BioReg­num: The mob is back. And what's to stop them from go­ing af­ter the en­tire bio­phar­ma in­dus­try?

John Car­roll, Ed­i­tor

My­lan to­day is at the cen­ter of the lat­est on­line mael­strom to erupt over drug pric­ing and ac­cess to ther­a­pies. And while it hun­kers down to see if it can wait out the storm that has erupt­ed over the EpiPen, the one man who can do it the most dam­age is rid­ing to its res­cue.

En­ter Mar­tin Shkre­li, for­mer biotech CEO and the most wide­ly hat­ed in­di­vid­ual in bio­phar­ma his­to­ry. Shkre­li spe­cial­ized in taunt­ing the dig­i­tal lynch mob that formed af­ter he raised the price of an an­cient drug called dara­prim by more than 5000% af­ter snatch­ing it up for a song. And he kept the crowd stirred by reneg­ing on a promise to roll back the price.

The Shkre­li de­fense — re­lax, in­sur­ance can pay for most of it — won’t fly. But that didn’t stop him from rolling it back out any­way.

“My­lan’s a good guy,” Shkre­li told CBS. “They have one prod­uct where they’re fi­nal­ly start­ing to make a lit­tle bit of mon­ey and every­one’s go­ing crazy over it….That’s up to in­sur­ance to pay for them,” he replied to a ques­tion. “It’s $300 a pen. $300. My iPhone is $700…. It’s $300 and 90% of Amer­i­cans are in­sured.”

This is some­thing like hav­ing a se­r­i­al killer ap­pear as a char­ac­ter wit­ness at your mur­der tri­al.

Mar­tin Shkre­li at the De­vel­op­ments in the Pre­scrip­tion Drug Mar­ket: Over­sight hear­ing, 2016

My­lan got in­to this po­si­tion by fol­low­ing the same strat­e­gy as Shkre­li, buy­ing an old prod­uct and jack­ing up the price, but fol­lowed a less risky ap­proach by tak­ing some time to do it. It blew up in­to a scan­dal in any case. And now the com­pa­ny is tak­ing the place of Valeant, which fol­lowed the same ap­proach and got shred­ded by the fall­out.

My­lan, Valeant and the rest of Big Phar­ma rou­tine­ly en­gaged in price goug­ing for the sim­ple rea­son that they can. No law ex­ists that pre­vents it. But hop­ing that the mob won’t sud­den­ly ap­pear at its door — along with Shkre­li — isn’t a vi­able strat­e­gy any more.

Bio­phar­ma needs to swear off this ad­dic­tion to price goug­ing and en­gage in some roll­backs, start­ing with My­lan. Com­pa­nies can pub­licly state that they’re op­posed to it and will stop the prac­tice, be­cause it is un­eth­i­cal. And if they don’t take the pledge, they can bear the con­se­quences. Oth­er­wise, every­one can face off against the grow­ing clam­or for drug pric­ing re­form in Con­gress.

This is an in­dus­try cri­sis. And the in­dus­try needs to re­spond. This whole pric­ing struc­ture for drugs is un­sus­tain­able in the US. It’s nice to ex­per­i­ment with new ap­proach­es like pay­ing for the right out­comes, but change has to hap­pen now. The al­ter­na­tive is to face con­trols on pric­ing for new and im­por­tant drugs, where com­pa­nies have a very good ar­gu­ment for charg­ing some very high rates.

Some­thing’s got to give.

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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Pfizer, South San Francisco — Jeff Rumans for Endpoints News

Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.
Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.
JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.
As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.
Here’s what physicians and patients are likely to be confronted with in the not too distant future, as Pfizer goes about the tricky business of getting a JAK inhibitor past regulators at the FDA and EMA.
Lined up side by side we see:
IGA response rate (clear or nearly clear skin)

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

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A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

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Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

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