Kite adds promis­ing leukemia re­spons­es in small CAR-T stud­ies, then fires the start­ing gun on its rolling sub­mis­sion

Kite Phar­ma­ceu­ti­cals $KITE has lined up a fresh batch of pos­i­tive ef­fi­ca­cy da­ta for its lead CAR-T for ad­vanced cas­es of acute lym­phoblas­tic leukemia at ASH. And then lat­er Sun­day evening the biotech added that it has launched its rolling sub­mis­sion for a first ap­proval.

David Chang, Kite

In­ves­ti­ga­tors turned up at the big ASH meet­ing to say that the drug spurred a com­plete re­mis­sion in 9 of 11 evalu­able pa­tients — 82% — en­rolled in the ZU­MA-3 and ZU­MA-4 Phase I stud­ies for ALL in a pre­lim­i­nary re­view.

But the treat­ment was not with­out dan­ger. Five of the 13 had se­ri­ous bouts of cy­tokine re­lease syn­drome as well as 5 who had grade three or above cas­es of neu­ro­log­i­cal events. There were no cas­es of cere­bral ede­ma, which have be­dev­iled ri­val Juno, but one of the pa­tients in the tri­als died from CRS.

Kite had long hoped to file its first ap­pli­ca­tion for KTE-C19 be­fore the end of the year, but was forced to de­lay un­til Q1 2017 af­ter some in­ter­ac­tions with the FDA, pre­sum­ably so it would have time to in­clude da­ta on the 6-month re­spons­es of pa­tients in ZU­MA-1. Some an­a­lysts have been skep­ti­cal of the dura­bil­i­ty of the treat­ment af­ter see­ing the ef­fect de­cline over a few months of treat­ment, but com­pa­ny ex­ecs ex­pect to see that re­sponse lev­el plateau at a sig­nif­i­cant lev­el.

Soon af­ter Kite post­ed the lat­est da­ta on their ther­a­py Sun­day, the com­pa­ny an­nounced that it had launched the rolling sub­mis­sion and would get it com­plet­ed by the end of the first quar­ter of 2017. But No­var­tis now may have an edge in fil­ing first, and look­ing for the first ever ap­proval of a CAR-T ther­a­py.

The drug re­mains one of the lead­ing CAR-T ther­a­pies head­ing to the FDA. No­var­tis is now hus­tling along its own CAR-T af­ter reg­is­ter­ing an im­pres­sive 82% com­plete re­mis­sion rate among the 50 chil­dren and young adults with B-cell acute lym­phoblas­tic leukemia.

“In the Phase I por­tion of these stud­ies, we on­ly en­rolled pa­tients with high dis­ease bur­den to rig­or­ous­ly eval­u­ate safe­ty and ef­fi­ca­cy of KTE-C19,” said Kite R&D di­rec­tor David Chang. “We look for­ward to ini­ti­at­ing the Phase II por­tions of these stud­ies in 2017.”

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.