Christi Shaw, JPM19 (Credit: Jeff Rumans)

Gilead­'s Kite keeps adding to CAR-T Yescar­ta's bag of tricks with FDA ap­proval in fol­lic­u­lar lym­phoma — a first

Sev­er­al months af­ter cre­at­ing the first com­mer­cial CAR-T port­fo­lio with the FDA’s nod for Tecar­tus, Gilead’s Kite has racked up a sought-af­ter new in­di­ca­tion for its flag­ship cell ther­a­py Yescar­ta.

The FDA on Fri­day grant­ed Yescar­ta — al­so known as axi­cab­ta­gene ciloleu­cel — an ac­cel­er­at­ed ap­proval as a third-line ther­a­py for adults with re­lapsed or re­frac­to­ry fol­lic­u­lar lym­phoma (FL).

The de­ci­sion was based on the Phase II ZU­MA-5 tri­al, an open-la­bel study in which 146  in­do­lent non-Hodgkin’s lym­phoma pa­tients re­ceived a sin­gle in­fu­sion of Yescar­ta af­ter two or more lines of treat­ment — in­clud­ing 124 with FL and 22 with mar­gin­al zone lym­phoma.

Of the FL pa­tients, 91% pa­tients re­spond­ed to Yescar­ta, 60% of whom achieved a com­plete re­mis­sion, ac­cord­ing to Kite. And 13 of the 25 pa­tients who achieved a par­tial re­mis­sion met the imag­ing cri­te­ria for a com­plete re­mis­sion, but hadn’t been con­firmed by a neg­a­tive bone mar­row biop­sy af­ter treat­ment.

The OK came right on time for Yescar­ta’s PDU­FA date, mak­ing it the first CAR-T ap­proved for FL. Gilead’s stock $GILD was up 2.2% ear­ly Mon­day morn­ing, pric­ing at $64.62 a share.

Reg­u­la­tors did, how­ev­er, slap a boxed warn­ing on the la­bel for cy­tokine re­lease syn­drome and neu­ro­log­ic tox­i­c­i­ties. In a safe­ty analy­sis of the 146 pa­tients, Grade 3 or high­er CRS and neu­ro­log­ic tox­i­c­i­ties oc­curred in 8% and 21%, re­spec­tive­ly.

At ASH20, Kite said ad­verse events of any grade oc­curred in 99% of pa­tients, with Grade 3 or high­er events oc­cur­ring in 86%. The most com­mon were neu­trope­nia (33%), de­creased neu­trophil count (27%), and ane­mia (23%), the com­pa­ny said.

Last month, Kite tapped Take­da vet Frank Neu­mann to head glob­al clin­i­cal de­vel­op­ment, re­plac­ing Ken Takeshi­ta who’s jump­ing to Dai­ichi Sankyo on April 1.

“Ad­vanc­ing CAR T ther­a­pies for pa­tients across lym­phomas re­mains a cor­ner­stone of our cell ther­a­py de­vel­op­ment pro­gram, and we are ex­cit­ed about the po­ten­tial of Yescar­ta for pa­tients with in­do­lent fol­lic­u­lar lym­phoma,” Kite CEO Christi Shaw said in a state­ment.

FL is the most com­mon form of in­do­lent lym­phoma, with a five-year sur­vival rate of on­ly 20% for pa­tients in the third line. Pa­tients with FL have ma­lig­nant tu­mors that grow slow­ly and can be­come more ag­gres­sive over time.

“Once a fol­lic­u­lar lym­phoma pa­tient’s dis­ease re­laps­es, the du­ra­tion of re­sponse to care short­ens with each round of ther­a­py,” said Caron Ja­cob­son, med­ical di­rec­tor of the Dana-Far­ber Can­cer In­sti­tute’s Im­mune Ef­fec­tor Cell Ther­a­py Pro­gram and as­sis­tant pro­fes­sor at Har­vard Med­ical School.

Gilead be­came an overnight CAR-T leader when it bought Kite for $12 bil­lion back in 2017, snatch­ing up clin­i­cal-stage Yescar­ta in the process. It nabbed a quick OK in re­lapsed or re­frac­to­ry large B-cell lym­phoma two weeks lat­er — not long af­ter No­var­tis’ Kym­ri­ah be­came the world’s first ap­proved CAR-T ther­a­py. This past Ju­ly, Kite nabbed its sec­ond ap­proval with Tecar­tus for treat­ment for re­lapsed or re­frac­to­ry man­tle cell lym­phoma.

While Yescar­ta was first out the gate in FL, Kyr­mi­ah isn’t far be­hind. No­var­tis said back in Au­gust that it got the in­ter­im re­sults it was look­ing for from a Phase II tri­al in FL, and an­nounced plans to file with the FDA in 2021.

Up­on read­ing out the ZU­MA-5 re­sults at ASH, Kite’s VP and head of med­ical af­fairs Ibrahim El-Houssieny said Gilead was al­ready talk­ing about de­vel­op­ing Yescar­ta for ear­li­er lines of ther­a­py.

“We are hav­ing these dis­cus­sions right now… about ear­li­er lines of ther­a­py and whether that would re­quire a Phase III or a Phase II,” he told End­points News at the time. “That’s still in dis­cus­sion. We haven’t yet reached a fi­nal con­clu­sion on the study de­sign but it’s some­thing that’s in the plan­ning,” he said.

Spe­cial re­port: Meet 20 ex­tra­or­di­nary women who are su­per­charg­ing bio­phar­ma R&D

Even though many biopharma leaders have come together in recent years to address its gender gap, the consensus is clear: We still have a long way to go.

