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.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.