Blue­bird bio or­ches­trates a se­ries of deals and a buy­out to guar­an­tee gene ther­a­py man­u­fac­tur­ing

A day af­ter The New York Times spot­light­ed the man­u­fac­tur­ing and sup­ply prob­lems be­set­ting a boom­ing gene ther­a­py field, with key play­ers scram­bling to main­tain a stock of the be­nign virus­es need­ed to make their prod­ucts, blue­bird bio $BLUE has come up with what it sees as a de­fin­i­tive so­lu­tion to the prob­lem.

The late-stage biotech has snagged a par­tial­ly com­plete man­u­fac­tur­ing com­plex in Durham, NC to do its own in-house man­u­fac­tur­ing work while ex­e­cut­ing a se­ries of con­tracts with sup­pli­ers. And blue­bird ex­ecs say the bas­ket of deals places them in con­trol of their own des­tiny — at least as far as man­u­fac­tur­ing is con­cerned.

The cost of the fa­cil­i­ty is rel­a­tive­ly small, says blue­bird CFO Jeff Walsh, com­ing in at $11.5 mil­lion. (Walsh isn’t say­ing who they bought it from, just that the orig­i­nal own­ers weren’t able to fin­ish it.) But the longterm com­mit­ment, which in­cludes hir­ing 50 staffers for the new in­ter­nal op­er­a­tion while ink­ing deals with out­side op­er­a­tors, is any­thing but small.

Derek Adams

“Solv­ing man­u­fac­tur­ing is a crit­i­cal step,” says blue­bird man­u­fac­tur­ing and tech chief Derek Adams. “We’ve been work­ing quite ex­ten­sive­ly over the last year or more to come up and ex­e­cute on a longterm an strat­e­gy.”

The re­sult in­volves a net­work of sup­pli­ers around the world, start­ing with Lon­za and apceth Bio­phar­ma to make Lenti-D and Lenti­Glo­bin and three more con­tract op­er­a­tions: Bram­mer Bio (Cam­bridge, MA), No­vasep (Gos­selies, Bel­gium) and SAFC (Carls­bad, CA) for lentivi­ral vec­tor across the pipeline.

Jeff Walsh

As the CAR-T pi­o­neers know on­ly too well, work­ing with pa­tient cells in cre­at­ing a per­son­al­ized ther­a­py is no easy task. In gene ther­a­py you have to ex­tract the cells, in­clude a vi­ral vec­tor and cor­rec­tive gene and get it back in­to the veins of pa­tients in a mat­ter of weeks. Any in­ter­rup­tion along that process — or loss of qual­i­ty — can be fa­tal for a gene ther­a­py com­pa­ny like blue­bird. And man­u­fac­tur­ing is­sues in gen­er­al re­main one of the most sig­nif­i­cant threats to any biotech look­ing to take break­through prod­ucts in­to the mar­ket, as the FDA has high­light­ed time and again in re­cent years.

Giv­en the stakes, and the com­plex­i­ty, I asked Adams what keeps him up at night.

“The thing that keeps me up at night,” says Adams, “is that the over­all sup­ply chain for gene ther­a­py prod­ucts is so com­plex” it can be hard to make sure all the pieces are un­der con­trol.

“I sus­pect that Derek is sleep­ing a lit­tle bet­ter now that we closed the deal,” says Walsh.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.