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.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

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