Just in time to as­sure a de­ci­sion be­fore CVR dead­line, Bris­tol My­ers Squibb files NDA for Cel­gene/blue­bird CAR-T

A new CAR-T ther­a­py may be com­ing. And maybe $9 per share for Cel­gene in­vestors, too.

Bris­tol My­ers Squibb an­nounced they sub­mit­ted an NDA for the mul­ti­ple myelo­ma “ide-cel” CAR-T ther­a­py Cel­gene de­vel­oped in part­ner­ship with blue­bird. The ther­a­py is one of the three that has to be ap­proved to un­lock the BMS-Cel­gene con­tin­gent val­ue agree­ment that would give share­hold­ers of the ab­sorbed NJ com­pa­ny $9 per share. The first, ozan­i­mod, was ap­proved last week. The sec­ond, a CAR-T treat­ment for non-Hodgkin’s lym­phoma called liso-cel, was sub­mit­ted to the FDA in De­cem­ber.

As Mizuho’s Sal­im Syed point­ed out in a note to in­vestors, the tim­ing is not ran­dom. The March 31 sub­mis­sion means that even un­der stan­dard re­view, the FDA will is­sue a de­ci­sion by March 31, 2021, the dead­line for ap­proval un­der the con­tin­gent val­ue agree­ment.

Strong num­bers bol­ster BMS’s sub­mis­sion. At ASH in De­cem­ber, they re­leased Phase II da­ta show­ing that in 140 pa­tients with re­lapsed and re­frac­to­ry mul­ti­ple myelo­ma, around three quar­ters re­spond­ed and around a third showed a com­plete re­sponse. In the most ef­fec­tive dos­ing arm, 81.5% re­spond­ed and 35.2% went in­to com­plete re­mis­sion. The me­di­an du­ra­tion was 11.3 months.

BMS isn’t alone in the hunt, al­though they are for now the fur­thest along. At the same con­fer­ence, J&J un­veiled a batch of ear­li­er stage da­ta. It was small­er — 29 pa­tients — but every pa­tient re­spond­ed by month 6. Both ther­a­pies tar­get BC­MA, or B-cell mat­u­ra­tion agent, a pro­tein ex­pressed al­most ex­clu­sive­ly on ma­lig­nant cells.

The ther­a­py, though, comes with the same po­tent side ef­fects as pre­vi­ous CAR-T treat­ments. Cy­tokine re­lease syn­drome, the po­ten­tial­ly dead­ly side ef­fect in which ac­ti­vat­ed white blood cells send the im­mune sys­tem in­to in­flam­ma­to­ry over­drive, oc­curred in 83% of pa­tients in the ide-cel tri­al. How­ev­er, on­ly 5.5% were clas­si­fied as “se­vere.”

Liso-cel and ide-cel would be the third and fourth CAR-T treat­ments ap­proved since No­var­tis’s Kym­ri­ah and Gilead/Kite Phar­ma’s Yescar­ta and were ap­proved in 2017, al­though a sec­ond CAR–T ther­a­py from Kite Phar­ma al­so cur­rent­ly sits be­fore the FDA.

Af­ter the ASH da­ta, an­a­lysts ex­pect both BMS CAR-Ts to clear the fin­ish line, al­though Syed raised ques­tions about whether, amid Covid-19, the com­pa­ny’s cell ther­a­py man­u­fac­tur­ing fa­cil­i­ty in Wash­ing­ton state had been and would be able to be FDA-in­spect­ed. But not­ing that liso-cel al­ready was sub­mit­ted in De­cem­ber and giv­en pri­or­i­ty re­view in Feb­ru­ary, he gave a 90% chance of ide-cel ap­proval and wrote that “we would ar­gue there is a de­cent chance here the plant has al­ready been in­spect­ed and the con­cerns may be overblown.”

How long the ther­a­py re­mains a top op­tion for mul­ti­ple myelo­ma pa­tients is a dif­fer­ent ques­tion. At ASH, BMS al­so showed da­ta for CC-93269, a BC­MA-tar­get­ing bis­pe­cif­ic an­ti­body that put up “crazy num­bers,” as Baird an­a­lyst Bri­an Sko­r­ney put it: 89% ORR, 33% CR. Re­gen­eron al­so showed off its BC­MA bis­pe­cif­ic. Safer and dra­mat­i­cal­ly sim­pler to ad­min­is­ter, these an­ti­bod­ies may ul­ti­mate­ly win out, Sko­r­ney said.

”We think the true com­mer­cial op­por­tu­ni­ty is re­al­ized by the most safe­ly com­bin­able as­set with gener­ic Revlim­id/Vel­cade,” he wrote. “As such, al­though fur­ther be­hind than BC­MA-CART, we see these drugs leap­ing ahead in the treat­ment par­a­digm, bar­ring ma­jor safe­ty is­sues.”

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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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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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

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

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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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A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.