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.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

Daphne Zohar (PureTech)

PureTech bags $200M from sale of Karuna shares — still siz­zling from promis­ing schiz­o­phre­nia da­ta

Cashing in on the exuberance around Karuna Therapeutics and its potential blockbuster CNS drug, PureTech has sold a chunk of the biotech’s shares to Goldman Sachs for $200 million.

Boston-based PureTech had helped Eli Lilly vet Steve Paul launch Karuna and invent its lead program, which combines two old drugs that both act on the muscarinic receptor and balances each other out. Xanomeline, a discard from Lilly, stimulates the M1 and M4 receptors; trospium is an muscarinic receptor antagonist approved to treat overactive bladders.

UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.