Blue­bird bio adds a key process to pi­o­neer­ing gene ther­a­py as it ramps up a piv­otal PhI­II

David David­son, blue­bird CMO

Blue­bird bio be­lieves it has de­vel­oped a new process that will make a marked im­prove­ment in their pi­o­neer­ing gene ther­a­py pro­grams, promis­ing to de­liv­er a sig­nif­i­cant im­pact on pa­tient out­comes in a new­ly-launched piv­otal study for Lenti­Glo­bin.

Late last year, blue­bird bio ex­ecs were forced to ex­plain why the lat­est da­ta on Lenti­Glo­bin had proved dis­ap­point­ing in a small set of sick­le cell pa­tients be­ing test­ed. In­stead of the cure that blue­bird $BLUE was look­ing for, sev­er­al pa­tients ex­pe­ri­enced a less than op­ti­mal re­sponse in pro­duc­ing an­ti-sick­ling he­mo­glo­bin, which caused more than a few in­vestors in the com­pa­ny to do a dou­ble take on the com­pa­ny’s prospects.

The prob­lem, blue­bird said at the time, was that the vec­tor copy num­ber mea­sur­ing the lev­el of cells which had been cor­rect­ed by gene ther­a­py fell short of ex­pec­ta­tions. In an in­ter­view with TheStreet’s Adam Feuer­stein at the time, CMO David David­son said that that prob­lem might be at­trib­uted to the fact that re­searchers had not re­moved enough of the dis­eased bone mar­row.

To­day, blue­bird says it has be­gun the piv­otal Phase III study for be­ta-tha­lassemia. And David­son tells me that they’re mak­ing a cru­cial cor­rec­tion in the man­u­fac­tur­ing process, adding two uniden­ti­fied small mol­e­cules — a pair of “trans­duc­tion en­hancers” added to its stem cell man­u­fac­tur­ing process — that the bell­wether biotech has ev­i­dence to be­lieve can have a marked im­pact on the vec­tor copy num­ber and the po­ten­tial to cure its top slate of dis­ease tar­gets.

The mar­ket bid up blue­bird’s shares by 9% Thurs­day morn­ing.

“We’re very ex­cit­ed about the two small mol­e­cules,” David­son tells me this morn­ing, with in vit­ro ev­i­dence of an im­prove­ment in the vec­tor copy num­bers as well as in vi­vo da­ta from a mouse mod­el to back it up.

“We’re plan­ning to ap­ply the same man­u­fac­tur­ing process with the im­prove­ment across the Lenti­Glo­bin pro­gram, in­clud­ing sick­le cell,” he adds. That in­volves the piv­otal study in pa­tients with trans­fu­sion-de­pen­dent be­ta-tha­lassemia and non-β0/β0 geno­types as well as an up­com­ing study with β0/β0 geno­types.

Blue­bird isn’t re­veal­ing ex­act­ly what these two mol­e­cules are, in part for com­pet­i­tive rea­sons, says David­son. They’ll be able to re­veal more about it at ASH at the end of the year, and the in­ves­ti­ga­tor says that they’ll be able to get firm pa­tient re­spons­es 6 to 12 months out, mak­ing this more of a 2017 sto­ry.

It’s par­tic­u­lar­ly sig­nif­i­cant for blue­bird that the com­pa­ny can pro­ceed right in­to Phase III with­out get­ting a new IND, says Wed­bush’s David Nieren­garten, who’s bull­ish on the com­pa­ny’s prospects.

Blue­bird helped make gene ther­a­py hot with its first, ear­ly snap­shots of some dra­mat­ic ef­fects on in­di­vid­ual pa­tients. Tar­get­ing some ex­treme­ly rare dis­eases, one or two pa­tients can tell a lot about a com­pa­ny’s prospects. But it’s al­so be­com­ing clear­er that not all gene ther­a­pies are cre­at­ed equal­ly, mak­ing these kinds of on­go­ing im­prove­ments cru­cial to the longterm suc­cess of the com­pa­nies.

The ju­ry is still out on blue­bird, but the com­pa­ny isn’t sit­ting still with the tech­nol­o­gy it has.

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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II 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.

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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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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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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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

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.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.