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. 

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Roche scraps one of two schiz­o­phre­nia PhII tri­als af­ter fail­ing the pri­ma­ry end­point

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

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(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

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Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

David Veitch, Basilea CEO

Basilea toss­es an on­col­o­gy drug back to Mer­ck, dou­bling down on an­tibi­otics in re­vamp

Swiss biotech Basilea Pharmaceutica has shed one of its last pipeline ties with oncology as it makes a move to concentrate on antibiotics in a race to profitability next year. And they’ve followed up on that with news of a Phase III success they believe can help expand prospects for a key franchise player.

The company put out word on Monday that the execs are handing rights to the FGFR inhibitor derazantinib back to Merck by the end of this year as it shuts down clinical work on the tumor checkpoint controller lisavanbulin. They’re still working on partnering that out along with their TTK/PLK1-inhibitor BAL0891 and preclinical oncology assets.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.