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.

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.

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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.

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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.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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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.

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Bris­tol My­ers Squib­b's just-launched MS drug Zeposia makes the cut in key ul­cer­a­tive col­i­tis tri­al

In March, Zeposia became the third oral S1P modulator to secure US approval for multiple sclerosis. Now, the drug has succeeded in a key ulcerative colitis study.

The immunomodulator, akin to others in its class, controls lymphocyte trafficking by limiting the white blood cells to the lymphatic system, in the lymph nodes, and thwarting their ability to jam up lymph nodes — precluding their ability to penetrate the bloodstream and the central nervous system.

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.

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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.

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