Kite grabs a stem cell bi­ol­o­gy ‘break­through’ in race for off-the-shelf T cell ther­a­pies

Look­ing for an edge in the race to de­vel­op block­buster off-the-shelf cell ther­a­pies to fight can­cer, Kite Ther­a­peu­tics has grabbed a li­cense for new T cell tech from UCLA that the biotech be­lieves can play a break­through role in the field of al­lo­gene­ic drugs.

Kite’s rep in R&D has been based on the first gen­er­a­tion of CAR-T drugs that ex­tracts cells from pa­tients and then cus­tomizes them with chimeric anti­gen re­cep­tors. Once in­fused back in­to pa­tients, they’re de­signed to track down and elim­i­nate can­cer cells. And Kite has one of the most ad­vanced pro­grams now in a piv­otal study.

But be­hind the first-gen drugs, Cel­lec­tis and oth­ers have been work­ing on al­lo­gene­ic ther­a­pies, adapt­ing a stock­pile of cells to do the same work with­out the ex­pen­sive per­son­al­iza­tion process.

The hunt for that new, more ef­fi­cient cell ther­a­py led Kite to the lab of Gay M. Crooks at  UCLA. Ac­cord­ing to Kite, the plat­form us­es stem cell bi­ol­o­gy to cu­rate an ide­al se­lec­tion of T cells which can then be ge­net­i­cal­ly en­gi­neered to make ef­fec­tive can­cer ther­a­pies.

The first CAR-Ts from Juno, Kite and No­var­tis are all en­ter­ing the last leg of clin­i­cal de­vel­op­ment. But as re­cent deaths in Juno’s JCAR015 study demon­strat­ed, there are still se­ri­ous safe­ty is­sues to con­sid­er as well as lim­i­ta­tions in terms of the type of can­cers that can be treat­ed and the ex­tent of the pa­tients who will ben­e­fit.

“This ATO sys­tem rep­re­sents a sig­nif­i­cant break­through in stem cell bi­ol­o­gy that will dri­ve our long-term strat­e­gy to de­vel­op best-in-class al­lo­gene­ic T-cell ther­a­pies,” said David Chang, M.D., Ph.D., Kite’s R&D chief. “This plat­form pro­vides a re­new­able source of T-cells and can be fur­ther ex­ploit­ed with gene en­gi­neer­ing, in­clud­ing chimeric anti­gen re­cep­tors, T-cell re­cep­tors and oth­er gene mod­i­fi­ca­tions of in­ter­est, to gen­er­ate po­tent T-cell prod­ucts that have the po­ten­tial to be re­sis­tant to re­jec­tion and to bear no risk of graft-ver­sus-host dis­ease.”

There’s no word on terms, but all the lead­ers in this field have been will­ing to pay top dol­lar for new tech.

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

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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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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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.”

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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