Kite Phar­ma hits its Q1 dead­line on fil­ing a BLA for its pi­o­neer­ing CAR-T, posts im­proved da­ta

Kite CEO Arie Bellde­grun

With the clock tick­ing down the fi­nal hours of Q1, Kite Phar­ma­ceu­ti­cals $KITE has kept its pledge to file an ap­pli­ca­tion for its pi­o­neer­ing CAR-T ther­a­py in the first quar­ter. The move puts Kite one step be­hind No­var­tis $NVS, which two days ago an­nounced that it had the FDA’s com­mit­ment for a speedy re­view of its own CAR-T.

The ques­tion now is whether the FDA will give the Kite’s ap­pli­ca­tion a pri­or­i­ty re­view as well, per­haps lin­ing up back-to-back ad­vi­so­ry com­mit­tee meet­ings to ex­am­ine the pros and cons of this new wave of can­cer ther­a­py.

Kite filed the BLA for axi­cab­ta­gene ciloleu­cel (KTE-C19, des­ig­nat­ed as a break­through ther­a­py by the FDA) as a treat­ment for pa­tients with re­lapsed or re­frac­to­ry ag­gres­sive non-Hodgkin lym­phoma who are in­el­i­gi­ble for au­tol­o­gous stem cell trans­plant.

Kite was held up a few months by the need to com­plete 6-month da­ta on axi­cab­ta­gene ciloleu­cel, a goal it ac­com­plished a month ago.

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, which helped qui­et some dogged crit­i­cism of the drug’s abil­i­ty to pro­vide a durable re­sponse.

On Fri­day, Kite al­so up­dat­ed its da­ta at AACR in an oral ple­nary pre­sen­ta­tion. And the num­bers had im­proved with more pa­tients to re­port on:

The ORR in the mITT analy­sis set of 101 pts was 82% (CR 54%, PR 28%), and was con­sis­tent across key co­vari­ates in­clud­ing dis­ease sub­type, re­frac­to­ry sta­tus, stage, and IPI score. At a me­di­an fol­low up of 8.7 m, 44% of pts were in re­sponse and 39% were in CR.

That’s up from 41% and 36% re­port­ed in Feb­ru­ary.

Kite — led by CEO Arie Bellde­grun — and No­var­tis are rac­ing to be right at the fore­front of in­tro­duc­ing per­son­al­ized cell ther­a­pies that reengi­neer pa­tients’ im­mune cells to tar­get can­cer. And it’s of­fered some sig­nif­i­cant re­spons­es for a num­ber of pa­tients with ad­vanced cas­es of blood can­cer.

Now Kite and No­var­tis will try to out­do each oth­er on man­u­fac­tur­ing and de­liv­ery, with the phar­ma gi­ant and the biotech need­ing to show that they can pro­vide quick and re­li­able treat­ment.

That may be a par­tic­u­lar­ly dif­fi­cult ques­tion for No­var­tis. As we re­port­ed Thurs­day af­ter­noon, the phar­ma gi­ant’s head of cell man­u­fac­tur­ing — Karen Walk­er, re­spon­si­ble for CAR-T — is leav­ing the com­pa­ny to­day af­ter a lengthy stint. Her de­par­ture fol­lows the un­ex­pect­ed move at No­var­tis to dis­solve its cell ther­a­py unit last sum­mer, lay­ing off staffers and ab­sorb­ing the group in its on­col­o­gy R&D di­vi­sion.

Walk­er and two oth­er ex­ecs were put in charge of No­var­tis’ ef­forts on CAR-T last fall, af­ter the com­pa­ny chopped up the sep­a­rate cell and gene ther­a­py group that had been cre­at­ed specif­i­cal­ly to dri­ve these new ther­a­pies to com­mer­cial­iza­tion. That move fol­lowed the de­par­ture of Us­man “Oz” Azam, who left No­var­tis in the wake of the re­or­ga­ni­za­tion.

Left on the side­lines to­day: Juno Ther­a­peu­tics. Once a leader in the CAR-T area, Juno was forced to shelve its lead ther­a­py af­ter it killed a string of pa­tients. Two of those pa­tients died af­ter a brief R&D halt or­dered by the FDA. But Juno nev­er was able to firm­ly es­tab­lish why the CAR-T caused lethal cas­es of brain swelling. Now the biotech has moved on to a fol­low-up pro­gram, putting the com­pa­ny well be­hind the fron­trun­ners.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Terrie Curran, Phathom CEO (Credit: Arcutis)

Phath­om's old Take­da drug bests Pre­vacid in a PhI­II GI tri­al. Next stop? The FDA

There’s no time for rest in biopharma — at least not at Phathom Pharmaceuticals. Just over a month after submitting two NDAs for its lead acid-fighter vonoprazan, the biotech is already lining up a third, and collecting an extra $50 million to push things along.

Vonoprazan met its primary non-inferiority endpoints in a Phase III study comparing it to standard-of-care Prevacid in a type of gastroesophageal reflux disease (GERD) called erosive esophagitis (EE). It also proved superior to the popular heartburn drug by multiple measures, including healing rate and maintenance of healing.

Thomas Lingelbach, Valneva CEO

Small biotech says its Covid-19 vac­cine spurs more an­ti­bod­ies than As­traZeneca’s. Will sup­ply deals come now?

In a first, a small runner-up vaccine developer says its own Covid-19 jab has induced “superior neutralizing antibody titer levels” over AstraZeneca’s AZD1222 when pitted head-to-head in a Phase III trial.

That and non-inferiority in seroconversion rate were the co-primary endpoints of the trial, which recruited 4,012 adult volunteers across the UK.

But on the exploratory endpoint of Covid-19 case counts, Valneva notes that both treatment groups saw a similar number of infections.