Re­gen­eron leaps in­to the block­buster race to de­vel­op off-the-shelf im­mune cell can­cer ther­a­pies

A year ago, Sanofi agreed to pay Re­gen­eron $1.8 bil­lion-plus to part­ner on its an­ti-PD1 check­point pro­gram. To­day, biotech heavy­weight Re­gen­eron is jump­ing in­to CAR-Ts and TCR tech, de­ter­mined to leap di­rect­ly in­to a block­buster brawl with a plan to emerge as a leader in the fast-grow­ing im­muno-on­col­o­gy field. And it’s made the lat­est in a hand­ful of rare biotech deals to part­ner on the tech­nol­o­gy it needs.

Re­gen­eron se­lect­ed the start­up Adicet Bio for its col­lab­o­ra­tion. A new face in im­muno-on­col­o­gy, the biotech popped up on­ly last Jan­u­ary with a hefty $51 mil­lion A round to get start­ed. But it’s helmed by Aya Jakobovits, the found­ing pres­i­dent and CEO of Kite Phar­ma, a leader in de­vel­op­ing per­son­al­ized CAR-Ts.

Aya Jakobovits, Adicet Bio

If it was any oth­er biotech oth­er than Re­gen­eron, the news might spur a lit­tle mock­ing from some of the side­line ob­servers who have been watch­ing the fron­trun­ners present a slate of top per­son­al­ized CAR-Ts down the fi­nal stretch to a like­ly near-term set of ap­provals. It may ap­pear late to the par­ty, but Re­gen­eron is a top in­dus­try R&D play­er; a ma­jor biotech out­fit that is well fi­nanced, re­lent­less­ly fo­cused with a track record that now in­cludes a list of ma­jor drugs that are ei­ther on the mar­ket or close to it. And it’s look­ing to catch the next wave form­ing off­shore of I/O.

Jakobovits gets a mod­est $25 mil­lion up­front to get start­ed on en­gi­neer­ing a pipeline of im­mune cells with chimeric anti­gen re­cep­tors and T cell re­cep­tors, a one-two ap­proach aimed at ze­ro­ing in on both sur­face anti­gens found specif­i­cal­ly on tar­get­ed can­cer cells as well as in­tra­cel­lu­lar tar­gets. There’s a sup­port pack­age for re­search as well, with ad­di­tion­al funds to back up the col­lab­o­ra­tion, but no one is dis­clos­ing any of that this morn­ing.

Both com­pa­nies em­pha­size that they’re con­cen­trat­ing on off-the-shelf drugs that will be pri­mar­i­ly fo­cused on sol­id tu­mors, the new new thing in im­muno-on­col­o­gy where first-gen hema­to­log­i­cal im­munother­a­pies have en­coun­tered hard bar­ri­ers.

“There’s a lot of room left to wind up as a leader,” says Michael Aber­man, the strat­e­gy ex­ec at Re­gen­eron. His com­pa­ny wasn’t the first to tack­le check­points, nor the first in cell ther­a­pies. But “it’s very ear­ly in the first wave to know how that’s go­ing to play out. There’s a lot of chal­lenges left ahead and a lot of op­por­tu­ni­ties. And we’re talk­ing a nov­el ap­proach.”

The pact with Re­gen­eron cov­ers a “broad pipeline,” Jakobovits tells End­points, em­pha­siz­ing that the key to cre­at­ing a safe, ef­fec­tive im­mune cell ther­a­py is be­ing “spe­cif­ic to the tu­mor tar­get.” But Jakobovits, who set up the com­pa­ny as a part­ner at Or­bimed, is very care­ful to stay in­side some nar­row bound­aries on ex­plain­ing ex­act­ly what dis­tin­guish­es their work.

