Mer­ck in­creas­es grip on its lead in lung can­cer, win­ning ap­proval for Keytru­da/chemo com­bo as first-line ther­a­py

Mer­ck has won its bid to ad­vance its lead­er­ship in treat­ing the cru­cial non-small cell lung can­cer mar­ket, gain­ing an FDA ap­proval to start mar­ket­ing a com­bi­na­tion of its PD-1 drug Keytru­da with chemother­a­py as a first-line ther­a­py. And its shares im­me­di­ate­ly popped, surg­ing 4% on the news in af­ter-mar­ket trad­ing while ri­val Bris­tol-My­ers Squibb took an­oth­er beat­ing, slid­ing 2%.

Mer­ck built its bid for an ac­cel­er­at­ed ap­proval around Phase II da­ta demon­strat­ing that the chemo/check­point com­bo spurred a much high­er con­cen­tra­tion of over­all re­spons­es for NSCLC than chemo alone — 55% vs. 29%, P = 0.0016. High PD-L1 ex­pressers did even bet­ter, though the ap­proval comes through for all pa­tients re­gard­less of bio­mark­er sta­tus. Here’s the link to the new la­bel.

That all trans­lates in­to bil­lions in rev­enue, with con­sen­sus sales fore­cast of $3.6 bil­lion for 2017 rev­enue. And climb­ing.

Roger Perl­mut­ter, Mer­ck

Bris­tol-My­ers un­wit­ting­ly gave Mer­ck’s ri­val ther­a­py a big leg up when its big bid for a front­line ap­proval col­lapsed in Phase III, forc­ing the big biotech to shake up its R&D group and re­fo­cus on its biggest sin­gle pipeline ef­fort. While the check­points have been muscling in­to a va­ri­ety of mar­kets, Mer­ck’s R&D team un­der Roger Perl­mut­ter has won at least a tem­po­rary ad­van­tage in a block­buster are­na. And an­a­lysts have been acute­ly aware of the rev­enue po­ten­tial — though noth­ing is writ­ten in stone in this fast-chang­ing can­cer field.

That les­son was re­in­forced Wednes­day morn­ing, when Roche an­nounced the stun­ning fail­ure of Tecen­triq in a Phase III blad­der can­cer study, af­ter it had al­ready won an ac­cel­er­at­ed ap­proval.

Sea­mus Fer­nan­dez at Leerink re­cent­ly not­ed just how sig­nif­i­cant this de­ci­sion was for Mer­ck, writ­ing:

We con­tin­ue to look to­wards the 5/10/17 PDU­FA da­ta for the Keytru­da (pem­brolizum­ab; an­ti-PD-1) + chemo com­bo in first-line (1L) non-small cell lung can­cer (NSCLC) for a mean­ing­ful ac­cel­er­a­tion in the drug’s US sales. Al­though this will help to dri­ve top- and bot­tom-line growth to­wards the end of the year and in­to 1H:18, the large num­ber of IO+IO and IO+chemo tri­als re­port­ing dur­ing that pe­ri­od could dra­mat­i­cal­ly al­ter the 1L NSCLC land­scape.

When it comes to check­points, with 5 ap­proved and more pil­ing in, every new goal is at­tract­ing a swarm of ri­vals.

As­traZeneca, which just gained an un­ex­cep­tion­al first ap­proval for dur­val­um­ab in blad­der can­cer, has been putting all of its chips on the MYS­TIC tri­al, which com­bines dur­val­um­ab with its ex­per­i­men­tal CT­LA-4 ther­a­py treme­li­mum­ab for lung can­cer. But an­a­lysts have been in­creas­ing­ly leery about CT­LA-4 as a com­bi­na­tion, not­ing its high lev­els of tox­i­c­i­ty — which may prove an easy tar­get for a next-gen suc­ces­sor.

“This ap­proval marks an im­por­tant mile­stone in the treat­ment of lung can­cer. Now, pem­brolizum­ab in com­bi­na­tion with peme­trexed and car­bo­platin can be pre­scribed in the first-line set­ting for pa­tients with metasta­t­ic non­squa­mous non-small cell lung can­cer, ir­re­spec­tive of PD-L1 ex­pres­sion,” said Dr. Corey Langer, di­rec­tor of tho­racic on­col­o­gy and pro­fes­sor of med­i­cine at the Hos­pi­tal of the Uni­ver­si­ty of Penn­syl­va­nia, in a state­ment. “Physi­cians should con­tin­ue to use each pa­tient’s in­di­vid­ual char­ac­ter­is­tics – in­clud­ing bio­mark­er sta­tus, his­tol­ogy, and oth­er clin­i­cal fac­tors – to de­ter­mine the best treat­ment plan for each per­son.”

To­day Mer­ck winds up one big step for­ward in NSCLC. How long it keeps that lead, though, is any­body’s guess.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.