Gilead wa­gers $109M — cash — that this biotech can il­lu­mi­nate a drug dis­cov­ery path point­ed straight in­to the kid­ney

By any ob­jec­tive mea­sure, Gilead has learned as much or more than vir­tu­al­ly any oth­er bio­phar­ma play­er about the liv­er. Now they’re go­ing deep­er, much deep­er, in­to the kid­ney with the help of a Cam­bridge, MA-based biotech you may have for­got­ten about.

Ab­bie Cel­niker

In a new dis­cov­ery pact be­ing un­veiled Wednes­day morn­ing, the big biotech $GILD is com­mit­ting $109 mil­lion — in cash — to start work with a biotech few in the glob­al in­dus­try will like­ly re­call off hand. Goldfinch Bio, a clas­sic Third Rock start­up craft­ed un­der the lead­er­ship of Ab­bie Cel­niker, has been build­ing what it rather grand­ly calls the Kid­ney Genome At­las to gath­er to­geth­er a trea­sure trove of ge­net­ics and clin­i­cal da­ta de­vot­ed en­tire­ly to the kid­ney. And they’ve added a bi­ol­o­gy plat­form of kid­ney cells and “organoids” to find drugs that can hit the pre­ci­sion tar­gets they’ve iden­ti­fied.

Tony John­son

Now helmed by Tony John­son, an As­traZeneca vet­er­an who had worked with Mene Pan­ga­los’ group in the UK, this is Goldfinch’s first big re­search al­liance. Af­ter get­ting $55 mil­lion in A round mon­ey close to 3 years ago, the com­pa­ny qui­et­ly added $29 mil­lion more to ex­tend the run­way through the tech build­out. 

They are aim­ing at the clin­ic, us­ing their ge­net­ics ex­per­tise and bi­ol­o­gy to avoid the mul­ti­ple mishaps that have oc­curred with kid­ney drug R&D in the past.

“We an­tic­i­pate val­i­dat­ing the first tar­get next year,” John­son tells me, then it’s on to the first mol­e­cule and in­to the clin­ic.

Gilead’s cash com­mit­ment — $50 mil­lion up­front, $5 mil­lion for eq­ui­ty and $54 mil­lion for re­search sup­port to flesh out its work specif­i­cal­ly on di­a­bet­ic kid­ney dis­ease — is an un­usu­al­ly large cash com­mit­ment for a dis­cov­ery deal, which helps ex­plain the over­sized $1.95 bil­lion in biobucks on the ta­ble for up to 5 pro­grams. 

The deal doesn’t cap the work at 5 drugs, but it’s a handy way to out­line the up­side.

For Gilead, it’s a way to build out a seg­ment of the pipeline that hasn’t re­ceived a lot of at­ten­tion. John­son notes a mid-stage pro­gram for an ASK1 drug in fi­bro­sis, where the com­pa­ny has been cen­tered on a va­ri­ety of ther­a­pies. A Gilead spokesman tells me:

We have been study­ing kid­ney dis­ease pre­clin­i­cal­ly and clin­i­cal­ly for sev­er­al years. Our pipeline builds up­on our ex­per­tise in the fields of in­flam­ma­tion and fi­bro­sis bi­ol­o­gy. These are process­es in­volved in a num­ber of kid­ney dis­eases in­clud­ing DKD, lu­pus nephri­tis and oth­er con­di­tions. Our aim is to de­vel­op nov­el dis­ease mod­i­fy­ing ther­a­pies for pa­tients with kid­ney dis­eases as we grow our pres­ence in in­flam­ma­tion and fi­bro­sis.

Gilead’s $5 mil­lion for eq­ui­ty makes it part of the syn­di­cate now. John­son says they’ll be think­ing more about the right time for an IPO as they get clos­er to proof of con­cept da­ta on new drugs. In the mean­time, they’ve been build­ing some strength on the AI side, look­ing to cre­ate a com­pu­ta­tion­al group that can plumb the da­ta they’ve been gath­er­ing to help spot­light tar­gets and drugs. 

If this all works out, the next 3 years at Goldfinch will be a lot more noisy than the last 3.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin and UBS in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.

UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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