In a stun­ner, FDA im­me­di­ate­ly drops its hold on Juno’s lead CAR-T

So much for bu­reau­cra­cy. In what may well be the short­est high-pro­file FDA clin­i­cal hold in the agency’s his­to­ry, Juno re­port­ed to­day that the FDA has lift­ed the halt on its lead CAR-T pro­gram, sat­is­fied that it found the cause for three pa­tient deaths.

Juno’s bat­tered shares $JUNO im­me­di­ate­ly rock­et­ed back up 28%.

Juno con­found­ed in­vestors last week when it re­port­ed the deaths and the sur­pris­ing hold, cit­ing the re­cent ad­di­tion of the chemother­a­py agent flu­dara­bine to its pre­con­di­tion­ing of pa­tients as a like­ly cul­prit. Some ex­perts, who had used the drug in oth­er stud­ies to help clear the path for a fol­lowup ther­a­py, pub­licly ques­tioned the ra­tio­nale. But the agency had no prob­lem pro­vid­ing a quick green light to re­sume the piv­otal study.

The Phase II ROCK­ET study will now con­tin­ue in adult pa­tients with re­lapsed or re­frac­to­ry B-cell acute lym­phoblas­tic leukemia (r/r ALL), us­ing on­ly cy­clophos­phamide in pre-con­di­tion­ing pa­tients for JCAR015.

Not im­me­di­ate­ly clear is whether Juno will be able to gain an ap­proval in 2017, as it had hoped be­fore the hold, putting it back in a race with ri­val Kite.

CAR-T ther­a­pies like this take cells from pa­tients and reengi­neer them with chimeric anti­gen re­cep­tors so they can be rein­ject­ed in­to pa­tients, mul­ti­ply and swarm can­cer cells. Ear­ly stud­ies have im­pressed in­ves­ti­ga­tors with their po­ten­tial for blood can­cers. But Juno roiled the field just days ago with news that a new kind of neu­ro­tox­i­c­i­ty had killed sev­er­al can­cer pa­tients in a study.

Some in­ves­ti­ga­tors, though, note that flu­darib­ine has been used in many oth­er stud­ies, with­out the kind of tox­i­c­i­ty Juno’s ther­a­py trig­gered. And be­cause it was used to help amp up the ef­fect of the drug, some won­der if Juno’s lead ther­a­py may not mea­sure up to Kite’s, which now ap­pears to be in the lead for the first, pi­o­neer­ing ap­proval.

Brad Lon­car, who man­ages an in­dex of the top 30 im­munother­a­py com­pa­nies on the mar­ket, not­ed that Juno CEO Hans Bish­op in­di­cat­ed that he would up­date in­vestors on the time­line for JCAR015 in Au­gust. Kite, mean­while, is push­ing full steam ahead on their piv­otal study. And No­var­tis in­di­cat­ed back in late May that it is on track to file an ap­proval for CTL019 in ear­ly 2017.


Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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Eye­ing quick ap­proval, Ab­b­Vie of­fers a close-up on their pres­by­opia drug da­ta

AbbVie picked up some bonus points earlier this year as one of its pipeline adds from the $63 billion Allergan buyout hit its top-line marks. And now the researchers have produced the detailed data on the case they are making with regulators, with an eye on a major new market and a hoped-for approval before New Year’s.

AGN-190584 is aiming to be the first easy-on eyedrop for presbyopia, a common ailment for large numbers of people who find it harder and harder to read things like a watch or cell phone close up. Anyone who’s held a book out at arm’s length in order to read it will be very familiar with the condition, if not the exact diagnosis.

Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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UP­DAT­ED: Pan­el of neu­ro­science ex­perts lays out the com­pli­ca­tions with us­ing Bio­gen's new Alzheimer's drug

Treatment of early Alzheimer’s patients with Biogen’s new drug Aduhelm should closely resemble how the drug was studied in its pivotal clinical trials, according to new recommendations from a panel of neuroscience experts led by UNLV’s Jeffrey Cummings.

“Those considering aducanumab therapy should understand that the expected benefit is slowing of cognitive and functional decline; improvement of the current clinical state is not anticipated,” they wrote Tuesday in The Journal of Prevention of Alzheimer’s Disease, noting that some of their recommendations are more specific or more restrictive than the information provided in the FDA’s prescribing information.

Christophe Weber, Takeda CEO (Kyodo via AP Images)

Take­da flesh­es out CNS pact with pep­tide drug­mak­er, set­ting aside $3.5B in fu­ture mile­stones

One of a suite of drugmakers looking to reinvest in the neuroscience space, Takeda has been aggressive in signing on new partners to help build up its pipeline in that space. But sometimes the best partner is the one you already have.

Takeda will set aside $3.5 billion in future milestones and an undisclosed upfront payment to build out its drug discovery deal with Japanese peptide conjugate maker PeptiDream, adding neurodegeneration to the partnership’s list of CNS targets, the companies said Tuesday.

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