In the glare of an in­tense gene ther­a­py spot­light, blue­bird con­tin­ues to tout each small step for­ward for its pi­o­neer­ing Lenti­Glo­bin

blue­bird bio CEO Nick Leschly. GET­TY

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As one of the pi­o­neers in the new wave of gene ther­a­py de­vel­op­ers, blue­bird bio $blue has spent con­sid­er­able time pro­vid­ing in­cre­men­tal ev­i­dence that it can step up re­sults for Lenti­Glo­bin, im­prov­ing the man­u­fac­tur­ing process and beef­ing up pos­i­tive out­comes for a ther­a­py that’s had its prob­lems along the way.

To­day, the re­search team has two new up­dates to of­fer on a hand­ful of pa­tients af­flict­ed by an in­abil­i­ty to pro­duce suf­fi­cient quan­ti­ties of healthy he­mo­glo­bin, couched in the re­lent­less­ly op­ti­mistic terms that has helped CEO Nick Leschly build their mar­ket cap to close to $10 bil­lion. Prepar­ing for an up­date this week­end at the 23rd Con­gress of the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion, they need to im­press an­a­lysts in the lead-up to a cru­cial mar­ket­ing ap­pli­ca­tion.

Blue­bird’s shares inched up to the $200 mark in ear­ly trad­ing Fri­day, with a gen­er­al­ly pos­i­tive spin on the lat­est da­ta. But they couldn’t hold on in the short run. By mid-morn­ing the stock was down 4% as the EHA gen­er­at­ed a touch crowd of ob­servers.

David David­son

In sick­le cell dis­ease, their fo­cus is on Group C, where 4 pa­tients past the 3-month fol­lowup stage are mak­ing more than 30% of an­ti-sick­ling HbAT87Q  — their de­f­i­n­i­tion of suc­cess. And one of those pa­tients who’s gone past 6 months has a nor­mal to­tal he­mo­glo­bin lev­el of 14.2 g/dL with 60%-plus pro­duc­tion.

These pa­tients are get­ting the new and im­proved ver­sion of the gene ther­a­py af­ter the orig­i­nal fell well short of a cure, and in­ves­ti­ga­tors are stay­ing on each pa­tient as blue­bird builds its case for the steep price they have in mind for Lenti­Glo­bin.

Dura­bil­i­ty is key here.

Blue­bird has made enough progress with its two stud­ies for trans­fu­sion-de­pen­dent β-tha­lassemia that CMO David David­son says to­day that they are on track to file for Eu­ro­pean ap­proval lat­er this year — even though it isn’t per­fect.

The key da­ta point here: Sev­en of 8 pa­tients are pro­duc­ing ≥ 7.6 g/dL of HbAT87Q and are main­tain­ing to­tal he­mo­glo­bin lev­els of 11.1 – 13.3 g/dL by 6 months. 

Blue­bird has be­come a fa­vorite tar­get for some ob­servers, ready to catch them out for hyp­ing less-than-per­fect num­bers in a field try­ing to achieve a near-per­fect record for a once-and-done ther­a­py. Their lat­est round of da­ta on the lead­ing BC­MA-tar­get­ing CAR-T caught some ear­ly flak at AS­CO a few days ago, ding­ing the stock. But the un­der­ly­ing pos­i­tives turned it around with in­vestors who re­main be­liev­ers.

“Con­sis­tent­ly high­er in vi­vo vec­tor copy num­bers and HbAT87Q he­mo­glo­bin lev­els in pa­tients in­di­cate that Lenti­Glo­bin man­u­fac­tur­ing re­fine­ments have re­sult­ed in im­proved gene ther­a­py char­ac­ter­is­tics and may en­able sus­tained trans­fu­sion in­de­pen­dence for a great ma­jor­i­ty of pa­tients,” said Pro­fes­sor Fran­co Lo­catel­li, the lead in­ves­ti­ga­tor of the North­star-2 study. 

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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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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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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Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.

Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.

Partners Innovation Fund

David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.

UP­DAT­ED: Roche fields first ap­proval for Ro­z­lytrek in the run-up to a show­down with Bay­er, Pfiz­er

While it’s wait­ing to hear back from FDA reg­u­la­tors, Roche is be­gin­ning the vic­to­ry lap for en­trec­tinib in Japan.

Roche is giv­ing Bay­er a run for their mon­ey with this tu­mor-ag­nos­tic drug, which tar­gets NTRK gene fu­sions. Now dubbed Ro­z­lytrek, it’s sanc­tioned to treat adult and pe­di­atric pa­tients in Japan with neu­rotroph­ic ty­ro­sine re­cep­tor ki­nase fu­sion-pos­i­tive, ad­vanced re­cur­rent sol­id tu­mors.