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.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,600+ biopharma pros reading Endpoints daily — and it's free.

Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,600+ biopharma pros reading Endpoints daily — and it's free.

AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,600+ biopharma pros reading Endpoints daily — and it's free.

Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,600+ biopharma pros reading Endpoints daily — and it's free.

Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,600+ biopharma pros reading Endpoints daily — and it's free.

So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,600+ biopharma pros reading Endpoints daily — and it's free.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,600+ biopharma pros reading Endpoints daily — and it's free.