The 15 top R&D spenders in the glob­al bio­phar­ma busi­ness: 2016

Just about every play­er on this list has trig­gered a ma­jor over­haul of R&D in the past decade

Over the past nine years that I’ve been an­a­lyz­ing Big Phar­ma’s R&D num­bers, there’s been some fluc­tu­a­tion in the top ten, but over­all spend­ing has re­mained fair­ly steady at rough­ly $70 bil­lion a year.

Some big trends, though, emerge af­ter you move be­yond the top line num­ber. Just about every play­er on this list has trig­gered a ma­jor over­haul of R&D in the past decade, shut­ter­ing fa­cil­i­ties, lay­ing off staffers, or at the very least chop­ping out core re­search ar­eas and ei­ther nar­row­ing their fo­cus or adding new ones. (And it’s not over yet, as you can see from yes­ter­day’s Mer­ck re­or­ga­ni­za­tion sto­ry.) The tur­bu­lence has helped per­suade a long list of top ex­ecs to leave the biggest com­pa­nies and leap to the helm of biotech star­tups, which have been en­riched by an IPO boom (now strug­gling) and a VC romp of his­toric pro­por­tions (not over yet). Can­cer re­mains the Big Kahu­na as new drugs of­fer dra­mat­ic break­throughs, and some high dra­ma, which is one rea­son why there is still lin­ger­ing un­cer­tain­ty over GSK’s de­ci­sion to swap its late-stage and com­mer­cial as­sets for No­var­tis’s vac­cines op­er­a­tion. Im­muno-on­col­o­gy in par­tic­u­lar­ly is dri­ving bil­lions of dol­lars in new in­vest­ments, and not just by Bris­tol-My­ers and Mer­ck.

John Car­roll, Ed­i­tor

We’ve seen the ar­rival of the first wave of biosim­i­lars from the largest play­ers, many of which are hap­py to in­vest in big Phase II­Is with an eye to grab­bing a mar­ket niche for some of the world’s megablock­busters. Over the next 5 years, or less, that could de­stroy some old fran­chis­es, a no­tion which is light­ing a fire un­der com­pa­nies like Ab­b­Vie. While some of the biggest com­pa­nies were wait­ing out the boom years, Ab­b­Vie couldn’t af­ford to wait, and it’s reached the point some an­a­lysts are start­ing to think they’re over­pay­ing for new deals.

There are some big changes to note. An Ag­gres­sive J&J just bare­ly edged out No­var­tis for the num­ber 2 spot, a re­flec­tion of its glob­al am­bi­tions in drug R&D. Bris­tol-My­ers al­so saw its spend­ing spike as it en­gi­neered an over­haul aimed at re­tain­ing the lead in im­muno-on­col­o­gy.

I specif­i­cal­ly want­ed to add the last 5 com­pa­nies to this list. Here you’ll find the Big Biotechs, com­pa­nies like Cel­gene and Gilead. Backed by some in­cred­i­bly prof­itable brands, these com­pa­nies are demon­strat­ing how small­er R&D bud­gets can of­ten be used to trans­late in­to more ex­cit­ing pipelines. Cel­gene is run by deal­mak­ers, which hasn’t dis­ap­point­ed, and every­one is wait­ing to see what Gilead has to be work­ing on.

Add it all up and you have $87 bil­lion of R&D clout.

Ex­pec­ta­tions are high for all the com­pa­nies on this list. Re­cent years have al­so seen a surge in new drug ap­provals. And the sin­gu­lar drugs that threat­en to dis­rupt glob­al mar­kets still ex­cite. The next 10 years could well be even bet­ter than the last.

— John Car­roll, fol­low me on Twit­ter: @John­Cendpts



1. Roche: Be­ing no. 1 means try­ing hard­er

  • 2015:  $9.67B (9.3B CHF over­all, $8.1B phar­ma plus di­ag­nos­tics)
  • 2014: $9.25B (8.9B CHF over­all, $7.9B phar­ma)
  • Change: 4.5%
  • As a per­cent­age of rev­enue: 19%
  • CMO: San­dra Horn­ing

The big pic­ture: Roche is fac­ing off against loom­ing biosim­i­lar com­pe­ti­tion against three of its main­stay drugs—Her­ceptin, Rit­ux­an and Avastin—by steadi­ly fund­ing one of the most am­bi­tious R&D ef­forts in the world. And it’s been pay­ing off.

San­dra Horn­ing, Roche Chief Med­ical Of­fi­cer

In April there was an ac­cel­er­at­ed OK for vene­to­clax (Ven­clex­ta) for pa­tients with chron­ic lym­pho­cyt­ic leukemia who have a chro­mo­so­mal ab­nor­mal­i­ty called 17p dele­tion. Vene­to­clax has racked up three break­through drug des­ig­na­tions from the FDA, which helped it get whisked along the reg­u­la­to­ry path with the FDA’s VIP pro­gram for top can­cer drugs in the pipeline. This ther­a­py is ex­pect­ed to earn in the rather ex­clu­sive neigh­bor­hood of $2 bil­lion a year.

One of its biggest late-stage ef­forts has been re­served for Genen­tech’s ate­zolizum­ab, the first PD-L1 check­point in­hibitor re­cent­ly ap­proved as Tecen­triq for blad­der can­cer. Like PD-1 ri­vals from Bris­tol-My­ers Squibb and Mer­ck, the phar­ma gi­ant has been fund­ing a slate of ad­di­tion­al stud­ies.

But it could do even bet­ter with ocre­lizum­ab, which has wide­ly been tapped as the biggest thing in the in­dus­try’s late-stage pipeline. Round­ing out the block­buster line­up is ACE910 for hema­tol­ogy and lam­pal­izum­ab, which could play a big role in dry AMD. Deutsche Bank an­a­lysts have pro­ject­ed lam­pal­izum­ab’s peak sales po­ten­tial at a megablock­buster lev­el $6 bil­lion.

That’s all mu­sic to the ears of Roche CEO Sev­erin Schwan, who told Reuters re­cent­ly that he was able to sleep bet­ter at night af­ter the suc­cess of ate­zo along with progress on some of its oth­er fran­chise drugs. And the phar­ma gi­ant has been mak­ing sig­nif­i­cant progress with its fo­cus on tar­get­ed can­cer drugs, with a com­bi­na­tion of Cotel­lic and Zelb­o­raf mak­ing progress in Eu­rope last year as Ale­cen­sa moved for­ward at the FDA for ALK-pos­i­tive non-small cell lung can­cer.

