Siddhartha Mukherjee, AP Images

Sid Mukher­jee's Vor taps a CAR-T de­vel­op­ment part­ner to strap to its at­tempt at bet­ter stem cell trans­plants

In the buzzy world of on­col­o­gy, few names shine brighter than Co­lum­bia on­col­o­gist and best­selling au­thor Sid­dhartha Mukher­jee, whose Vor Bio­phar­ma is look­ing to rewrite the rules of stem cell trans­plants. If his team’s tech works as planned, it would al­so rewrite the rules on how CAR-Ts can be de­ployed in blood can­cer — and now it’s part­ner­ing up with a lit­tle-known biotech to test that hy­poth­e­sis.

Robert Ang

Vor has inked a mul­ti-year dis­cov­ery deal with Abound Bio to ac­cess its plat­form of sin­gle- and mul­ti-tar­get CAR-Ts to com­bine with Vor’s pipeline of en­gi­neered blood stem cells, the part­ners said Thurs­day.

The biotechs will ini­tial­ly tar­get acute myeloid leukemia and think that a mul­ti-tar­get­ed CAR-T paired with an HSC trans­plant could be a path to bet­ter treat­ing hard-to-hit blood can­cer. The Pitts­burgh-based on­col­o­gy play­er has shown pre­clin­i­cal da­ta for a CD19/20-tar­get­ed CAR that caught Vor’s in­ter­est, CEO Robert Ang told End­points News.

Fi­nan­cial terms of the deal were not dis­closed.

The prob­lem with us­ing mul­ti-tar­get­ed CARs for years has been the off-tar­get side ef­fects on healthy blood cells. Vor is work­ing on trans­plant­i­ng stem cells in­to pa­tients’ bone mar­row that re­move or mod­i­fy the tar­get­ed anti­gens, the­o­ret­i­cal­ly al­low­ing a CAR con­struct to bet­ter tar­get tu­mors with­out the dele­te­ri­ous safe­ty risks.

Ang was un­equiv­o­cal when talk­ing about Vor’s aims: The biotech is look­ing to com­plete­ly re­place stan­dard-of-care stem cell trans­plants with its plat­form. While he couldn’t dis­cuss what fu­ture mile­stones look like for the Abound deal, Ang said the de­vel­op­ment phase of that pact would run “in par­al­lel” with Vor’s work on its stem cell plat­form.

“(Abound) is re­al­ly a team that’s al­ready demon­strat­ed sci­en­tif­ic prowess in this are­na, and we think it’s a mar­velous op­por­tu­ni­ty to work with them to­wards tar­gets that we can uti­lize in our own ap­proach,” Ang said.

John Mel­lors

Abound Bio was co-found­ed back in 2019 by Dimiter “Mitko” Dim­itrov, the com­pa­ny’s chief sci­en­tif­ic of­fi­cer and a se­nior in­ves­ti­ga­tor at the Na­tion­al Can­cer In­sti­tute, and CEO John Mel­lors. CAR-T ther­a­py re­searcher Ri­mas Orentas serves as the biotech’s VP for cel­lu­lar ther­a­py. On top of its nov­el CD19/20 bis­pe­cif­ic CAR-T, the team re­cent­ly post­ed pre­clin­i­cal da­ta on a trispe­cif­ic CAR con­struct that could show promise far down the road.

Vor, mean­while, is the brain­child of Mukher­jee, who found­ed the com­pa­ny back in 2016 and even­tu­al­ly took it pub­lic in Feb­ru­ary. The com­pa­ny’s lead pro­gram is VOR33, an HSC the biotech is study­ing so­lo and as a pair­ing with CD33 tar­get­ing CAR-T can­di­date VCAR33, which it in-li­censed from the NIH, in IND en­abling stud­ies in AML.

Ang was quick to point out the Abound deal wasn’t to be viewed as an in­ter­rup­tion or re­place­ment for the VOR33/VCAR33 pro­gram, where ini­tial da­ta is ex­pect­ed lat­er in the year to ear­ly next year.

“We’ve worked re­al­ly hard on one tar­get, and that’s CD33 … but to take this to an­oth­er lev­el to look at tar­gets out­side of CD33 and to look at tar­get­ing mul­ti­ple epi­topes … we think that is a re­al­ly at­trac­tive ap­proach,” Ang said.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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Harith Rajagopalan (Fractyl)

Af­ter a decade in the Type 2 di­a­betes game, Fractyl Lab­o­ra­to­ries recharges with a fresh $100M and a new name

Harith Rajagopalan compared the way Type 2 diabetes is managed to sticking your fingers in a dam that’s leaking from a number of places.

You can take drugs to lower your blood sugar, cholesterol, or blood pressure, but you’re not addressing what he says is the core issue — the metabolic abnormality that causes the disease.

“We’re so busy plugging the holes in the dam, we don’t have time to see that the whole infrastructure is at risk,” he said. “That infrastructure is a full-body systemic metabolic abnormality called metabolic syndrome, that we’re ignoring while we’re so busy trying to treat all of the individual symptoms of the condition.”

Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia spends Soft­Bank's cash in bid to edge out AI ri­vals

Exscientia is sprinting to win the great AI biotech race.

The UK company, having long labored on small discovery deals with large pharmas, raised up to $525 million in a Series D led by the infamous Japanese conglomerate SoftBank in April and followed it up less than a month later with a Bristol Myers Squibb deal that paid $50 million cash and $1.2 billion in milestones.

Now, the Oxford spinout is splurging on a shiny new tool. On Monday they announced they purchased the three-year-old molecule-screening biotech Allcyte, a longtime collaborator, for $60.6 million in cash and stock.

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Alexander Vos, VectorY CEO

Start­ing fresh in man­u­fac­tur­ing, For­bion start­up re­fu­els to steer next-gen gene ther­a­py ap­proach for ALS, Alzheimer's in­to clin­ic

Forbion laid out its case for a next-generation gene therapy approach when it took the wraps off VectorY Therapeutics and its vectorized antibody tech in February. Now, the Dutch VC has tapped an experienced hand at cell and gene therapy manufacturing to steer the ship — and pulled a marquee syndicate for a €31 million ($37.6 million) seed round.

Alexander Vos, the new CEO, is a venture partner at BioGeneration Ventures and jumps immediately from VarmX, a BGV portfolio company developing an anticoagulant. But before that, he had led Dutch CDMO PharmaCell for eight years until it was bought out by Lonza.

Lynn Fitch, Mississippi Attorney General (Rogelio V. Solis/AP Images)

Mis­sis­sip­pi sues Eli Lil­ly, Sanofi and No­vo over in­sulin prices as in­ter­change­able biosim­i­lars may ar­rive soon

Mississippi Attorney General Lynn Fitch last week sued the top three insulin manufacturers, which collectively cover almost the entire US insulin market, alleging that they’ve colluded to raise their prices in lockstep, and in some cases by more than 1,000% for drugs that are decades old.

“Because of Manufacturer Defendants’ collusive price increases, nearly a century after the discovery of insulin, diabetes medications have become unaffordable for many diabetics,” the lawsuit says.

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