FDA fol­lows through with a ground­break­ing OK for Yescar­ta, Gilead­'s new CAR-T break­through

Just two weeks af­ter Gilead closed on its $12 bil­lion Kite buy­out, the FDA has fol­lowed through with a ground­break­ing ap­proval of Yescar­ta (axi-cel), putting its CAR-T drug neck-and-neck with No­var­tis’ pi­o­neer Kym­ri­ah as the two drugs are prepped for a launch.

In a vir­tu­al heart­beat, Gilead used its con­sid­er­able cash re­serves to reach a deal to buy out Kite and axi-cel 6 weeks ago, ac­quir­ing its new­ly ap­proved CAR-T and all the next-gen tech­nol­o­gy now in the works. The ac­qui­si­tion made Gilead an overnight leader in CAR-T. While beat­en to a his­toric first FDA ap­proval for a CAR-T by an ag­gres­sive group at No­var­tis — ini­tial­ly green-light­ed at the end of Au­gust for pe­di­atric and young adult pa­tients with a form of acute lym­phoblas­tic leukemia — Gilead is re­tain­ing most of the Kite gang and hon­ing its man­u­fac­tur­ing ef­fort, shav­ing off the time it takes to turn around these per­son­al­ized ther­a­pies.

In clas­sic ag­gres­sive form, Gilead came out gun­ning, of­fer­ing the ther­a­py at a price of $373,000 — $102,000 less than its ri­val at No­var­tis. On the oth­er hand, No­var­tis of­fered a val­ue-based deal to a num­ber of pay­ers in which they on­ly get re­im­burse­ment for pa­tients who re­spond to ther­a­py, evening the play­ing field.

Once again, FDA com­mis­sion­er Scott Got­tlieb took the hon­ors in con­grat­u­lat­ing the de­vel­op­ers, as he did when No­var­tis came out in front. And this time, he promised to lend the agency’s help for the rest of the bur­geon­ing cell ther­a­py field. Said Got­tlieb:

“To­day marks an­oth­er mile­stone in the de­vel­op­ment of a whole new sci­en­tif­ic par­a­digm for the treat­ment of se­ri­ous dis­eases. In just sev­er­al decades, gene ther­a­py has gone from be­ing a promis­ing con­cept to a prac­ti­cal so­lu­tion to dead­ly and large­ly un­treat­able forms of can­cer. This ap­proval demon­strates the con­tin­ued mo­men­tum of this promis­ing new area of med­i­cine and we’re com­mit­ted to sup­port­ing and help­ing ex­pe­dite the de­vel­op­ment of these prod­ucts. We will soon re­lease a com­pre­hen­sive pol­i­cy to ad­dress how we plan to sup­port the de­vel­op­ment of cell-based re­gen­er­a­tive med­i­cine. That pol­i­cy will al­so clar­i­fy how we will ap­ply our ex­pe­dit­ed pro­grams to break­through prod­ucts that use CAR-T cells and oth­er gene ther­a­pies. We re­main com­mit­ted to sup­port­ing the ef­fi­cient de­vel­op­ment of safe and ef­fec­tive treat­ments that lever­age these new sci­en­tif­ic plat­forms.”

The CD19 T cell im­munother­a­py — which reengi­neers pa­tient cells in­to a po­tent can­cer ther­a­py — has been ap­proved for adult pa­tients with re­lapsed or re­frac­to­ry large B-cell lym­phoma af­ter two or more lines of sys­temic ther­a­py. That la­bel in­cludes dif­fuse large B-cell lym­phoma (DL­B­CL), pri­ma­ry me­di­asti­nal large B-cell lym­phoma, high-grade B-cell lym­phoma, and DL­B­CL aris­ing from fol­lic­u­lar lym­phoma.

Alessan­dro Ri­va

The R&D work will now be led by Alessan­dro Ri­va, a for­mer No­var­tis sci­en­tist pro­mot­ed yes­ter­day to ex­ec­u­tive vice pres­i­dent in charge of on­col­o­gy R&D, with a seat on the lead­er­ship team.

Left be­hind in the race to CAR-T dom­i­nance, for now at least, is Juno Ther­a­peu­tics. Its ri­val drug was mired down by a lethal tox­i­c­i­ty is­sue that killed 5 pa­tients, forc­ing the biotech to switch over to its num­ber 2 pro­gram.

No­var­tis man­aged to sur­prise quite a few an­a­lysts by peg­ging Kym­ri­ah at $475,000, which is sig­nif­i­cant­ly low­er than the $600,000 max­i­mum pro­vid­ed in some of the spreads. In a sto­ry we pub­lished yes­ter­day, how­ev­er, a num­ber of ex­perts said the full cost of ther­a­py may well end up at $1 mil­lion to $1.5 mil­lion.

The da­ta, though, are jaw drop­ping.

The FDA’s ap­proval comes through on the da­ta for ZU­MA-1, which demon­strat­ed that 72% of the pa­tients in­volved had an ob­jec­tive re­sponse rate to the ther­a­py. And 51% demon­strat­ed a com­plete re­mis­sion, with no sign of the can­cer left.

In a re­cent farewell let­ter to the staff, Kite CEO Arie Bellde­grun summed it up by writ­ing:

In a span of just a few short years, we grew from few­er than 10 em­ploy­ees to al­most 700. The com­pa­ny’s val­ue in­creased 2300% from the time of our IPO to near­ly $12 bil­lion with the ac­qui­si­tion by Gilead Sci­ences. Our clos­ing $180 per share price rep­re­sents not just a 960% ap­pre­ci­a­tion from the IPO price of $17 per share, but the largest ever pre-com­mer­cial bio­phar­ma ac­qui­si­tion.

Now Gilead can see if it’s ag­gres­sive team can make the most of the block­buster peak sales es­ti­mates that have yet to be climbed.

To­day, Bellde­grun had this to say:

“The FDA ap­proval of Yescar­ta is a land­mark for pa­tients with re­lapsed or re­frac­to­ry large B-cell lym­phoma. This ap­proval would not have been pos­si­ble with­out the coura­geous com­mit­ment of pa­tients and clin­i­cians, as well as the on­go­ing ded­i­ca­tion of Kite’s em­ploy­ees. We must al­so rec­og­nize the FDA for their abil­i­ty to em­brace and sup­port trans­for­ma­tion­al new tech­nolo­gies that treat life-threat­en­ing ill­ness­es. We be­lieve this is on­ly the be­gin­ning for CAR T ther­a­pies.”

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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II 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.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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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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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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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

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.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.