Rac­ing past a wound­ed Juno, Kite aims to file lead CAR-T for OK by end of 2016

With Juno Ther­a­peu­tics se­ri­ous­ly de­layed by a brief but painful clin­i­cal hold by the FDA, ri­val Kite Phar­ma out­lined plans to­day to shoot for an ac­cel­er­at­ed ap­proval af­ter it gath­ers the first round of da­ta on 50 pa­tients from its piv­otal study of a ri­val CAR-T, due in just a few months. And com­pa­ny ex­ec­u­tives say their man­u­fac­tur­ing op­er­a­tions will be ready to start pro­duc­ing this ther­a­py in com­mer­cial quan­ti­ties be­fore the end of this year.

If suc­cess­ful, Kite could be the first to the mar­ket, es­pe­cial­ly if the FDA opts to act quick­ly for one of its ‘break­through’ ther­a­pies, which pro­vides for a swift re­view. Juno has al­ready said that it won’t be able to reach the mar­ket be­fore 2018. And No­var­tis has yet to de­tail what kind of time­line it is look­ing at.

Their SEC fil­ing in­cludes this state­ment:

“We have com­plet­ed en­roll­ment of all 72 pa­tients in the DL­B­CL (dif­fuse large B cell lym­phoma) co­hort and 20 pa­tients in the PM­B­CL (pri­ma­ry me­di­asti­nal B cell lym­phoma) and TFL (trans­formed fol­lic­u­lar lym­phoma) co­hort in ZU­MA-1. We plan to open an ad­di­tion­al co­hort in ZU­MA-1 to al­low us to con­tin­ue to dose pa­tients with KTE-C19 in the Unit­ed States and to ex­pand the clin­i­cal tri­al to Eu­rope. We plan to re­port ZU­MA-1 top-line da­ta from the first 50 DL­B­CL pa­tients with at least three-months of fol­low-up by the end of the third quar­ter of 2016. If we be­lieve the da­ta are com­pelling, we plan to dis­cuss with the FDA the fil­ing of a Bi­o­log­ics Li­cense Ap­pli­ca­tion, or BLA, for ac­cel­er­at­ed ap­proval of KTE-C19 as a treat­ment for pa­tients with re­frac­to­ry DL­B­CL, PM­B­CL and TFL. Sub­ject to the in­ter­im re­sults and dis­cus­sions with the FDA, we plan to sub­mit the BLA at the end of 2016. If ap­proved, we plan to com­mer­cial­ly launch KTE-C19 in 2017.  We plan to re­port da­ta from ZU­MA-2 and the Phase 2 por­tions of ZU­MA-3 and ZU­MA-4 in 2017. If we be­lieve the da­ta are com­pelling, we plan to pur­sue FDA ap­proval for these ad­di­tion­al in­di­ca­tions.”

The race be­tween Kite and Juno for a pi­o­neer­ing FDA OK has be­come one of the most close­ly fol­lowed ri­val­ries in R&D. Both had been shoot­ing for a 2017 launch. But just days ago Juno ac­knowl­edged in its quar­ter­ly re­view that a 6-day hold on JCAR015 would post­pone any com­mer­cial ef­fort un­til 2018.

An ag­gres­sive Kite is clear­ly tak­ing ad­van­tage of every an­gle it can think of. CAR-T ther­a­pies re­ly on cells ex­tract­ed from pa­tients which are then reengi­neered to in­clude a chimeric anti­gen re­cep­tor that can hunt down can­cer cells.

Juno has said that its prob­lems with JCAR015 were trig­gered by the ad­di­tion of flu­dara­bine to the reg­i­men used to prep pa­tients to bet­ter re­spond to their drug. The biotech fin­gered flu­dara­bine for the deaths of four pa­tients, which spurred the hold. No­tably, while Juno was grap­pling with the FDA, Kite put out an an­nounce­ment that it was stay­ing on track with its lead pro­gram.

Kite CEO Arie Bellde­grun

In a call with an­a­lysts Mon­day evening, Kite CEO Arie Bellde­grun al­so said that while in­ves­ti­ga­tors are us­ing flu­dara­bine in their pre­con­di­tion­ing reg­i­men, they have yet to see any of the un­usu­al­ly lethal ad­verse events with neu­ro­tox­i­c­i­ty that tripped up Juno. “I have to say that ad­verse event pro­file has been very much on par with what we have been pre­dict­ing based on the ear­li­er stud­ies,” the CEO told an­a­lysts.

Less than two months ago, Kite held a rib­bon cut­ting cer­e­mo­ny for its new man­u­fac­tur­ing fa­cil­i­ty, a 43,500-square-foot plant that will be used to make its per­son­al­ized KTE-C19.

“Our com­mer­cial fa­cil­i­ty will have the ca­pac­i­ty to pro­duce up to 5,000 pa­tient ther­a­pies per year and we ex­pect it to be op­er­a­tional in pro­duc­ing clin­i­cal ma­te­ri­als by year-end,” not­ed Bellde­grun. “Over­all, we have con­tin­u­ous­ly been op­ti­miz­ing key as­pect of our man­u­fac­tur­ing, sup­ply chain, and qual­i­ty con­trol and pos­sess a pro­pri­etary process that dra­mat­i­cal­ly re­duces the time to ap­prox­i­mate­ly 14 days for when a pa­tients ma­te­r­i­al are shift to our fa­cil­i­ty to when the en­gi­neered T-cells are re­leased to the pa­tient. This is one of the fastest rates in the in­dus­try.”

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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Cue the M&A chat­ter: UniQure is scout­ing for a buy­out deal as gene ther­a­py field siz­zles — re­port

All the en­thu­si­asm that’s been whipped up in the gene ther­a­py field this past year has helped stoke the ru­mor mill about all sorts of pos­si­bil­i­ties for uniQure $QURE, which has seen a quick run-up on its share price. And now the biotech’s back­ers are get­ting a big boost from Bloomberg to keep the run go­ing.

There’s no deal to re­port, but sources are telling the busi­ness news ser­vice that uniQure has brought in ad­vis­ers to see what might be done — in­clud­ing a sale — with the phar­ma gi­ants now prowl­ing the clin­i­cal play­ers for part­ners and ac­qui­si­tions.

'We kept at it': Jef­frey Blue­stone plots late-stage come­back af­ter teplizum­ab shown to de­lay type 1 di­a­betes

Late-stage da­ta pre­sent­ed at the Amer­i­can Di­a­betes As­so­ci­a­tion an­nu­al meet­ing in 2010 pushed Eli Lil­ly to put a crimp on teplizum­ab as the phar­ma gi­ant found it un­able to re­set the clock on new­ly di­ag­nosed type 1 di­a­betes. At the same con­fer­ence but in dif­fer­ent hands nine years lat­er, the drug is mak­ing a crit­i­cal come­back by scor­ing suc­cess in an­oth­er niche: de­lay­ing the on­set of the dis­ease.

In a Phase II tri­al with 76 high-risk in­di­vid­u­als — rel­a­tives of pa­tients with type 1 di­a­betes who have di­a­betes-re­lat­ed au­toan­ti­bod­ies in their bod­ies — teplizum­ab al­most dou­bled the me­di­an time of di­ag­no­sis com­pared to place­bo (48.4 months ver­sus 24.4 months). The haz­ard ra­tio for di­ag­no­sis was 0.41 (p=0.006).