Vir chief Scan­gos picks Her­bert ‘Skip’ Vir­gin to run R&D; Arde­lyx forges $160M deal with Ky­owa Hakko Kirin

Her­bert “Skip” Vir­gin

A month ago George Scan­gos blazed through a biotech buy­out, two bil­lion-dol­lar drug de­vel­op­ment deals and more than $500 mil­lion in fi­nanc­ing to build a pipeline of new ther­a­pies for in­fec­tious dis­eases at his start­up biotech Vir. Now he’s se­lect­ed Her­bert “Skip” Vir­gin as R&D chief and CSO. Vir­gin is join­ing Vir from Wash­ing­ton Uni­ver­si­ty School of Med­i­cine in St. Louis, Mis­souri, where he has served as the Ed­ward Mallinck­rodt Pro­fes­sor and Chair of the De­part­ment of Pathol­o­gy & Im­munol­o­gy since 2006.

Arde­lyx $ARDX is pick­ing up a $30 mil­lion up­front in a pact with Ky­owa Hakko Kirin cov­er­ing the Japan­ese rights to its late-stage drug tena­panor for car­diore­nal dis­eases. The li­cens­ing deal al­so in­cludes $130 mil­lion in mile­stones and high-teens for the roy­al­ties.

PNC Bank is ac­quir­ing The Trout Group, the in­vestor re­la­tions and strate­gic ad­vi­so­ry firm that boasts a glob­al list of clients in­clud­ing Toca­gen, Gen­fit and Zai Lab. Un­der the de­fin­i­tive agree­ment, an­nounced Mon­day, Trout will be com­bined with PNC sub­sidiary Sole­bury Com­mu­ni­ca­tions — a sim­i­lar but not in­dus­try-spe­cif­ic firm — to be­come Sole­bury Trout Com­mu­ni­ca­tions. Trout founder/CEO Jonathan Fass­berg will be re­tained as one of three co-CEOs, serv­ing along­side Sole­bury’s Jeff Gross­man and Lisa Wol­ford. The new com­pa­ny will be dou­ble the size of 30-strong Trout, and will like­ly ex­pand ac­cess to biotech bank­ing busi­ness for PNC.

→ Lim­it­ed sup­ply of cus­tomized virus­es cru­cial to the de­liv­ery of gene ther­a­py is hold­ing up biotechs de­vel­op­ing such nov­el treat­ment, The New York Times re­ports. With an ex­pen­sive and ar­du­ous pro­ce­dure, the few firms ca­pa­ble of man­u­fac­tur­ing these dis­abled virus­es for clin­i­cal tri­als are “swamped with or­ders and re­quests.” A GEN col­umn pub­lished ear­li­er this month de­scribes the is­sue as a “ca­pac­i­ty crunch,” with Bio­Plan As­so­ci­ates es­ti­mat­ing that the CMO short­fall in world­wide cell/gene ther­a­py is 5 times cur­rent ca­pac­i­ty. A ready source of virus is be­com­ing para­mount; so much so that Bio­Marin has de­cid­ed to build its own man­u­fac­tur­ing plant. As CMO Mil­li­pore­Sig­ma’s Udit Ba­tra tells the NYT, “It’s a re­al is­sue.”

Scot­land’s Sym­promics is col­lab­o­rat­ing with UCL on a gene ther­a­py for Parkin­son’s dis­ease. Their first tar­get look­ing to con­trol gene ex­pres­sion will be Young-On­set Parkin­son’s dis­ease.

Gilead’s Solval­di is fi­nal­ly sell­ing in Chi­na, and the biotech gi­ant $GILD is charg­ing just shy of $9,000 for a 12-week reg­i­men — a frac­tion of the US price of $84,000. The launch, first re­port­ed by Chi­nese out­lets, comes two months af­ter the he­pati­tis C drug nailed an ap­proval from the CF­DA. Chi­na ac­counts for 10 out of 130 mil­lion hep C pa­tients world­wide, Caix­in ob­serves. Solval­di is the first di­rect an­ti-vi­ral agent in Chi­na, but it won’t be the last. Bris­tol-My­ers Squibb $BMY and John­son & John­son $JNJ are al­ready rac­ing to mar­ket their own prod­ucts af­ter nab­bing CF­DA ap­provals on Gilead’s heels.

→ The FDA has giv­en Cy­tomX $CT­MX the OK on its IND for the com­pa­ny’s CT­LA-4 pro­body ther­a­peu­tic, be­ing de­vel­oped in part­ner­ship with Bris­tol-My­ers Squibb. CT­LA-4 is the tar­get of BMS’ check­point in­hibitor Yer­voy, and is the first tar­get to get to the clin­ic un­der the com­pa­nies’ part­ner­ship formed in 2014. The IND ac­cep­tance gets Cy­tomX a $10 mil­lion mile­stone pay­ment, bring­ing the to­tal pay­ments from the deal to over $275 mil­lion — with $4.8 bil­lion still avail­able in po­ten­tial mile­stones.

→ Cincin­nati biotech Cin­Rx just closed $10 mil­lion in a Se­ries B to ad­vance sev­er­al drug can­di­dates in di­verse in­di­ca­tions, rang­ing from large mar­kets to or­phan in­di­ca­tions. The com­pa­ny plans to file an IND in the first half of 2018 for its most ad­vanced pro­gram, CIN-102, for the treat­ment of gas­tro­pare­sis. This lat­est round, led by undis­closed new and ex­ist­ing in­vestors, fol­lows a $26 mil­lion Se­ries A closed in 2015. The com­pa­ny’s pipeline in­cludes treat­ments for gas­troin­testi­nal dis­or­ders in­clud­ing IBS, along with car­dio­vas­cu­lar dis­ease and der­ma­tol­ogy.


Brit­tany Meil­ing and John Car­roll con­tributed to this re­port.

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.

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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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.