Out of jobs, a pair of ear­ly cell ther­a­py ex­ec­u­tives went to Seoul, came back with a new com­pa­ny, $70M and a plan to leapfrog nat­ur­al killer com­peti­tors

Tom Far­rell didn’t have much to do af­ter Bel­licum an­nounced in Jan­u­ary 2017 that they were bring­ing in a new CEO. He had led the CAR-T com­pa­ny for over a decade, since be­fore Carl June’s New Eng­land Jour­nal of Med­i­cine pa­per had made cell ther­a­py the hottest thing in can­cer re­search. Now he was fac­ing an 18-month non-com­pete.

So he worked quick­ly when, not long af­ter that clock ex­pired in 2018, a banker who helped take Bel­licum pub­lic told him about a South Ko­re­an com­pa­ny called Green Cross Lab­Cell that had built a nat­ur­al killer cell fac­to­ry and was look­ing to de­vel­op ther­a­pies off it. Far­rell hopped a plane to Seoul.

Tom Far­rell

It was “huge­ly im­pres­sive,” Far­rell told End­points News. “There was noth­ing [else] I came across that was tru­ly dis­rup­tive from a busi­ness mod­el per­spec­tive.”

A year and a half lat­er, Far­rell has his new com­pa­ny. Called Arti­va, it launch­es with $78 mil­lion in Se­ries A fund­ing and an ex­clu­sive deal with Green Cross to push some of their nat­ur­al killer cell tech­nol­o­gy in­to the clin­ic. They’ll start with a ther­a­py that com­bines NKs with an ap­proved an­ti­body ther­a­py like rit­ux­imab to im­prove the an­ti­body’s ef­fec­tive­ness. Be­hind that, they’re work­ing on CAR-NK ther­a­py and, longer term, gene-edit­ed CAR-NK cells. RA Cap­i­tal Man­age­ment, ven­Bio and 5AM Ven­tures led the round.

Arti­va joins what, af­ter many years, has re­cent­ly be­come a boom­ing field. In Feb­ru­ary, MD An­der­son showed that a Take­da-li­censed CAR-NK ther­a­py cleared tu­mors com­plete­ly in 7 of 11 non-Hodgkin’s lym­phoma pa­tients. Two months lat­er, J&J gave Fate Ther­a­peu­tics, one of the ear­li­est biotechs in the field, an up-to $3.1 bil­lion deal for their CAR-NK and CAR-T ther­a­pies. The Big Phar­mas are joined by a slate of re­cent up­starts, in­clud­ing Celu­lar­i­ty, Nkar­ta, Nan­tK­west, and Cy­tovia.

Un­like the oth­er new­com­ers, Arti­va makes vir­tu­al­ly no claim on hav­ing orig­i­nal sci­ence. In fact, Far­rell said, biotech’s em­pha­sis on nov­el tech­nolo­gies is part of why cell ther­a­py has ad­vanced on­ly “in­cre­men­tal­ly” since the ap­proval of the first two CAR-T ther­a­pies. In­dus­try hasn’t fo­cused enough on ad­dress­ing the man­u­fac­tur­ing is­sues that have made ther­a­pies so cost­ly and dif­fi­cult to scale, he said.

Lewis Lanier, an im­mu­nol­o­gist at the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co and an ear­ly pi­o­neer in NK cell re­search, said Arti­va would still face the same ques­tions oth­er drug de­vel­op­ers face — will some pa­tient re­ject the cells? Will the nat­ur­al killer cells ac­tu­al­ly last a sig­nif­i­cant amount of time af­ter in­fu­sion? — but the col­lab­o­ra­tion could give them an edge.

“The Ko­re­an Green Cross man­u­fac­tur­ing fa­cil­i­ty is re­al­ly first rate, that’s where the ad­van­tage is,” Lanier, who is not in­volved in Arti­va, told End­points. “The sci­ence is re­al­ly rou­tine, they’re not do­ing any­thing par­tic­u­lar­ly in­no­v­a­tive.”

