Deaths de­rail Juno's launch count­down, giv­ing Kite and No­var­tis the lead

The FDA’s hold on Juno Ther­a­peu­tics’ lead CAR-T pro­gram, JCAR015, last­ed on­ly six days. But the de­rail­ment was se­ri­ous enough to push its ex­pect­ed ap­proval date from 2017 back in­to 2018, leav­ing Kite Phar­ma and No­var­tis an­gling for the first ap­provals in the field in 2017.

Juno CEO Hans Bish­op

“Re­gard­ing the ROCK­ET tri­al,” Juno CEO Hans Bish­op told an­a­lysts Thurs­day evening, “the process of get­ting IRB ap­proval across mul­ti­ple sites along with the gat­ed en­roll­ment for the next six pa­tients leaves us to now es­ti­mat­ing ap­proval as ear­ly as the first half of 2018.”

The new H1 2018 pro­jec­tion marks a set­back for Juno, which had been seen as run­ning neck and neck with Kite in the race to get the land­mark ap­proval for a new can­cer ther­a­py that takes cells from pa­tients and reengi­neers them in­to a can­cer cell at­tack ve­hi­cle.

The hold last month, fol­low­ing the death of sev­er­al pa­tients from lethal cas­es of cere­bral ede­ma, stunned long­time ob­servers of Juno. The biotech quick­ly and suc­cess­ful­ly ap­pealed to the agency to lift the hold, say­ing they be­lieved that adding flu­dara­bine to its pre­con­di­tion­ing reg­i­men for pa­tients — prep­ping them to bet­ter re­spond to their CAR-T — had cre­at­ed a tox­ic com­bi­na­tion with the ther­a­py, killing 4 pa­tients. Reg­u­la­tors, who had al­so placed the same ther­a­py briefly on hold af­ter cy­tokine re­lease syn­drome al­so killed some pa­tients ear­ly on, were quick to re­spond af­fir­ma­tive­ly.

The de­lay leaves JCAR015 poised to en­ter the mar­ket just a year ahead of JCAR017. Bish­op de­scribes the sec­ond drug as “the back­bone of our CD19 fran­chise,” which is aimed at NHL, pe­di­atric and adult ALL and CLL.

Juno, though, is al­so mov­ing a va­ri­ety of pro­grams across a broad R&D front, and is now go­ing af­ter mul­ti­ple myelo­ma in a new pact that ropes in long­time col­lab­o­ra­tors at Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter.

In a sep­a­rate press re­lease, the Seat­tle-based biotech an­nounced Thurs­day evening that it had struck a deal with MSK and Eu­re­ka Ther­a­peu­tics for IP on bind­ing do­mains need­ed to com­mer­cial­ize a CAR-T for mul­ti­ple myelo­ma. These bind­ing do­mains were de­vel­oped in a pact that Eu­re­ka and MSK had struck ear­li­er.

That deal is par­tic­u­lar­ly sig­nif­i­cant, as it in­volves a ful­ly-hu­man bind­ing do­main tar­get­ing B-cell mat­u­ra­tion anti­gen (BC­MA), along with bind­ing do­mains against two ad­di­tion­al undis­closed mul­ti­ple myelo­ma tar­gets. The BC­MA pact puts Juno on a po­ten­tial col­li­sion course with blue­bird bio, which al­so chose that tar­get in a col­lab­o­ra­tion its pur­su­ing with Cel­gene. Those two play­ers re­struc­tured their deal last year to con­cen­trate on BC­MA and Cel­gene lat­er signed a ma­jor pact with Juno as well.

Pfiz­er and Cel­lec­tis are al­so aim­ing at BC­MA in their col­lab­o­ra­tion, but they’re con­cen­trat­ing on a sec­ond-gen, off-the-shelf prod­uct.

Juno CSO Hy Lev­it­sky

“We ex­pect the BC­MA CAR to en­ter hu­man test­ing as ear­ly as the first half 2017,” Juno CSO Hy Lev­it­sky told an­a­lysts. “We’re op­ti­mistic the CAR T cell ther­a­py can be an im­por­tant com­po­nent in treat­ing pa­tients with mul­ti­ple myelo­ma. And we are pleased to bring ad­di­tion­al ful­ly hu­man body do­main against BC­MA and oth­er tar­gets in­to our pro­gram. We be­lieve that a mul­ti-pronged ap­proach may be nec­es­sary to treat this dis­ease and hence the im­por­tance of ac­cess to sev­er­al hu­man con­structs spe­cif­ic for more than one tar­get.”

This isn’t the first such pact that Juno and Eu­re­ka have struck. Back at the be­gin­ning of the year the two com­pa­nies struck a deal on a ful­ly hu­man bind­ing do­main that tar­gets MUC16. These bind­ing do­mains play a key role in im­prov­ing cell per­sis­tence, amp­ing up their ef­fect on pa­tients. And the in­tense ri­val­ry to dom­i­nate the first wave of com­mer­cial CAR-Ts to hit the mar­ket has spurred an arms race for the best tech with the most po­ten­tial.

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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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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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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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.