Nick Leschly, bluebird CEO (Jeff Rumans/JPM20)

Lenti­Glo­bin con­tin­ues to de­liv­er in lat­est sick­le cell up­date as blue­bird out­lines path to ac­cel­er­at­ed ap­proval, de­spite pa­tient death

Still reel­ing from the FDA’s stun­ning re­fusal to file its CAR-T ther­a­py ide-cel, blue­bird is ea­ger to win back some cred­i­bil­i­ty with a new slice of the Lenti­Glo­bin da­ta they will be tak­ing to the FDA for an ac­cel­er­at­ed ap­proval.

To be sure, the BLA sub­mis­sion won’t hap­pen un­til the sec­ond half of 2021 — giv­ing blue­bird plen­ty of time to fig­ure out the man­u­fac­tur­ing. But an up­date pre­sent­ed at the EHA vir­tu­al con­gress has ap­par­ent­ly helped them reach “gen­er­al agree­ment” with reg­u­la­tors and boost­ed ex­ecs’ con­fi­dence.

Build­ing on pre­vi­ous­ly re­port­ed da­ta, the pre­sen­ta­tion cen­ters around Group C of the HGB-206 study, com­pris­ing a to­tal of 25 pa­tients who re­ceived a gene ther­a­py pro­duced on a “re­fined man­u­fac­tur­ing process that was de­signed to in­crease vec­tor copy num­ber (VCN) and im­prove en­graft­ment po­ten­tial of gene-mod­i­fied stem cells.”

Among 14 pa­tients with at least six months of fol­low-up and a his­to­ry of va­so-oc­clu­sive crises and acute chest syn­drome — five more than the last dis­clo­sure — the mean re­duc­tion in an­nu­al­ized rate of those painful episodes was 99.5%. It marks a slight im­prove­ment from the al­ready stel­lar 99% in­ves­ti­ga­tors ob­served at ASH last De­cem­ber.

These were pa­tients who had a me­di­an of eight events in the two years pri­or to treat­ment, ac­cord­ing to blue­bird.

One of the pa­tients who had com­plete res­o­lu­tion of va­so-oc­clu­sive events died sud­den­ly 20 months af­ter treat­ment, al­though it was at­trib­uted to car­dio­vas­cu­lar and oth­er un­der­ly­ing is­sues and thus deemed “un­like­ly re­lat­ed” to Lenti­Glo­bin.

Zoom­ing out to the 16 who made the six-month cut­off point, me­di­an lev­els of a key bio­mark­er known as gene ther­a­py-de­rived he­mo­glo­bin were main­tained and con­tributed at least 40% of all he­mo­glo­bin.

At last vis­it re­port­ed, to­tal he­mo­glo­bin ranged from 9.6 – 16.2 g/dL and HbAT87Q lev­els ranged from 2.7 – 9.4 g/dL. At Month 6 the pro­duc­tion of HbAT87Q was as­so­ci­at­ed with a re­duc­tion in the pro­por­tion of HbS in to­tal he­mo­glo­bin. Pa­tients had a me­di­an of ≤ 60% HbS. All pa­tients in Group C were able to stop reg­u­lar blood trans­fu­sions and re­main off trans­fu­sions at three months post-treat­ment.

What mat­ters most, though, will be the rate of com­plete res­o­lu­tion of se­vere va­so-oc­clu­sive events at 18 months post-treat­ment — which blue­bird said would ce­ment the planned BLA sub­mis­sion.

Then there’s the com­mer­cial man­u­fac­tur­ing is­sues that led to pre­vi­ous de­lays for the launch of its oth­er EU-ap­proved gene ther­a­py, Zyn­te­glo, and re­view of ide-cel.

In an earn­ings call last month, CEO Nick Leschly promised an­a­lysts that they have “learned a lot” and are pre­pared for the tran­si­tions in­to new pro­duc­tion meth­ods, in­clud­ing sus­pen­sion lentivi­ral vec­tor. “(Q)uite hon­est­ly, we would not be sign­ing up for a reg­u­la­to­ry time line or range of time line if we didn’t feel that we had the ex­e­cu­tion in­fra­struc­ture be­hind it,” he said.

Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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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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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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George Yancopoulos (Regeneron)

UP­DAT­ED: 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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Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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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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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Jan van de Winkel, Genmab CEO

Seat­tle Ge­net­ics, Gen­mab turn on TV for a high­light reel in cer­vi­cal can­cer — but a ri­val biotech promis­es a bet­ter show

Seattle Genetics $SGEN and their partners at Genmab $GMAB polished up some positive Phase II numbers for their antibody drug conjugate tisotumab vedotin — you can call it TV — for recurrent cervical cancer. And while they mapped out a shortcut to a potential quick approval, the big challenge for this team is being presented by a rival biotech which muscled its way into the spotlight for the same indication a year ago.

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