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.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK; First plas­ma tri­al sug­gests mor­tal­i­ty re­duc­tion

Count NIH director Francis Collins out for any wager that the FDA would authorize a Covid-19 vaccine in October.

The discussion came up during a call with reporters because some states and local governments have been told by the CDC to have vaccination plans ready to go by Oct. 1. Pharma execs, most notably from Pfizer and BioNTech, have raised hopes about a licensure during that month; President Donald Trump last week sounded an optimistic note about having a vaccine on the market “right around” Election Day on Nov. 3 — or possibly before.

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Charlie Silver (Mission Bio)

'We want to be every­where.' Mis­sion Bio rais­es $70M be­hind re­sis­tance-hunt­ing se­quenc­ing plat­form

Charlie Silver wants to look really, really closely at a lot of your cells. And he just got a lot of money to do so.

Silver’s startup, Mission Bio, raised $70 million in a Series C round Thursday led by Novo Holdings. The money, which brings Mission Bio to $120 million raised since its 2012 founding, will be used to advance the single-cell sequencing platform they built to detect early response or resistance to new cancer therapies.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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