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.

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

Learn more about Avance ClinicReady here.
Contact us about your next study.
Download our Frost & Sullivan APAC CRO Report here. 

The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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