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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Hervé Affagard, MaaT Pharma CEO

One year in­to clin­i­cal hold, FDA has more ques­tions about 'pooled' mi­cro­bio­me ther­a­py

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”

Feud be­tween two biotechs left near­ly 12M dos­es of mon­key­pox an­tivi­ral on the ta­ble

As the US embarks on a new delivery strategy to stretch out its thinning supply of monkeypox vaccines, the need for treatments could pick up as cases of the virus rise. And the amount of courses of one potential antiviral, soon to be clinically tested for efficacy in humans, was almost 12 million more than it is today, according to SEC filings.

While not greenlit for treating monkeypox, SIGA’s FDA-approved smallpox antiviral, Tpoxx, can be requested by physicians under an expanded use program. As of Monday, HHS tells Endpoints News it had tapped into more than 15,000 of the 1.7 million courses of Tpoxx that have been stockpiled, but with cases climbing over the past few weeks, demand will likely not peter out in the near future, especially if the vaccine supply runs dry.

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