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.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.