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.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Charlie Fuchs, Roche and Genentech global head of product development for oncology and hematology (Yale Cancer Center)

Yale can­cer spe­cial­ist Char­lie Fuchs tapped as new glob­al de­vel­op­ment chief for Roche/Genen­tech

Roche and their big sub Genentech have just recruited a top cancer specialist at Yale to head up global product development in oncology and hematology.

I just got word that the pharma giant, which leads one of the most active cancer research operations in the world, recruited Charlie Fuchs, director of the Yale Cancer Center and physician-in-chief of Smilow Cancer Hospital. He’ll join the global operation March 1 and will be based in South San Francisco, where Genentech is based.

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Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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Glax­o­SmithK­line scraps a LAG3 study, mark­ing an­oth­er fail­ure for the pipeline af­ter a crit­i­cal set­back

Another gap has appeared in GlaxoSmithKline’s pipeline.

Friday morning the Australian biotech Immutep put out word that Hal Barron’s R&D group at GSK had decided to scrap a Phase II proof-of-concept study in ulcerative colitis for their anti-LAG3 therapy GSK2831781. According to the biotech, the program didn’t survive an interim review.

The trial was stopped by GSK based on the assessment of clinical data as part of a planned interim analysis conducted in consultation with the trial’s Data Review Committee. GSK is conducting further reporting, assessment and analyses of the efficacy and safety data and evaluating the biology to determine next steps for the GSK2831781 development program.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.