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.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

Thank you, next: Take­da hands Ovid $196M cash to rein back in Phase III-ready seizure drug, re­viv­ing bat­tered stock

Soticlestat made it.

Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones.

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Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepreneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film “Fantastic Voyage,” the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his and his co-founders’ own accounts, along with some seed cash from friends and family.

Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.