Kite CEO Arie Bellde­grun preps for a loom­ing date with the FDA as he tack­les first-mover ad­van­tage in CAR-T

New York — Arie Bellde­grun has a date with the FDA “with­in a month” that will dic­tate just how fast Kite Phar­ma $KITE will be able to file for an ap­proval of its lead CAR-T. But as of now, the biotech has ear­ly da­ta ready to go to meet a dead­line the CEO set for him­self in 2014: To file for an ap­proval of KTE-C19 be­fore the end of this year.

In a sit­down short­ly af­ter the end of Kite’s an­nu­al R&D meet­ing with an­a­lysts in Man­hat­tan, Bellde­grun re­it­er­at­ed one of the key themes dur­ing the ses­sion. He wants to nail down the first-mover ad­van­tage in CAR-T, and he’s ready to go with pos­i­tive three-month re­sults on the ther­a­py for ad­vanced dif­fuse large B-cell lym­phoma (DL­B­CL).

Their up­com­ing pre-ap­pli­ca­tion meet­ing with reg­u­la­tors will pro­vide the clar­i­ty Kite is look­ing for on sub­mit­ting the da­ta. And even if the FDA de­cides that it wants to wait for 6-month re­sults, he adds, that will be in hand in Feb­ru­ary, with a sub­mis­sion ready to go be­fore the end of Q3.

If all goes ac­cord­ing to plan with the ac­cel­er­at­ed timetable, Bellde­grun reck­ons that he has “a 12-month lead for sure” over the com­pe­ti­tion, though he’s al­so quick to add that the gi­ant No­var­tis — which re­cent­ly stunned out­side ob­servers by dis­solv­ing its cell ther­a­py unit — can be hard to read. That’s enough time to play a lead­ing role with pay­ers as well as the physi­cians at dozens of sites where KTE-C19 would be ad­min­is­tered to a mar­ket of up to about 7,000 treat­ment-re­sis­tant pa­tients like­ly to qual­i­fy for ther­a­py. And he is keen to play the lead­ing role in cre­at­ing this mar­ket, ahead of Juno and No­var­tis.

Much of the pre­sen­ta­tion Tues­day was de­vot­ed to demon­strat­ing that Kite is ready to seize the mo­ment. The heads of re­search, man­u­fac­tur­ing and com­mer­cial op­er­a­tions all took their turns on stage to as­sert their readi­ness to roll on a quick green light from the FDA. And right be­hind the pi­o­neers, Kite has lined up a slate of new drugs as well as new col­lab­o­ra­tions on next-gen treat­ments that are de­signed to keep the lead, adding new safe­ty switch­es and bet­ter tech­nolo­gies aimed at keep­ing the lead when the fo­cus shifts to CAR-T 2.0.

Kite CMO and R&D chief David Chang ham­mered that next-gen point by un­veil­ing four new near-term clin­i­cal pro­grams, spot­light­ing KITE-796 for acute myeloid leukemia and KITE-585 for mul­ti­ple myelo­ma, which tar­gets BC­MA. Those drugs al­so flag an evo­lu­tion at the biotech, as they look be­yond the close part­ner­ship they have at the NCI and look to de­vel­op in-house treat­ments which re­ly on new tech­nolo­gies de­signed to im­prove safe­ty and ef­fi­ca­cy.

Kite re­ceived mixed re­views for its 3-month KTE-C19 da­ta when it was re­leased a few weeks ago. The three-month snap­shot was clear­ly pos­i­tive and the FDA has proved ready and will­ing to push out new can­cer drugs at a record pace, op­ti­mists not­ed, well ahead of the late-stage da­ta that was once es­sen­tial. But a vo­cal crowd of skep­tics on Twit­ter ze­roed in on the de­clin­ing re­sponse rates tracked among the pa­tients tak­ing KTE-C19, which re­lies on a process in which T cells are ex­tract­ed from pa­tients, reengi­neered to hunt down can­cer cells and then re­in­fused back in­to pa­tients.

The ini­tial over­all re­sponse rates as well as com­plete re­spons­es de­clined from an ini­tial 76% and 47% re­spec­tive­ly to 39% and 33% at three months, the crit­ics hoot­ed. And the fact that CAR-T is still very much a rad­i­cal­ly new ther­a­peu­tic ap­proach to can­cer gives oth­ers pause as well. But Bellde­grun him­self re­mains con­fi­dent that the num­bers will plateau, as they have be­fore, in this pa­tient group, re­tain­ing a promis­ing pro­file for reg­u­la­tors as well as pa­tients. More da­ta is ex­pect­ed at ASH that will al­so help il­lu­mi­nate its ther­a­peu­tic im­pact.

Kite caught a big ad­van­tage in this race in Ju­ly, when the FDA clamped a brief clin­i­cal hold on Juno’s ri­val ther­a­py. And even though the biotech quick­ly had the hold lift­ed af­ter promis­ing to drop flu­dara­bine from the pre­con­di­tion­ing reg­i­men — help­ing to ease con­cerns about the tox­ic re­sponse that killed 4 pa­tients in two dif­fer­ent stud­ies — it’s ap­proval time­line was knocked back from 2017 to 2018, hand­ing Kite a clear lead.

Bellde­grun is do­ing every­thing he can to hang on to that lead, and plans to use every minute to get his com­pa­ny rec­og­nized as the leader in cre­at­ing the mar­ket to come. It’s all up to the FDA now to de­cide just how soon that may hap­pen.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.