It hasn’t all been a bed of ros­es in R&D, though.

Roche has tried, and large­ly failed, to make sig­nif­i­cant head­way in Alzheimer’s dis­ease. But the com­pa­ny has per­sist­ed with a kind of grim de­ter­mi­na­tion. It was greet­ed with a ma­jor set­back on crenezum­ab, part­nered with AC Im­mune, which failed a re­cent study. But the com­pa­ny was in­spired by in­di­ca­tions of its po­ten­tial as well as Bio­gen’s work in the field to push ahead in­to a piv­otal study. It even chose to con­tin­ue work on gan­tenerum­ab, a Mor­phoSys drug that flat failed.

Roche took a ma­jor risk when it ac­quired Genen­tech back in 2009. But it’s paid off, al­low­ing the com­pa­ny to play a big role in on­col­o­gy dur­ing a pe­ri­od of up­heaval.


 

2. J&J: Glob­al am­bi­tions spur a world­wide hunt for new drugs

  • 2015 R&D spend­ing: $9 bil­lion
  • 2014 R&D spend­ing: $8.5 bil­lion
  • Change: +6%
  • As a per­cent­age of rev­enue ($70B): 13%
  • R&D chief: Paul Stof­fels

The Big Pic­ture: Over the past year, J&J has been push­ing hard on the drug de­vel­op­ment front. Now that its glob­al deal teams are all in place, the phar­ma gi­ant has reached in­to all the ma­jor, and many mi­nor, biotech hubs around the plan­et to source new pro­grams.

For the drug de­vel­op­ment team, that meant an ex­tra $600 mil­lion in ex­pens­es in 2015 when com­pared to 2014. Spend­ing on the de­vices side of the re­search busi­ness re­mained flat as J&J went on to re­or­ga­nize that dis­ap­point­ing side of the or­ga­ni­za­tion.

Paul Stof­fels, Ex­ec­u­tive Vice Pres­i­dent, Chief Sci­en­tif­ic Of­fi­cer, John­son & John­son

R&D chief Paul Stof­fels has re­mained a busy play­er in the can­cer drug are­na. In March, the com­pa­ny fol­lowed up with its sec­ond deal with Macro­Gen­ics, com­mit­ting $75 mil­lion up­front as it sought to pur­sue an al­ter­na­tive to the CAR-Ts that ri­vals have been ad­vanc­ing in adop­tive T cell re­search. And a month lat­er it struck a $500 mil­lion pack­age deal for Tesaro’s late-stage can­cer ther­a­py as it looked to beef up its work in prostate can­cer, where it’s made ma­jor in­roads with Zyti­ga.

J&J could on­ly have been pleased to see Tesaro’s im­pres­sive piv­otal da­ta in ovar­i­an can­cer, which leaves it as the fron­trun­ner among a group of 4 PARP in­hibitors.

J&J’s R&D group, though, likes to play some cards close to its vest, par­tic­u­lar­ly when they’re play­ing in a com­pet­i­tive late-stage field. Two years ago it bought out Aragon in a heav­i­ly front­ed $1 bil­lion deal so it could put its hands on ARN-509, al­so for prostate can­cer. But they’ve been re­luc­tant to dis­cuss it much as the drug plays out in two big Phase III stud­ies. Mean­while, its late-stage pipeline al­so in­cludes the an­ti-in­flam­ma­to­ry IL-23 drug guselkum­ab, in a crowd­ed field, as well as es­ke­t­a­mine, its own take on ke­t­a­mine for de­pres­sion.

Demon­strat­ing once again how J&J on­ly likes to talk about cer­tain things, the com­pa­ny re­cent­ly stopped work on the an­ti-NGF pain drug ful­ranum­ab with no ex­pla­na­tion.

There are plen­ty of ear­ly-stage deals work­ing as well. Stof­fels en­joys an­nounc­ing these pacts in big batch­es as J&J con­tin­ues to play a lead­ing role on the BD front. Its grow­ing, $9 bil­lion bud­get 2015 R&D bud­get sig­nals that there are many more con­tracts in the works.


 

3. No­var­tis: Qui­et, fast and big

  • 2015 R&D spend­ing: $8.9B
  • 2014 R&D spend­ing: $9B
  • Change: – 1%
  • As a per­cent­age of rev­enue ($49.4B): 18%
  • R&D chiefs: NI­BR chief Jay Brad­ner, CMO Vas Narasimhan

The big pic­ture: For a Big Phar­ma com­pa­ny with the sec­ond largest R&D bud­get in the in­dus­try, No­var­tis can move very fast, if it de­cides to.

Vas­ant Narasimhan, Glob­al Head Drug De­vel­op­ment and Chief Med­ical Of­fi­cer for No­var­tis

So it was no great sur­prise to see the phar­ma gi­ant wrap a late-stage pro­gram for its CDK 4/6 can­cer drug LEE011 (ri­bo­ci­clib), now head­ed to a show­down with Pfiz­er (on the mar­ket with pal­bo­ci­clib) as well as Eli Lil­ly. It’s clear that No­var­tis’ San­doz will bull ahead in­to the biosim­i­lars mar­ket, look­ing to top­ple some ma­jor fran­chis­es with less ex­pen­sive copy­cats.

No­var­tis al­so has been achiev­ing re­sults for drugs like PKC412 (mi­dostau­rin) and oth­er ther­a­pies that you’ve prob­a­bly heard much less of. While No­var­tis moves quick­ly on deals, like its in­vest­ment and col­lab­o­ra­tion with In­tel­lia, part­ner­ing on a promis­ing CRISPR Cas9 ap­proach to gene edit­ing, the com­pa­ny can al­so be ex­tra­or­di­nar­i­ly low key about its top drug prospects.

