Roche’s he­mo­phil­ia drug slash­es bleed­ing rate, boost­ing block­buster pro­jec­tions — but doubts on safe­ty linger

Roche HQ in Basel on May 21, 2017 / John Mc­Carter

Roche served up an­oth­er por­tion of late-stage da­ta on its he­mo­phil­ia drug emi­cizum­ab (ACE910), un­der­scor­ing why an­a­lysts be­lieve the Swiss phar­ma gi­ant has an­oth­er block­buster drug on its hands.

Prep­ping for an up­com­ing sci­ence con­fer­ence, in­ves­ti­ga­tors re­port­ed that 62.9% of the he­mo­phil­ia A pa­tients who had de­vel­oped in­hibitors to stan­dard ther­a­pies and were on their drug had ze­ro bleed­ing episodes at 31 weeks, com­pared to on­ly 5.6% of pa­tients on by­pass­ing agents. The pa­tients saw bleed­ing rates slashed for all kinds of bleeds re­viewed, from all bleeds to joint bleeds, with a clear win on a qual­i­ty of life mea­sure­ment as well. And in a small sub­set of pa­tients pro­vid­ed a pre­emp­tive by­pass­ing agent and then emi­cizum­ab, the risk of bleed­ing dropped 79%.

But the two Phase III stud­ies Roche re­port­ed on were not with­out dan­ger. There were 2 cas­es of throm­boem­bol­ic events and 3 in­stances of throm­bot­ic mi­croan­giopa­thy. 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. That will soon be han­dled by reg­u­la­tors. 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.

The drug re­mains one of Roche’s top Phase III prospects and one of the po­ten­tial­ly most valu­able or­phan prod­ucts now in the late-stage pipeline. Eval­u­atePhar­ma has es­ti­mat­ed 2022 sales at close to $1.5 bil­lion with the most op­ti­mistic an­a­lysts ready to mul­ti­ply that by at least two. With Roche’s PD-L1 check­point drug Tecen­triq came un­der a cloud af­ter fail­ing a Phase III, suc­cess on this score is crit­i­cal.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

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.

Iron­wood kicks de­layed-re­lease Linzess for­mu­la­tion to the curb af­ter tri­al fail­ure

The delayed-release formulation of Ironwood and Allergan’s bowel drug Linzess will not see the light of day.

The experimental drug, MD-7246, failed to help patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) in a mid-stage study, prompting the partners to abandon the therapy.

First approved in 2012, Linzess (known chemically as linaclotide) enhances the activity of the intestinal enzyme guanylate cyclase-C to increase the secretion of intestinal fluid and then transit through the intestinal tract, as well as reduce visceral pain, to relieve pain and constipation associated with IBS.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.