Blue­bird’s gene ther­a­py for be­ta-tha­lassemia falls short of a cure, but still wows in 22 pa­tients

Af­ter go­ing back to the draw­ing board to come up with a bet­ter gene ther­a­py for be­ta-tha­lassemia, a dis­ease that in­ter­feres with the body’s abil­i­ty to pro­duce he­mo­glo­bin, in­ves­ti­ga­tors work­ing for blue­bird bio $BLUE have pro­duced some com­pelling da­ta to demon­strate that their ther­a­py can elim­i­nate or re­duce the need for blood trans­fu­sions.

But they don’t have a cure — yet.

Pub­lish­ing in the New Eng­land Jour­nal of Med­i­cine, the re­searchers re­port that they test­ed the treat­ment in 22 pa­tients. In the 9 pa­tients with the most se­vere form of the dis­ease, they re­port they were able to dra­mat­i­cal­ly re­duce the need for blood trans­fu­sions by 73% and elim­i­nate it for now in 3. Six of those pa­tients, though, have need­ed to con­tin­ue trans­fu­sions. And 12 of 13 pa­tients with a less se­vere form of the dis­ease cur­rent­ly no longer re­quire blood trans­fu­sions af­ter a sin­gle treat­ment — with a me­di­an of 26 months trans­fu­sion-free.

The in­ter­im da­ta great­ly ex­pands on the pa­tient ex­pe­ri­ences they can now bank on to demon­strate the ef­fi­ca­cy of the at­tempt at a once-and-done ther­a­py which af­fects hun­dreds of thou­sands of pa­tients. And the in­ves­ti­ga­tors have not aban­doned an at­tempt to find a com­plete cure for all.

“There is room for im­prove­ment, as we’d like to see the elim­i­na­tion of de­pen­den­cy on trans­fu­sion even for pa­tients with the most se­vere form of the dis­ease,” Har­vard Med­ical’s Philippe Leboulch told their in-house pub. “But there is al­so hope with pro­to­col mod­i­fi­ca­tions we have in­tro­duced in our Phase III tri­als.”

Blue­bird — run by CEO Nick Leschly — was forced to go back and change the man­u­fac­tur­ing process on Lenti­Glo­bin af­ter some un­even re­spons­es ear­ly on. Last sum­mer, the com­pa­ny came up with some en­cour­ag­ing da­ta on the first three pa­tients.

The gene ther­a­py is made by ex­tract­ing blood stem cells from pa­tients and in­sert­ing a func­tion­al be­ta-glo­bin gene in­to them, trans­plant­i­ng them af­ter a round of chemo to clear their bone mar­row.

Jerome Groop­man in The New York­er writes:

The chal­lenge now is find­ing ways to im­ple­ment a com­plex, po­ten­tial­ly life-sav­ing treat­ment in parts of the world where med­ical care is lim­it­ed. Es­tab­lished fa­cil­i­ties for au­tol­o­gous mar­row trans­plan­ta­tion al­ready ex­ist in many de­vel­oped na­tions, as do lab­o­ra­to­ries that can in­tro­duce a healthy glo­bin gene in­to stem cells. But, in some parts of the world where be­ta tha­lassemia is most com­mon, these fa­cil­i­ties do not yet ex­ist.

Con­trary to what the en­thu­si­asts may be pre­sent­ing to­day, there’s clear­ly room for im­prove­ment. And blue­bird is clear­ly not alone in the field try­ing to pro­duce a cure. CRISPR Ther­a­peu­tics, for ex­am­ple, has been us­ing its gene edit­ing tech to see if it can find a last­ing cure for the dis­ease. And those oth­er pro­grams, in­clud­ing ef­forts at Bel­licum, will like­ly con­tin­ue to see if they can leapfrog blue­bird with some­thing bet­ter.


blue­bird bio CEO Nick Leschly Get­ty

Brent Saunders [Getty Photos]

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Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

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Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

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Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

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An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

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Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

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Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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