EU stamps his­toric OK on blue­bird’s gene ther­a­py for β-tha­lassemia — now sit back and wait for the price

It’s of­fi­cial: blue­bird bio has scored its first drug ap­proval with an EU mar­ket­ing au­tho­riza­tion for its gene ther­a­py for trans­fu­sion-de­pen­dent β-tha­lassemia.

Zyn­te­glo is the new name for a treat­ment once known as Lenti­Glo­bin, a process that takes CD34+ cells from a pa­tient’s hematopoi­et­ic stem cells, in­serts func­tion­al copies of the βA-T87Q-glo­bin gene, then re­in­fus­es the mod­i­fied HSCs. Pa­tients are giv­en chemother­a­py to prime their bone mar­rows be­fore the re­in­fu­sion.

It’s a one-shot ther­a­py de­signed to re­place a life­time of trans­fu­sions and med­ica­tion, in­di­cat­ed for pa­tients 12 years or old­er. The ap­proval fol­lows an EMA rec­om­men­da­tion in March.

The Cam­bridge, MA-based biotech plans to pro­vide an up­date on the roll out plans and pric­ing by the end of next week, a spokesper­son con­firmed.

No mat­ter the fig­ure, the stick­er price is like­ly to trig­ger a fresh wave of de­bate on pric­ing of sup­pos­ed­ly once-and-done ther­a­pies while No­var­tis’ de­ci­sion to charge $2.1 mil­lion for its spinal mus­cu­lar at­ro­phy treat­ment still re­ver­ber­ates.

While blue­bird has pro­duced com­pelling re­sults — dra­mat­i­cal­ly re­duc­ing the need for blood trans­fu­sions in a 22-pa­tient study and elim­i­nat­ing it in most of the pa­tients with a less se­vere form of the dis­ease — Zyn­te­glo has fall­en short of a cure for more se­vere pa­tients. Phase III tri­als are on­go­ing.

Chief ex­ec­u­tive Nick Leschly has pre­vi­ous­ly pro­posed a plan to pay for the ther­a­py by in­stall­ment and stat­ed that Zyn­te­glo will be priced low­er than its “in­trin­sic val­ue” — co­in­ci­den­tal­ly es­ti­mat­ed at $2.1 mil­lion

SVB Leerink an­a­lysts have pre­vi­ous­ly mod­eled the launch price of Lenti­Glo­bin as $1.2 mil­lion in the US and $0.9 mil­lion in the EU.


Im­age: Nick Leschly. In­ter­na­tion­al So­ci­ety for Phar­ma­ceu­ti­cal En­gi­neer­ing

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Days af­ter In­ter­cept re­jec­tion, Akero surges on ‘un­prece­dent­ed‘ NASH da­ta

A year and a half after scoring a $70 million Series B and a top Gilead executive as CEO, Akero Therapeutics has announced new data on their NASH drug. And with the field still reeling from a surprise FDA rejection this week, the news was enough to send their stock surging.

Akero had already said in March that its lead drug had beaten placebo in its Phase II trial, reducing liver fat by 14% in the highest dose group compared to 0.3% in placebo, according to MRI scans. But although NASH is an obesity-related condition and results from fatty buildup in the liver, the real immediate question for any therapy is whether it can resolve the fibrosis and inflammation that results from that buildup. Those data require biopsying the patients, a longer and more invasive process that was further complicated by a pandemic.

Covid-19 has roiled clin­i­cal tri­al plans around the world, rais­ing con­cerns over the in­dus­try’s fu­ture on new drug ap­provals

Over the past 4 months, a group of analysts at GlobalData tracked 322 biopharma companies — biotechs, pharmas, CROs and such — reporting on the trouble Covid-19 has caused for their clinical development plans.

Slightly more than half — 179 — are US operations, with about 1 in 4 scattered throughout Europe and in Canada. And the disruptions are clustered around mid-stage development, though a hefty number of late-stage derailments may well blunt the stream of approvals down the road.

New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”