James Brad­ner, Pres­i­dent, No­var­tis In­sti­tutes for Bio­Med­ical Re­search (NI­BR)

That might be chang­ing, though, now that Jay Brad­ner has tak­en the top po­si­tion at the No­var­tis In­sti­tutes for Bio­med­ical Re­search in Cam­bridge, MA, with Vas Narasimhan mov­ing in­to the top job in de­vel­op­ment. Brad­ner, the young, im­pres­sive ex-Har­vard pro­fes­sor knows his stuff, and demon­strates lots of en­thu­si­asm for top projects. Narasimhan is an up-and-com­er with some big tasks to per­form, help­ing ex­e­cute an over-arch­ing strat­e­gy to win­kle out a bil­lion dol­lars in an­nu­al costs. (That’s clas­sic No­var­tis style: spend big but leave no rock un­turned in search of greater ef­fi­cien­cy.)

We’ll keep our fin­gers crossed here at End­points that some greater trans­paren­cy may lie ahead of No­var­tis.

Last year No­var­tis scored ap­provals for En­tresto and Cosen­tyx, plus the very first FDA nod for a biosim­i­lar. It may not do as well this year. But big things are cook­ing at No­var­tis.


 

4. Pfiz­er: The big one keeps get­ting away

  • 2015 R&D spend­ing: $7.7 bil­lion
  • 2014 R&D spend­ing: $8.4 bil­lion
  • Change: -8%
  • As a per­cent­age of rev­enue: 15.7%
  • R&D chief: Mikael Dol­sten

The big pic­ture: Pfiz­er gets lit­tle re­spect for its R&D ef­fort, but it’s im­por­tant to re­mem­ber the phar­ma gi­ant de­liv­ered the re­al goods in 2015 with an ac­cel­er­at­ed ap­proval for the CDK 4/6 drug pal­bo­ci­clib (Ibrance). The drug was every bit the break­through the FDA had des­ig­nat­ed it as, and there are some ma­jor sales goals in mind, even though com­pe­ti­tion is loom­ing.

Pal­bo­ci­clib will like­ly one day face com­pe­ti­tion from Lil­ly and No­var­tis. Lil­ly in par­tic­u­lar is aim­ing at prov­ing it can do pal­bo one bet­ter with abe­maci­clib.

Mikael Dol­sten,
Pres­i­dent, World­wide Re­search & De­vel­op­ment, Pfiz­er

So what has Pfiz­er done for in­vestors re­cent­ly? It ac­quired Hos­pi­ra and a pipeline of late-stage biosim­i­lars that are look­ing in­creas­ing­ly de-risked. There’s a pain drug, tanezum­ab (an an­ti-NGF drug part­nered with Eli Lil­ly), that has re­al po­ten­tial now that the sec­tor is back on track. There’s a PD-L1 drug, avelum­ab, which may qual­i­fy as the 5th check­point drug to make it on­to the mar­ket.

Pfiz­er paid a record $850 mil­lion up­front to part­ner with Mer­ck KGaA on avelum­ab, sig­nal­ing its will­ing­ness to pay top dol­lar for every­thing it deems wor­thy. But wher­ev­er Pfiz­er turns now, it’s look­ing at some ma­jor league com­pe­ti­tion, from biosims through pain to can­cer.

Pfiz­er CEO Ian Read has set his sights on a megamerg­er twice, and twice been forced to re­treat. The com­pa­ny will be forced to prove at some point that it can pull off a string of im­por­tant ap­provals on its own. They em­ploy some of the smartest peo­ple in the drug de­vel­op­ment busi­ness. And even af­ter a top-to-bot­tom R&D re­org a few years ago, Pfiz­er still ranks com­fort­ably among the top 10 spenders.

But at this rate, Pfiz­er has a long way to go be­fore it earns the kind of re­spect a $7.7 bil­lion bud­get should war­rant.


 

5. Mer­ck: Af­ter a big year, still look­ing to be No. 1

  • 2015 R&D spend­ing: $6.7 bil­lion
  • 2014 R&D spend­ing: $7.2 bil­lion
  • Change: -7%
  • As a per­cent­age of rev­enue: 17%
  • R&D chief: Roger Perl­mut­ter

The big pic­ture: 2015 proved to be a big year for Mer­ck and its re­search chief Roger Perl­mut­ter. The com­pa­ny scored a big win with the ap­proval for Keytru­da, a trail­blaz­ing PD-1 check­point drug that promis­es to help rev­o­lu­tion­ize the way can­cer is treat­ed. The com­pa­ny fi­nal­ly got sug­am­madax ap­proved at the end of 2015 — af­ter three straight re­jec­tions over the years. And it had rolled up to the thresh­old of an ap­proval for Zepati­er, a com­bo cure for he­pati­tis C, which came soon af­ter the New Year ar­rived.

Mer­ck has been mak­ing a game fight over the check­point mar­ket with the leader in the field, Bris­tol-My­ers Squibb. It hus­tled out stel­lar lung can­cer da­ta just days ago and CEO Ken Fra­zier re­cent­ly not­ed that the com­pa­ny will spend $2 bil­lion this year alone on Keytru­da-re­lat­ed re­search, but a lot of an­a­lysts be­lieve the mar­ket-lead­ing Bris­tol will con­tin­ue to over­shad­ow the com­pa­ny.

In the nov­el drug ball­park, Mer­ck is tak­ing some home run swings.

It has a huge, and what is wide­ly con­sid­ered near­ly hope­less, at­tempt to get anace­trapib over the fin­ish line. This is the last of four CETP in­hibitors, and the last to size up da­ta from a mas­sive CV out­comes study. The oth­er three all went down in flames. And Perl­mut­ter won’t get the blame if this lega­cy pro­gram flops as well.

MK-8931 (verube­ce­s­tat) is the lead­ing BACE drug for Alzheimer’s, look­ing to make a dif­fer­ence in what is per­haps the largest po­ten­tial mar­ket for any new drug. Alzheimer’s though, has proven to be a rid­dle that has de­feat­ed every sig­nif­i­cant R&D ef­fort of the past decade.

There’s even a large­ly for­got­ten os­teo­poro­sis drug, odanacat­ib, in the pipeline.

Roger M. Perl­mut­ter, Mer­ck, Ex­ec­u­tive Vice Pres­i­dent and Pres­i­dent of Mer­ck Re­search Lab­o­ra­to­ries

Mer­ck went through a long dry spell in the clin­ic be­fore it start­ed to turn things around with Keytru­da. But the drug didn’t ar­rive in time to stop an R&D re­or­ga­ni­za­tion that claimed thou­sands of jobs, and which was fol­lowed up this week with news of an­oth­er round as the phar­ma gi­ant con­cen­trates its forces in San Fran­cis­co and Cam­bridge, MA. Now, though, it has to see if it can do bet­ter than a sol­id sec­ond, or weak third, in the mar­kets it tack­les.

