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.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.