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.”

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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Jason Foster, Ori Biotech CEO

UK up­start rais­es $100M in bid to dig­i­tize and stan­dard­ize cell and gene ther­a­py man­u­fac­tur­ing

There’s a giant need for cell and gene therapy manufacturing options going forward, as companies move to invest massively into that space. Ori Biotech is the latest, as the UK-based biotech announced it has secured more than $100 million in its oversubscribed Series B funding round Tuesday.

Novalis LifeSciences led the round. The Boston-based company is led by chairman Marijn Dekkers and partner Paul Meister, who built Thermo Fisher Scientific. Dekkers went on to become the CEO of Bayer, and is currently also the chairman at Ginkgo Bioworks.