In the mean­time, the deal team hasn’t been par­tic­u­lar­ly busy since the $10 bil­lion Cu­bist buy­out, though there has been the oc­ca­sion­al pact to un­der­score Perl­mut­ter’s en­thu­si­asms for new tech­nol­o­gy.

The ju­ry is still out on Mer­ck. A big win, or two, would go a long way to re­solv­ing lin­ger­ing doubts.


 

6. Bris­tol-My­ers Squibb: The im­muno-on­col­o­gy leader is still think­ing big

  • 2015 R&D spend­ing: $5.9B
  • 2014 R&D spend­ing: $4.5B
  • Change: 31%
  • As a per­cent­age of rev­enue ($16.5B): 36%
  • R&D chief: Fran­cis Cuss

The big pic­ture: About a year ago, Bris­tol-My­ers Squibb joined just about every ma­jor phar­ma group in pulling off a coun­try­wide re­or­ga­ni­za­tion of its R&D or­ga­ni­za­tion. Ear­ly-stage vi­rol­o­gy re­search went out the win­dow as Bris­tol-My­ers looks to con­cen­trate more of its staff in Cam­bridge, MA and the Bay Area – cov­er­ing the two big hubs on both coasts.

It’s been beef­ing up its staff in par­tic­u­lar on the West Coast, where it’s been de­vot­ing a sig­nif­i­cant ex­tra ef­fort on im­muno-on­col­o­gy. It’s clear that Bris­tol-My­ers plans to re­main the big dog in check­points and check­point com­bos. And it’s no sur­prise that their check­point drug Op­di­vo (nivolum­ab) has won 6 break­through drug des­ig­na­tions.

Fran­cis Cuss, Bris­tol-My­ers Squibb Chief Sci­en­tif­ic Of­fi­cer, R&D

They demon­strat­ed that com­mit­ment again re­cent­ly when Bris­tol-My­ers com­plet­ed a $520 mil­lion deal to buy Cor­morant and its IL-8 tu­mor mi­croen­vi­ron­ment drug. The push now is to find new com­bos that can re­al­ly amp up the at­tack on can­cer cells.

Bris­tol-My­ers’ deal­mak­ers like to stay prac­ti­cal. If they see a com­pet­ing pro­gram that’s fur­ther ahead of theirs, they’ll buy it. That’s what led to the Pad­lock ac­qui­si­tion.

They’ve been a very fo­cused, very re­sults-ori­ent­ed op­er­a­tion for years now. That’s why they’re dis­en­gag­ing from vi­rol­o­gy. If they’re not get­ting the re­sults, they’ll look for new op­por­tu­ni­ties to ei­ther build on a lead or cre­ate a mar­ket-lead­ing op­por­tu­ni­ty.

It’s al­so clear from the big surge in R&D spend­ing last year that the com­pa­ny is will­ing to spend big to fu­el growth.


 

7. As­traZeneca: One step for­ward, two steps back

  • 2015 R&D spend­ing: $5.6 bil­lion (non-GAAP, core spend­ing)
  • 2014 R&D spend­ing: $4.9 bil­lion
  • Change: 14%
  • Spend­ing as a per­cent­age of rev­enue: 25% (23.6B)
  • R&D chiefs: Sean Bo­hen, Bahi­ja Jal­lal, Mene Pan­ga­los

The big pic­ture: With As­traZeneca, it al­ways seems that every step for­ward is dogged by two steps back. And it’s been do­ing a lot of step­ping in the 4 years since Pas­cal So­ri­ot took over.

Mene Pan­ga­los, Ex­ec­u­tive Vice-Pres­i­dent, IMED Biotech Unit

The R&D group gained a huge win with the ap­proval of Tagris­so (AZD9291), gain­ing an ac­cel­er­at­ed ap­proval in record time. Ri­val Clo­vis un­in­ten­tion­al­ly helped by or­ches­trat­ing a midair ex­plo­sion at the FDA.

Mean­while, As­traZeneca’s PD-L1 check­point in­hibitor dur­val­um­ab has slid to a weak fourth place po­si­tion, which con­tin­ues to nar­row its po­ten­tial as the three lead­ers – Bris­tol-My­ers, Mer­ck and now Roche – surge for­ward in the mar­ket. Fourth place is not where As­traZeneca needs to be right now.

Sean Bo­hen, Ex­ec­u­tive Vice Pres­i­dent of Glob­al Med­i­cines De­vel­op­ment and Chief Med­ical Of­fi­cer.

It was down­right em­bar­rass­ing, though, to have the FDA re­ject ZS-9 af­ter buy­ing the drug dur­ing the reg­u­la­to­ry re­view phase. As­traZeneca is ev­i­dent­ly go­ing to have to re­solve some man­u­fac­tur­ing is­sues ahead of a for­mal de­ci­sion, but this is one big play­er that can ill af­ford to wait for any­thing.

There can be no doubt of the ur­gency of the sit­u­a­tion. As­traZeneca has been pum­meled by gener­ic com­pe­ti­tion and So­ri­ot was faced with the weak­est pipeline in the top 10 when he took over. He’s steered the com­pa­ny in­to a bliz­zard of new deals, and pre­sent­ed man­age­ment with a near-im­pos­si­ble goal of achiev­ing $45 bil­lion in sales in 2023.

A few days ago, As­traZeneca made a des­per­ate bid to block gener­ic com­pe­ti­tion to Crestor, ar­gu­ing that the ap­proval to use the drug to treat ho­mozy­gous fa­mil­ial hy­per­c­ho­les­terolemia, dan­ger­ous­ly high lev­els of cho­les­terol, should give it an ex­tra 7 years of mar­ket pro­tec­tion. They made a weak safe­ty ar­gu­ment, but most an­a­lysts saw it for what it was: a latch-ditch ef­fort to safe­guard the source of $5 bil­lion of the $23 bil­lion it made last year.

Bahi­ja Jal­lal, Ex­ec­u­tive Vice-Pres­i­dent of Med­Im­mune, a sub­sidiary of As­traZeneca

As­traZeneca has a bad habit of in­sist­ing that it loves its drugs, right up un­til they dump them. That hap­pened to bro­dalum­ab and it part­ly just oc­curred with tralok­inum­ab, now that it out­li­censed the rights for atopic der­mati­tis, where it looked weak.