For years, NK cells have been viewed as one of the key po­ten­tial ways of mak­ing off-the-shelf cell ther­a­py. Part of the in­nate im­mune sys­tem, im­plant­i­ng these cells from donors doesn’t lead to the same re­sis­tance that donor T cells can. One of the prob­lems, though, is that NKs are “finicky,” as Lanier puts it, vast­ly more dif­fi­cult to grow and ma­nip­u­late in a lab. On­ly re­cent­ly have a cou­ple com­pa­nies fig­ured out ways to do it con­sis­tent­ly. Fate, for in­stance, us­es mas­ter lines of iP­SC stem cells.

At the Green Cross fa­cil­i­ty Far­rell toured two No­vem­bers ago, the South Ko­re­an com­pa­ny had re­fined a process to de­rive NK cells from do­nat­ed um­bil­i­cal cord blood and cryo-pre­serve it. A week af­ter his tour, Far­rell flew to San Diego for the ASH con­fer­ence, where he ran in­to Pe­te Fly­nn, an­oth­er long­time biotech ex­ec­u­tive out of a job. Fly­nn had run ear­ly de­vel­op­ment for Fate in its ear­ly years be­fore leav­ing to run R&D for the an­ti-obe­si­ty com­pa­ny Orex­i­gen, which had just gone bank­rupt.

Far­rell ex­plained what he saw in Seoul and the two de­bat­ed dif­fer­ent ap­proach­es to off-the-shelf ther­a­py. They fig­ured the man­u­fac­tur­ing base could be a launch­ing pad.

“Even though we’re a Se­ries A com­pa­ny, we’re look­ing to be­come the go-to NK cell,” Fly­nn, now COO, told End­points. “Ba­si­cal­ly all the pieces are in place al­ready, where­as for some of those oth­er com­pa­nies, there might still be some work to do.”

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Sin­gu­lar fo­cus on ROR1 earns Velos­Bio $137M to fund PhI ADC and oth­er pro­grams

Years after selling Acerta to AstraZeneca for $7 billion, largely on the promise of its BTK inhibitor, Dave Johnson has once again gathered hefty financial support behind a new cancer target.

Matrix Capital Management and Surveyor Capital are leading a $137 million round for VelosBio, which has recently begun a Phase I study for its lead antibody-drug conjugate targeted against ROR1. Johnson took up the CEO post in October 2018.

Alexander Vos, VarmX CEO

'Fun­da­men­tal­ly dif­fer­en­t' from Por­to­la, Dutch biotech lands €32M to steer an­ti-an­ti­co­ag­u­lant through the clin­ic

Portola may not have had much success proving the commercial value of an anti-anticoagulant, but that’s not stopping European investors from pouring $36.2 million (€32 million) into what they see as a superior approach put forth by a Dutch biotech.

VarmX’s blood thinner reversal agent stems from research done by founder and CSO Pieter Reitsma at Leiden University Medical Center. A modified recombinant form of factor X, VMX-C001 “has an insertion of 16 amino acids that replaces a stretch of 7 amino acids in the so-called serine protease domain” compared to the native coagulation factor, CEO Alexander Vos told Endpoints News.

Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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Covid-19 roundup: Mod­er­na sticks to Ju­ly for its Phase III as ru­mors swirl; Fol­low­ing US lead, EU buys up Covid-19 treat­ments

The Phase III might be delayed from its original early July goal, but Moderna says it will still kick off the pivotal study for what could ultimately be the first Covid-19 vaccine before the end of the month.

A day after Reuters reported that squabbling between the Cambridge biotech and government regulators had held up the trial by about two weeks, Moderna released a statement saying that they had completed enrollment of their 650-person Phase II trial and were on track to begin Phase III by the end of the month. The protocol for that study, which is meant to prove whether or not the vaccine can prevent people from becoming sick, has been finalized, they said.

Adrian Gottschalk, Foghorn CEO

Mer­ck dan­gles up to $425 mil­lion to team with Flag­ship’s Foghorn Ther­a­peu­tics on drug­ging the shape of DNA

Two years after it first emerged from stealth mode, Flagship’s Foghorn Therapeutics has nabbed its first Big Pharma partner as Merck signs on to the biotech’s vision of drugging the very shape of DNA.

The deal, worth up to $425 million but with the upfront cash undisclosed, comes as Foghorn nears a pivot to a clinical stage biotech. The Cambridge-based company has added nearly 60 staffers from the 25 it had when it first emerged out of Flagship and, CEO Adrian Gottschalk said, they have finally refined the screening technology at the heart of the company, with plans to file their first IND towards the end of the year.