In­ves­ti­ga­tors de­tail some of the hopes – and fears – be­hind Cel­lec­tis' off-the-shelf CAR-T

Cel­lec­tis is right at the cut­ting edge of de­vel­op­ing a new, off-the-shelf CAR-T that hopes to dis­rupt a mar­ket that has yet to be cre­at­ed. So far, about the on­ly tan­gi­ble hu­man ev­i­dence of its po­ten­tial dura­bil­i­ty has been seen in a pair of in­fants who were giv­en Cel­lec­tis’ $CLLS lead ther­a­py to ward off lethal cas­es of leukemia. And while an up­date on their con­di­tion shows that the two young pa­tients are still alive and do­ing well months af­ter treat­ment, one had to fight off graft-vs-host dis­ease trig­gered by the ther­a­peu­tic, a key sign of tox­i­c­i­ty that in­ves­ti­ga­tors are watch­ing close­ly.

Writ­ing in Sci­ence Trans­la­tion­al Med­i­cine, the in­ves­ti­ga­tors han­dling these two cas­es of­fered de­tails on their pi­o­neer­ing cas­es, us­ing TAL­EN en­gi­neered cells to di­rect­ly tar­get B cell leukemia. Both chil­dren had re­lapsed, drug-re­sis­tant cas­es of B cell acute lym­phoblas­tic leukemia.

In the first case, the in­ves­ti­ga­tors were al­lowed to use Cel­lec­tis’ off-the-shelf treat­ment on an 11-month-old child. Grade 2 skin GVHD de­vel­oped but was suc­cess­ful­ly treat­ed with steroids, and 18 months lat­er the in­fant re­mains in re­mis­sion.

An­oth­er ba­by girl was treat­ed in much the same way af­ter re­laps­ing in late 2015 and re­spond­ed well, with­out GVHD. An ag­gres­sive ther­a­peu­tic in­ter­ven­tion leaves her “clin­i­cal­ly well at home.”

The sur­vival times for these girls is not new. The da­ta were first dis­cussed at a sci­en­tif­ic meet­ing in De­cem­ber, along with oth­er cas­es in­volv­ing short­er pe­ri­ods of post-treat­ment sur­vival times. (Dura­bil­i­ty will be a key fo­cus.) But this new re­port does pro­vide more in­sights in­to the ther­a­py, un­der­scor­ing Cel­lec­tis’ ra­tio­nale that the lead CAR-Ts now in late-stage de­vel­op­ment – which ex­tract T cells from pa­tients, en­gi­neer them and then in­fuse them back in­to pa­tients – leave a lot to be de­sired. In some cas­es, the in­ves­ti­ga­tors note, pa­tients aren’t go­ing to have enough T cells left to reengi­neer in­to ther­a­pies, mak­ing an off-the-shelf al­ter­na­tive us­ing cells from healthy donors es­sen­tial.

The threat of GVHD, though, once again spot­lights the se­vere ad­verse events that have been tied to these ther­a­pies. Juno’s $JUNO CAR-Ts have been tied to sev­en deaths from cere­bral ede­ma, in­clud­ing a tal­ly of five for their lead pro­gram, which is now in lim­bo. Kite $KITE and No­var­tis $NVS, mean­while, are rac­ing to win the first ap­provals in the field.

Cel­lec­tis isn’t in the frank ranks of that race. But it’s an­gling to come in not far be­hind, if it can es­cape a cat­a­stro­phe.

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

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.

Bull­ish biotech mar­ket pro­pels Pli­ant to $144M IPO — as No­var­tis pro­vides a $10M boost

After pharma partner Novartis boosted its IPO with a $10 million private placement, Pliant Therapeutics has wrapped its journey to the Nasdaq on a high note.

Pliant had penciled in a $86 million raise back in May. But as has become the norm in recent months, that initial number has turned out to be a mere placeholder, making way for the final haul of $144 million.

The South San Francisco biotech did so by pricing at $16, the high end of the range, while bringing the number of shares offered up to 9 million.