Cel­lec­tis signs $760M mile­stone deal with NK up­start; Im­muno­core brings on As­traZeneca vet to shep­herd bis­pe­cif­ic launch plans

Af­ter near­ly two decades as of­ten one of the on­ly com­pa­nies de­vel­op­ing off-the-shelf CAR-Ts, Cel­lec­tis has in re­cent years racked up big-name part­ner­ships, in­clud­ing with Arie Bellde­grun’s Al­lo­gene and French gi­ant Servi­er.

Now they’ve teamed with a lit­tle known start­up that has yet to an­nounce a Se­ries A: New York-based Cy­tovia Ther­a­peu­tics. The two com­pa­nies an­nounced on Tues­day a deal to de­vel­op uni­ver­sal gene-edit­ed nat­ur­al killer cells and CAR-NK cells for sol­id tu­mors and oth­er can­cers.

Cy­tovia will be re­ly­ing on tech­nol­o­gy Cel­lec­tis has built over the last 20 years as they look to build their own nat­ur­al killer cell plat­form. To make their uni­ver­sal CAR-Ts, Cel­lec­tis takes T cells and ma­nip­u­lates them with TAL­ENs, a gene edit­ing sys­tem that de­vel­oped be­fore the ad­vent of CRISPR and still has ad­van­tages in some ar­eas.

An­dré Chouli­ka

Cy­tovia will give Cel­lec­tis $15 mil­lion in cash or eq­ui­ty, with $760 mil­lion in mile­stones, to de­vel­op TAL­ENs that they can use to en­gi­neer iP­SC stem cells in­to nat­ur­al killer cell ther­a­pies. Cel­lec­tis CEO An­dré Chouli­ka said the deal was a chance to ex­pand their gene edit­ing tech­nol­o­gy in­to a new do­main.

“Down the road, this col­lab­o­ra­tion should al­low for NK cell ther­a­pies to be made avail­able to can­cer pa­tients, which is very much in line with Cel­lec­tis’ mis­sion to pro­vide life-sav­ing prod­uct can­di­dates to ad­dress un­met pa­tient needs in this field,” he said in a state­ment.

Daniel Te­per, a long­time in­vestor with Phar­mD, launched Cy­tovia in 2019, a few months be­fore re­searchers at MD An­der­son would show some of the first strong ev­i­dence that CAR-NKs could work in hu­mans. Like T cells, NK cells have the abil­i­ty to rec­og­nize and de­stroy can­cer­ous cells. Cru­cial­ly, though, they don’t trig­ger im­mune re­ac­tions when trans­ferred be­tween pa­tients, mak­ing them a po­ten­tial plat­form for off-the-shelf cell ther­a­py.

Most NK de­vel­op­ers, such as Celu­lar­i­ty, de­rive their NK cells from donor um­bil­i­cal cord blood. Cy­tovia will in­stead try to de­vel­op them in the lab from stem cells, re­ly­ing on col­lab­o­ra­tions with the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co, the New York Stem Cell In­sti­tute and now Cel­lec­tis. They are al­so de­vel­op­ing a set of an­ti­bod­ies de­signed to bridge a pa­tient’s own NK cells to their tu­mors.

The first two pro­grams are set to be in the clin­ic lat­er this year, with two more fol­low­ing in 2022. — Ja­son Mast

Im­muno­core brings on an As­traZeneca vet as bis­pe­cif­ic launch nears

As it pre­pares for the po­ten­tial com­mer­cial launch of tebenta­fusp, its I/O T cell re­cep­tor bis­pe­cif­ic, UK-based Im­muno­core has tapped an As­traZeneca vet­er­an to help shep­herd the process.

Im­muno­core has added Ralph Tor­bay as its head of com­mer­cial, hop­ing to lean on his Big Phar­ma ex­per­tise. Dur­ing a four-year stint at As­traZeneca, Tor­bay helped launch three on­col­o­gy meds, Im­muno­core said, and helped de­vel­op the drug­mak­er’s hema­tol­ogy port­fo­lio.

Tor­bay led the glob­al launch for lung can­cer med Imfinzi as well as Tagris­so, an­oth­er lung can­cer drug tar­get­ing EGFR. While serv­ing as glob­al head of hema­tol­ogy at the com­pa­ny, Tor­bay al­so helped build the com­mer­cial in­fra­struc­ture for Calquence.

Pri­or to As­traZeneca, Tor­bay led “sev­er­al high-pro­file pipeline in­te­gra­tions and prod­uct launch­es” with No­var­tis On­col­o­gy.

“The im­pres­sive qual­i­ty of da­ta al­ready pub­lished for tebenta­fusp clear­ly demon­strates its po­ten­tial as a new break­through treat­ment for metasta­t­ic uveal melanoma pa­tients, an un­der­served group where new ther­a­pies are ur­gent­ly need­ed,” Tor­bay said in a state­ment. “This is a tes­ta­ment to the ex­cel­lence of Im­muno­core’s pi­o­neer­ing sci­ence and un­der­lines the po­ten­tial of our bis­pe­cif­ic im­munother­a­pies across a range of in­di­ca­tions.”

