Shire blasts Roche over its 'mis­lead­ing' case for trail­blaz­ing he­mo­phil­ia drug emi­cizum­ab, scor­ing in­junc­tion

Over the past year, Shire $SH­PG has seen its shares buf­fet­ed re­peat­ed­ly as Roche moved steadi­ly ahead with its late-stage pro­gram for its close­ly-watched he­mo­phil­ia A drug emi­cizum­ab (ACE910). Now it’s fight­ing back, tak­ing Roche to court over the week­end, ahead of any reg­u­la­to­ry de­ci­sion for a se­ries of what it claims are dam­ag­ing and mis­lead­ing com­ments about the drug’s rel­a­tive safe­ty and ef­fi­ca­cy. But Roche im­me­di­ate­ly fired back Mon­day morn­ing with more Phase III da­ta, with no sign that Shire’s as­sault had in­flu­enced its lat­est up­date.

One day be­fore Roche was slat­ed to re­lease new da­ta at a sci­en­tif­ic con­fer­ence in Berlin, Shire an­nounced on Sun­day that it ob­tained a pre­lim­i­nary in­junc­tion from a court in Ham­burg, Ger­many against Roche aimed at the way it’s po­si­tioned the da­ta and shunt­ed blame for throm­bot­ic events – blood clot­ting – away from emi­cizum­ab and on­to by­pass­ing agents, in­clud­ing one from Shire.

Flem­ming Orn­skov

While it’s com­mon for drug mak­ers to wran­gle over in­tel­lec­tu­al prop­er­ty among ri­val ther­a­pies, IP has noth­ing to do with this le­gal coun­ter­at­tack. Shire is ar­gu­ing that Roche has been mak­ing “in­ac­cu­rate and mis­lead­ing” state­ments about the se­ri­ous ad­verse events tracked in its Phase III – events which have con­spired to keep this would-be block­buster un­der a cloud.

Shire claims that Roche has been mis­lead­ing­ly of­fer­ing a look at “treat­ed bleeds” — a sec­ondary end­point — in­stead of the “num­ber of bleeds over time,” orig­i­nal­ly laid out as the pri­ma­ry end­point in HAVEN 1. And by blam­ing throm­bot­ic in­ci­dents on its by­pass­ing agent, Shire adds, it’s been dam­aged.

Roche isn’t say­ing any­thing in di­rect re­sponse and de­clined to re­spond to my ques­tion of how the pre­lim­i­nary in­junc­tion could af­fect to­day’s pre­sen­ta­tion. In a state­ment to End­points News, the Basel com­pa­ny not­ed:

We are not com­ment­ing on press re­leas­es from oth­er com­pa­nies.

We stand be­hind the emi­cizum­ab da­ta, our clin­i­cal tri­al pro­to­col, in­ves­ti­ga­tors and the he­mo­phil­ia com­mu­ni­ty.
We are ex­cit­ed to be here at the In­ter­na­tion­al So­ci­ety on Throm­bo­sis and Haemosta­sis (ISTH) Con­gress to be a part of the sci­en­tif­ic dis­cus­sion around the emi­cizum­ab Phase III HAVEN 1 da­ta. Our de­ci­sions and ac­tions are al­ways based on do­ing what is right for pa­tients. 

As promised, Roche rolled out HAVEN 1 da­ta bright and ear­ly on Mon­day. Emi­cizum­ab, they say, not on­ly re­duced the rate of treat­ed bleeds by 87%, hit­ting the pri­ma­ry end­point, the drug al­so hand­i­ly outscored by­pass­ing agents like Shire’s. Their ther­a­py slashed “on-de­mand BPAs across all bleed mea­sure­ments, in­clud­ing ze­ro treat­ed bleeds (62.9 per­cent vs. 5.6 per­cent), ze­ro treat­ed spon­ta­neous bleeds (68.6 per­cent vs. 11.1 per­cent), ze­ro treat­ed joint bleeds (85.7 per­cent vs. 50.0 per­cent), ze­ro treat­ed tar­get joint bleeds (94.3 per­cent vs. 50.0 per­cent) and ze­ro bleeds over­all, which in­cludes all treat­ed and non-treat­ed bleeds (37.1 per­cent vs. 5.6 per­cent).”

Roche, though, has al­so ac­knowl­edged that there were 2 cas­es of throm­boem­bol­ic events and 3 in­stances of throm­bot­ic mi­croan­giopa­thy, ac­cord­ing to re­searchers en­gaged in the late-stage pro­gram. Back in Feb­ru­ary Roche rat­tled its most bull­ish fol­low­ers on this drug with the re­port that a pa­tient died from a rec­tal he­m­or­rhage af­ter be­ing treat­ed with emi­cizum­ab and a by­pass­ing agent dur­ing a break­through bleed­ing episode. The in­ci­dents were at­trib­uted to the BPAs.

As a re­sult of the ad­verse events, an­a­lysts back­ing Shire and No­vo Nordisk – both of which have he­mo­phil­ia fran­chis­es threat­ened by Roche’s drug – have been spec­u­lat­ing about a pos­si­ble di­rect tie to the Roche drug. While Roche would ap­pear to have the in­side track among pa­tients who had de­vel­oped in­hibitors to stan­dard drugs, it could face re­sis­tance to its use among oth­ers with­out in­hibitors, re­strict­ing its use and lim­it­ing its com­mer­cial ap­peal.

Shire made it clear that it’s been rat­tling its le­gal sword be­hind the scenes, to no avail.

From Shire’s state­ment:

Based on Roche’s pub­licly avail­able in­for­ma­tion to date (as of 7 Ju­ly 2017), physi­cians, pa­tients and care­givers may be mis­in­formed about the ap­pro­pri­ate man­age­ment of break­through bleeds un­con­trolled by emi­cizum­ab. In ad­di­tion, through these ac­tions, Shire be­lieves Roche has un­law­ful­ly dis­par­aged Shire’s proven by­pass­ing agent, FEI­BA (An­ti-In­hibitor Co­ag­u­lant Com­plex). Shire has is­sued mul­ti­ple un­heed­ed re­quests to Roche in an ef­fort to re­solve these con­cerns in an ap­pro­pri­ate man­ner. As a re­sult, Shire made the de­ci­sion to seek court in­ter­ven­tion.

There’s a lot at stake here for these com­pa­nies. Roche bad­ly needs to push through a slate of ma­jor new prod­uct ap­provals as biosim­i­lar com­pe­ti­tion to its fran­chise drugs starts to carve in­to its rev­enue flow. An­a­lysts have of­fered a va­ri­ety of es­ti­mates on peak sales for emi­cizum­ab, in­clud­ing one of $1.5 bil­lion by 2022, if Roche can es­tab­lish this as a new stan­dard of ther­a­py for he­mo­phil­ia.

Roche is slat­ed to of­fer a new pre­sen­ta­tion on its drug on Mon­day at the 26th In­ter­na­tion­al So­ci­ety on Throm­bo­sis and Haemosta­sis (ISTH) Meet­ing in Berlin, Ger­many. The phar­ma gi­ant did not im­me­di­ate­ly re­spond to a query from End­points News on Sun­day.

Shire CEO Flem­ming Orn­skov got in­to the he­mo­phil­ia mar­ket in a big way with its ac­qui­si­tion of Bax­al­ta. But it’s al­so been af­flict­ed with wa­ver­ing sales, which has hit the bot­tom line.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.