With one BLA at the FDA, Kite hus­tles along im­pres­sive new leukemia da­ta and sounds a note of cau­tion on safe­ty

CHICA­GO — With its lead pro­gram for non-Hodgkin lym­phoma in front of FDA re­view­ers, Kite Phar­ma $KITE to­day up­dat­ed their Phase I CAR-T study on re­lapsed cas­es of acute lym­phoblas­tic leukemia, not­ing a strik­ing­ly high com­plete re­mis­sion rate of 73% as they con­tin­ue to work on im­prov­ing the safe­ty pro­file of their ther­a­py.

The study in­clud­ed 11 pa­tients in the Phase I por­tion of ZU­MA-3. Com­plete re­mis­sions — with the re­spon­ders all test­ing neg­a­tive for min­i­mal resid­ual dis­ease with few or no can­cer cells in cir­cu­la­tion — were be­ing tracked that last­ed from 2 to more than 7.4 months. And the Phase II tri­al will get un­der­way as Kite looks to ex­pand use of its drug KTE-C19.

David Chang, Kite

“In many ways it sort of con­firms what we thought we’d see in the clin­i­cal study; very rapid re­sponse in 2 to 4 weeks,” Kite CMO David Chang tells me.

Safe­ty, though, re­mains a key con­cern. These CAR-T stud­ies have been marred from the be­gin­ning by com­mon cas­es of cy­tokine re­lease syn­drome. In this tri­al, there were three cas­es of grade three or high­er CRS with one death and six cas­es of grade three or high­er neu­ro­log­i­cal re­ac­tions.

That left Kite look­ing at who should re­ceive tocilizum­ab with­in 36 hours post‐KTE‐C19 in­fu­sion, and a low­er dose of 0.5×10(6) CAR T cells/kg rather than the two dos­es of 2.0×10(6)/kg and 1.0×10(6)/kg used in the first round.

Kite re­cent­ly re­port­ed that one pa­tient died re­cent­ly from cere­bral ede­ma, or brain swelling, in a sep­a­rate tri­al, rat­tling in­vestors who didn’t like the com­par­isons to the five cas­es of cere­bral ede­ma that forced Juno to scrap their led pro­gram, leav­ing Kite and No­var­tis alone in the race to a po­ten­tial first ap­proval lat­er this year.

Kite has learned a lot about con­trol­ling safe­ty, says Chang. Dos­ing, get­ting rid of in­fec­tions be­fore treat­ment, prepar­ing to bat­tle back CRS are all part of the game plan now.

Cas­es of cere­bral ede­ma have al­so sparked a de­bate over whether the CD28 cos­tim­u­la­to­ry do­main Kite us­es in their CAR-Ts — in­stead of the 4-1BB do­main No­var­tis us­es — could be re­spon­si­ble. Juno’s ini­tial lead ther­a­py al­so used the CD28 cos­tim­u­la­to­ry do­main.

“In a way I can see why some peo­ple would say that, but there’s re­al­ly no ba­sis to draw that con­clu­sion,” says Chang. He adds that there have been cas­es of cere­bral ede­ma with 4-1BB go­ing back to the ear­ly aca­d­e­m­ic stud­ies.

Chang al­so isn’t keen on mak­ing cross-tri­al com­par­isons.

“There’s a lot of false con­clu­sions that one can draw,” he says. But out­comes can be al­tered by the char­ac­ter­is­tics of the pa­tients re­cruit­ed, whether they were tru­ly chemo re­frac­to­ry or sim­ply re­lapsed, how many times they had re­lapsed on mul­ti­ple lines of ther­a­py and the dis­tri­b­u­tion of dis­ease types, all of which play in­to the da­ta.

Kite has al­so been hon­ing its man­u­fac­tur­ing skills. In this lat­est Phase I the man­u­fac­tur­ing team was able to get the time it takes to make the ther­a­py from pa­tient cells down to six days. Chang says that the com­pa­ny has the vein-to-vein time down to about 17 days, which he be­lieves can still like­ly be im­proved by two or three days.

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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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 clinicaltrials.gov. 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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Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

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Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Years af­ter link­ing arms with Bris­tol My­ers and both Mer­cks, Sutro finds its lat­est part­ner in Tokyo

Astellas and Sutro Biopharma are linking arms on a new field of antibody-drug conjugates that they hope will improve upon existing cancer immunotherapies.

The Tokyo pharma will dole out $90 million in cash for the collaboration, the companies said Monday afternoon. That upfront payment will extend the South San Francisco biotech’s runway from late 2023 into the first half of 2024, Cowen analysts noted.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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