M&A is off to a hot start in 2017; Too bad we can't say the same for gen­der par­i­ty

End­points News as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.


M&A trans­ac­tions are look­ing brighter this year, and val­u­a­tions re­main high

Two big M&A deals Thurs­day, the $30 bil­lion ac­qui­si­tion of Acte­lion and Cel­gene’s pact to ac­quire new­born Delinia for $300 mil­lion up­front, are rais­ing hopes that 2017 will be rich in buy­outs. The still-pre­clin­i­cal Delinia had just raised a $35 mil­lion A round a few months ago, mean­ing that Sofinno­va Part­ners and At­las Ven­ture made out like ban­dits on that one. Acte­lion CEO Jean-Paul Clozel al­so proved that he could get just about every­thing he want­ed from J&J: A rich cash deal plus a new com­pa­ny that will spin out Acte­lion’s promis­ing pipeline. Ear­li­er, Take­da proved how much biotech as­sets are go­ing for when it paid $5.2 bil­lion for Ari­ad — bid­ding against it­self in the process. There’s al­so plen­ty of pent up de­mand for M&A. Throw in some com­pa­nies that al­most have to shoot for a ma­jor deal this year — Gilead, Bio­gen, Sanofi, among oth­ers – and you have the mak­ings for some brisk bid­ding wars ahead. That kind of M&A en­vi­ron­ment will do two things: Help buoy IPOs, as we’ve al­ready seen in an ear­ly glimpse, and keep ven­ture cash pump­ing in­to biotech. M&A is a very at­trac­tive al­ter­na­tive to IPOs now. And un­less the Trump ad­min­is­tra­tion does some­thing soon to re­al­ly douse the mar­ket en­vi­ron­ment for drug­mak­ing, 2017 is shap­ing up as a very in­ter­est­ing year.


We have to do a lot bet­ter about gen­der par­i­ty in biotech, now

I found it hard to be­lieve just how slow­ly women are gain­ing ground in biotech. A new study from Lift­stream’s Karl Simp­son shows that women hold on­ly one in 10 board seats at the new­ly pub­lic biotechs he re­viewed. And if you look at their rate of im­prove­ment in 2016, it will take some 40 years to achieve par­i­ty. Those are the kind of stats you’d ex­pect to see in the con­struc­tion field. A high-tech en­ter­prise like drug de­vel­op­ment, cen­tered in pro­gres­sive ur­ban ar­eas like the Bay Area and Cam­bridge/Boston, with one of the best ed­u­cat­ed work­forces of any in­dus­try? No way. And yet it un­der­scores just how male-ori­ent­ed the biotech in­dus­try is. CEOs re­cruit CEOs, large­ly men, or the men who dom­i­nate VCs go on the board – and stay on the board. A year ago, there was a mo­ment when the dirty lit­tle se­cret of biotech’s en­trenched sex­ism be­came some­thing of a cause cele­bre when some of the par­ties at JP Mor­gan in­clud­ed fe­male mod­els to help fa­cil­i­tate the frat boy flair they were look­ing for. Par­ties may have changed as a re­sult, but it’s a mean­ing­ful im­prove­ment in rep­re­sen­ta­tion in po­si­tions of in­flu­ence that will dic­tate re­al change. There has to be a tip­ping point at hand to make that hap­pen much, much soon­er. And I’m not talk­ing about 20 years from now. Di­ver­si­fied man­age­ment and boards have a proven, pos­i­tive im­pact on com­pa­ny per­for­mance. It’s time for biotech to get out of the ice age.


PhRMA is dream­ing if it be­lieves it can re­form the drug in­dus­try’s tat­tered im­age with an ad cam­paign

PhRMA be­lieves it has an an­swer to what ails the in­dus­try. Af­ter drug com­pa­nies emerged from 2016 paint­ed with the same brush of scan­dal for rapid­ly ris­ing prices – for new and old drugs alike – the in­dus­try lob­by­ing group be­lieves that spend­ing 10s of mil­lions of dol­lars on a pub­lic­i­ty cam­paign aimed at giv­ing phar­ma a bet­ter im­age with the pub­lic should help turn the ta­bles. But the chances of it work­ing are al­most nil. At­tack­ing some­thing, like man­aged care’s old Har­ry and Louise ads, is a lot more ef­fec­tive than in­di­rect­ly at­tempt­ing a makeover of an in­dus­try’s rep. At this stage, when Don­ald Trump at­tacks phar­ma, it’s be­cause he knows he’s on to a pop­ulist ap­proach that will work like mag­ic to build steam for some sort of re­form. One Tweet from the pres­i­dent is eas­i­ly worth many times what PhRMA’s mem­ber­ship will get from a mil­lion dol­lars of ad­ver­tis­ing. PhRMA’s best hopes lie in what it does best; in­flu­enc­ing mem­bers of Con­gress to back bio­phar­ma on a bi­par­ti­san ba­sis. That is what de­liv­ered the 21st Cen­tu­ry Cures Act and that is how the group can best in­flu­ence re­form. Truth is, change is com­ing, one way or the oth­er. It’s time PhRMA got ahead of that bus, rather than try to step in front of it. And don’t look for the clas­sic red her­rings to work this time. Drug prices are a much eas­i­er tar­get than com­plex hos­pi­tal costs. And so­lu­tions, like Medicare ne­go­ti­a­tions, are go­ing to be dif­fi­cult to re­sist.


It’s time to move along from the Mar­tin Shkre­li sideshow

PhRMA CEO Stephen Ubl had al­ready tried his mock­ing dig at Mar­tin Shkre­li — we need few­er hood­ies and more lab coats — be­fore he rolled it back out for CN­BC this week. I heard it in San Fran­cis­co at our ex­ec­u­tive break­fast. Shkre­li, even though he’s been banned from Twit­ter, still man­aged to stage a high-pro­file come­back with a quick­ie web site and YouTube rant. Shkre­li will soon have oth­er fish to fry this sum­mer, when he’s sched­uled to go on tri­al on fraud charges un­re­lat­ed to the 5000% price hike on Dara­prim that made him a fa­vorite whip­ping boy for the pub­lic, as well as phar­ma ex­ecs anx­ious to draw a dis­tinc­tion. But the in­dus­try needs to do some soul search­ing now. Con­ve­nient scape­goat­ing – even of some­one as odi­ous as Shkre­li makes him­self out to be – isn’t go­ing to play.

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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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. 

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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Sil­i­con Val­ley's most an­tic­i­pat­ed slide deck just dropped. What does it mean for bio­phar­ma's dig­i­tal teams?

These aren’t the typ­i­cal slides you’d see at End­points — no mol­e­cules, clin­i­cal pro­grams, or p-val­ues. In­stead, we’ll talk dig­i­tal and in­ter­net trends, fac­tors that elite glob­al brands — re­gard­less of in­dus­try — must first mea­sure and un­der­stand be­fore de­ploy­ing prod­ucts in­to the world. That’s a con­cept that most of our Big Phar­ma au­di­ence is in tune with. Dig­i­tal aware­ness is key to suc­cess in the dis­cov­ery, de­vel­op­ment, and mar­ket­ing of new bio­phar­ma­ceu­ti­cals, and most of the ma­jors now have a chief dig­i­tal of­fi­cer: No­var­tis, Sanofi, and Pfiz­er, just to name a few.

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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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.

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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