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.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.