Samit Hirawat. Bristol-Myers Squibb

Bris­tol-My­ers is mak­ing a bee-line to the FDA with pos­i­tive liso-cel da­ta — but is it too late in the CAR-T game?

Bris­tol-My­ers Squibb came to ASH this past week­end with a va­ri­ety of mes­sages on the new can­cer drugs they had ac­quired in the big Cel­gene buy­out, in­clud­ing liso-cel, the lead CAR-T pro­gram picked up in the $9 bil­lion Juno ac­qui­si­tion. And one of the most im­por­tant was that they had the piv­otal ef­fi­ca­cy and safe­ty da­ta need­ed to snag an ap­proval from the FDA next year, with the BLA on track for a fil­ing this month.

Pro­vid­ed there are no sna­fus or even mod­est stum­bles now, they should get an OK with­in the 2020 time­line lined out in their $9 CVR for Cel­gene — the first in a tri­fec­ta of ap­provals re­quired for a pay­out.

Whether they can go on to make it in­to a vi­able com­mer­cial ther­a­py, though, is a whole oth­er thing.

At first glance, there isn’t any­thing about the safe­ty and ef­fi­ca­cy da­ta that would force a CRL or de­lay. In a large tri­al of pa­tients with re­lapsed/re­frac­to­ry large B-cell lym­phomas in­ves­ti­ga­tors tracked an out­stand­ing 73% re­sponse rate and 53% com­plete re­sponse rate in heav­i­ly pre­treat­ed pa­tients with few op­tions.

That’s in line with Yescar­ta from Gilead’s Kite, which snagged an ap­proval more than 2 years ago, just af­ter No­var­tis’ Kym­ri­ah came through.

Then there’s safe­ty. To be sure, the drug has safe­ty is­sues. There were 4 pa­tients in the study who died af­ter treat­ment. Sev­er­al oth­ers died for un­re­lat­ed is­sues. But…with on­ly a 2% rate of cy­tokine re­lease syn­drome, Bris­tol-My­ers has a shot at a dif­fer­en­ti­at­ed safe­ty pro­file.

“There is a po­ten­tial these pa­tients can be treat­ed on an out­pa­tient ba­sis,” says Samit Hi­rawat, the chief med­ical of­fi­cer at Bris­tol-My­ers. He not­ed that 26% of pa­tients were nev­er ad­mit­ted, while 76% were ad­mit­ted 4 days or lat­er af­ter ther­a­py. The key is to treat them at a hos­pi­tal with the in­fra­struc­ture to pro­vide care 24/7, so a pa­tient can be ad­mit­ted at any time lat­er if need­ed.

How that will fly with pay­ers af­ter ri­vals have been on the mar­ket for about 3 years, with new ev­i­dence that ear­li­er use of steroids can dra­mat­i­cal­ly re­duce CRS and con­sid­er­able dura­bil­i­ty of re­sponse, will have to be seen.

But time is not on Bris­tol-My­ers’ side. Liso-cel is the long de­layed fol­lowup to the dis­as­trous JCAR015, which killed a num­ber of pa­tients. Their set­back threw them years off sched­ule. And ri­vals are ad­vanc­ing off-the-shelf al­ter­na­tives or oth­er new ap­proach­es that could knock the pi­o­neers com­plete­ly out of the game. In the mean­time, Bris­tol-My­ers is gath­er­ing its own dura­bil­i­ty da­ta and will grad­u­al­ly see if their mix of CD4 and CD8 cells can do bet­ter.

The main in­ter­est now is in the time­line around the ap­proval. Hi­rawat says they’ll ask for pri­or­i­ty re­view, and there’s every rea­son to be­lieve that reg­u­la­tors will move swift­ly — bar­ring a nasty sur­prise.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.