An­oth­er biotech games the sys­tem, and every­one gets to pay for the fall­out

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

The FDA got played – again

As I write, our sto­ry on Marathon and its old, cheap steroid — de­flaza­cort — that it steered to an FDA ap­proval yes­ter­day is gain­ing some se­ri­ous trac­tion on so­cial me­dia out­lets like Red­dit. That’s where the on­line mob formed for Mar­tin Shkre­li, af­ter word spread that he and Tur­ing had hiked the price of Dara­prim for HIV users by 5000%-plus. And it’s where you can feel the vis­cer­al pub­lic anger that’s been build­ing over drug prices.

Once again, we have a com­pa­ny that is play­ing the sys­tem, grab­bing a steroid that’s been sold around the world for decades and jack­ing up the rate by a jaw-drop­ping amount. $89,000, in this case, up more than $88,000 over what it costs in Cana­da. Marathon al­so got a pri­or­i­ty re­view vouch­er worth hun­dreds of mil­lions of dol­lars for its slight trou­ble, to rec­og­nize a con­tri­bu­tion for R&D that in­cludes buy­ing rights to a study that was done in 1995 — be­fore the 6-minute walk test be­came a stan­dard gauge of suc­cess.

The FDA gave Marathon every ad­van­tage in the books, and then some. Tak­ing an­oth­er page from the Shkre­li hand­book, Marathon says it will make sure that on­ly in­sur­ers will get gigged, pa­tients shouldn’t have to wor­ry. So un­less you pay for in­sur­ance, you should have no re­al qualms. Right?

It’s all per­fect­ly le­gal. And it’s all per­fect­ly wrong. Un­til Con­gress un­der­stands and acts de­ci­sive­ly to stop it from hap­pen­ing again, the next pric­ing scan­dal will be wait­ing right around the next cor­ner, with some­one at the FDA hold­ing its hand. And each of these erup­tions will se­ri­ous­ly dam­age bio­phar­ma’s rep­u­ta­tion at a time it needs to be seen as play­ing fair.

Scott Got­tlieb re­mains the best choice for FDA com­mish

Ear­ly on Fri­day Tom Price got the nod to run HHS, as ex­pect­ed. So we should hear any­time now who Don­ald Trump has se­lect­ed to run the FDA.

We fear that he’s go­ing to go ahead with Jim O’Neill, a Lib­er­tar­i­an with some wacky ideas about ap­prov­ing drugs based on safe­ty alone. That old de­bate about a drug’s safe­ty/ben­e­fit ra­tio? That would go out the door, along with the rest of the stan­dards that have made US drug de­vel­op­ment the guid­ing force for the world.

We hope that Trump choos­es Scott Got­tlieb. The for­mer deputy com­mis­sion­er un­der­stands what it takes to run the agency, and has the med­ical cre­den­tials to un­der­stand the stakes. As John Maraganore said re­cent­ly, this isn’t like mar­ket­ing a soft drink, where you choose be­tween Pep­si and Coke. It’s lit­er­al­ly a mat­ter of life and death, and the FDA was put in place to make it more about life.

The Marathon scan­dal just proves once again that too many com­pa­nies are ready to start fleec­ing the pub­lic at the ear­li­est pos­si­ble mo­ment. Who­ev­er runs the FDA needs to be pre­pared for that.

Thank you, biotech, for tak­ing a prin­ci­pled stand

As we re­port­ed more than a week ago, it was clear from our snap poll that the biotech in­dus­try was solid­ly op­posed to Trump’s trav­el ban from sev­en pri­mar­i­ly Mus­lim na­tions. This week, 166 ex­ecs lodged their for­mal, for-the-record op­po­si­tion to the ban in a let­ter pub­lished in Na­ture Biotech­nol­o­gy.

This isn’t just about whether these com­pa­nies can re­cruit staffers from these na­tions. The ban strikes fear in the hearts of every­one work­ing in the Unit­ed States on an H1-B visa, which is com­mon through­out sci­ence-ori­ent­ed in­dus­tries in the US. The ban makes all for­eign­ers un­wel­come, re­gard­less of the con­tri­bu­tion they make to sci­ence and these com­pa­nies. And it threat­ens the easy move­ment of tal­ent and ideas, all of which has been an enor­mous ad­van­tage for the US.

Un­for­tu­nate­ly, BIO and PhRMA along with most Big Phar­ma CEOs see no up­side in at­tack­ing Trump’s ex­ec­u­tive or­der, now stayed by the courts. We are not naive. These groups are made to qui­et­ly seek in­flu­ence and lob­by for ben­e­fits, and there are some huge tax ben­e­fits on the ta­ble right now. Un­der any oth­er ad­min­is­tra­tion, at any oth­er time, si­lence could be un­der­stood.

But not this time.

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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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?

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.

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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