I/O in­vest­ment spe­cial­ist Brad Lon­car says the deal high­lights some key points. Re­gen­eron, he says:

1) Sees the promise in cell ther­a­py, which not all com­pa­nies do. For ex­am­ple, Gilead’s CEO said the idea makes him very un­com­fort­able. Many bears be­lieve it will nev­er be com­mer­cial­ly vi­able. So for a com­pa­ny with the sci­en­tif­ic cred­i­bil­i­ty of Re­gen­eron to take a small dive in says some­thing.

2) Un­der­stands that it’s too late to be a play­er in the au­tol­o­gous (per­son­al­ized) ap­proach or doesn’t be­lieve it can be eco­nom­i­cal­ly vi­able. All of the com­mer­cial op­por­tu­ni­ty has al­ready been tak­en up by ex­ist­ing play­ers.

3) Be­lieves the off the shelf ap­proach is sci­en­tif­i­cal­ly vi­able, which not every­one does.

I’d say the main point is that it’s a good sign for the whole field of cell ther­a­py when a com­pa­ny as cred­i­ble as Re­gen­eron feels like they need to be a play­er in this area. Most peo­ple don’t ap­pre­ci­ate the fact that cell ther­a­py is ba­si­cal­ly ver­sion 1.0 right now and there are many im­prove­ments that will be made over the com­ing years. It has the chance to be very spe­cial and I’m sure Re­gen­eron sees that.

A tech­nol­o­gy arms race of a kind has tak­en shape as var­i­ous com­pa­nies sort out how off-the-shelf ther­a­pies can even­tu­al­ly re­place the first gen­er­a­tion lab-craft­ed per­son­al­ized ther­a­pies that ex­tract cells from pa­tients, reengi­neer them in­to can­cer treat­ments and the in­fuse them back in­to pa­tients. Just days ago Jakobovits’s for­mer com­pa­ny Kite ex­e­cut­ed a deal with UCLA for al­lo­gene­ic tech from the lab of Gay M. Crooks.

Re­gen­eron’s not-so-se­cret weapon in the new front on can­cer in­cludes its unique Ve­locIm­mune mouse mod­els, which are de­scribed as “the largest mam­malian ge­net­ic en­gi­neer­ing project ever ac­com­plished.” It helped them de­vel­op REGN1979, a bis­pe­cif­ic an­ti­body ther­a­py that R&D chief George Yan­copou­los has high­light­ed ex­cit­ed­ly for its abil­i­ty to tar­get the B cell mark­er, CD20, and the CD3 com­po­nent of the T cell re­cep­tor, which trig­gers redi­rect­ed killing of B cells. Its an­ti-PD-1 an­ti­body is REGN2810. Any tar­get­ing mol­e­cules that come out of this new col­lab­o­ra­tion can be redi­rect­ed in­to any oth­er pro­grams Re­gen­eron has, in­clud­ing its pact with Sanofi.

Re­gen­eron doesn’t do many of these pacts, says Aber­man. There was the In­tel­lia deal back in April that brings CRISPR-Cas9 gene edit­ing tech, Avalanche’s gene ther­a­py pact, and now Adicet. But they ex­pect a lot out of the few deals they do with the se­lect biotechs they want to work with.

For Jakobovits, the deal al­so al­lows her to push the quick ex­pan­sion of he new biotech. The staff of 14 is slat­ed to dou­ble, with ad­di­tion­al in­put com­ing from her Is­raeli sub­sidiary.

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,000+ biopharma pros reading Endpoints daily — and it's free.

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.
Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.
JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.
As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.
Here’s what physicians and patients are likely to be confronted with in the not too distant future, as Pfizer goes about the tricky business of getting a JAK inhibitor past regulators at the FDA and EMA.
Lined up side by side we see:
IGA response rate (clear or nearly clear skin)

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,000+ biopharma pros reading Endpoints daily — and it's free.

First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,000+ biopharma pros reading Endpoints daily — and it's free.

Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,000+ biopharma pros reading Endpoints daily — and it's free.

UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,000+ biopharma pros reading Endpoints daily — and it's free.

A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,000+ biopharma pros reading Endpoints daily — and it's free.

Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 62,000+ biopharma pros reading Endpoints daily — and it's free.