Nick Leschly at Endpoints JPM20 (Jeff Rumans)

Blue­bird spins it­self in­to two com­pa­nies, sev­er­ing gene ther­a­py and can­cer units

Blue­bird bio, once one of biotech’s flashiest com­pa­nies, is tak­ing a sweep­ing step as it looks to right the ship af­ter a se­ries of high-pro­file set­backs: split­ting the com­pa­ny in two.

The Cam­bridge-based com­pa­ny will split it­self in­to a unit fo­cused on can­cer and a unit fo­cused on rare dis­ease, sev­er­ing the cell ther­a­py and gene ther­a­py units that the biotech rode to promi­nence. CEO Nick Leschly ex­plained the move as a prac­ti­cal one, re­flect­ing the dif­fer­ent kinds of ex­per­tise in the dis­ease ar­eas.

“You don’t build an on­col­o­gy com­pa­ny by hir­ing peo­ple who are ex­perts in se­vere ge­net­ic dis­ease, nor do you do vice ver­sa,” Leschly told WSJ. “A lot of this comes down to … pri­or­i­ties and fo­cus.”

Yet the move comes as blue­bird’s stock has lost much of its ini­tial lus­ter, as blue­bird has strug­gled to turn strong da­ta in­to com­mer­cial ther­a­pies and in­vestors moved on to new­er gene ther­a­py com­pa­nies such as CRISPR Ther­a­peu­tics.

And it will ef­fec­tive­ly end Leschly’s day-to-day in­volve­ment in the biotech he has be­come syn­ony­mous with and a gene ther­a­py field where he long served as the most promi­nent CEO. Leschly will lead the as-yet-un­named new­co, while An­drew Oben­shain, their long­time Eu­ro­pean chief, will lead blue­bird bio. Leschly will hang on as blue­bird’s ex­ec­u­tive chair.

An­a­lysts were skep­ti­cal that the ap­proach was the so­lu­tion. In a note to in­vestors, Piper San­dler’s Tyler Van Bu­ren said blue­bird had strug­gled to re­plen­ish its pipeline over the years, de­spite sig­nif­i­cant fund­ing, and he wor­ried that they didn’t have enough as­sets or cash to sus­tain mul­ti­ple com­pa­nies.

“Ul­ti­mate­ly, while these two fran­chis­es are dif­fer­ent, we are not con­vinced that their re­spec­tive pipelines are ro­bust enough to sus­tain the in­de­pen­dent en­ti­ties,” he wrote, “and we be­lieve some in­vestors ap­pre­ci­at­ed the bal­ance of the two fran­chis­es.”

The com­pa­ny’s stock $BLUE, which has fall­en dra­mat­i­cal­ly from its 2018 peak, when it was worth over $11 bil­lion, re­mained flat at just un­der $49.

Af­ter com­mand­ing at­ten­tion with cu­ra­tive da­ta for a sick­le cell gene ther­a­py and nu­mer­ous re­mis­sions in tri­als for a mul­ti­ple myelo­ma cell ther­a­py, blue­bird has strug­gled to bring both past the fin­ish line.

Af­ter their most re­cent de­lay, the com­pa­ny re­mains near­ly two years away from sub­mit­ting their sick­le cell gene ther­a­py to the FDA. The FDA is now re­view­ing the mul­ti­ple myelo­ma cell ther­a­py ide-cel, now part­nered with Bris­tol My­ers Squibb, but the agency ini­tial­ly served the com­pa­ny with a refuse-to-file let­ter for sub­mit­ting in­suf­fi­cient man­u­fac­tur­ing in­for­ma­tion.

The gene ther­a­py, known as Zyn­te­glo, was ap­proved in Eu­rope for an­oth­er rare blood dis­ease, be­ta tha­lassemia. But the $1.8 mil­lion price tag blue­bird placed on it shocked an­a­lysts and in­dus­try watch­ers and, with the pan­dem­ic hit­ting short­ly af­ter their of­fi­cial launch, the com­pa­ny had yet to sell a sin­gle unit as of their No­vem­ber Q3 fil­ing.

De­spite the set­backs, the com­pa­ny still re­mains at the front of a now crowd­ed pack to com­mer­cial­ize a sick­le cell cure, and an­a­lysts peg peak sales for ide-cel as high as $900 mil­lion. Blue­bird al­so has an im­munother­a­py for Merkel cell car­ci­no­ma and a gene ther­a­py for cere­bral adrenoleukody­s­tro­phy in clin­i­cal de­vel­op­ment.

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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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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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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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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Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

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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No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Andrea Pfeifer, AC Immune CEO (AC Immune)

Look­ing to repli­cate Covid-19 suc­cess in neu­ro, BioN­Tech back­ers bet on AC Im­mune and its new­ly-ac­quired Parkin­son's vac­cine

The German billionaires behind BioNTech have found a new vaccine project to back.

Through their family office Athos Service, twin brothers Thomas and Andreas Strüngmann are leading a $25 million private placement into Switzerland’s AC Immune — which concurrently announced that it’s shelling out $58.7 million worth of stock to acquire Affiris’ portfolio of therapies targeting alpha-synuclein, including a vaccine candidate, for Parkinson’s disease.