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

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.