In blow to Shire, Roche bags an FDA OK for its block­buster he­mo­phil­ia A prospect

Guy Young, USC Keck School of Med­i­cine. Im­age: USC

De­spite be­ing dogged by ques­tions about safe­ty and an un­usu­al le­gal coun­ter­at­tack from ri­val Shire, Roche to­day scored an FDA ap­proval for emi­cizum­ab (ACE910), its he­mo­phil­ia A ther­a­py which will now hit the mar­ket as Hem­li­bra.

The drug is de­signed to bring to­geth­er fac­tor IXa and fac­tor X pro­teins to re­store the blood clot­ting process for he­mo­phil­ia A pa­tients, re­duc­ing the threat of bleeds. And an­a­lysts have pegged po­ten­tial sales at up to $5 bil­lion, though Reuters‘ most re­cent con­sen­sus was a con­sid­er­ably less frothy $1.6 bil­lion.

Sig­nif­i­cant­ly, the ap­proval comes more than three months ahead of its late-Feb­ru­ary PDU­FA date, reg­is­ter­ing as the 40th new mol­e­c­u­lar en­ti­ty of the year as the in­dus­try looks ready to cruise past the cur­rent-era record 44 new ap­provals achieved at CDER in 2015.

The ap­proval was based on some im­pres­sive da­ta on a drug that Roche has stead­fast­ly main­tained is a near cer­tain block­buster-to-be. But it al­so comes with a boxed warn­ing on a threat of blood clots and dam­age to blood ves­sels, which could al­so crimp sales.

Last sum­mer the phar­ma gi­ant laid out da­ta de­tail­ing how the drug 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).”

Along the way, Roche blamed Shire’s by­pass­ing agents for throm­boem­bol­ic events that they saw in their study, ad­vis­ing doc­tors to stop us­ing FEI­BA.

Shire, to put it mild­ly, flipped.

“To im­ply a cause-and-ef­fect of FEI­BA hav­ing caused the se­vere ad­verse events is mis­lead­ing,” Shire hema­tol­ogy chief Ju­liana Dierks told Reuters. “We are look­ing for­ward to trans­paren­cy. Give us the da­ta, give us the facts.”

Shire al­so claimed 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 throm­boem­bol­ic in­ci­dents in the study — with one pa­tient dy­ing of a rec­tal he­m­or­rhage — on its by­pass­ing agent, Shire al­so main­tained that it had been dam­aged. Shire sought an in­junc­tion against Roche in Ger­many, but the ac­tion was re­ject­ed by the court and the com­plaint hasn’t slowed its Big Phar­ma ri­val at all.

How bad could this get for Shire? Bern­stein an­a­lysts say Hem­li­bra along with new ther­a­pies from No­vo Nordisk and Bay­er could shrink its share of the he­mo­phil­ia mar­ket from 49% to 29%

Roche has nev­er done any­thing but shrug the as­sault off. The phar­ma gi­ant is los­ing patent pro­tec­tion on three fran­chise drugs, and it needs to field ma­jor new drugs to fill the gap.

Hem­li­bra is priced to do just that. Roche, like a grow­ing num­ber of com­pa­nies, is us­ing an av­er­age whole­sale price based on av­er­age weight. At an av­er­age weight of about 127 pounds, Roche says, the cost “is ap­prox­i­mate­ly $482,000 for the first year of treat­ment and then ap­prox­i­mate­ly $448,000 per year, which is less than half of the WAC of the on­ly oth­er ap­proved pro­phy­lac­tic treat­ment for these pa­tients.”

The price fits in at the low­er rung of the list of the 10 most ex­pen­sive drugs in the world, all among the rare dis­ease crowd.

“To­day’s ap­proval pro­vides a new pre­ven­ta­tive treat­ment that has been shown to sig­nif­i­cant­ly re­duce the num­ber of bleed­ing episodes in pa­tients with he­mo­phil­ia A with Fac­tor VI­II in­hibitors,” said Richard Paz­dur, whose ti­tles in­clude act­ing di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts. “In ad­di­tion, pa­tients treat­ed with Hem­li­bra re­port­ed an im­prove­ment in their phys­i­cal func­tion­ing.”

“Peo­ple with he­mo­phil­ia A who de­vel­op in­hibitors face sig­nif­i­cant chal­lenges pre­vent­ing bleeds and typ­i­cal­ly re­quire in­fu­sions of med­i­cine mul­ti­ple times a week, which can be es­pe­cial­ly dif­fi­cult for young chil­dren and their fam­i­lies,” said Guy Young, di­rec­tor of he­mo­sta­sis and throm­bo­sis pro­gram, Chil­dren’s Hos­pi­tal Los An­ge­les, and pro­fes­sor of pe­di­atrics, Uni­ver­si­ty of South­ern Cal­i­for­nia Keck School of Med­i­cine. “This new med­i­cine has been shown to re­duce the fre­quen­cy of bleeds com­pared to the cur­rent­ly avail­able med­i­cines and on­ly needs to be in­ject­ed once a week. This could make a mean­ing­ful dif­fer­ence for these chil­dren.”

Im­age: Roche, John Mc­Carter

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Tillman Gerngross, Adagio CEO

Q&A: Till­man Gern­gross ex­plains why his Covid mAb will have an edge over an al­ready crowd­ed field

If anyone knows about monoclonal antibodies, it’s serial entrepreneur, Adimab CEO, and Dartmouth professor of bioengineering Tillman Gerngross.

Even the name of Gerngross’ new antibody startup Adagio Therapeutics is meant to reflect his vision behind the development of his Covid-19 mAb: slowly, he said, explaining that “everyone else, whether it’s Regeneron, Lilly, or AstraZeneca, Vir, they all valued speed over everything.”

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Susan Galbraith speaking at Endpoints News' virtual EUBIO21 summit

Imfinzi/treme­li­mum­ab com­bo scores As­traZeneca an­oth­er OS win — this time in liv­er can­cer

Is the tide turning on AstraZeneca’s battered PD-L1/CTLA4 combo?

A single priming dose of the experimental tremelimumab, followed by Imfinzi every four weeks, beat Nexavar (sorafenib) in helping a group of liver cancer patients live longer in a Phase III study, the company reported, meeting the primary endpoint.

Specifically, the two drugs extended overall survival for patients with unresectable hepatocellular carcinoma who had not received prior systemic therapy and were not eligible for localized treatment.

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Alex Gorsky, J&J CEO (Evan Vucci/AP Images)

As suits mount, J&J spins out talc li­a­bil­i­ties in­to Chap­ter 11 us­ing 'Texas two-step' ma­neu­ver

With lawsuits piling up alleging its talc baby powder products are unsafe, J&J has taken the controversial step of spinning out its related liabilities into a new company and filing for Chapter 11 bankruptcy, according to court papers filed late Thursday.

The procedures will now head to bankruptcy court in North Carolina, halting all lawsuits and their respective discovery processes. It’s a move that comes a few months after the Wall Street Journal and Reuters both reported J&J was exploring this option, and after a federal judge said the process could move forward in August.