Pi­o­neer­ing Mer­ck makes an­oth­er break­through with PD-1 star Keytru­da, grab­bing first OK for a can­cer bio­mark­er

Mer­ck con­tin­ues to be the trend­set­ter to watch in im­muno-on­col­o­gy. On Tues­day the phar­ma gi­ant nabbed a unique first in the field, grab­bing an FDA ap­proval to use Keytru­da to fight can­cer with a par­tic­u­lar bio­mark­er sig­na­ture of its own.

Specif­i­cal­ly, the agency has pro­vid­ed a green light to pre­scribe Keytru­da to fight tu­mors with a bio­mark­er called mi­crosatel­lite in­sta­bil­i­ty-high or mis­match re­pair de­fi­cient.

Richard Padzur

No­tably, can­cer drug czar Richard Paz­dur did the hon­ors in rec­og­niz­ing the achieve­ment in the FDA’s state­ment.

“This is an im­por­tant first for the can­cer com­mu­ni­ty,” Richard Paz­dur, act­ing di­rec­tor of the of­fice of hema­tol­ogy and on­col­o­gy prod­ucts in the FDA’s CDER and di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence, said in a state­ment. “Un­til now, the FDA has ap­proved can­cer treat­ments based on where in the body the can­cer start­ed — for ex­am­ple, lung or breast can­cers. We have now ap­proved a drug based on a tu­mor’s bio­mark­er with­out re­gard to the tu­mor’s orig­i­nal lo­ca­tion.”

The agency sin­gled out tu­mors with ab­nor­mal­i­ties that play a role in DNA re­pair, high­light­ing col­orec­tal, en­dome­tri­al and gas­troin­testi­nal can­cers, but al­so less com­mon­ly in can­cers aris­ing in the breast, prostate, blad­der, thy­roid gland and more.

In this case Mer­ck was able to draw on da­ta from five stud­ies show­ing Keytru­da’s abil­i­ty to shrink a sig­nif­i­cant num­ber of tu­mors.

Roger Perl­mut­ter, Mer­ck

Mer­ck has been rolling up a string of im­pres­sive wins on the I/O front with its PD-1 check­point drug Keytru­da. While Roger Perl­mut­ter was lucky enough to have it in the pipeline when he ar­rived as R&D chief, he has pressed every ad­van­tage in ex­pand­ing on its pi­o­neer­ing first ap­proval.

A week ago the agency fol­lowed through with an OK for blad­der can­cer, its sec­ond in a few weeks. Just this morn­ing the phar­ma gi­ant scored an ac­cel­er­at­ed re­view for stom­ach can­cer, a clear sig­nal of fu­ture suc­cess. And Mer­ck re­searchers will present da­ta on 16 can­cer types at AS­CO in ear­ly June.

Re­cent­ly the phar­ma gi­ant leapfrogged Bris­tol-My­ers Squibb on non-small cell lung can­cer, win­ning an OK to use a com­bi­na­tion of Keytru­da and chemo as a front­line ther­a­py. And there are hun­dreds of ad­di­tion­al stud­ies un­der­way to test its ef­fi­ca­cy in com­bo ther­a­pies.

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrate their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk that uses the same basic ingredient as Genotropin, which the Danish company sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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Sofinno­va-backed Abi­vax touts longer term mid-stage da­ta in ul­cer­a­tive col­i­tis

Two months after Abivax convinced Sofinnova to bankroll several mid-stage studies of its lead drug — ABX464 — with a €12 million stock purchase, the Paris-based biotech has rolled out more data on the anti-inflammatory molecule for all investors to see.

In a Phase IIa maintenance study involving 22 patients with moderate to severe ulcerative colitis who have been failed by previous treatments, 12 achieved clinical remission as assessed by endoscopy. But since only 19 completed the full one-year trial, 16 of whom had an endoscopy, investigators scored the remission rate at 75%. Although there’s no comparator arm, execs were pleased with improvements over an initial two-month, placebo-controlled induction phase by a number of measures ranging from remission to Mayo score and a fecal biomarker.

Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

FDA ap­proval lets Foamix set its maid­en ac­ne ther­a­py on course for US mar­ket launch

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.