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.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.