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.

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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