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.”

Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Pascal Soriot, AstraZeneca CEO (AP Images)

Pas­cal So­ri­ot cash­es in As­traZeneca’s chips on Mod­er­na for $1.2B cash in­jec­tion

While still working to prove its own Covid-19 vaccine, AstraZeneca has reportedly capitalized on the success of another.

The company has sold off its 7.7% stake in Moderna and turned it into $1.2 billion in cash, according to the Times, beefing up the reserves just as Pascal Soriot is wrapping up his $39 billion acquisition of Alexion and its rare disease pipeline.

AstraZeneca’s stock sale follows a similar move by Merck in December. But like its pharma brethren, the British giant is keeping its R&D collaborations with Moderna.

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CEO Marco Taglietti (Scynexis)

'N­ev­er been more ur­gent:' Scynex­is looks to tack­le su­per­bug cri­sis with late-stage read­out for an­ti­fun­gal hope­ful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

“The need for new anti-infectives capable of fighting the most resistant pathogens has never been more urgent as we confront the ongoing COVID-19 global pandemic,” CEO Marco Taglietti said in a statement.

A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Takeda spun out a pipeline of experimental psychiatry drugs to Neurocrine in a $2 billion deal amid a post-merger shakeout, R&D chief Andy Plump described the therapies as “very interesting but still difficult.”

On Tuesday, we got some idea of how difficult.

San Diego-based Neurocrine revealed that one of the three spotlight clinical programs they’d acquired failed the primary endpoint in a Phase II trial for schizophrenia, registering a negative outcome on the change from baseline in the positive and negative syndrome scale/negative symptom factor score (PANSS NSFS).

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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