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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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ProFound Therapeutics founding team

Flag­ship's lat­est biotech could turn some of the thou­sands of new pro­teins it dis­cov­ered in­to ther­a­pies — and it has $75M to start

Flagship Pioneering, the incubator of Moderna and dozens of other biotechs, says it has landed upon tens of thousands of previously undiscovered human proteins. The VC shop wants to potentially turn them into therapeutics.

Like other drug developers that have turned proteins into therapeutics (think insulin for diabetes), Flagship’s latest creation, ProFound Therapeutics, wants to tap into this new trove of proteins as part of its mission to treat indications ranging from rare diseases to cancer to immunological diseases.

Richard Silverman, Akava Therapeutics founder and Northwestern professor

This time around, Lyri­ca's in­ven­tor is de­vel­op­ing his North­west­ern dis­cov­er­ies at his own biotech

Richard Silverman was left in the dark for the last five years of clinical development of the drug he discovered. The Northwestern University professor found out about the first approval of Lyrica, in the last few days of 2004, like most other people: in the newspaper.

What became one of Pfizer’s top-selling meds, at $5 billion in 2017 global sales before losing patent protection in 2019, started slipping out of his hands when Northwestern licensed it out to Parke-Davis, one of two biotechs that showed interest in developing the drug in the pre-email days, when the university’s two-person tech transfer team had to ship out letters to garner industry appetite.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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Up­dat­ed: US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Simba Gill, CEO of Evelo Biosciences

While down 87% YOY, Evelo gets Flag­ship and oth­ers to in­fuse new cap­i­tal for come­back hope

Just four years after Flagship spinout Evelo Biosciences went public in an IPO worth $85 million, the biotech has seen its share price tank from $13 a share this time last year (ultimately reaching a peak of over $17) to now under $1.50. And today, it looks like Flagship still thinks the fledging biotech, in a down market, is still worth something after initial pre-IPO backing from the likes of Google’s GV, Celgene, Mayo Clinic and Alexandria Venture.

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Peter Thompson, Terremoto Biosciences interim CEO

For­mer Prin­cip­ia team looks to shake up co­va­lent small mol­e­cules again, this time at 'earthquake' scale

Terremoto Biosciences goes back a long ways, in a sense, to about a dozen years ago when Principia Biopharma was founded by UCSF professor Jack Taunton. Peter Thompson initially helmed the biotech.

The company helped expand covalent small molecule inhibitors beyond oncology and into autoimmune disease by targeting cystine. But that amino acid is uncommon in a lot of proteins, offering fewer drug targets than, say, lysine, which is present in most proteins of interest. So, over the years, Taunton went back to the drawing board to check out that second amino acid.

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