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.”

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: As­traZeneca signs 400M vac­cine sup­ply deal with EU; 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK

Amid talks with multiple players, the European Commission has reached its first vaccine supply deal with AstraZeneca, securing 400 million doses of the Oxford candidate for all of its member countries.

The pharma giant said in a press release that the deal builds on the existing agreement with Germany, France, Italy and the Netherlands, announced in June. It was unclear, however, whether that means simply extending the same 400 million doses to all EU countries or doubling the reservation to 800 million doses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

Charlie Silver (Mission Bio)

'We want to be every­where.' Mis­sion Bio rais­es $70M be­hind re­sis­tance-hunt­ing se­quenc­ing plat­form

Charlie Silver wants to look really, really closely at a lot of your cells. And he just got a lot of money to do so.

Silver’s startup, Mission Bio, raised $70 million in a Series C round Thursday led by Novo Holdings. The money, which brings Mission Bio to $120 million raised since its 2012 founding, will be used to advance the single-cell sequencing platform they built to detect early response or resistance to new cancer therapies.

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.