Quin Wills (L) and Jack O’Meara

A UK biotech looks to up­set the pre­clin­i­cal mod­el for liv­er drug de­vel­op­ment, and now it's gear­ing up for the clin­ic

Liv­er dis­ease is one of the hard­est ther­a­peu­tic ar­eas to hit, and some re­searchers have point­ed to a dearth of mean­ing­ful pre­clin­i­cal mod­els dur­ing drug de­vel­op­ment. Now, a UK biotech has fig­ured out a workaround for its own siR­NA liv­er drugs, and it all starts with dis­card­ed hu­man or­gans.

Ochre Bio an­nounced the clos­ing of a $9.6 mil­lion seed fi­nanc­ing round, led by Khosla Ven­tures. Backed VC, Apol­lo Health Ven­tures, Selvedge, Ho­ton and Her­mes Epitek al­so par­tic­i­pat­ed in the fundrais­ing.

Ochre, which is led by co-founders Quin Wills and Jack O’Meara, us­es a strat­e­gy called “deep phe­nomics” to scan do­nat­ed liv­ers and dig­i­tize that in­fo in­to a crunch­able da­ta set. So far, the group says it has iden­ti­fied 200 new tar­gets for liv­er dis­ease.

In­stead of work­ing with an­i­mal mod­els, the com­pa­ny goes straight to hu­man liv­ers that have been do­nat­ed and deemed un­fit for use in trans­plants. The group tests its siR­NA ther­a­pies di­rect­ly on those “ex­plant­ed” liv­ers main­tained out­side the body. But the com­pa­ny has more planned: By 2022, the biotech says, it will have de­vel­oped a com­plete­ly in sil­i­co liv­er that can be used to dig­i­tal­ly per­turb a range of genes to iden­ti­fy new tar­gets at an even high­er scale.

While Wills was work­ing as the head of cel­lu­lar and sys­tems ge­nomics for No­vo Nordisk, he was pitch­ing a pared-down ver­sion of the work Ochre is do­ing, and he in­creas­ing­ly re­al­ized that dis­cov­ery in the space was go­ing the same way as a lot of car­diometa­bol­ic dis­eases.

“You can find a new tar­get if you want, but where do you go with this? Be­cause there are very poor in vit­ro mod­els, ter­ri­ble an­i­mal mod­els of un­clear rel­e­vance, the clin­i­cal tri­als have un­clear end­points, lack of bio­mark­ers,” he said in an in­ter­view with End­points News. “It’s just a messy messy space, and I don’t want to do tar­get dis­cov­ery for the next 10 years and not see it go any­where.”

The com­pa­ny is aim­ing to head to clin­i­cal tri­als by 2023. This fund­ing will help on­board five trans­plant cen­ter part­ners on dis­card­ed donor liv­ers. The com­pa­ny will go through an­oth­er round of fund­ing be­fore it heads to clin­i­cal tri­als, O’Meara said in an in­ter­view.

In a state­ment, Alex Mor­gan, part­ner of Khosla Ven­tures, said:

I have been im­pressed with the progress this very tal­ent­ed team has made to ac­cel­er­ate de­vel­op­ment of their deep phe­no­typ­ing plat­form. The liv­er is a ma­jor con­ver­gence point in hu­man me­tab­o­lism and many as­pects of health through­out the body, and the com­pa­ny’s ap­proach of de­vel­op­ing treat­ments that can im­prove the trans­plant suc­cess of liv­ers and then us­ing those in­sights to cre­ate ther­a­pies for the larg­er mar­ket of gen­er­al dis­eases as­so­ci­at­ed with the liv­er is a cre­ative and unique ap­proach.

Alex Mor­gan

The com­pa­ny, which re­lies on RNA, has been able to ride the suc­cess that has come with the tech­nol­o­gy’s jour­ney in­to be­com­ing a house­hold name, Wills said, thanks in part to Mod­er­na and Pfiz­er’s Covid-19 vac­cines.

“It’s def­i­nite­ly helped in terms of per­cep­tion, that’s very im­por­tant for us in terms of the fund­ing we’ve had, and it’s im­por­tant in terms of peo­ple who’ve want­ed to work with us,” Wills said. “If I had said even a year ago, peo­ple would have been far less re­cep­tive.”

The com­pa­ny al­so re­cent­ly opened up a Tai­wanese lab to study liv­er dis­ease in Asia, in ad­di­tion to its glob­al net­work of trans­plant part­ners that it has es­tab­lished in Eu­rope and North Amer­i­ca.

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.

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.

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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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.

Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

UP­DAT­ED: Sanofi buys mR­NA play­er Trans­late Bio for $3.2B. And the price fits a pop­u­lar range for biotech M&A

Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio $TBIO a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines, Sanofi closed the deal with a buyout early Tuesday, spending $38 a share in a $3.2 billion buyout.

Translate’s stock $TBIO soared after the market closed Monday when Reuters reported the first word of the acquisition just hours ahead of the formal announcement. The wire service, though, didn’t have a price to report in its scoop, and investors chased the stock up 78% in the wild ride that followed. Once the price was announced, gains shriveled to 29% ahead of the bell.

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