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.

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Suf­fer­ing No­var­tis part­ner Cona­tus is pack­ing it in on NASH af­ter a se­ries of un­for­tu­nate tri­al events

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.

Ab­b­Vie gets a green light to re­sume re­cruit­ing pa­tients for one myelo­ma study — but Ven­clex­ta re­mains un­der a cloud

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.

Bris­tol-My­ers star Op­di­vo fails sur­vival test in a matchup with Nex­avar aimed at shak­ing up the big HCC mar­ket

Bris­tol-My­ers Squibb has suf­fered an­oth­er painful set­back in its years-long quest to ex­pand the reach of Op­di­vo. The phar­ma gi­ant this morn­ing not­ed that their Check­mate-459 study com­par­ing Op­di­vo with Bay­er’s Nex­avar in front­line cas­es of he­pa­to­cel­lu­lar car­ci­no­ma — the most com­mon form of liv­er can­cer — failed to hit the pri­ma­ry end­point on over­all sur­vival.

This was a sig­nif­i­cant mile­stone in Bris­tol-My­ers’ tal­ly of PD-1 cat­a­lysts this year. Nex­avar (so­rafenib) has been the stan­dard of care in front­line HCC for the past decade, though Op­di­vo has been mak­ing head­way in sec­ond-line HCC cas­es, where it’s go­ing toe-to-toe with Bay­er’s Sti­var­ga (re­go­rafenib) af­ter re­cent ap­provals shook up the mar­ket.

Dean Hum. Nasdaq via YouTube

Gen­fit goes to Chi­na with a deal worth up to $228M for NASH drug

Fresh off the high of its Nas­daq IPO de­but, and the low of com­par­isons to Cymabay — whose NASH drug re­cent­ly stum­bled — Gen­fit on Mon­day un­veiled an up to $228 mil­lion deal with transpa­cif­ic biotech Terns Phar­ma­ceu­ti­cals to de­vel­op its flag­ship ex­per­i­men­tal liv­er drug — elafi­bra­nor — in Greater Chi­na.

The deal comes more than a week af­ter Gen­fit $GN­FT is­sued a fiery de­fense of its dual PPAR ag­o­nist elafi­bra­nor, when com­peti­tor Cymabay’s PPARδ ag­o­nist, se­ladel­par, fiz­zled in a snap­shot of da­ta from an on­go­ing mid-stage tri­al. The main goal at the end of 12 weeks was for se­ladel­par to in­duce a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in liv­er fat con­tent, but da­ta showed that pa­tients on the place­bo ac­tu­al­ly per­formed bet­ter.