Kite scores a land­mark win with promis­ing 6-month lym­phoma da­ta for lead CAR-T

Jeff Wiezorek, Kite

Kite Phar­ma’s close­ly-watched lead CAR-T turned in pos­i­tive six-month re­sults for ag­gres­sive non-Hodgkin lym­phoma, hit­ting a key goal as the biotech rolls up to the FDA with its ap­pli­ca­tion for mar­ket­ing ap­proval.

A group of skep­tics had lashed the com­pa­ny af­ter its three-month re­sults, which demon­strat­ed a clus­ter of re­laps­es and a sharp drop in ob­jec­tive re­sponse rates and com­plete re­spons­es for axi­cab­ta­gene ciloleu­cel (KTE-C19). But CEO Arie Bellde­grun told me last fall that in­ves­ti­ga­tors be­lieved that the six-month re­sults would show that the ef­fi­ca­cy plateaued, demon­strat­ing its dura­bil­i­ty.

And that’s what hap­pened.

At six months, the ORR in dif­fuse large B-cell lym­phoma (DL­B­CL) hit 36%, down on­ly three points from month three. The CR rate was 31%, down two points. Big­ger drops were al­so record­ed for pri­ma­ry me­di­asti­nal B-cell lym­phoma (PM­B­CL) and trans­formed fol­lic­u­lar lym­phoma (TFL) en­rolled in Co­hort 2, but the six-month num­bers post­ed were all clear­ly sta­tis­ti­cal­ly sig­nif­i­cant.

“These re­sults with axi­cab­ta­gene ciloleu­cel are ex­cep­tion­al and sug­gest that more than a third of pa­tients with re­frac­to­ry ag­gres­sive NHL could po­ten­tial­ly be cured af­ter a sin­gle in­fu­sion of axi­cab­ta­gene ciloleu­cel,” said Jeff Wiezorek, MD, Se­nior Vice Pres­i­dent of Clin­i­cal De­vel­op­ment at Kite. “The ZU­MA-1 study was built on a foun­da­tion of sup­port and com­mit­ment from Dr. Steven Rosen­berg and the Na­tion­al Can­cer In­sti­tute and our ZU­MA-1 clin­i­cal tri­al in­ves­ti­ga­tors who be­lieved in the po­ten­tial for CAR-T ther­a­py to change the par­a­digm of can­cer treat­ment.”

Its stock shot up more than 20% this morn­ing.

Kite will now com­plete its rolling sub­mis­sion for this drug next month, be­fore the end of Q1. That leaves it neck-and-neck with No­var­tis, which has its own pi­o­neer­ing ap­pli­ca­tion for its CAR-T head­ed to reg­u­la­tors as well.

The key point to­day is that Kite’s CAR-T looks durable, for now. As Leerink’s Michael Schmidt not­ed: “Im­por­tant­ly, the re­spons­es seem to be durable based on this land­mark analy­sis…”

“Giv­en the da­ta, we see the chances of axi-cel ac­cel­er­at­ed ap­proval as like­ly,” wrote Jef­feries’ Biren Amin.

But some ob­servers were ran­kled by Kite’s claim of a po­ten­tial cure, not­ing that the ther­a­py still has a long way to go be­fore its dura­bil­i­ty can be ful­ly as­sessed. Still, there’s plen­ty enough here for a quick OK.

Arie Bellde­grun, Kite CEO

CAR-T drugs burst on the biotech scene a few years ago as Kite, Juno and No­var­tis all an­gled for the lead spot in search of the first mar­ket­ing ap­proval. These first-gen­er­a­tion drugs ex­tract T cells from pa­tients and then reengi­neer them to at­tack can­cer cells.

It’s been an ar­du­ous and of­ten dan­ger­ous process for the late-stage can­cer pa­tients en­rolled in these stud­ies. Kite notes that the deaths of three pa­tients have been linked to their drug. Juno’s lead pro­gram is still in lim­bo, af­ter it was forced to sus­pend work af­ter a string of deaths caused by brain swelling.

The ad­verse events they record­ed are well known to the field and the agency, Bellde­grun told me last year. And in­ves­ti­ga­tors saw break­through ben­e­fits for their ther­a­py. Based on SCHOL­AR-1 da­ta, these pa­tients would nor­mal­ly ex­pect to see an 8% com­plete re­sponse rate. Kite, he added, al­so proved that it could man­u­fac­ture the per­son­al­ized ther­a­py for 22 cen­ters, most of which had nev­er han­dled the ther­a­py be­fore.

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

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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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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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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New safe­ty da­ta ex­pose po­ten­tial weak­ness as Pfiz­er's abroc­i­tinib takes on Dupix­ent in eczema

Last September, when Pfizer celebrated positive data from a second Phase III study of abrocitinib, many watchers applauded the efficacy but were still waiting to see whether the JAK1 inhibitor is “safe enough to be a formidable competitor to Dupixent,” the clear leader in the atopic dermatitis field. The full slate of safety data are now out and, according to one analyst, the answer is: probably not.