Cel­lec­tis re­cruits ex-Servi­er re­search chief De­pil; Are­na inks $224M deal on new drugs; JLABS heads to Shang­hai; As­traZeneca vet takes Goldfinch helm

→ French CAR-T de­vel­op­er Cel­lec­tis has re­cruit­ed Stéphane De­pil, the for­mer R&D chief of Servi­er, to lead the com­pa­ny’s clin­i­cal de­vel­op­ment. De­pil will serve as se­nior vice pres­i­dent of R&D and chief med­ical of­fi­cer. The new ap­point­ment brings De­pil back to the in­dus­try af­ter a break while he di­rect­ed the can­cer im­munother­a­py pro­gram at the Léon Bérard Can­cer Cen­ter in Ly­on, France. But back when he was at Servi­er, he over­saw 20 can­cer pro­grams, sev­er­al of which were in clin­i­cal tri­als. Cel­lec­tis hopes to ben­e­fit from De­pil’s track record in the field to move its CAR-T as­sets in­to the clin­ic af­ter a rough start in­volv­ing a pa­tient death. “Stéphane De­pil’s deep med­ical, aca­d­e­m­ic, and clin­i­cal reg­u­la­to­ry on­col­o­gy ex­pe­ri­ence – specif­i­cal­ly in R&D for all phas­es with­in the phar­ma­ceu­ti­cal, biotech­nol­o­gy, and clin­i­cal re­search spaces – will be in­valu­able as he leads Cel­lec­tis’ strat­e­gy and pro­motes aware­ness of the break­through work that we are do­ing as a leader and in­no­va­tor in the gene-edit­ing field,” said An­dré Chouli­ka, Cel­lec­tis’ CEO, in a state­ment. “His strate­gic al­liance-build­ing, col­lab­o­ra­tion skills, un­der­stand­ing of the glob­al en­vi­ron­ment with on­co­log­i­cal clin­i­cal re­search, and first­hand ex­pe­ri­ence run­ning a phar­ma com­pa­ny all add a great de­gree of abil­i­ty and depth to our lead­er­ship team.”

→ Are­na Phar­ma­ceu­ti­cals, the San Diego drug mak­er best known for its di­et pill Belviq, has inked a deal worth up to $224M with Ever­est Med­i­cines to de­vel­op and com­mer­cial­ize its two new pipeline drugs in main­land Chi­na, Tai­wan, Hong Kong, Macau, and South Ko­rea. The two drugs, ra­linepag and es­trasi­mod, are a far cry from Are­na’s ear­ly work in obe­si­ty drugs. Ra­linepag is a Phase III-ready oral, se­lec­tive prosta­cy­clin re­cep­tor ag­o­nist for the treat­ment of pul­monary ar­te­r­i­al hy­per­ten­sion. The drug’s Phase II da­ta got Are­na’s in­vestors pret­ty jazzed ear­li­er this year. The drug is de­signed to be more po­tent and ef­fec­tive than Acte­lion’s Up­travi. Then there’s etrasi­mod, a Phase II oral S1P re­cep­tor mod­u­la­tor be­ing eval­u­at­ed for mul­ti­ple au­toim­mune dis­eases, in­clud­ing ul­cer­a­tive col­i­tis, a form of in­flam­ma­to­ry bow­el dis­ease. Ear­li­er this year, Amit Mun­shi said the drug was safer and po­ten­tial­ly more ef­fi­ca­cious than Ozan­i­mod, the drug de­vel­oped by San Diego’s Re­cep­tos (pur­chased by Cel­gene for $7.2 bil­lion). In the new deal with Ever­est, a com­pa­ny backed by $50M from Chi­na’s C-Bridge Cap­i­tal, Are­na gets $12M in an up­front pay­ment and is el­i­gi­ble to re­ceive up to $212M in mile­stones, along with roy­al­ties.

John­son & John­son is launch­ing a JLABS in Shang­hai — the first lo­ca­tion out­side of North Amer­i­ca — af­ter reach­ing agree­ments with the Shang­hai Mu­nic­i­pal Gov­ern­ment, Pudong New Area Gov­ern­ment and Shang­hai Phar­ma En­gine Com­pa­ny. Sched­uled to open in Q2 2019, the fa­cil­i­ty in Zhangjiang Hi-Tech Park will span 4,400 sq. me­ter (47,000+ sq. ft.) and can host up to 50 life sci­ence and health care star­tups. “Chi­na, and Shang­hai specif­i­cal­ly, have be­come a glob­al hotspot for health­care in­no­va­tion,” said Vladimir Makat­saria, John­son & John­son Chi­na Group chair­man, in a state­ment. J&J is cur­rent­ly ac­cept­ing ap­pli­ca­tions from biotech, phar­ma­ceu­ti­cal, med­ical de­vice, con­sumer and dig­i­tal health com­pa­nies to this site as well as its eight oth­er JLABS lo­ca­tions.

→ In­dus­try vet An­tho­ny John­son is tak­ing the helm at Goldfinch Bio, a Cam­bridge, MA-based biotech look­ing to de­vel­op new drugs for ge­net­i­cal­ly de­fined kid­ney dis­eases. Goldfinch launched a year ago with $55 mil­lion in Third Rock mon­ey. John­son is the lat­est in a long line of Big Phar­ma R&D ex­ecs mak­ing a move to a start­up, leav­ing his post as head of ear­ly clin­i­cal de­vel­op­ment for the In­no­v­a­tive Med­i­cines and Ear­ly De­vel­op­ment Biotech Unit. His bio in­cludes 25 pos­i­tive proof-of-con­cept stud­ies. In ad­di­tion, Michael Brox­son is join­ing Goldfinch as chief busi­ness and op­er­at­ing of­fi­cer.

Shire $SH­PG has struck a col­lab­o­ra­tion deal with Rani Ther­a­peu­tics that gives the rare dis­ease gi­ant ex­clu­sive rights to re­search, eval­u­ate and pur­sue a world­wide li­cense for the oral de­liv­ery of fac­tor VI­II to pa­tients with he­mo­phil­ia A. This in­vest­ment, the terms of which were not dis­closed, comes just a few weeks af­ter ri­val Roche’s Hem­li­bra nabbed an FDA ap­proval, some­thing that an­a­lysts said could help shrink Shire’s share of the he­mo­phil­ia mar­ket from 49% to 29%. The Rani Pill tech­nol­o­gy brings a nov­el in­testi­nal de­liv­ery ap­proach to the treat­ment of he­mo­phil­ia, which is dom­i­nat­ed by in­jecta­bles, though the com­pa­ny is al­so do­ing work on rheuma­toid arthri­tis, can­cer and Crohn’s dis­ease. As part of the pact, Shire has al­so made an eq­ui­ty in­vest­ment in­to Rani Ther­a­peu­tics.

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.


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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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.

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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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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Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.

Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.

Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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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.