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.”

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.