Armed with $55M in Third Rock cash, Goldfinch Bio sets out to build a kid­ney dis­ease pipeline

Three years ago, Pe­ter Mundel at Har­vard Med­ical School pub­lished an in­trigu­ing study in the New Eng­land Jour­nal of Med­i­cine that high­light­ed the promise of abat­a­cept in treat­ing a threat­en­ing kid­ney dis­ease. Like many in the field, he was look­ing for ways to adapt ex­ist­ing drugs to fight kid­ney dis­ease.

Now, though, Mundel has shut down his lab at Har­vard and joined a Third Rock start­up that is com­ing out of stealth mode to­day. But in­stead of adapt­ing old drugs for kid­ney dis­ease, the goal is to de­vel­op a pipeline of nov­el ther­a­pies, armed with new in­sights in­to the ge­net­ics of what trig­gers these ail­ments, and how they can be treat­ed.

It’s a clas­sic Third Rock mod­el. The com­pa­ny was seed­ed for months as ven­ture in­vestors drew to­geth­er a kind of who’s who in kid­ney dis­ease to ad­vise it. There’s a start­up staff of 10, which will soon grow to about 25, and some key po­si­tions are be­ing filled by Third Rock play­ers who will grad­u­al­ly hand over their jobs as the start­up builds its team. And there’s an A round of $55 mil­lion from Third Rock to get the team to some proof-of-con­cept da­ta af­ter they get in­to the clin­ic.

“Our first tar­get we think is in­volved in the main path­way dri­ving FS­GS (fo­cal seg­men­tal glomeru­loscle­ro­sis, char­ac­ter­ized by scar­ring of the kid­ney’s fil­ter­ing units),” says Third Rock part­ner Ab­bie Cel­niker, who will be tak­ing the helm on an in­ter­im ba­sis for her first CEO role as a new part­ner at Third Rock. That’s a fa­mil­iar tar­get for Mundel. And the com­pa­ny will be ex­plor­ing the broad­er set of dis­eases that FS­GS is in­volved in as it ramps up new pro­grams.

“I think its im­por­tant to rec­og­nize there has nev­er been a re­al kid­ney spe­cif­ic med­i­cine,” Mundel tells me. Kid­ney drugs are typ­i­cal­ly de­vel­oped for oth­er con­di­tions and then tried in the kid­ney. “We are re­al­ly go­ing at the root; kid­ney spe­cif­ic tar­gets com­ing out of our ge­nom­ic in­for­ma­tion.”

Mundel has joined Goldfinch as the se­nior vice pres­i­dent of bi­ol­o­gy. Jean-Christophe Har­mange is the se­nior vice pres­i­dent of drug dis­cov­ery. Clin­i­cal ge­neti­cist and Third Rock ven­ture part­ner Phil Reil­ly will serve as the com­pa­ny’s in­ter­im chief med­ical of­fi­cer. Third Rock part­ner Neil Ex­ter will serve as in­ter­im chief op­er­at­ing of­fi­cer.

“I think this is a great time to ful­fill the dream of mak­ing kid­ney spe­cif­ic drugs,” says Mundel. “And it’s great to be in this Third Rock en­vi­ron­ment.”

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Evotec CEO Werner Lanthaler, File Photo

Ox­ford, Evotec ramp up LAB10x with AI ex­perts at Sen­syne — fo­cused on biotech spin­outs

Ox­ford is al­ly­ing it­self with Evotec and ar­ti­fi­cial in­tel­li­gence out­fit Sen­syne Health to ramp up some new biotech spin­outs while look­ing to “ac­cel­er­ate da­ta-dri­ven drug dis­cov­ery and de­vel­op­ment.”

The big idea here is that Ox­ford sci­en­tists — some of the best drug hunters in the world — can uti­lize Sen­syne’s AI plat­form for their work, re­ly­ing on the chemists and hands-on de­vel­op­ers at Evotec to push ahead to a crit­i­cal proof of con­cept mo­ment. And they’ll do it through a project leader called LAB10x, which gets £5 mil­lion over the next three years to fund the work.

Suf­fer­ing No­var­tis part­ner Cona­tus is pack­ing it in on NASH af­ter a se­ries of un­for­tu­nate tri­al events

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Dean Hum. Nasdaq via YouTube

Gen­fit goes to Chi­na with a deal worth up to $228M for NASH drug

Fresh off the high of its Nas­daq IPO de­but, and the low of com­par­isons to Cymabay — whose NASH drug re­cent­ly stum­bled — Gen­fit on Mon­day un­veiled an up to $228 mil­lion deal with transpa­cif­ic biotech Terns Phar­ma­ceu­ti­cals to de­vel­op its flag­ship ex­per­i­men­tal liv­er drug — elafi­bra­nor — in Greater Chi­na.

The deal comes more than a week af­ter Gen­fit $GN­FT is­sued a fiery de­fense of its dual PPAR ag­o­nist elafi­bra­nor, when com­peti­tor Cymabay’s PPARδ ag­o­nist, se­ladel­par, fiz­zled in a snap­shot of da­ta from an on­go­ing mid-stage tri­al. The main goal at the end of 12 weeks was for se­ladel­par to in­duce a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in liv­er fat con­tent, but da­ta showed that pa­tients on the place­bo ac­tu­al­ly per­formed bet­ter.

Alex­ion wins pri­or­i­ty re­view for Ul­tomiris' aHUS in­di­ca­tion; FDA ex­pands ap­proval of Ver­tex's Symdeko

→ Alex­ion $ALXN has scored a speedy re­view for Ul­tomiris for pa­tients with atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) af­ter post­ing pos­i­tive da­ta from a piv­otal study in Jan­u­ary. The drug is the rare dis­ease com­pa­ny’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks. The de­ci­sion date has been set to Oc­to­ber 19. Late last year, Ul­tomiris se­cured ap­proval for noc­tur­nal he­mo­glo­bin­uria (PNH) pa­tients.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.