But you can’t de­ny that the elim­i­nate-the-weak­est strat­e­gy makes sense. If some­thing in R&D doesn’t cut it, it has to go, which is why As­traZeneca down­sized ear­ly-stage an­tibi­ot­ic re­search in Mass­a­chu­setts and spun the rem­nant out as a biotech com­pa­ny.

As­traZeneca does have ben­ral­izum­ab be­ing prepped for a reg­u­la­to­ry fil­ing af­ter wrap­ping a pos­i­tive Phase III pro­gram, but most an­a­lysts on­ly see it of­fer­ing a frac­tion of the $2 bil­lion in peak sales that the com­pa­ny pro­vid­ed in 2014.

Tak­en al­to­geth­er, this is not a pic­ture of a com­pa­ny on the way to meet­ing a very am­bi­tious set of sales goals. On the oth­er hand, it still has the kind of clout need­ed to make a ma­jor dif­fer­ence in the R&D are­na. The right buy­out (Medi­va­tion, per­haps?) would help its com­mer­cial op­er­a­tions as well as its late-stage pipeline.

Don’t count it out.


 

8. Sanofi: The mas­ter R&D part­ner at work

  • 2015 R&D spend­ing: $5.6B (5B eu­ros)
  • 2014 R&D spend­ing: $5.15B (4.6B eu­ros)
  • Change: 8.7%
  • As a per­cent­age of rev­enue ($38.6B):  14.7%
  • R&D chief: Elias Zer­houni

The big pic­ture: On its own, Sanofi R&D hasn’t had a heck of a lot to boast about in re­cent years. In part­ner­ship with Re­gen­eron, though, the phar­ma gi­ant has been scor­ing some ground­break­ing wins on the R&D side. And it’s look­ing to use its col­lab­o­ra­tive mod­el to ex­pand on its plat­form strat­e­gy with more big wins in the years ahead.

Elias Zer­houni, Pres­i­dent of Glob­al Re­search and De­vel­op­ment, Sanofi

EP Van­tage list­ed their IL-4/IL-13 con­tender dupilum­ab as a top 5 Phase III drug, which some an­a­lysts be­lieve can earn as much as $2.5 bil­lion for eczema and asth­ma. An­oth­er part­nered ther­a­py, the IL-6 drug sar­ilum­ab, is al­so of­ten tapped as a po­ten­tial block­buster.

Both of these drugs fol­low the pi­o­neer­ing ap­proval of Pralu­ent, their PC­SK9 cho­les­terol drug that has been chal­lenged by Am­gen, which claims the ri­val drug in­fringed on its patents.

Sanofi R&D chief Elias Zer­houni is a big be­liev­er in plat­form part­ner­ships. And it many ways, he has had to be. The com­pa­ny tried, and large­ly failed, to re­struc­ture its un­pro­duc­tive French re­search op­er­a­tions un­der Chris Viehbach­er’s lead. Since then, it’s been lean­ing heav­i­ly on its U.S. op­er­a­tions and part­ners for in­no­va­tion.

The com­pa­ny was forced to down­size its in­ter­nal on­col­o­gy ef­forts af­ter a key set­back and lit­tle progress. But now the com­pa­ny’s new CEO, Olivi­er Brandi­court, has his eyes set on a takeover at Medi­va­tion, which would de­liv­er Xtan­di as well as a close­ly-watched late-stage PARP in­hibitor (ta­la­zoparib) in a hot­ly con­test­ed sec­tor of can­cer drug de­vel­op­ment.

Sanofi Elias Zer­houni is adamant that part­ner­ing with plat­form com­pa­nies of­fers the best path to new drug ap­provals. His rule:“Every com­pa­ny that ac­quires a plat­form com­pa­ny kills it.” And he has big hopes that a top-to-bot­tom re­or­ga­ni­za­tion of in­ter­nal R&D op­er­a­tions will make their own pipeline ops more pro­duc­tive as well. But ei­ther way, Zer­houni has found a strat­e­gy that works. And he’s stick­ing with it.


 

9. Eli Lil­ly: Progress, but is it enough?

  • 2015 R&D spend­ing: $4.8 bil­lion
  • 2014 R&D spend­ing: $4.73 bil­lion
  • Change: +1%
  • As a % of rev­enue: 24%
  • R&D chief: Jan Lund­berg

The big pic­ture: A cou­ple of years ago, Eli Lil­ly had gone so long with­out a sig­nif­i­cant drug ap­proval, it was easy to dis­miss this top-10 R&D spender as an in­signif­i­cant force. But re­cent­ly, Lil­ly has been mak­ing some se­ri­ous noise in the de­vel­op­ment are­na, and it’s no longer just forced en­thu­si­asm in the wake of fail­ure.

Jar­diance helped enor­mous­ly when the SGLT2 proved last fall that it could sig­nif­i­cant­ly low­er the risk of car­dio events by 14% among high-risk di­a­bet­ics. The da­ta on re­duced hos­pi­tal­iza­tion and re­duced mor­tal­i­ty were even bet­ter. But then there’s the, yes, but, that of­ten fol­lows progress at Lil­ly.

Jan Lund­berg, Ex­ec­u­tive Vice Pres­i­dent, Sci­ence and Tech­nol­o­gy, and Pres­i­dent, Lil­ly Re­search Lab­o­ra­to­ries

Yes, but: Lil­ly and its part­ner Boehringer In­gel­heim bare­ly man­aged to eke out a nar­row pan­el vote fa­vor­ing a change of la­bel that could make an enor­mous dif­fer­ence in Jar­diance sales. Some of the ex­perts were ran­kled they didn’t get two con­fir­ma­to­ry stud­ies, as was nor­mal­ly re­quired.

In ad­di­tion, there was a big win for ix­ek­izum­ab, a po­ten­tial block­buster ap­proved for pso­ri­a­sis and sold as Taltz. But – it fol­lows No­var­tis in­to the mar­ket and still has ri­vals from J&J and Mer­ck in hot pur­suit.

Great things are ex­pect­ed of baric­i­tinib for rheuma­toid arthri­tis. But, this is one of the most hot­ly com­pet­i­tive fields in R&D.

