Blue­bird has a promis­ing new up­date on its BC­MA CAR-T for mul­ti­ple myelo­ma, but is it still the leader?

Nick Leschly / Boston Globe, End­points News

Blue­bird bio $BLUE un­veiled its lat­est up­date on its close­ly-watched BC­MA-tar­get­ing CAR-T for mul­ti­ple myelo­ma to­day of­fer­ing a slate of sol­id ev­i­dence that demon­strates the promise that at­tract­ed Cel­gene $CELG to their am­bi­tious col­lab­o­ra­tion on this drug. But with a ri­val Chi­nese group mak­ing a splash at AS­CO this year, the biotech may be see­ing its sta­tus as the fron­trun­ner in the field start to fade a bit.

In the first new da­ta to come out since late last year, blue­bird of­fered an up­date on 18 pa­tients in four dif­fer­ent dos­ing co­horts in search of in­sights on dura­bil­i­ty, dos­ing and safe­ty for bb2121.

With 18 pa­tients evalu­able for ef­fi­ca­cy, there were 15 in what blue­bird termed ac­tive dos­ing reg­i­mens above the min­i­mum for bb2121, and all of them achieved an ob­jec­tive re­sponse. Twelve achieved a very good par­tial re­sponse to com­plete re­sponse, as­so­ci­at­ed with longer sur­vival times. And of the 4 pa­tients evalu­able for min­i­mal resid­ual dis­ease sta­tus, all were MRD neg­a­tive with on­ly a few or no myelo­ma cells in cir­cu­la­tion.

That is all ex­cel­lent.

Up un­til now, blue­bird and Cel­gene were wide­ly viewed as the lead­ers in the BC­MA field. But Nan­jing Leg­end’s da­ta out this morn­ing may well put that sta­tus in ques­tion.

I asked blue­bird CEO Nick Leschly about that over the week­end.

His re­sponse, with a smile: “We are com­plete­ly, con­struc­tive­ly para­noid no mat­ter what.”

Short trans­la­tion: Blue­bird is hap­py where they are and con­fi­dent about mov­ing to next steps as their part­ner Cel­gene lays the foun­da­tion for a ground­break­ing reg­is­tra­tional study that may well get start­ed lat­er this year. But they all know it won’t be easy or free of chal­lenges — now or in the fu­ture as ri­val ther­a­pies line up to chal­lenge them.

Da­ta from tri­als that don’t in­volve head-to-head de­signs with­in the con­struct of the same study are no­to­ri­ous­ly dif­fi­cult to com­pare. In Leg­end’s case, says Leschly, it seems ev­i­dent that the pa­tients weren’t as sick and hadn’t failed the same mul­ti­ple of drugs that blue­bird re­cruit­ed for.

That makes it “ap­ples and or­anges,” he says, adding “that doesn’t mean it isn’t great da­ta.”

All of it, notes Leschly, helps val­i­date the tar­get, while blue­bird is sat­is­fied that it has the right drug to take for­ward. Its next-gen drug, bb21217, will come up be­hind it as Cel­gene preps for the piv­otal BC­MA study to some. “It’s all go, go, go” on bb2121, says Leschly, whose of­fi­cial ti­tle is chief blue­bird.

“All these pa­tients are do­ing in­cred­i­bly well,” says the CEO about his lead ther­a­py, point­ing to par­tial re­spons­es in their study that re­flect­ed a near era­sure of can­cer. These drugs face a high bar on demon­strat­ing ef­fi­ca­cy, he adds, and bb2121 comes through with fly­ing col­ors.

Blue­bird came to AS­CO with a few things to prove. It need­ed to prove that its drug works in a broad­er num­ber of pa­tients. And it need­ed to prove that the safe­ty is­sues around cy­tokine re­lease syn­drome were man­age­able.

Blue­bird’s CEO is com­ing out of AS­CO be­liev­ing that he’s an­swered those chal­lenges.

I asked Leschly about the de­bate over the 4-1BB cos­tim­u­la­to­ry do­main its drug us­es, and the the­o­ry that it could be safer than the CD28 al­ter­na­tives on the mar­ket.

Leschly’s re­mark: he’s learned to “be care­ful of open­ing your mouth be­fore you know what you’re talk­ing about.”

The 4-1BB do­main does seem to help dura­bil­i­ty, he adds, but he’s hes­i­tant to say that it ex­tends to safe­ty at this point. It’s just too ear­ly.

Blue­bird’s ex­ecs say they still have to de­cide ex­act­ly which dose to take in­to the next phase of their study (and I pressed them on it), as they add new pa­tients and pre­pare for the piv­otal study to come.

Cel­gene is clear that they want to start en­rolling for the piv­otal study this year, says Leschly. And with 50% of the US com­mer­cial rights re­sid­ing at blue­bird, he’s ready for the next step.

Michel Younatsos, 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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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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Covid-19 roundup: Pfiz­er teams with Gilead on remde­si­ivr; Japan, Brazil, Switzer­land, In­dia get vac­cines

Pfizer has joined the list companies helping Gilead manufacture remdesivir. The pharma giant announced today they signed a multi-year agreement to provide Gilead with contract manufacturing services at their McPherson, Kansas plant. The deal is part of a broad effort by Gilead to scale up the drug, the only currently authorized therapy for Covid-19, to 2 million doses this year.

That effort now includes 40 different companies on 3 continents, according to a press release the biotech put out yesterday, not including the generic drugmakers the company has allowed to produce the anti-viral for low and middle-income countries. Dozens of state governments, though, have said those efforts have not been extensive enough to keep up with demand and have called upon the federal government to sidestep Gielad’s patents and begin scaling the drug itself.  – Jason Mast

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In sur­pris­ing set­back, com­bo of Roche’s Tecen­triq and chemo fails to help pa­tients with triple-neg­a­tive breast can­cer

Roche broke ground last year when they secured the first FDA approval for a checkpoint therapy in triple-negative breast cancer, a notoriously difficult-to-treat indication that has been passed over by the wave of targeted therapies.

Now, though, doctors are puzzling over why a combination of drugs meant to make that therapy more potent instead appeared to make it less effective.

Roche said Thursday that in a Phase III trial, combining their PD-1/L1 checkpoint therapy Tecentriq with the chemotherapy paclitaxel, did not significantly improve progression-free survival for patients with locally advanced or metastatic triple-negative breast cancer over giving those patients chemotherapy alone. In fact, patients on the Tecentriq-chemo arm had lower overall survival than patients on chemo, although the drugmaker cautioned that the trial was not powered for that endpoint and the data were immature.

Alex­ion cre­ates new post for chief di­ver­si­ty of­fi­cer; Bar­ry Greene step­ping down at Al­ny­lam, Yvonne Green­street named as suc­ces­sor

Alexion has carved out a new position for chief diversity officer and filled it with an inside promotion.

Uzair Qadeer will now be responsible for their “diversity, inclusion and belonging” strategy, looking to reshape the biotech’s corporate culture. A veteran of Deloitte and Bristol Myers Squibb, Qadeer was working on executive coaching and helping create the diversity program he now leads.

President Trump (AP Images)

FDA takes the lead on defin­ing es­sen­tial un­der Trump's 'Buy Amer­i­can' ex­ec­u­tive or­der — as in­dus­try warns of sup­ply chain dis­rup­tion

President Donald Trump has signed an executive order detailing how the federal government should help on-shore drug manufacturing — and the FDA will play a central role.

The agency now has three months to draw up the list of “essential medicines, medical countermeasures, and their critical inputs” that the US must have available at all times. Various departments and agencies are then directed to buy these drugs and their ingredients from American manufacturers.

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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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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