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.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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