A lot of Lil­ly’s rep in R&D will al­so rest on its late-stage CDK 4/6 can­cer drug abe­maci­clib. If that comes in as a pre­ferred win­ner in the field, the drug could make a ma­jor dif­fer­ence for Lil­ly. But – it’s not a fore­gone con­clu­sion.

Then there’s Alzheimer’s, which is rid­dled with caveats for Lil­ly. Solanezum­ab al­ready failed, spec­tac­u­lar­ly, in Phase III and the BACE drug in the pipeline is an­oth­er spec­u­la­tive play. Cu­ri­ous­ly, Lil­ly is al­ready try­ing to back off a key end­point in its so­la stud­ies, with no as­sur­ances that the FDA will play along. But, if ei­ther of these drugs works in late-stage tri­als, even mar­gin­al­ly, it would have a swift and po­ten­tial­ly enor­mous im­pact on the com­pa­ny’s bot­tom line.

Lil­ly’s R&D op­er­a­tion has made a come­back. But an­a­lysts are look­ing for much bet­ter things to come. The ju­ry is still out, and the ver­dict can’t be as­sumed.


 

10. Glax­o­SmithK­line: Still can’t win re­spect

  • 2015 R&D spend­ing: $4.38B (3.09B pounds)
  • 2014 R&D spend­ing: $4.4B  (3.1B)
  • Spend­ing as a per­cent­age of rev­enue: 12.9%
  • R&D di­rec­tor: Patrick Val­lance

The big pic­ture: Glax­o­SmithK­line knows what it’s like to ex­pe­ri­ence bit­ter set­backs. But when in­ves­ti­ga­tors fail to find what they hoped for, this com­pa­ny al­so doesn’t hes­i­tate to dump a fail­ure and move on.

In that spir­it, dris­apersen, MAGE A3 and dara­pladib  all dis­ap­peared. But that was awhile ago, now. In the fol­lowup, GSK joined the list of com­pa­nies un­der­go­ing an R&D over­haul in the U.S. Its as­set swap with No­var­tis left it with a less ex­cit­ing, but re­li­able, vac­cines group, where Mon­cef Slaoui is now the chief, un­til his re­tire­ment next year.

Patrick Val­lance, Pres­i­dent, Phar­ma­ceu­ti­cals R&D

With­out a lot to ex­cite in­vestors, GSK wise­ly opt­ed to hang on to its big HIV play at Vi­iV, where it holds a ma­jor­i­ty in­ter­est and con­tin­ues to make progress in R&D. It’s al­so scored sol­id Phase III da­ta for sirukum­ab, its IL-6 drug for RA, with the kind of late-stage pro­gram that vir­tu­al­ly as­sures a near-term ap­proval. GSK al­so re­cent­ly be­came the first Big Phar­ma to win an ap­proval for a gene ther­a­py — in the EU — mark­ing a sig­nif­i­cant ad­vance in the field. And it beefed up its res­pi­ra­to­ry group with last fall’s FDA OK for the asth­ma drug mepolizum­ab, which al­so has po­ten­tial in oth­er lu­cra­tive in­di­ca­tions.

None of that, though, has soothed an­a­lysts, who see a ma­jor cor­po­ra­tion with­out the kind of pipeline that can de­liv­er megablock­buster drug can­di­dates. By nor­mal stan­dards, that would in­di­cate that some­thing big should be in the deal works. But with the fo­cus now on a ma­jor shift with a new CEO and top ex­ecs ex­pect­ed to move in, there’s no guar­an­tee that GSK will do some­thing seis­mic this year. Pa­tience is wear­ing thin.


 

11. Ab­b­Vie: A com­pa­ny in a hur­ry

  • 2015 R&D spend­ing: $4.28B
  • 2014 R&D spend­ing: $3.3b
  • Change: 30%
  • As a per­cent­age of rev­enue: 18%
  • R&D di­rec­tor: Michael Sev­eri­no

The big pic­ture:  It’s no sur­prise see­ing Ab­b­Vie’s R&D bud­get shoot up in 2015. This com­pa­ny has the $14 bil­lion drug Hu­mi­ra go­ing for it – and against it. With gener­ic com­pe­ti­tion (like­ly) on the hori­zon, Ab­b­Vie has to move fast to build the pipeline, win ap­provals and build new sales. Hu­mi­ra de­liv­ers well over half of its an­nu­al rev­enue.

That’s a big task.

Michael Sev­eri­no, Ex­ec­u­tive Vice Pres­i­dent, Chief Sci­en­tif­ic Of­fi­cer, Ab­b­Vie

Ab­b­Vie in turn has been wheel­ing and deal­ing its way through the in­dus­try. Its big, $10 bil­lion play for Stem­cen­trx and Ro­va-T (roval­pituzum­ab tesirine) im­me­di­ate­ly raised eye­brows at the cost and the po­ten­tial ben­e­fit to pa­tients.

Ab­b­Vie and its part­ner Bio­gen won a re­cent ap­proval for the MS drugZ­in­bry­ta (da­clizum­ab), but it won’t be an overnight won­der, more like­ly to build sales over time. The com­pa­ny (with its part­ner Roche) will like­ly do much bet­ter with Ven­clex­ta (vene­to­clax), ap­proved in April for chron­ic lym­pho­cyt­ic leukemia for pa­tients who have a chro­mo­so­mal ab­nor­mal­i­ty called 17p dele­tion. It’s won three break­through des­ig­na­tions and an­a­lysts ex­pect it to earn $2 bil­lion a year.

Then there have been a string of on­col­o­gy deals, in­clud­ing a pact with ar­genx, stretch­ing back to its $21 bil­lion deal to ac­quire Phar­ma­cyclics.

Ab­b­Vie doesn’t mind walk­ing away from a deal that no longer suits its im­me­di­ate needs. In­fin­i­ty Phar­ma­ceu­ti­cals found that out the hard way when their lead drug for in­do­lent non-Hodgkin lym­phoma hit the end­point in a crit­i­cal study, but fell short of ex­pec­ta­tions for what would be need­ed in the mar­ket­place. And now it faces the same kind of fish-or-cut-bait de­ci­sion af­ter Abl­ynx de­liv­ered pos­i­tive but ho-hum da­ta for their part­nered drug on rheuma­toid arthri­tis, the IL-6 drug vo­bar­il­izum­ab (ALX-0061).

