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

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,600+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,600+ biopharma pros reading Endpoints daily — and it's free.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,600+ biopharma pros reading Endpoints daily — and it's free.

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,600+ biopharma pros reading Endpoints daily — and it's free.

Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.