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.

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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