Arch joins Cel­gene, Chi­na in­vestors bankrolling a pi­o­neer­ing New York biotech start­up fo­cused on a new ap­proach to metas­ta­sis

Julio Aguirre-Ghiso has steered the work in his lab at Mount Sinai down some of the less-trav­eled path­ways of can­cer re­search. And it’s led him to launch a biotech — which is hav­ing its com­ing-out par­ty to­day in New York — that has now set out to de­vel­op new med­i­cines tai­lored for the field he’s been pi­o­neer­ing.

While a mas­sive amount of can­cer re­search has been de­vot­ed to a bet­ter un­der­stand­ing of tu­mors and new and bet­ter ways to kill tu­mor cells, Aguirre-Ghiso’s re­search has cen­tered on “dor­mant” dis­sem­i­nat­ed tu­mor cells, or DTCs, that can re­ac­ti­vate long af­ter drugs have wiped out all ap­pear­ances of can­cer — fu­el­ing a metasta­t­ic re­sponse that comes back to kill the pa­tient with near cer­tain­ty.

Ari Nowacek

That lab work has be­come the foun­da­tion of Hi­ber­Cell, a new com­pa­ny seed­ed by Arch last sum­mer and now de­but­ing with a $60.75 mil­lion launch round. Ari Nowacek, a prin­ci­pal at Arch who has helped cham­pi­on the com­pa­ny, is step­ping in as BD chief for the start­up, which has a small, vir­tu­al team of 5 now run­ning the show.

The syn­di­cate Nowacek helped form is telling about the way mon­ey comes to­geth­er in the bio­phar­ma world these days. Arch al­lied it­self, as it has be­fore, with Hill­house and 6 Di­men­sions out of Chi­na. Cel­gene, still op­er­at­ing as an in­de­pen­dent com­pa­ny in the lead-up to fi­nal­iz­ing the Bris­tol-My­ers buy­out, stepped in here as well. The NYC Life Sci­ences Fund, ea­ger to help fos­ter a Big Ap­ple hub, al­so con­tributed cash, along­side a group of uniden­ti­fied in­sti­tu­tion­al in­vestors and in­di­vid­u­als.

There isn’t any­thing par­tic­u­lar­ly new about the no­tion of dis­sem­i­na­tion in can­cer, says Alan Rig­by, the co-founder and CSO of the com­pa­ny. That dates back decades. But Aguirre-Ghiso’s lab has made some im­por­tant break­throughs in the bi­ol­o­gy of dis­sem­i­na­tion, find­ing that “soli­tary cells or mi­cro-clus­ters break away at ear­ly stages and dis­sem­i­nate ear­ly.” 

On his home page on Mount Sinai’s web­site, the sci­en­tist fur­ther ex­plains:

My lab has al­so de­signed an epi­ge­net­ic re­pro­gram­ming ther­a­py to in­duce dor­man­cy of DTCs, which is be­ing de­vel­oped in­to a clin­i­cal tri­al. We al­so dis­cov­ered that UPR sig­nal­ing can pro­mote the sur­vival of dor­mant tu­mor cells and that macrophages are key play­ers in the reg­u­la­tion of ear­ly dis­sem­i­na­tion and dor­man­cy. With mul­ti­ple col­lab­o­ra­tors we run an NCI-Tu­mor Mi­croen­vi­ron­ment Net­work Cen­ter that stud­ies the mi­croen­vi­ron­men­tal stress and dor­man­cy and de­vel­ops new tech­nolo­gies to im­age and tar­get metas­ta­sis. We al­so col­lab­o­rate to char­ac­ter­ize dor­man­cy in hu­man breast, prostate and head and neck can­cer DTCs and we study the epi­ge­net­ic reg­u­la­tion of DTC dor­man­cy. A ma­jor ef­fort in our lab is al­so to de­vel­op a trans­la­tion­al pro­gram with the phar­ma­ceu­ti­cal in­dus­try to iden­ti­fy po­ten­tial drugs to tar­get dor­mant dis­ease.

The re­search they’re do­ing has di­rect ap­pli­ca­tions that are par­tic­u­lar­ly suit­ed for the Chi­na mar­ket, adds Rig­by, cit­ing esophageal, gas­tric and liv­er can­cers. He adds that the launch round should get them in­to 2022, in­clud­ing a nice stretch af­ter they’re slat­ed to get in­to the clin­ic in 2020 or ear­ly ’21 with their first drug.

Rig­by al­so says that the team, which is like­ly to grow some­what, in-li­censed late pre­clin­i­cal drug can­di­dates that they’re work­ing with now. He de­clined to of­fer de­tails about those drugs, not un­usu­al for a start­up look­ing to get out ahead of any po­ten­tial ri­vals that may ap­pear now.


Im­age: Julio Aguirre-Ghiso and Alan Rig­by. HI­BER­CELL

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Adding mar­quee in­vestors, Black­Thorn bags $76M to back an AI-dri­ven strat­e­gy for pre­ci­sion neu­ro med­i­cine

As ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing loom ever larg­er in drug dis­cov­ery and de­vel­op­ment, a biotech op­er­at­ing at the “nexus” of tech­nol­o­gy and neu­ro­sciences has cashed in with $76 mil­lion in fresh fi­nanc­ing.

The big idea at Black­Thorn Ther­a­peu­tics is to do for neu­robe­hav­ioral dis­or­ders what ge­net­i­cal­ly tar­get­ed ther­a­py has done for on­col­o­gy: Re­de­fine pa­tient pop­u­la­tions by the un­der­ly­ing bi­ol­o­gy — dys­reg­u­lat­ed brain cir­cuits, or neu­rotypes — in­stead of symp­toms, there­by find­ing the pa­tients who are most like­ly to ben­e­fit at en­roll­ment phase.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.

News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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Ab­b­Vie touts new da­ta for Hu­mi­ra suc­ces­sor; Gilead inks dis­cov­ery deal

→ Ab­b­Vie is tout­ing new pos­i­tive da­ta com­par­ing their ag­ing block­buster Hu­mi­ra with their hoped-for block­buster upadac­i­tinib. Over 48 weeks a larg­er pro­por­tion of pa­tients tak­ing the ex­per­i­men­tal drug ex­pe­ri­enced clin­i­cal re­mis­sion than in the con­trol arm with Hu­mi­ra. Their drug brought in $20 bil­lion last year, top­ping the scales in the num­ber 1 slot.

→ Gilead has turned to Van­cou­ver-based Ab­Cellera for its lat­est dis­cov­ery deal. Ab­Cellera will use its know-how in “sin­gle-cell screen­ing of nat­ur­al im­mune sources” to find an­ti­body can­di­dates for Gilead to pur­sue in the in­fec­tious dis­ease field. The deal in­cludes an up­front and mile­stones.