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.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

Top Har­vard chemist caught up in FBI’s 'T­hou­sand Tal­ents' drag­net, ac­cused of ly­ing about Chi­nese con­nec­tions, pay

The FBI’s probe into the alleged theft of R&D secrets by Chinese authorities has drawn Harvard’s top chemist into its net.

The agency accused Charles M. Lieber, who chairs the university’s chemistry and chemical biology department, with lying about his involvement in China’s Thousand Talents campaign, which was established as a way of drawing in innovators from around the world. And the scientist, 60, was charged with making false statements about his ties to China.

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Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Passage Bio — Jim Wilson’s self-described “legacy company” — has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.

UP­DAT­ED: Ac­celeron res­ur­rects block­buster hopes for so­tater­cept with pos­i­tive PhII — and shares rock­et up

Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

“We’re thrilled to report such positive topline results from the PULSAR trial,” Acceleron CEO Habib Dable said in a statement. The company said in a conference call they plan on discussing a Phase III trial design with regulators.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”