Ab­b­Vie al­ready has an in-house pro­gram they’re hot about.

The point here is that Ab­b­Vie is a com­pa­ny on the move. It il­lus­trat­ed its com­mit­ment to speed with the $350 mil­lion pur­chase of a pri­or­i­ty re­view vouch­er last year that shaves 4 months off the re­view process.

Time is a com­mod­i­ty that Ab­b­Vie can­not af­ford to squan­der.

A few days ago the FDA es­sen­tial­ly blessed Am­gen’s biosim­i­lar of Hu­mi­ra, giv­ing it a clean bill of health for the full slate of in­di­ca­tions now ap­proved in the U.S. The day is com­ing when the com­pa­ny will face off against cheap knock­offs. And that sets the stage for even more deal­mak­ing in the year to come.


 

12. Am­gen: Ready for an­oth­er buy­out

  • 2015 R&D spend­ing: $4.07 bil­lion
  • 2014 R&D spend­ing: $4.27 bil­lion
  • Change: – 4%
  • As a per­cent­age of rev­enue: 18.5%
  • R&D di­rec­tor: Sean Harp­er

The big pic­ture: There aren’t a lot of ma­jor ex­per­i­men­tal drugs to watch in Am­gen’s late-stage pipeline, but they do present a pos­i­tive pic­ture.

Sean Harp­er, Ex­ec­u­tive Vice Pres­i­dent, Re­search and De­vel­op­ment

Erenum­ab, or AMG 334, is now in Phase III, scor­ing pos­i­tive Phase II da­ta for re­duc­ing mi­graines and pos­si­bly po­si­tion­ing it­self as a leader in a Cal­ci­tonin-Gene-Re­lat­ed-Pep­tide (CGRP) re­cep­tor race that pits the Big Biotech against Te­va, Alder and Eli Lil­ly. Late-stage da­ta is due lat­er in the year and you can ex­pect Am­gen to take it straight to the FDA.

Then there’s its os­teo­poro­sis drug ro­mosozum­ab, an­oth­er ri­val-in­fest­ed are­na where Am­gen hopes to do well. This is an­oth­er po­ten­tial block­buster, un­less Ra­dius Health’s ($RDUS) abaloparatide over­shad­ows it on the mar­ket. And its biosim­i­lar of Hu­mi­ra looks to be on a glide path to an ap­proval, with Ab­b­Vie’s le­gal coun­ter­at­tack loom­ing as the biggest ob­sta­cle to get­ting it on the mar­ket.

Am­gen has de­liv­ered ap­provals for drugs like Repatha, its PC­SK9 trail­blaz­er, as well as Im­ly­g­ic, the first on­colyt­ic vi­ral ther­a­py — but al­most cer­tain­ly not the last.

That’s all well and good, but Am­gen hasn’t done a ma­jor deal since it ac­quired Onyx for $10 bil­lion three years ago. Since then, it shut down the old Onyx cam­pus and re­or­ga­nized R&D, cut­ting re­search jobs in the process. And its CEO is a care­ful deal­mak­er who has reined in R&D ex­pens­es while wait­ing for some equal­ly good deals to come his way. That’s why the com­pa­ny is rou­tine­ly men­tioned among the hunters in the mar­ket. Chances are, when Am­gen does strike, it will look to make a play on a mar­ket­ed drug, or a very late-stage as­set that looks like a win­ner at the FDA. Or prefer­ably both.

Val­u­a­tions are down in biotech, mak­ing this as good a time as any for pur­su­ing a deal. I wouldn’t ex­pect Am­gen to sit around for much longer.


 

13. Cel­gene: No fair prices, but lots of deals

  • 2015 R&D spend­ing: $3.7 bil­lion
  • 2014 R&D spend­ing: $2.4 bil­lion
  • Change: +54%
  • As a per­cent­age of rev­enue: 40%
  • R&D di­rec­tor: Ru­pert Vessey

Cel­gene cit­ed its big up­front pay­ments as one of the rea­sons for the big surge in R&D spend­ing in 2015. And that can come as no sur­prise to any­one track­ing one of the most am­bi­tious Big Biotechs in the in­dus­try.

The biotech has been blaz­ing a big, wide trail on the deal­mak­ing front. Re­cent­ly, it re­tooled its col­lab­o­ra­tion with Agios, spend­ing $200 mil­lion up­front on the new arrange­ment. But that’s just the lat­est in a se­ries of high-pro­file pacts.

Ru­pert Vessey, Ex­ec­u­tive Vice Pres­i­dent and Pres­i­dent, Re­search and Ear­ly De­vel­op­ment

Cel­gene paid $7.2 bil­lion to ac­quire Re­cep­tos, bag­ging RPC1063, a promis­ing oral treat­ment now in Phase III de­vel­op­ment for ul­cer­a­tive col­i­tis and mul­ti­ple scle­ro­sis. And a year ago Cel­gene paid a bil­lion dol­lars in a big up­front and eq­ui­ty deal to align it­self with Juno Ther­a­peu­tics, which is un­der a cloud now fol­low­ing the death of three pa­tients in their most ad­vanced CAR-T study.

In the mean­time, George Golumbes­ki has been steadi­ly ink­ing small­er deals, of­ten pre­fer­ring to take an op­tion in de­vel­op­ment ef­forts and wait­ing to see how the biotechs fare in the clin­ic.

Cel­gene Ex­ec­u­tive Chair­man Bob Hug­in likes to say that there’s re­al­ly no such thing as a fair price in biotech. Just about what­ev­er you pay, if it fails it was way too ex­pen­sive, and if it works, it was cheap. The one sin in his book is do­ing noth­ing, es­pe­cial­ly when you have a fran­chise drug (Revlim­id earned $5.8 bil­lion last year) to pay the way for R&D to do more and bet­ter work.

There’s no sign that that ag­gres­sive spir­it is tak­ing a hol­i­day any­time soon.


 

14. Take­da: A cul­ture shift pre­cedes R&D over­haul

  • 2015 R&D spend­ing: $3.5 bil­lion
  • 2014 R&D spend­ing: $2.9 bil­lion
  • Change: +20%
  • As a per­cent­age of rev­enue: 21%
  • R&D di­rec­tor: An­drew Plump

These num­bers are a bit dat­ed, frankly. Take­da re­ports on a fis­cal year ba­sis that ends mid-year, so this has a lot to do with 2014.

