Heart at­tack while on Repatha? Am­gen will give you your mon­ey back

Wash­ing­ton, DC —Am­gen has over­come one of the tough­est chal­lenges in car­dio re­search, out­lin­ing a clear health ben­e­fit for its PC­SK9 cho­les­terol drug Repatha. But be­fore pay­ers get a chance to gnaw at the num­bers be­ing put on dis­play to­day, look­ing for holes or find­ing fault with the lev­el of ben­e­fit, the phar­ma gi­ant wants to send them a sim­ple mes­sage: it’s ready to of­fer a ne­go­ti­at­ed truce to get them to drop their de­fens­es and pull out of the trench­es.

Af­ter find­ing ac­cess to Repatha blocked and locked by blan­ket pay­er re­jec­tions, cor­ralling this drug far from the main­stream, Am­gen $AMGN wants to par­lay.

If pay­ers will just con­sid­er the ben­e­fits Am­gen is demon­strat­ing to­day and take down their elab­o­rate bar­ri­ers, com­pa­ny ex­ecs say they are not on­ly will­ing to of­fer a re­fund when the drug fails, they’ll al­so ne­go­ti­ate an­nu­al pay­ment caps and risk shar­ing deals with in­sur­ers so pay­ers can be sure to keep con­trol of their bud­gets.

Sean Harp­er, EVP Re­search and De­vel­op­ment

Drug re­funds have be­gun to make their way in­to the sys­tem, but they’re still rare enough to make this a stand­out pro­pos­al for a man­u­fac­tur­er try­ing to es­tab­lish a big drug.

“We will work with pay­ers if they’re will­ing to just stop block­ing pa­tients from get­ting the drug,” Sean Harp­er, head of R&D at Am­gen, tells me.

Harp­er and his team showed up at Amer­i­can Col­lege of Car­di­ol­o­gy con­fer­ence in Wash­ing­ton DC on Fri­day to lay their case out with some pi­o­neer­ing da­ta. Study­ing this drug in 27,564 pa­tients over two years, re­searchers say, Repatha was able to low­er a com­pos­ite of car­dio risks by an av­er­age of 20%. And the im­prove­ment in­creased with time, grow­ing from a 16% risk ad­van­tage in year one to 25% af­ter 12 months.

That’s large enough to hit key end­points, but it’s al­so small enough to leave many an­a­lysts won­der­ing if this drug can ever make it to big block­buster sta­tus, which Am­gen is cer­tain it de­serves.

Break­ing down the da­ta, which every­one will do, re­searchers not­ed a 27% re­duc­tion in the risk of heart at­tacks, a 21% risk re­duc­tion for stroke and a 22% re­duc­tion on coro­nary revas­cu­lar­iza­tion. But sig­nif­i­cant­ly there was no im­pact on mor­tal­i­ty or un­sta­ble angi­na.

This was the first time that a tri­al has shown a clear car­dio ben­e­fit from PC­SK9 in­hi­bi­tion.

Sums up Harp­er: “This is huge that we can do this.”

In­vestors, though, were clear­ly not im­pressed. Am­gen’s shares were down 6% af­ter the head­lines hit. And this Tweet should give you a fla­vor for what’s dri­ving the skep­ti­cism:

Umer Raf­fat at Ever­cor­eISI not­ed that most of in­vestors were look­ing for a mor­tal­i­ty ben­e­fit and were clear­ly dis­ap­point­ed in not see­ing one. Am­gen’s bull­ish­ness on the da­ta ahead of the de­tails al­so in­flat­ed ex­pec­ta­tions. And the same bit­ter taste al­so ex­tend­ed to the rest of the play­ers in the field, in­clud­ing Re­gen­eron $REGN and Sanofi $SNY, which both got hit, as well as The Med­i­cines Com­pa­ny {$MD­CO -20%} which has a next-gen ther­a­py in the clin­ic it’s been see­ing suc­cess with.

Baird’s Bri­an Sko­r­ney called it a let down. He added:

The pri­ma­ry end­point just hit stat sig with a HR of 0.85 (be­low ex­pec­ta­tions), while the MACE sec­ondary end­point saw a 20% risk re­duc­tion (in-line). Even more dis­ap­point­ing, CV death failed to sep­a­rate from place­bo. Though we do think that to­day’s da­ta is ev­i­dence that PC­SK9 in­hi­bi­tion works, the ben­e­fit isn’t as pro­found as in­vestors were look­ing for and pay­ers are like­ly to lever­age this dis­ap­point­ment.

Am­gen has bet big on Repatha, bankrolling sev­er­al huge stud­ies to demon­strate the drug’s abil­i­ty to dra­mat­i­cal­ly slash LDL lev­els, which its in­ves­ti­ga­tors say is clear­ly a tox­in. Now it has a win on car­dio out­comes to make its point to physi­cians and a large po­ten­tial pa­tient pop­u­la­tion. But the tri­al suc­cess is not big enough by it­self to get pay­ers to stop re­ject­ing pre­scrip­tions “over and over and over again,” as Harp­er de­scribes the treat­ment to date.