In tan­dem with Tor­bay’s hir­ing, Im­muno­core will al­so add Roy Herb­st, a di­rec­tor at the Yale Can­cer Cen­ter, to its board of di­rec­tors. Herb­st pre­vi­ous­ly served on Im­muno­core’s sci­en­tif­ic ad­vi­so­ry board. — Kyle Blanken­ship

Ite­ri­on rais­es $17M Se­ries B for sol­id tu­mors

Ite­ri­on has spent the past few years try­ing to drug β-catenin, a com­mon onco­gene that drug de­vel­op­ers have long strug­gled to hit, be­cause it lacks an easy bind­ing site. They will now have some ex­tra cash to do so, rais­ing $17 mil­lion in a Se­ries B led by Lu­mi­ra Ven­tures.

Ite­ri­on will use the cash to ad­vance their drug, tega­vivint, in tri­als for a hand­ful of can­cers, in­clud­ing desmoid tu­mors, AML and non-small cell lung can­cer.

Rather than go af­ter β-catenin di­rect­ly, Ite­ri­on tries to block TBL1, a pro­tein β-catenin en­gages to dri­ve tu­mor growth. — Ja­son Mast

Be­yond Air wins $2M+ grant for ni­tric ox­ide treat­ment

Be­yond Air has some new grant mon­ey to play with.

The Gar­den City, NY-based com­pa­ny an­nounced Tues­day it has re­ceived a $2.17 mil­lion grant from the Cys­tic Fi­bro­sis Foun­da­tion to ad­vance de­vel­op­ment for an in­haled ni­tric ox­ide treat­ment for NTM pul­monary dis­ease. Funds will be di­rect­ed to­ward a pi­lot study of a high con­cen­tra­tion for­mu­la­tion of the pro­gram.

“We are grate­ful to re­ceive fund­ing sup­port from the Cys­tic Fi­bro­sis Foun­da­tion,” CEO Steve Lisi said in a state­ment. “To date, our NTM pro­gram has pro­duced da­ta from four com­pas­sion­ate use pa­tients and nine pa­tients from a pre­vi­ous pi­lot study.”

The new study is a 12-week, sin­gle-arm tri­al in Aus­tralia ex­pect­ed to en­roll about 20 CF or non-CF bronchiec­ta­sis pa­tients with cer­tain types of lung in­fec­tions. Be­yond Air said it ex­pects to re­port in­ter­im da­ta around the mid­dle of 2021, which will be fol­lowed by topline da­ta rough­ly six months lat­er. — Max Gel­man

Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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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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Gala­pa­gos posts a safe­ty win for fil­go­tinib, but is it too lit­tle, too late?; Bio-Techne inks $320M mol­e­c­u­lar di­ag­nos­tics buy­out

Once a promising $725 million play in immunology, Gilead’s big bet on filgotinib effectively disintegrated in December when the drugmaker reworked its partnership with Galapagos. Now, Galapagos is sporting safety data that will come as a relief — but will it make a difference on filgotinib’s chances in the US?

In a study designed to compare filgotinib’s effect on sperm count with placebo, Galapagos’ JAK inhibitor saw fewer patients post a 50% or more reduction in sperm concentration after 13 weeks of treatment, according to data from the MANTA and MANTA-RAy studies unveiled Thursday.

In the lat­est big in­vest­ment in gene ther­a­py man­u­fac­tur­ing, Bio­gen com­mits $200M to a ma­jor new fa­cil­i­ty in NC

You’d be forgiven for thinking that the only R&D effort of any consequence at Biogen belongs to aducanumab, its controversial Alzheimer’s drug. But behind the uproar around that drug, the big biotech has a full scale pipeline in play that includes a growing focus on developing gene therapies.

Now Biogen plans to build up the kind of manufacturing muscle that will give it an advantage in gaining FDA approvals — where CMC is always key — and then marketing them around the world.

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Eli Lil­ly claims a TKO in its long-run­ning ti­tle fight with No­vo Nordisk for the block­buster di­a­betes mar­ket — but there’s a hitch

Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now.

Let’s start with the latest data from Lilly.

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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.

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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Cedric Francois, Apellis CEO (Apellis)

Apel­lis joins the grow­ing num­ber of bio­phar­mas scrap­ping a failed Covid-19 pro­gram af­ter an ear­ly flop

The global pandemic set off a frenzy of R&D activity as biotechs around the world scrambled to see if they could come up with a new medication or vaccine to help fight back. But even as the mRNA standouts are highlighting the market El Dorado open to successful teams, the failures are starting to pile up.

Thursday afternoon it was Apellis’ $APLS turn to deep-six a new drug.

The biotech reports that their C3 therapy APL-9 had failed to move the needle on mortality when combined with standard of care, as compared to SOC alone.