a­rate Phase III study — dubbed KEYNOTE-590 — pit­ting Keytru­da in com­bi­na­tion with chemother­a­py as a first-line treat­ment in pa­tients with esophageal car­ci­no­ma. The tri­al is ex­pect­ed to wrap up next month.

Mean­while, oth­er check­point ri­vals — from Bris­tol-My­ers Squibb $BMY and BeiGene — are lin­ing up to tack­le the no­to­ri­ous­ly hard-to-treat can­cer.

Keytru­da, which is ap­proved for a pletho­ra of can­cers, has ce­ment­ed its crown in the field of im­muno-on­col­o­gy. It gen­er­at­ed $2.6 bil­lion in sec­ond-quar­ter rev­enue, up 58% over the same pe­ri­od a year ago.

The drug is on track to eclipse $10.6 bil­lion in sales by the end of the year — which is more than 50% over what it gen­er­at­ed in 2018 — and to sur­pass $20 bil­lion by 2024, Mizuho an­a­lysts wrote in a note.

Keytru­da was once seen as an un­der­dog to Bris­tol-My­ers’ pi­o­neer­ing Op­di­vo, but a string of tri­al fail­ures left Op­di­vo in the back­seat and Keytru­da as the dri­ving force. Now, some wor­ry that Mer­ck may be lean­ing too heav­i­ly on its key­stone as­set, but the com­pa­ny’s R&D chief Roger Perl­mut­ter is work­ing on but­tress­ing the com­pa­ny’s pipeline with a string of ac­qui­si­tions to mit­i­gate those con­cerns.

“While KEYTRU­DA is grow­ing in dom­i­nance, both in the mar­ket and on MRK’s P&L, we see MRK as hav­ing con­sid­er­able run­way (2028 LOE) to ad­dress con­cerns re­gard­ing con­cen­tra­tion of rev­enue,” Mizuho an­a­lysts added.

So­cial im­age: Shut­ter­stock

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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