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.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

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For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
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FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

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The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Mer­ck KGaA spin­out gets first fund­ing to bring dual-act­ing can­cer mol­e­cules in­to the clin­ic

Two and a half years after launch, Merck KGaA spinout iOnctura is getting its first major round of funding.

The oncology startup raised €15 million ($16.6 million) to put its lead drug into the clinic and get its second drug past IND-enabling tests. INKEF Capital and VI Partners co-led the round and were joined by the biotech’s longtime backer M Ventures, an arm of Merck KGaA, and Schroder Adveq.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

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