Hal Barron at Endpoints News' UKBIO 2019

FDA ap­proves GSK's BC­MA drug, mark­ing a rocky re­turn to can­cer for the British gi­ant

De­spite con­cerns on both safe­ty and ef­fi­ca­cy, the FDA ap­proved Glax­o­SmithK­line’s mul­ti­ple myelo­ma drug be­lan­tam­ab mafodotin, de­cid­ing that for the sick­est pa­tients the ben­e­fits out­weighed the risk. It will be mar­ket­ed as Blenrep and sold for $23,900 per month.

The ap­proval is no­table both for the mul­ti­ple myelo­ma field and for GSK it­self. It’s the first ap­proval for a drug that tar­gets BC­MA, a pro­tein over­ex­pressed on mul­ti­ple myelo­ma cells that re­searchers have been try­ing to tar­get for over a decade. A flur­ry of oth­er BC­MA drugs, hav­ing al­ready shown promise in ear­ly tri­als, are like­ly to fol­low in the next few years.

For GSK, it’s the first can­cer drug the British gi­ant has de­vel­oped in-house since they trad­ed al­most the en­tire­ty of their on­col­o­gy pipeline to No­var­tis in ex­change for a port­fo­lio of vac­cines and in­fec­tious dis­ease drugs in 2014. And the com­pa­ny, un­der R&D chief Hal Bar­ron, wants it to be a sig­nal of things to come.

Ax­el Hoos

“GSK is back in on­col­o­gy,” Ax­el Hoos, GSK’s se­nior vice pres­i­dent of on­col­o­gy R&D, told End­points News.

Still, Blenrep’s im­pact may be lim­it­ed, both from a med­ical and mar­ket per­spec­tive. Both are like­ly to be blunt­ed by the an­ti­body-drug con­ju­gate’s tox­i­c­i­ties and the stronger re­sults oth­er late-stage an­ti-BC­MA drugs have shown in re­cent tri­als.

GSK has said pa­tients in its piv­otal tri­al lived longer than you would ex­pect pa­tients at that stage to live, but in its in­ter­nal re­view, the FDA called the com­pa­ny’s sur­vival end­points “un­in­ter­pretable” be­cause there was no con­trol group. Still, the agency not­ed the 31% re­sponse rate in pa­tients who had failed nu­mer­ous drugs — in­clud­ing J&J’s block­buster Darza­lex — and said the ther­a­py “may be ben­e­fi­cial.”

The ques­tion was whether the ben­e­fit out­weighed the risk: The drug caused a form of cornea de­gen­er­a­tion called ker­atopa­thy, blur­ring some pa­tients’ vi­sion and caus­ing “se­vere vi­sion loss” in oth­ers.

An­a­lysts tend­ed to think that for pa­tients on their last line of ther­a­py, the cal­cu­lus made sense, and sure enough, the FDA ad­vi­so­ry com­mit­tee vot­ed 12-0 to rec­om­mend ap­proval. But they ar­gued that ear­li­er-stage pa­tients would be less like­ly to ac­cept those risks and that oth­er ther­a­pies now in clin­i­cal de­vel­op­ment could out­shine it.

“Many of the fac­tors raised by re­view­ers, while they mit­i­gate the risk of a CRL from the FDA,” Cowen’s Steve Scala wrote af­ter the Ad­Com, “high­light the very is­sues that could make com­mer­cial launch of the drug some­thing of an up­hill bat­tle.”

He pro­ject­ed $500 mil­lion in peak sales in 2025.

Those oth­er ther­a­pies in­clude the CAR-T ther­a­py joint­ly de­vel­oped by J&J and Leg­end Biotech and the “ide-cel” CAR-T ther­a­py de­vel­oped by blue­bird and Bris­tol My­ers Squibb. In ear­li­er-stage pa­tients, those showed re­sponse rates of 100% and 89%, re­spec­tive­ly. Re­gen­eron and Am­gen each have BC­MA bi-specifics, with the Re­gen­eron one al­ready show­ing ear­ly promise.

For now, GSK will have the field to them­selves, but in the long run they’re bank­ing on the mar­ket ad­van­tage of be­ing first, the ease of sup­ply­ing and ad­min­is­ter­ing an an­ti­body as op­posed to a CAR-T ther­a­py, and the po­ten­tial to com­bine Blenrep with oth­er ther­a­pies. Hoos al­so point­ed to the drug’s dura­bil­i­ty and he ar­gued that, al­though the safe­ty is­sues were re­al, doc­tors could man­age them with eye drops and oth­er mod­i­fi­ca­tions.

This “is the first tar­get­ing agent for BC­MA, which a lot of peo­ple have been wait­ing for,” he said. “We think this can add a lot of val­ue.”

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; 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.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Robert Davis, Merck CEO

Mer­ck shares slide as re­sponse to its oral Covid an­tivi­ral plunges on lat­est up­date

After a second look, Merck says that its big breakthrough on the Covid front doesn’t look so good.

The pharma giant updated its data on its oral antiviral molnupiravir on Friday, and said that its drug reduced the rate of hospitalization and death by 30% — a sharp plunge from the 50% rate reported at the beginning of October during their interim readout.

And the news sparked a sharp retreat on Merck’s shares, which fell 4% ahead of the bell on Friday.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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