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. 

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.