Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J at­tempts to jos­tle past Bris­tol-My­ers Squibb and blue­bird for a land­mark ap­proval of its an­ti-BC­MA CAR-T — and while Glax­o­SmithK­line maps a quick path to the FDA rid­ing on its own BC­MA-tar­get­ing an­ti­body-drug con­ju­gates — the bis­pecifics are ar­riv­ing on the scene to stake a claim for a mar­ket that could cross $10 bil­lion per year.

The main ri­val­ry in mul­ti­ple myelo­ma is shap­ing up to be one be­tween Re­gen­eron and Bris­tol-My­ers, which picked up a bis­pe­cif­ic an­ti­body to BC­MA through its re­cent­ly closed $74 bil­lion takeover of Cel­gene. Both pre­sent­ed promis­ing first-in-hu­man da­ta at the ASH 2019 meet­ing.

Bri­an Sko­r­ney

“Al­though Cel­gene has both a time and myelo­ma ex­pe­ri­ence ad­van­tage, we be­lieve Re­gen­eron is close be­hind and could even catch up if the megamerg­er leads to some pro­gram de­lays” was Baird an­a­lyst Bri­an Sko­r­ney’s take as he scored the first myelo­ma da­ta for REGN5458.

With bis­pecifics the gen­er­al idea is to bridge T cells and tu­mors cells, there­by in­duc­ing the killing of can­cer. REGN5458, in par­tic­u­lar, binds to the CD3 re­cep­tor on the im­mune cells and BC­MA on mul­ti­ple myelo­ma cells.

In­ves­ti­ga­tors had da­ta on the first two dose groups to re­port from the on­go­ing Phase I/II dose es­ca­la­tion study, in which 3 pa­tients were giv­en 3mg week­ly dos­es and 4 pa­tients re­ceived 6mg. Over­all, 4 of them record­ed a re­sponse — giv­ing rise to a 57% re­sponse rate over­all — 3 of them from the 6mg group (75% re­sponse rate there). Among those three tak­ing the high­er dose, two passed the cru­cial test of be­ing min­i­mal resid­ual dis­ease (MRD) neg­a­tive, a high bar sug­gest­ing no can­cer cells were de­tectable in their bone mar­row.

Is­rael Lowy

No­tably, these pa­tients have had a me­di­an of sev­en lines of pri­or ther­a­py and had all failed CD38 an­ti­body treat­ment. And at the 6mg dose lev­el, the me­di­an num­ber of pri­or treat­ment was even high­er at 9.5, Sko­r­ney not­ed.

Cy­tokine re­lease syn­drome, ane­mia and lym­phope­nia were all doc­u­ment­ed, though Re­gen­eron said the ad­verse events were not dose lim­it­ing.

Their re­sults come on the heels of Bris­tol-My­ers/Cel­gene’s da­ta re­veal, in which CC-93269 showed an 89% re­sponse rate in the high­est dose of 10mg af­ter be­ing in­ves­ti­ga­tors went up from 0.15mg with 30 pa­tients to­tal.

“CC-93269 shows promis­ing dose-de­pen­dent ef­fi­ca­cy, in­clud­ing MRD-neg­a­tive sCRs, with a con­ve­nient ad­min­is­tra­tion sched­ule in pa­tients with heav­i­ly pre­treat­ed re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma.”

Most of the grade 3 or high­er ad­verse events were neu­trope­nia, ane­mia and in­fec­tions, the in­ves­ti­ga­tors not­ed, though cy­tokine re­lease syn­drome al­so cropped up in 76% of all pa­tients in the tri­al.

In the Re­gen­eron tri­al, three pa­tients ex­pe­ri­enced CRS, amount­ing to a 42% oc­cur­rence.

“We are en­cour­aged to see promis­ing, rapid clin­i­cal ac­tiv­i­ty even at the ini­tial two dos­es of REGN5458 in heav­i­ly pre­treat­ed pa­tients with mul­ti­ple myelo­ma,” Is­rael Lowy, head of clin­i­cal and trans­la­tion­al sci­ences for on­col­o­gy at Re­gen­eron, said in a state­ment. “We are ac­tive­ly re­cruit­ing pa­tients in­to high­er dose groups in this tri­al and look for­ward to shar­ing fur­ther re­sults in 2020.”

Lowy added that the Tar­ry­town, NY-based biotech has ini­ti­at­ed a tri­al for its sec­ond BC­MA/CD3 bis­pe­cif­ic, which binds dif­fer­ent­ly. They are mov­ing fast — and they need to in a field brim­ming with com­peti­tors.

Pfiz­er is ad­vanc­ing its own ver­sion, al­though the ab­sence of equiv­a­lent­ly promis­ing da­ta at ASH pushed Sko­r­ney to sur­mise “not all bis­pecifics are cre­at­ed equal­ly.” Ab­b­Vie al­so got its hands on a bis­pe­cif­ic hook­ing to the same tar­gets from a $90 mil­lion deal with Teneo­bio.

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.

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.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

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.