FDA re­jects Am­gen’s os­teo­poro­sis drug ro­mo as ri­val Ra­dius sur­pris­es with a new CEO

Af­ter miss­ing a key sec­ondary end­point for its big os­teo­poro­sis drug ro­mosozum­ab last fall and then run­ning in­to a dis­turb­ing safe­ty is­sue two months ago, Am­gen $AMGN said Sun­day that the FDA has hand­ed the big biotech a re­jec­tion on its mar­ket­ing ap­pli­ca­tion, a wide­ly an­tic­i­pat­ed set­back for the drug de­vel­op­er that will give ri­val Ra­dius a shot at es­tab­lish­ing a big lead in what had once been thought of as a horse race. And with­in hours of the CRL news from Am­gen, Ra­dius put out the word that it is mak­ing a change at the helm, with long­time CEO Bob Ward out and No­vo US pres­i­dent Jes­per Høi­land step­ping in to or­ches­trate the cam­paign for Ra­dius’ first mar­ket launch.

Jes­per Høi­land

Ac­cord­ing to the com­pa­ny’s state­ment, the agency says they want Am­gen to in­clude the da­ta from two late-stage stud­ies – the ARCH study as well as a Phase III in men called the BRIDGE tri­al, sig­nif­i­cant­ly de­lay­ing a come­back. That’s al­so bad news for UCB, which is part­nered with Am­gen.

BRIDGE was com­plet­ed more than a year ago. I asked a spokesper­son why the da­ta hadn’t al­ready been sup­plied to the FDA, and she said in re­ply that the ex­ec­u­tive crew at Am­gen would of­fer some added con­text dur­ing their earn­ings re­view Ju­ly 25.

In a fol­lowup on Mon­day af­ter­noon, Am­gen added: “Based on orig­i­nal dis­cus­sions with FDA, the FRAME study was suf­fi­cient for ini­tial BLA sub­mis­sion. The piv­otal 7,180-pa­tient FRAME tri­al was the first to eval­u­ate ver­te­bral frac­ture risk re­duc­tion as ear­ly as one year as a pri­ma­ry end­point.”

Ro­mo — which tar­gets the scle­rostin pro­tein — fol­lowed by Am­gen’s Pro­lia (deno­sum­ab) clear­ly vault­ed the bar in Phase III for re­duc­ing ver­te­bral frac­tures, with a hefty 75% risk re­duc­tion com­pared to a place­bo plus deno­sum­ab. In­ves­ti­ga­tors al­so were able to show a bet­ter safe­ty pro­file in its pre­sen­ta­tion at the an­nu­al con­fab of the Amer­i­can So­ci­ety for Bone Min­er­al Re­search. And there was an in­crease in bone min­er­al den­si­ty among the drug arm in the study.

But the drug al­so missed a key sec­ondary end­point in the piv­otal pro­gram. The drug did not sig­nif­i­cant­ly im­prove pa­tients’ risk of non-ver­te­bral frac­tures, leav­ing Ra­dius Health $RDUS with a pos­si­ble dis­tinct ad­van­tage on that score af­ter win­ning ap­proval in late April. Then in May, Am­gen pre­saged the re­jec­tion with a note spelling out a dis­turb­ing car­dio risk im­bal­ance be­tween ro­mo and Fos­amax; 2.5% for ro­mo and 1.9% for Fos­amax.

Reg­u­la­tors made it clear they want­ed to eval­u­ate the full set of da­ta in siz­ing up an ap­proval, de­rail­ing an ex­pect­ed sum­mer OK.

The board at Waltham, MA-based Ra­dius of­fered Ward a slap on the back and con­grat­u­la­tions for or­ga­niz­ing the IPO and Tym­los ap­proval. Chair­man Kurt Graves al­so made it clear that the com­pa­ny want­ed to go with an ex­pe­ri­enced hand at jump-start­ing new drug brands, giv­ing Høi­land a cheer for his work in that field at No­vo.

While both Am­gen and Ra­dius have an­gled for an ap­proval, com­mer­cial suc­cess is a com­plete­ly dif­fer­ent is­sue. As The New York Times re­port­ed re­cent­ly, pa­tients are gen­er­al­ly start­ed on bis­pho­s­pho­nates like Fos­amax, which are old and cheap. But they’re al­so lim­it­ed, un­able to build bone the way For­teo and the two new drugs are de­signed to do.

Lil­ly, mean­while, has been rapid­ly jack­ing up the price of For­teo ahead of its loss of patent pro­tec­tion. The Times re­ports the whole­sale price has soared to $3,100 a month, more than three times its price in 2010. Lil­ly has been in­creas­ing the price twice a year, for six years.

Sean Harp­er

Am­gen has had its ups and downs over the past year. Parsabiv was ap­proved ear­li­er this year, but isn’t ex­pect­ed to be­come a block­buster. A Phase III CGRP mi­graine drug – now part­nered with No­var­tis – has done well, but so have oth­er ri­val ther­a­pies that have been crowd­ing in. It’s had more suc­cess in the courts, but still faces an on­go­ing le­gal brawl with Re­gen­eron and Sanofi over the PC­SK9 mar­ket, while nei­ther group has been able to gain much trac­tion with pay­ers. Go­ing back to the draw­ing board on ro­mo won’t help, but can’t be avoid­ed as the drug faces an un­cer­tain fu­ture.

“Dur­ing our in­ter­ac­tions with the FDA, we agreed that the ARCH da­ta should be con­sid­ered in the reg­u­la­to­ry re­view pri­or to the ini­tial mar­ket­ing au­tho­riza­tion and, as a re­sult, an­tic­i­pat­ed this re­quest. We look for­ward to work­ing through the re­view process with the Agency,” said Sean Harp­er, ex­ec­u­tive vice pres­i­dent of R&D at Am­gen. “We re­main com­mit­ted to help­ing pa­tients with os­teo­poro­sis and will use the ad­di­tion­al time to bet­ter un­der­stand the ben­e­fit:risk pro­file of Eveni­ty.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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