Samit Hirawat. Bristol-Myers Squibb

Bris­tol-My­ers is mak­ing a bee-line to the FDA with pos­i­tive liso-cel da­ta — but is it too late in the CAR-T game?

Bris­tol-My­ers Squibb came to ASH this past week­end with a va­ri­ety of mes­sages on the new can­cer drugs they had ac­quired in the big Cel­gene buy­out, in­clud­ing liso-cel, the lead CAR-T pro­gram picked up in the $9 bil­lion Juno ac­qui­si­tion. And one of the most im­por­tant was that they had the piv­otal ef­fi­ca­cy and safe­ty da­ta need­ed to snag an ap­proval from the FDA next year, with the BLA on track for a fil­ing this month.

Pro­vid­ed there are no sna­fus or even mod­est stum­bles now, they should get an OK with­in the 2020 time­line lined out in their $9 CVR for Cel­gene — the first in a tri­fec­ta of ap­provals re­quired for a pay­out.

Whether they can go on to make it in­to a vi­able com­mer­cial ther­a­py, though, is a whole oth­er thing.

At first glance, there isn’t any­thing about the safe­ty and ef­fi­ca­cy da­ta that would force a CRL or de­lay. In a large tri­al of pa­tients with re­lapsed/re­frac­to­ry large B-cell lym­phomas in­ves­ti­ga­tors tracked an out­stand­ing 73% re­sponse rate and 53% com­plete re­sponse rate in heav­i­ly pre­treat­ed pa­tients with few op­tions.

That’s in line with Yescar­ta from Gilead’s Kite, which snagged an ap­proval more than 2 years ago, just af­ter No­var­tis’ Kym­ri­ah came through.

Then there’s safe­ty. To be sure, the drug has safe­ty is­sues. There were 4 pa­tients in the study who died af­ter treat­ment. Sev­er­al oth­ers died for un­re­lat­ed is­sues. But…with on­ly a 2% rate of cy­tokine re­lease syn­drome, Bris­tol-My­ers has a shot at a dif­fer­en­ti­at­ed safe­ty pro­file.

“There is a po­ten­tial these pa­tients can be treat­ed on an out­pa­tient ba­sis,” says Samit Hi­rawat, the chief med­ical of­fi­cer at Bris­tol-My­ers. He not­ed that 26% of pa­tients were nev­er ad­mit­ted, while 76% were ad­mit­ted 4 days or lat­er af­ter ther­a­py. The key is to treat them at a hos­pi­tal with the in­fra­struc­ture to pro­vide care 24/7, so a pa­tient can be ad­mit­ted at any time lat­er if need­ed.

How that will fly with pay­ers af­ter ri­vals have been on the mar­ket for about 3 years, with new ev­i­dence that ear­li­er use of steroids can dra­mat­i­cal­ly re­duce CRS and con­sid­er­able dura­bil­i­ty of re­sponse, will have to be seen.

But time is not on Bris­tol-My­ers’ side. Liso-cel is the long de­layed fol­lowup to the dis­as­trous JCAR015, which killed a num­ber of pa­tients. Their set­back threw them years off sched­ule. And ri­vals are ad­vanc­ing off-the-shelf al­ter­na­tives or oth­er new ap­proach­es that could knock the pi­o­neers com­plete­ly out of the game. In the mean­time, Bris­tol-My­ers is gath­er­ing its own dura­bil­i­ty da­ta and will grad­u­al­ly see if their mix of CD4 and CD8 cells can do bet­ter.

The main in­ter­est now is in the time­line around the ap­proval. Hi­rawat says they’ll ask for pri­or­i­ty re­view, and there’s every rea­son to be­lieve that reg­u­la­tors will move swift­ly — bar­ring a nasty sur­prise.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Roche scraps one of two schiz­o­phre­nia PhII tri­als af­ter fail­ing the pri­ma­ry end­point

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

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(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

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Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

David Veitch, Basilea CEO

Basilea toss­es an on­col­o­gy drug back to Mer­ck, dou­bling down on an­tibi­otics in re­vamp

Swiss biotech Basilea Pharmaceutica has shed one of its last pipeline ties with oncology as it makes a move to concentrate on antibiotics in a race to profitability next year. And they’ve followed up on that with news of a Phase III success they believe can help expand prospects for a key franchise player.

The company put out word on Monday that the execs are handing rights to the FGFR inhibitor derazantinib back to Merck by the end of this year as it shuts down clinical work on the tumor checkpoint controller lisavanbulin. They’re still working on partnering that out along with their TTK/PLK1-inhibitor BAL0891 and preclinical oncology assets.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.