An­drew Plump, Take­da Chief Med­ical and Sci­en­tif­ic Of­fi­cer

Take­da has been scour­ing the plan­et to new drugs for its gas­tro port­fo­lio. That quest re­cent­ly led it to an op­tion deal to ac­quire Al­tos Ther­a­peu­tics. More re­cent deals in­clude a pact with Ther­a­vance as well as a $400 mil­lion part­ner­ship with TiGenix for its stem cell ther­a­py Cx601, in stud­ies for treat­ing fis­tu­las in pa­tients with Crohn’s dis­ease. And it set aside $65 mil­lion in near-term fund­ing to pay for a rare dis­ease al­liance with Ul­tragenyx.

Take­da marked a ma­jor cul­tur­al shift when it named Christophe We­ber as its new CEO in the spring of 2015. Japan­ese phar­ma com­pa­nies are not known for mak­ing even mod­est cul­tur­al ad­just­ments, so nam­ing a West­ern­er as its new chief con­found­ed some com­pa­ny ob­servers. You could make a case that We­ber has pro­ceed­ed, rather cau­tious­ly, to make the com­pa­ny over with a more am­bi­tious strat­e­gy fo­cused care­ful­ly on on­col­o­gy and gas­troen­terol­o­gy. But it will have to do much more in R&D to catch up with its U.S. and Eu­ro­pean ri­vals.


 

15. Gilead: The 600-pound go­ril­la goes hunt­ing

  • 2015 R&D spend­ing: $3 bil­lion
  • 2014 R&D spend­ing: $2.85 bil­lion
  • Change:  +5%
  • As a per­cent­age of rev­enue: 9%
  • R&D di­rec­tor:  Nor­bert W. Bischof­berg­er

Judged by mar­ket cap, Gilead is punch­ing way above its R&D weight. Cur­ing he­pati­tis C with a trail­blaz­ing new cock­tail comes with a hefty price, and this is one heavy­weight that will nev­er miss a punch on sales.

So­val­di got it all start­ed, but Gilead just days ago got an ap­proval for its lat­est cock­tail, Ep­clusa, which was re­mark­able pri­mar­i­ly be­cause it marked the en­try of an­oth­er sig­nif­i­cant dis­count as com­pe­ti­tion in the field con­tin­ues to heat up.

Nor­bert W. Bischof­berg­er, Ex­ec­u­tive Vice Pres­i­dent, Re­search and De­vel­op­ment and Chief Sci­en­tif­ic Of­fi­cer, Gilead

Gilead built its R&D house on a foun­da­tion of HIV drugs, and it’s con­tin­ued to make hay with new ar­rivals on that front as well. But Zy­delig (ide­lal­is­ib) got hit with a re­cent set­back af­ter Gilead was forced to halt 6 stud­ies fol­low­ing signs of se­ri­ous side ef­fects.

Gilead still knows how to do a splashy deal. It paid Gala­pa­gos $725 mil­lion in an up­front and eq­ui­ty buy to part­ner on fil­go­tinib, the rheuma­toid arthri­tis drug that Ab­b­Vie dropped in fa­vor of its in-house pro­gram.

So what’s left to talk about in the pipeline?

Not a huge amount, which is why all the an­a­lysts for months now have been buzzing about how bad­ly Gilead needs a ma­jor buy­out. Hep C has been huge, but it’s fad­ing. That leaves Wall Street won­der­ing how the com­pa­ny can jazz earn­ings in the fu­ture. Gilead has plen­ty of cash on hand to do a deal. And af­ter all these months, an­a­lysts want to see if they can do some­thing big, and ex­cit­ing.

Ex­pec­ta­tions are sky high, though, so any mis­step will be pun­ished in­stant­ly.

Gilead’s R&D group is no­to­ri­ous­ly hard to talk to. The com­pa­ny has been sub­ject­ed to harsh, pre­dictable crit­i­cism for its strate­gies and pric­ing poli­cies. But Gilead is the 600-pound go­ril­la in its sphere. It doesn’t ex­plain and it doesn’t com­plain. But it does per­form.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Large advertisements for the drug Vivitrol decorate the walls of Grand Central Station on June 15, 2017 in New York City. (Photo: Andrew Lichtenstein via Getty)

FDA slaps down Alk­er­mes for mis­lead­ing Viv­it­rol ads — don't for­get vul­ner­a­bil­i­ty to opi­oid over­dose

The ads piqued interest as soon as they started appearing in 2016: at Grand Central Station, on the Red Line in Cambridge, and on a billboard off the New Jersey Turnpike. All showed a young person, generally with his or her arms crossed, and the question, “what is Vivitrol?”

Vivitrol’s maker, Alkermes, was in the midst of a marketing and lobbying campaign to promote the anti-opioid addiction drug — a campaign that would face significant backlash for tarnishing competitors despite little evidence for Vivitrol’s superiority.

FDA in-house re­view spot­lights an is­sue with one of Hori­zon's end­points but notes ef­fi­ca­cy for lead drug

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.

Seat­tle Ge­net­ics de­tails pos­i­tive OS and PFS da­ta for tu­ca­tinib in breast can­cer

Seattle Genetics $SGEN is showing off more positive data around tucatinib, its pivotal-stage drug for HER2 positive breast cancer.

A month after hearing about solidly upbeat hazard ratios, we learned today that the estimated progression-free survival rate at one year was 33% in the tucatinib arm compared to 12% for patients taking trastuzumab and capecitabine alone.

Median PFS was 7.8 months (95% CI: 7.5, 9.6) in the tucatinib arm, compared to 5.6 months (95% CI: 4.2, 7.1) in the control arm.

Bat­tered, cash hun­gry In­tec feels the burn of No­var­tis re­jec­tion

It’s a case of some bad timing for Intec.

Just when a key trial testing the company’s Accordion drug delivery tech imploded in Parkinson’s disease, they handed Novartis data from a successful PK study of a custom Accordion pill engineered to deliver a Novartis compound to entice the Swiss drugmaker into signing a licensing agreement.

Novartis said thanks, but no thanks.

For the cash-strapped Israeli drug developer, the failure to clinch the deal marks a big blow. As of the third quarter, the company has $15.7 million in cash and equivalents, which HC Wainwright analysts estimate will keep the lights on into mid-2020.

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

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