Dai­ichi Sankyo lines up a $900M deal for rights to a cho­les­terol drug look­ing to dis­rupt a block­buster mar­ket

While Es­pe­ri­on’s in­vestors have been through a roller coast­er of re­ac­tions on whether or not the FDA was will­ing to ac­cept LDL low­er­ing as a suit­able bio­mark­er for an ap­proval — ahead of an out­comes study — the CEO says that Eu­ro­pean reg­u­la­tors have con­sis­tent­ly nod­ded their ap­proval through­out the de­vel­op­ment process. 

And that’s helped give Dai­ichi Sankyo con­fi­dence to of­fer $300 mil­lion in two pay­ments to li­cense EU and Swiss rights to their late-stage cho­les­terol drug be­mpe­doic acid as Es­pe­ri­on $ES­PR po­si­tions it­self for a Q1 FDA sub­mis­sion fol­lowed up by a quick Eu­ro­pean pitch.

“The EMA nev­er wa­vered,” Es­pe­ri­on CEO Tim Mayleben tells me in a pre­view to to­day’s an­nounce­ment. “There was nev­er any dra­ma from the EMA on that point.”

Mayleben and the Es­pe­ri­on team will now get a $150 mil­lion up­front from the Japan­ese com­pa­ny, with an­oth­er $150 mil­lion due on the first com­mer­cial sale of the drug in their new ter­ri­to­ry. To­tal mile­stones rack up to $900 mil­lion.

That gives Mayleben the cash to put to­geth­er the com­mer­cial op­er­a­tions he wants in the US, with some more time to fol­low up on ne­go­ti­a­tions for Chi­nese and Japan­ese rights.

The deal comes short­ly af­ter Es­pe­ri­on tout­ed da­ta from its 52-week safe­ty study, the last in a se­ries of Phase III hur­dles the biotech has had to clear as skep­tics picked over its strat­e­gy and pooh-poohed its prospects. An­a­lysts back­ing the com­pa­ny, though, have sup­port­ed Es­pe­ri­on’s pitch that be­mpe­doic acid can be priced un­der the PC­SK9 drugs from Am­gen and Re­gen­eron/Sanofi, hit­ting a sub­stan­tial mar­ket of peo­ple in need of sig­nif­i­cant, though not dra­mat­ic, low­er­ing of LDL.

Dai­ichi, says Mayleben, has had years of ex­pe­ri­ence mar­ket­ing car­di­ol­o­gy drugs, po­si­tion­ing them to play to a slate of sin­gle-pay­er sys­tems that he be­lieves will be re­cep­tive to their ap­proach, an­gling to un­der­cut PC­SK9 on price. In Eu­rope alone the com­mer­cial team amounts to about 1,000 staffers.

Whether the PC­SK9 pro­duc­ers will sit still as Es­pe­ri­on and Dai­ichi carve away mar­ket share, though, has been a reg­u­lar con­cern for crit­ics of Es­pe­ri­on, who be­lieve the big com­pa­nies be­hind those drugs are in a po­si­tion to crush be­mpe­doic acid at any time. In the mean­time, the PC­SK9 play­ers have been slash­ing their prices in an at­tempt to over­come a set of hur­dles pay­ers set up to pre­vent US mem­bers from ac­cess­ing a ther­a­py they see as far too ex­pen­sive.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Covid-19 roundup: Pfiz­er im­pos­es vac­cine man­date for US work­ers; WHO calls for mora­to­ri­um on boost­ers, while some coun­tries make plans any­way — re­port

As the US struggles to keep pace with the fast-spreading Delta variant, big companies like Walmart and Disney are imposing vaccine mandates for some workers. It may come as no surprise that Pfizer — the Big Pharma behind the US’ first authorized Covid-19 vaccine — is joining them.

Pfizer will start requiring all US employees and contractors to get vaccinated, or participate in weekly Covid-19 testing, spokesperson Pamela Eisele told Reuters. Workers outside the US are strongly urged to get a vaccine if they can, according to the report. And those with medical conditions or religious objections can seek accommodations.

Tien Lee, Aardvark Therapeutics CEO

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aardvark Therapeutics CEO Tien Lee started his work on the biotech’s lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see added benefits with lower blood glucose levels and anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced today that they’ve raised enough cash in the B round to cover mid-stage development work.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.