Months af­ter an­oth­er lethal set­back, Juno fi­nal­ly opts to kill lead CAR-T

Juno CEO Hans Bish­op

Months af­ter one of the worst set­backs in the re­cent his­to­ry of drug de­vel­op­ment left a string of pa­tients dead, Juno has fi­nal­ly de­cid­ed that it will ter­mi­nate its lead drug pro­gram, hop­ing for a bet­ter out­come with an­oth­er CAR-T ther­a­py in its pipeline.

Juno CEO Hans Bish­op claimed $JUNO that its de­ci­sion to shelve JCAR015 was all due to the “un­ex­pect­ed” tox­i­c­i­ty that it dis­cov­ered af­ter the drug killed five pa­tients due to cere­bral ede­ma, or brain swelling. The sec­ond tal­ly of deaths, though, oc­curred in No­vem­ber, af­ter the com­pa­ny con­vinced the FDA to swift­ly lift a clin­i­cal hold on the drug by mak­ing the du­bi­ous claim that elim­i­nat­ing flu­dara­bine from the pre­con­di­tion­ing reg­i­men for pa­tients would re­solve the safe­ty is­sue that had al­ready killed sev­er­al late-stage can­cer pa­tients.

Flu­dara­bine is still com­mon­ly used in most CAR-T stud­ies, need­ed to help these cell ther­a­pies take ef­fect in pa­tients. This first wave of drugs ex­tracts T cells from pa­tients and then reengi­neers them to go af­ter can­cer cells, and progress has been reg­u­lar­ly marred by se­ri­ous safe­ty is­sues. But there’s still been no clear ex­pla­na­tion of why JCAR015 de­railed.

Shares of Juno dropped 8% Wednes­day evening on the biotech’s re­minder of its se­ri­ous­ly flawed ef­forts.

“We con­tin­ue to ex­pe­ri­ence en­cour­ag­ing signs of clin­i­cal ben­e­fit in our tri­al ad­dress­ing NHL, but we al­so rec­og­nize the un­for­tu­nate and un­ex­pect­ed tox­i­c­i­ty we saw in our tri­al ad­dress­ing ALL with JCAR015,” said Bish­op in a state­ment. “We have de­cid­ed not to move for­ward with the ROCK­ET tri­al or JCAR015 at this time, even though it gen­er­at­ed im­por­tant learn­ings for us and the im­munother­a­py field.”

Juno threw out sev­er­al vague fac­tors that may have trig­gered the deaths. But they all added up to a re­turn to Phase I to prove their point. And there was no time to go back to the draw­ing board. From the state­ment:

Through the in­ves­ti­ga­tion Juno iden­ti­fied mul­ti­ple fac­tors that may have con­tributed to this in­creased risk, in­clud­ing pa­tient spe­cif­ic fac­tors, the con­di­tion­ing chemother­a­py pa­tients re­ceived, and fac­tors re­lat­ed to the prod­uct. Al­though Juno be­lieves there are pro­to­col mod­i­fi­ca­tions and process im­prove­ments that could en­able Juno to pro­ceed with JCAR015 in clin­i­cal test­ing in adult r/r ALL, Juno would first need to es­tab­lish pre­lim­i­nary safe­ty and dose in a Phase I tri­al. As a re­sult of the tim­ing de­lay that would en­tail and Juno’s be­lief that it has oth­er prod­uct can­di­dates in its pipeline that are like­ly to pro­vide im­proved ef­fi­ca­cy and tol­er­a­bil­i­ty,

As just about every­one who fol­lows this field has ex­pect­ed for some time now, Juno is turn­ing to JCAR017, hop­ing to get back on track af­ter falling far be­hind the two lead­ers in the field: Kite and No­var­tis. In its state­ment to­day, Juno out­lined plans to launch a piv­otal study for r/r dif­fuse large B cell lym­phoma lat­er this year. The biotech, though, has now fall­en be­hind ri­vals by more than a year.

At one point Juno was con­sid­ered a con­tender for a pi­o­neer­ing ap­proval, run­ning neck-and-neck with Kite. Yes­ter­day, though, Kite post­ed im­pres­sive 6-month re­sults for its CAR-T ahead of its ap­pli­ca­tion for ap­proval, as Juno was just get­ting ready to for­mal­ly ter­mi­nate its trou­bled lead pro­gram.

Added Bish­op:

Look­ing for­ward in­to 2017, we con­tin­ue to be op­ti­mistic about the progress we are mak­ing with JCAR017 and our pipeline more broad­ly. We ex­pect 2017 will be a da­ta-rich year of key in­sights, based on up to 20 on­go­ing tri­als by year end, and we plan to present da­ta from these tri­als as ap­pro­pri­ate through­out the year.

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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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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

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No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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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.