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.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Brex­it fears, Wood­ford woes over­shad­owed UK biotech and cut 2019 fi­nanc­ing by al­most half

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