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.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Turned back at the FDA, Im­muno­Gen is ax­ing 220 staffers, sell­ing pro­grams and hun­ker­ing down for a new PhI­II gam­ble

After being stymied by FDA regulators who were unconvinced by ImmunoGen’s $IMGN desperation shot at an accelerated OK based on a secondary endpoint, the struggling biotech is slashing its workforce, shuttering R&D projects and looking for buyers to pick up some of its experimental cancer assets as it goes back into a new Phase III with the lead drug.

We found out last month that the FDA had batted back their case for an accelerated approval of their antibody-drug conjugate mirvetuximab soravtansine, which had earlier failed a Phase III study for ovarian cancer. Now the other shoe is dropping.

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Bridge­Bio takes crown for biggest biotech IPO of 2019, as fel­low uni­corn Adap­tive rais­es of­fer­ing size and price

Bridge­Bio Phar­ma and Adap­tive Biotech­nolo­gies have not just up­sized IPO of­fer­ings — the pair of uni­corns have al­so raised their of­fer­ing prices above the range, haul­ing in a com­bined $648.5 mil­lion.

Neil Ku­mar’s Bridge­Bio Phar­ma, found­ed in 2015, has a sta­ble of com­pa­nies fo­cused on dis­eases that are dri­ven by de­fects in a sin­gle gene — en­com­pass­ing der­ma­tol­ogy, car­di­ol­o­gy, neu­rol­o­gy, en­docrinol­o­gy, re­nal dis­ease, and oph­thal­mol­o­gy — and can­cers with clear ge­net­ic dri­vers. The start­up mill birthed a pletho­ra of firms such as Ei­dos, Navire, QED Ther­a­peu­tics and Pelle­Pharm, which func­tion as its sub­sidiaries.

As­traZeneca chal­lenges Roche on front­line SCLC af­ter seiz­ing an in­ter­im win — and Mer­ck may not be far be­hind

The crowded playing field in the PD-1/L1 marketing game is about to get a little more complex.

This morning AstraZeneca reported that its CASPIAN study delivered a hit in an interim readout for their PD-L1 Imfinzi combined with etoposide and platinum-based chemotherapy options for frontline cases of small cell lung cancer, a tough target which has already knocked back Bristol-Myers’ shot in second-line cases. The positive data  — which we won’t see before they roll it out at an upcoming scientific conference — give AstraZeneca excellent odds of a quick vault to challenging Roche’s Tecentriq-chemo combo, approved 3 months ago for frontline SCLC in a landmark advance.

“This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options,” noted AstraZeneca cancer R&D chief José Baselga in a statement.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.