One of Kite Phar­ma’s CAR-T pa­tients died from cere­bral ede­ma, trig­ger­ing a safe­ty alarm

Kite Phar­ma re­vealed to­day that one of the pa­tients in their late-stage pro­gram for the CAR-T drug KTE-C19 died from cere­bral ede­ma, the same brain swelling con­di­tion that went on to scut­tle Juno Ther­a­peu­tics’ lead drug.

In a call with an­a­lysts for their Q1 re­port, the close­ly-watched biotech $KITE said that they had in­formed the FDA and there was no pause or halt to the study. The death in late April, though, clear­ly raised a red flag for an­a­lysts af­ter Kite had man­aged to get all the way through a piv­otal pro­gram with­out a death due to cere­bral ede­ma af­ter treat­ing hun­dreds of pa­tients.

David Chang, Kite

“It took about two days of pro­gres­sive­ly wors­en­ing neu­ro­log­i­cal events,” com­ment­ed Kite CMO David Chang. “In this time the pa­tient’s over­all con­di­tion was de­te­ri­o­rat­ing.”

“This pa­tient had re­frac­to­ry non-Hodgkin lym­phoma,” added Chang. “At the time of en­roll­ment he had ex­plo­sive dis­ease that was rapid­ly pro­gress­ing and had a lot of symp­toms from the tu­mor.” There was fever, con­cerns about un­der­ly­ing in­fec­tions – though tests came back neg­a­tive — and “pret­ty rapid­ly pro­gress­ing dis­ease.”

In an email, a spokesper­son for Kite not­ed that “we don’t see any safe­ty con­cerns. All axi-cel and KTE-C19 de­vel­op­ment stud­ies con­tin­ue as planned. As a re­minder, over­all in­ci­dence of KTE-C19 re­lat­ed grade 5 events stands at 2% in ap­prox­i­mate 200 pa­tients treat­ed in our study sup­ports the ben­e­fit of axi-cel and KTE-C19.  If pa­tients treat­ed in the NCI stud­ies are in­clud­ed, over 300 pa­tients have been treat­ed with KTE-C19.”

Kite’s shares dropped 10% as news of the death spread.

Kite had want­ed to fo­cus to­day pri­mar­i­ly on its com­mer­cial­iza­tion plans for this drug, look­ing to a pos­si­ble FDA ap­proval lat­er in the year. But af­ter Juno was forced to shelve JCAR015 af­ter it killed 5 pa­tients who suf­fered cere­bral ede­ma, the news clear­ly cap­tured an­a­lysts’ at­ten­tion.

The FDA quick­ly lift­ed their first clin­i­cal hold on Juno’s drug af­ter the first three deaths, in­di­cat­ing reg­u­la­tors’ al­lowance for the ad­vanced state most of these can­cer pa­tients are in when they get in­to a CAR-T study. When two more pa­tients died soon af­ter the hold was lift­ed, though, that ex­pe­ri­ence could raise ques­tions of whether reg­u­la­tors may have be­come more sen­si­tive to the safe­ty is­sues in­volved with these drugs.

Juno had ini­tial­ly blamed the first group of deaths on the use of flu­dara­bine dur­ing the pre­con­di­tion­ing reg­i­men pa­tients go through to make them more re­cep­tive to cell ther­a­py. So they dropped it, then saw more pa­tients die. The flu/cy com­bo, though, was used by Kite and oth­ers. Kite be­lieves it has just the right mix to gain ef­fi­ca­cy with­out cre­at­ing un­rea­son­able safe­ty is­sues.

These drugs — reengi­neered T cells tak­en from pa­tients and then re­in­fused — have had safe­ty is­sues from the very be­gin­ning, with a num­ber of pa­tients suf­fer­ing from cy­tokine storms that oc­ca­sion­al­ly turned lethal.

Chang said that they would con­tin­ue to con­sid­er the ef­fi­ca­cy and safe­ty of the drug as more stud­ies pro­ceed.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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