Blue­bird bio or­ches­trates a se­ries of deals and a buy­out to guar­an­tee gene ther­a­py man­u­fac­tur­ing

A day af­ter The New York Times spot­light­ed the man­u­fac­tur­ing and sup­ply prob­lems be­set­ting a boom­ing gene ther­a­py field, with key play­ers scram­bling to main­tain a stock of the be­nign virus­es need­ed to make their prod­ucts, blue­bird bio $BLUE has come up with what it sees as a de­fin­i­tive so­lu­tion to the prob­lem.

The late-stage biotech has snagged a par­tial­ly com­plete man­u­fac­tur­ing com­plex in Durham, NC to do its own in-house man­u­fac­tur­ing work while ex­e­cut­ing a se­ries of con­tracts with sup­pli­ers. And blue­bird ex­ecs say the bas­ket of deals places them in con­trol of their own des­tiny — at least as far as man­u­fac­tur­ing is con­cerned.

The cost of the fa­cil­i­ty is rel­a­tive­ly small, says blue­bird CFO Jeff Walsh, com­ing in at $11.5 mil­lion. (Walsh isn’t say­ing who they bought it from, just that the orig­i­nal own­ers weren’t able to fin­ish it.) But the longterm com­mit­ment, which in­cludes hir­ing 50 staffers for the new in­ter­nal op­er­a­tion while ink­ing deals with out­side op­er­a­tors, is any­thing but small.

Derek Adams

“Solv­ing man­u­fac­tur­ing is a crit­i­cal step,” says blue­bird man­u­fac­tur­ing and tech chief Derek Adams. “We’ve been work­ing quite ex­ten­sive­ly over the last year or more to come up and ex­e­cute on a longterm an strat­e­gy.”

The re­sult in­volves a net­work of sup­pli­ers around the world, start­ing with Lon­za and apceth Bio­phar­ma to make Lenti-D and Lenti­Glo­bin and three more con­tract op­er­a­tions: Bram­mer Bio (Cam­bridge, MA), No­vasep (Gos­selies, Bel­gium) and SAFC (Carls­bad, CA) for lentivi­ral vec­tor across the pipeline.

Jeff Walsh

As the CAR-T pi­o­neers know on­ly too well, work­ing with pa­tient cells in cre­at­ing a per­son­al­ized ther­a­py is no easy task. In gene ther­a­py you have to ex­tract the cells, in­clude a vi­ral vec­tor and cor­rec­tive gene and get it back in­to the veins of pa­tients in a mat­ter of weeks. Any in­ter­rup­tion along that process — or loss of qual­i­ty — can be fa­tal for a gene ther­a­py com­pa­ny like blue­bird. And man­u­fac­tur­ing is­sues in gen­er­al re­main one of the most sig­nif­i­cant threats to any biotech look­ing to take break­through prod­ucts in­to the mar­ket, as the FDA has high­light­ed time and again in re­cent years.

Giv­en the stakes, and the com­plex­i­ty, I asked Adams what keeps him up at night.

“The thing that keeps me up at night,” says Adams, “is that the over­all sup­ply chain for gene ther­a­py prod­ucts is so com­plex” it can be hard to make sure all the pieces are un­der con­trol.

“I sus­pect that Derek is sleep­ing a lit­tle bet­ter now that we closed the deal,” says Walsh.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Fabrice Chouraqui, Cellarity CEO-partner (LinkedIn)

Drug de­vel­op­er, Big Phar­ma com­mer­cial ex­ec, now an up­start biotech chief — Fab­rice Chouraqui is ready to try some­thing new as a ‘CEO-part­ner’ at Flag­ship

Fabrice Chouraqui’s career has taken some big twists along his life journey. He got his PharmD at Université Paris Descartes and jumped into the drug development game for a bit. Then he took a sharp turn and went back to school to get his MBA at Insead before returning to pharma on the commercial side.

Twenty years later, after steadily rising through the ranks and journeying the globe to nab a top job as president of US pharma for the Basel-based Novartis, Chouraqui exited in another career switch. And now he’s headed into a hybrid position as a CEO-partner at Flagship, where he’ll take a shot at leading Cellarity — one of the VC’s latest paradigm-changing companies of the groundbreaking model that aspires to deliver a new platform to the world of drug R&D.

Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.