Companies this year were 2.5 times more likely than last year to have a diversity and inclusion program in place, according to a recent BIO survey, but women are still largely absent from executive roles. Getting women to enter the industry isn’t the problem — studies show that they represent just under half of all biotech employees around the world. But climbing through the ranks can be challenging, as women still report facing stereotypes, and, unfortunately, harassment.

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Op­ti­miz­ing Oral Drug De­liv­ery us­ing Zy­dis® Oral­ly Dis­in­te­grat­ing Tablet Tech­nol­o­gy to Ad­dress Pa­tient Chal­lenges

KEY POINTS

Patients prefer oral dosing, but swallowing tablets can be a challenge for many patients.
The Zydis® orally disintegrating tablet (ODT) platform addresses challenges associated with oral dosing, expanding benefits for patients and options for healthcare providers.
A strong growth trajectory is expected for ODTs given therapeutic innovation and continued technology development.

Many patients prefer conventional tablets for the administration of medications, but some geriatric and pediatric patients and those with altered mental status and physical impairments find swallowing tablets to be difficult. Orally disintegrating tablets (ODTs), which dissolve completely without chewing or sucking, offer a patient-friendly dosage form for the administration of small-molecule drugs, peptides and proteins. With the potential for multiple sites of drug absorption, often faster onset action for the active pharmaceutical ingredient (API), and potentially greater bioavailability, ODTs are an attractive option for drug developers considering first-to-market formulations or product line extensions of existing drugs with compatible API. In this report, we look at how innovation in the industry-leading Zydis ODT platform is expanding oral formulation options and bringing benefits to patients.

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Geoffrey Porges (SVB Leerink)

The 2022 wave com­ing? Top an­a­lyst says Big Phar­ma will have more than $1T avail­able to sat­is­fy its grow­ing ap­petite for biotech M&A

All through this year you could practically feel the frustration of the biotech investor class as M&A activity continued to drag behind expectations — or desires. Buyouts of public companies provide the essential juice for keeping stocks lively, and there’s been a notable lack of juice in 2021.

So is all that about to change, big time?

SVB Leerink’s Geoffrey Porges, a longtime student of biotech M&A, thinks so. In a lengthy analysis he put out last week, Porges totted up the cash flow of the major pharmas and determined that there was a good long list of industry buyers who would have around a half trillion dollars of cash to play with in 2022. Leverage that up with added debt and you could get that deal cache to $1.6 trillion.

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Warren Huff, Reata CEO

FDA slams Reata's kid­ney drug as in­ef­fec­tive ahead of ad­comm meet­ing

The FDA on Monday offered an overwhelmingly negative opinion on Reata Pharmaceuticals’ potential drug to slow the loss of kidney function in those with the rare disease Alport syndrome, according to briefing documents released ahead of an advisory committee meeting Wednesday.

The agency’s review team said it “does not believe the submitted data demonstrate that bardoxolone is effective in slowing the loss of kidney function in patients with AS and reducing the risk of progression to kidney failure.” Originally developed as a cancer drug, bardoxolone was later scuttled by Reata almost a decade ago as a chronic kidney disease drug due to deaths in a Phase III trial.

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No­var­tis pe­ti­tions FDA to block 18 gener­ic com­peti­tors for its megablock­buster heart drug un­til 2024

Novartis is looking to milk one of its most prized possessions a little while longer without any generic competition, mostly because of a newly approved use that the Big Pharma won earlier this year for its megablockbuster heart drug Entresto.

In addition to battling (and winning) litigation last month to block a handful of Indian generics from entering the market early alongside Entresto, the company last week petitioned the FDA to try to stop a total of 18 Entresto generics from launching before 2024 at the earliest.

Mar­ket­ingRx Matchup: How Ab­b­Vie and Bio­haven ads rank in head-to-head mi­graine chal­lenge

Are you ready to rumble? DTC brands that is. MarketingRx is launching a new monthly feature today called MarketingRx Matchup. We’re pitting two pharma brands’ DTC advertising in the same therapeutic category against each other to find out what consumers and patients really think.

Market research company Leger is handling the polling and analysis each month, and I’ll be writing up the results — along with my own take — inside MRx on the first Tuesday of the month.

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Wendy Lund, Organon chief communications officer

Q&A: Organon chief com­mu­ni­ca­tions of­fi­cer Wendy Lund talks about the Mer­ck spin­off, women’s health and why it mat­ters

One of Wendy Lund’s earliest jobs was head of marketing at Planned Parenthood. As the youngest person on its management team, she introduced them to emerging new technologies, and in return, she learned the importance of fighting for what you believe in.

Now as chief communications officer at Organon, the women’s health company recently spun off by Merck, Lund is keeping that point top of mind. That’s in part because women’s health hasn’t been a spotlight therapy area for Big Pharma in years. Several companies have spun off, sold or at least considered selling women’s health assets to focus on “core” products.

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Mar­ket­ingRx roundup: Pfiz­er re-ups pneu­mo­nia ads as Mer­ck threat looms; Re­al Chem­istry founder CEO Jim Weiss steps back

Every autumn, leaves fall from the trees and people start holiday shopping – and for the last few years Pfizer debuts a new “Know Pneumonia” awareness TV ad. This year the commercial, launched a week ago, features different people who talk about why they got vaccinated against pneumococcal pneumonia. Actors portray a young female firefighter with asthma, a mechanic with heart disease and an older woman with her grandchild. A Pfizer spokesperson declined comment on the latest iteration of the long-running campaign.

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Mer­ck pumps the brakes on two more PhI­II tri­als for its lead an­ti-HIV drug

After trial investigators flagged a drop in immune cell counts that an external committee determined was related to treatment last month, Merck has been pausing HIV-related Phase II and III trials ever since.

On Monday, the biopharma company announced it’s pausing enrollment in two of its Phase III trials evaluating its leading anti-HIV drug candidate, which is the once-monthly, oral islatravir.

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