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.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Moncef Slaoui, Getty Images

When will it end? Big Phar­ma's top vac­cine ex­pert at OWS of­fers a speedy time­line for a Covid-19 vac­cine — ei­ther be­fore or right af­ter the elec­tion

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

Levi Garraway, Roche CMO (Source: Genentech)

UP­DAT­ED: FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

Per­cep­tive fields SPAC #3 as an­oth­er group of biotechs scoops up $364M in lat­est Nas­daq romp

There’s no sign that the windfall of cash dropping biotech’s way on Wall Street is abating. Three more biopharmas priced IPOs on Thursday and Friday morning, riding a historic boom with a $364 million payoff.

London-based biotech Freeline Therapeutics took home the lion’s share of the cash with $159 million after pricing 8,823,529 shares at $18 a pop. Checkmate Pharmaceuticals, of Cambridge, MA, raised $75 million with an offer of 5 million shares at $15 — right at the midpoint of its range. And Arya Sciences Acquisition Corp III, the third in a series from Perceptive, priced 13,000,000 shares at $10 per share.

Douglas Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

Roche-backed Dicer­na push­es in­to the pack rac­ing to­ward the block­buster hep B goal line, armed with PhI da­ta

Dicerna has lined up a set of proof-of-concept data from a small cohort of hepatitis B patients in a match-up against some heavyweight rivals which got out in front of this race. And right in the front row you’ll find a team from Roche, which paid $200 million in cash and offered another $1.5 billion in milestones to partner with Dicerna $DRNA on their RNAi program for hep B.

Right now it’s looking competitive, with lots of big challenges ahead.

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Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

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