Bris­tol My­ers Squibb earns long-await­ed FDA nod for liso-cel, join­ing short list of lym­phoma CAR-T win­ners

When Bris­tol My­ers Squibb bought out Cel­gene for $74 bil­lion back in late 2019, one of the pur­port­ed cen­ter­pieces in that deal was liso-cel, which hoped to join a small group of CAR-Ts for non-Hodgkin lym­phoma. Ex­pect­ing an ap­proval in 2020, Bris­tol has had its share of set­backs in get­ting liso-cel across the fin­ish line — but now it fi­nal­ly has the green light.

The FDA on Fri­day ap­proved Bris­tol’s Breyanzi (liso­cab­ta­gene mar­aleu­cel), a CAR-T to treat dif­fuse large B cell lym­phoma in pa­tients who have pre­vi­ous­ly re­ceived two pri­or rounds of sys­temic ther­a­py, the agency said in a re­lease.

The agency based its re­view on piv­otal Phase I da­ta show­ing 54% of pa­tients treat­ed with liso-cel achieve com­plete re­mis­sion. DL­B­CL makes up about 33% of all non-Hodgkin lym­phoma cas­es, the FDA said, of which there are around 77,000 new­ly di­ag­nosed each year.

The one-time ther­a­py will come with a $410,300 whole­sale price, Bris­tol said, say­ing it came up with that fig­ure af­ter con­sid­er­ing “many fac­tors in­clud­ing its med­ical and clin­i­cal val­ue, pa­tient val­ue and so­ci­etal val­ue.”

That’s a sig­nif­i­cant pre­mi­um over Gilead’s Yescar­ta, which launched with a $373,000. Kym­ri­ah al­so launched with a $373,000 WAC for B cell lym­phomas.

With the ap­proval will come a black-box warn­ing la­bel for both cy­tokine re­lease syn­drome, a po­ten­tial­ly fa­tal over­re­ac­tion of the im­mune sys­tem, as well as neu­ro­log­ic tox­i­c­i­ties. Those warn­ings are se­vere enough that the FDA has in­sti­tut­ed a Risk Eval­u­a­tion and Mit­i­ga­tion Strat­e­gy that will re­quire health­care fa­cil­i­ties and physi­cians ad­min­is­ter­ing the ther­a­py to be spe­cial­ly cer­ti­fied to iden­ti­fy and treat CRS and neu­ro tox­i­c­i­ties.

Those warn­ings and ad­min­is­tra­tion re­stric­tions are aren’t unique to liso-cel, how­ev­er. Yescar­ta and Kym­ri­ah, for in­stance, sport the same black box warn­ings, and the un­met clin­i­cal need will like­ly dri­ve up­take in what is an ex­treme­ly dif­fi­cult-to-treat pa­tient pop­u­la­tion.

Liso-cel is now the third CAR-T on the mar­ket for var­i­ous forms of non-Hodgkin lym­phoma, along­side Yescar­ta and Kym­ri­ah. Bris­tol aimed to have the ther­a­py ap­proved late last year, but de­lays from Covid-19 and an ug­ly in­spec­tion re­port at one of the com­pa­ny’s con­tract man­u­fac­tur­ing sites pushed the ap­proval in­to the new year.

Liso-cel was one of a group of pipeline can­di­dates tied to a $9 CVR from Bris­tol’s Cel­gene buy­out in 2019. With the ap­proval de­lay, in­vestors lost out on that pay­day when the year ex­pired — not a ma­jor is­sue for Bris­tol it­self but def­i­nite­ly a headache for the CVR traders.

In De­cem­ber, an FDA in­spec­tion at Lon­za Hous­ton’s plant found a raft of is­sues, in­clud­ing mis­la­bel­ing, prod­ucts for the US and EU drug mar­kets stored in the same bins, “poor­ly main­tained” freez­er units, and ex­pired batch­es of in­gre­di­ents that weren’t prop­er­ly dis­posed of, ac­cord­ing to a Form 483 let­ter pub­lished on­line late last month.

Lon­za, one of a group of con­trac­tors work­ing on liso-cel’s man­u­fac­tur­ing, said de­lays in the FDA’s in­spec­tion sched­ule made it dif­fi­cult to ad­just in time for an ap­proval with­in 2020. Bris­tol pre­vi­ous­ly said it re­spond­ed to the agency’s con­cerns with­in eight days of re­ceiv­ing its let­ter.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs-up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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President Joe Biden signs the Democrats' landmark climate change and health care bill. From L-R: Sen. Joe Manchin (D-WV), Senate Majority Leader Chuck Schumer (D-NY), House Majority Whip James Clyburn (D-SC), Rep. Frank Pallone (D-NJ) and Rep. Kathy Castor (D-FL). (Susan Walsh/AP Images)

Pres­i­dent Biden signs ma­jor drug pric­ing re­forms in­to law: What's com­ing for bio­phar­ma?

President Joe Biden yesterday afternoon signed into law historic, decades-in-the-making new drug pricing reforms as part of a wider reconciliation bill that will likely take a chunk out of biopharma companies’ profits for some blockbusters just prior to generic or biosimilar competition.

The partisan bill (all Democrats in the House and Senate voted for it, and all Republicans voted against it) includes not only Medicare price negotiations — which won’t kick off until 2026, leaving ample time for a legal challenge — but mandatory inflation-related rebates, and a $2,000 annual cap on what seniors’ pay for their prescription drugs.

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James Sabry, Roche global head of pharma partnering

Roche, Genen­tech plunk down $60M up­front to part­ner with Chi­nese phar­ma on PRO­TAC-based prostate can­cer drug

Roche and Genentech are always on the hunt for deals, and on Thursday they found their newest partner.

The pair will team up with the Chinese pharma company Jemincare to push forward a new program for prostate cancer, the companies announced. Roche is ponying up $60 million upfront to get its hands on the candidate and promising up to $590 million in biobucks, plus royalties, down the line.

In return, Genentech will get a worldwide license to develop the program, known as JMKX002992, and bring it to market.

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Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia ter­mi­nates Bay­er pact half a year ear­ly, col­lect­ing small por­tion of €240M promised

Bayer and Exscientia are winding down their three-year collaboration, leaving the big German pharma to take the AI-designed compounds born out of the pact further.

London-based Exscientia revealed in its Q2 update that the partners have “mutually agreed to end” their collaboration, which kicked off in early 2020, after recently achieving a drug discovery milestone. In an SEC filing, Exscientia said it terminated the pact on May 30, about six months early.