Baeuer­le-backed Cul­li­nan grows its pipeline of port­fo­lio plays, adopts a new can­cer drug with eye to clin­i­cal de­vel­op­ment

About 15 months af­ter Cul­li­nan On­col­o­gy de­buted with $150 mil­lion and in­tro­duced its part-fund/part-op­er­at­ing com­pa­ny mod­el for piec­ing to­geth­er a pipeline full of can­cer drugs un­der in­di­vid­ual in­vest­ment ve­hi­cles, the biotech is pub­licly spot­light­ing an ear­ly-stage on­col­o­gy drug that is be­ing adopt­ed af­ter mak­ing the cut.

The biotech struck a deal with Tai­ho Phar­ma­ceu­ti­cals to take the drug and put it in a ve­hi­cle dubbed Cul­li­nan Pearl, which will op­er­ate un­der the um­brel­la or­ga­ni­za­tion now manned by a lean-and-mean team of 15. Tai­ho in turn gets a chunk of eq­ui­ty and its ven­ture arm is in­vest­ing along­side Cul­li­nan in a launch round for Pearl.

The drug here is TAS6417, an EGFR ty­ro­sine ki­nase in­hibitor, which “spares wild type EGFR.” The think­ing is that fea­ture will help the team amp up the po­ten­cy of the drug to dis­tin­guish it from the rest of the drugs in the same field.

Owen Hugh­es

“We are a means to an end,” says Owen Hugh­es, the CEO at Cul­li­nan. Tai­ho had this one nes­tled in among its ear­ly-stage pipeline projects and is li­cens­ing it out for Cul­li­nan to see if it can beat a fast path through the clin­ic — ei­ther un­veil­ing some re­al val­ue or killing it as a los­er.

Back when I talked to Hugh­es in the fall of 2017 dur­ing the for­mal launch of Cul­li­nan, he out­lined a plan to build a pipeline en­tire­ly out of deals with oth­er com­pa­nies, turn­ing to ven­dors to do a lot of the heavy R&D lift­ing for the sub­sidiaries un­der di­rec­tion from the team. 

Since then, he’s had a first-hand les­son on just how com­pet­i­tive that mar­ket is. He’s al­so killed 2 op­tion deals where they had a shot at some promis­ing pro­grams but couldn’t repli­cate the pre­clin­i­cal re­search work that snagged their at­ten­tion.

Hugh­es, though, has an ace in the hole. Patrick Baeuer­le at MPM is the co-founder, fi­nan­cial backer and CSO of the com­pa­ny. He di­rect­ed the Mi­cromet BiTE plat­form, ac­quired by Am­gen, which com­plet­ed the de­vel­op­ment on the first BiTE. And af­ter help­ing launch a se­ries of biotechs in the can­cer space, he’s al­so been help­ing Cul­li­nan come up with a slate of home-grown drugs to add to the pipeline.

Cul­li­nan now has 8 pro­grams un­der the um­brel­la, in­clud­ing VK-2019, an Ep­stein-Barr Nu­clear Anti­gen 1 in­hibitor dis­cov­ered at The Wis­tar In­sti­tute. Hugh­es and the team set up Cul­li­nan Apol­lo for that drug. You can look for 1 or 2 new deals this year, he adds, as the biotech reach­es the high end of the 6 to 10 projects orig­i­nal­ly planned.

The CEO isn’t get­ting spe­cif­ic on the up­fronts and mile­stones for these ear­ly-stage drugs, which wouldn’t like­ly as­tound any­one in this free-flow­ing en­vi­ron­ment. But he did note that the key to cap­tur­ing these pro­grams has less to do with cash than eq­ui­ty. The part­ners get a com­pa­ny com­mit­ted to hus­tling ahead, and a chunk of eq­ui­ty — gen­er­al­ly 20% or less — that can pay off if they score pay dirt da­ta.

So how is the mon­ey hold­ing out? Hugh­es says they re­cent­ly banked their sec­ond $50 mil­lion tranche to push ahead with deal­mak­ing. The third tranche is wait­ing for them as they con­tin­ue to make progress.

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.

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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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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J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

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.

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.

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.

Turns out, Rudy Tanzi did­n't see much of a sto­ry about a hid­den link be­tween En­brel and Alzheimer's ei­ther

The Wash­ing­ton Post man­aged to whip up the quick­est in­dus­try con­sen­sus I’ve ever seen that one of its re­porters was pur­vey­ing overblown non­sense with a sto­ry that Pfiz­er was sit­ting on da­ta sug­gest­ing that En­brel could be an ef­fec­tive treat­ment for Alzheimer’s. 

In cov­er­ing that bit of an­ti-Big Phar­ma fan­ta­sy — there are lots of rea­sons to go af­ter phar­ma, but this piece was lu­di­crous — I not­ed com­ments in the sto­ry from some promi­nent peo­ple in the field crit­i­ciz­ing Pfiz­er for not pub­lish­ing the da­ta. I sin­gled out Rudy Tanzi at Har­vard and then ap­plied some added crit­i­cism for the things he’s done to hype — in my opin­ion — high­ly ques­tion­able as­sump­tions. You can see it in the link. 

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.