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.

Paul Hudson, Sanofi CEO (Getty Images)

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

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Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

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Roger Perlmutter, Merck R&D chief (YouTube)

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Covid-19 roundup: Buoyed by soar­ing shares, No­vavax inks a $167M deal to buy Covid-19 vac­cine-mak­ing fa­cil­i­ty; France cools on hy­drox­y­chloro­quine

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