Joshua J. Of­man, Am­gen

“The process on uti­liza­tion man­age­ment has been bro­ken,” says Am­gen val­ue man­age­ment chief Joshua J. Of­man. Ever since 2013’s in­tro­duc­tion of So­val­di pay­ers have been up­ping their game, fig­ur­ing out new ways to stop their mem­bers from get­ting ac­cess to new drugs like this. And in Repatha’s case, pay­ers are in over­drive, forc­ing physi­cians to go back and file over and over again.

The num­bers Am­gen is re­port­ing to­day are al­so be­ing pitched as far more con­ser­v­a­tive than what pa­tients see in the re­al world. All the pa­tients in the study got the best stan­dard of care, which is some­thing you won’t see in the re­al world. And Am­gen is hap­py to ne­go­ti­ate its next round of con­tracts based on re­al-world ex­pec­ta­tions.

So now Am­gen says they’ll guar­an­tee a low­er risk of heart at­tack and re­fund the cost of the drug for any pa­tient who has a heart at­tack. They’ll still come out well ahead if they can reach the at-risk pop­u­la­tion whose LDL lev­els can­not be con­trolled by cheap statins.

Am­gen’s full court press on Repatha has in­clud­ed a co­or­di­nat­ed at­tack on its big ri­val Pralu­ent from Re­gen­eron and Sanofi. At one point re­cent­ly a judge’s rul­ing threat­ened to push the com­pet­ing drug off the mar­ket as Am­gen — a ma­jor league lit­i­ga­tor — pressed its ar­gu­ment that Pralu­ent vi­o­lat­ed its PC­SK9 patents. But the part­ners man­aged to block that, for now.

Next up: Car­dio da­ta from Re­gen­eron and Sanofi. So far, these drugs have ap­peared very sim­i­lar, and it wouldn’t sur­prise many ob­servers if their com­peti­tor comes on around the same score. But every da­ta point will get care­ful scruti­ny as the gi­ants block and tack­le in search of every small ad­van­tage, while hop­ing to achieve a break­through on mar­ket­ing that can fi­nal­ly gen­er­ate some big num­bers.

So far, it’s not look­ing good.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Image: Shutterstock

Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Mer­ck scraps their $425M Covid-19 drug in lat­est pan­dem­ic set­back

Seven months after paying $425 million cash to acquire it, Merck is scrapping a Covid-19 drug they hoped could provide one of the only treatments for severe hospitalized patients.

Merck’s decision comes after they faced significant and unexpected regulatory delays in getting the drug, known as MK-7110 or CD24Fc, across the finish line. The Big Pharma licensed the drug under the belief that it had already shown sufficient benefit in severe patients and they could help scale it up far faster than OncoImmune, its former owner, could. But in February, the company reported that the FDA insisted Merck run a new trial before seeking authorization.

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As­traZeneca-Alex­ion merg­er slides through FTC re­view af­ter sup­posed M&A crack­down pos­es no bar­ri­ers

The AstraZeneca-Alexion megamerger received a good sign Friday, despite warning signs of the tides turning against large M&A pharma deals.

US regulators at the FTC have cleared the acquisition for approval, AstraZeneca announced, all but signing off on the deal to go through once it officially closes in the third quarter. AstraZeneca originally said it was planning to buy out Alexion back in December for $39 billion.

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Severin Schwan, Roche CEO (Georgios Kefalas/Keystone via AP Images)

Look­ing to ce­ment its lead in packed MS mar­ket, Roche's Ocre­vus un­corks new da­ta in ear­ly-stage pa­tients

Among a positively jam-packed multiple sclerosis market, Roche’s Ocrevus has managed to stand out for what the Swiss drugmaker is calling the most successful launch in its long history. But in order to press its advantage, Ocrevus is looking to earlier-stage patients, and new interim data should help build its case there.

After 48 weeks on Roche’s Ocrevus, 85% of newly diagnosed primary progressing or relapsing MS patients without a history of disease modifying therapy posted no disease activity, including disease progression or relapse, according to interim data set to be presented this weekend at the virtual American Academy of Neurology meeting.

J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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David Stack, Pacira Biosciences CEO

In high­ly un­usu­al move, Paci­ra sues med­ical jour­nal for li­bel over its non-opi­oid painkiller

A New Jersey biotech whose only approved drug is used as a painkiller after surgeries is suing a scientific journal, its editors and a handful of authors for libel after the publication printed numerous papers and editorials that the company says discredited the drug.

Pacira Biosciences filed the complaint against the American Society of Anesthesiologists in the US District Court for New Jersey on Wednesday afternoon. A February issue of the group’s journal Anesthesiology printed three articles and other content full of “bias” that “seriously disparaged” the drug Exparel, Pacira claimed.

Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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