RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Bio­phar­ma is muscling up.

Robert Ang

CEO Robert Ang, who was re­luc­tant to di­vulge the head­count when dis­cussing his move from Neon Ther­a­peu­tics to Vor last Au­gust, read­i­ly of­fered that the team has grown from 6 to 50 in less than a year. The biotech is mov­ing to a larg­er of­fice on Cam­bridge Park­way Dri­ve in weeks, giv­ing it more space to com­plete the IND-en­abling work and man­u­fac­tur­ing scale-up — con­duct­ed by a CD­MO part­ner — in prepa­ra­tion for clin­i­cal tri­als planned for the first half of 2021.

It’s al­so hauled in $110 mil­lion from its Se­ries B, led by RA Cap­i­tal Man­age­ment and fea­tur­ing new in­vestors like Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, Pagli­u­ca Fam­i­ly Of­fice and Alexan­dria Ven­ture In­vest­ments. 5AM Ven­tures, which co-led the $42 mil­lion Se­ries A with RA Cap­i­tal, al­so joined along­side old-timers PureTech Health, John­son & John­son In­no­va­tion — JJDC and Os­age Uni­ver­si­ty Part­ners.

“Vor has an el­e­gant ap­proach to en­gi­neer­ing hematopoi­et­ic stem cells that we be­lieve is amongst the most promis­ing in­no­va­tions in on­col­o­gy,” Joshua Resnick, man­ag­ing di­rec­tor at RA Cap­i­tal, said in a state­ment.

Sid­dhartha Mukher­jee

Pi­o­neered by Co­lum­bia on­col­o­gist Sid­dhartha Mukher­jee (who has chron­i­cled the promise and price of the first gen­er­a­tion of CAR-T ther­a­pies), the ap­proach flips tra­di­tion­al cell ther­a­py on its head. Rather than en­gi­neer­ing im­mune cells to at­tack can­cer­ous ones, or tar­get­ing tu­mor cells, Vor is tin­ker­ing with the healthy cells.

By edit­ing out tar­gets that are present in both healthy and ma­lig­nant cells, the rea­son­ing goes, sci­en­tists can un­leash the full pow­er of tar­get­ed ther­a­pies with­out hav­ing to wor­ry about side ef­fects.

They are start­ing out with CD33 for acute myeloid leukemia, but the new cash will fund some new re­search in­to oth­er undis­closed tar­gets.

Al­so known as Siglec-3, the pro­tein is a com­mon tar­get for ex­ist­ing treat­ments like Pfiz­er’s an­ti­body-drug con­ju­gate My­lotarg. Large ge­nom­ic data­bas­es sug­gest there are in­di­vid­u­als who have lived their whole lives with­out ever ex­press­ing the CD33, Ang said, and re­searchers have shown that knock­ing it out in mice and hu­man cells in vit­ro doesn’t seem to im­pact func­tion­al­i­ty.

The re­al test, of course, will have to be in the clin­ic.

“We hope to see that it’s en­tire­ly bi­o­log­i­cal­ly re­dun­dant and safe,” Ang told End­points News.

On top of that — prov­ing not on­ly that they can ed­it the hematopoi­et­ic stem cells with­out in­duc­ing ma­lig­nan­cy, but al­so re­tain their re­pro­ducibil­i­ty — Vor is al­so aim­ing to test how their pro­gram, VOR33, may com­ple­ment My­lotarg or a CD33-tar­get­ed CAR-T ther­a­py, which would be a “nov­el nov­el treat­ment com­bi­na­tion.”

Vor has had a chance to dis­cuss these and oth­er con­cerns with FDA reg­u­la­tors back in Jan­u­ary, Ang added.

The tri­al would al­so be a test for the man­u­fac­tur­ing process Vor has de­vel­oped, pack­ing every­thing from ex­trac­tion to re­in­fu­sion in 48 hours.

“We’re do­ing it not just be­cause we can do it, but al­so be­cause I think it’s best for pa­tients that we do it,” Ang said. Not on­ly is there an ur­gency in the clin­ic, but less ma­nip­u­la­tion al­so means bet­ter chance of en­graft­ment and health­i­er cells. “What that al­so does is it gives us hope­ful­ly flex­i­bil­i­ty fur­ther down the track to think about how it could, more im­por­tant­ly, roll us out on a late de­vel­op­ment or com­mer­cial ba­sis.”

It could mean a re­gion­al mod­el or on-site man­u­fac­tur­ing; ei­ther way, it’s a de­cen­tral­ized fu­ture that Vor is now prepar­ing for.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Steve Harr, Sana Biotechnology CEO

Four years in, Sana gets first FDA go-ahead to bring can­cer treat­ment in­to the clin­ic

Sana Biotechnology is finally headed to the clinic.

Thursday afternoon, the biotech announced the FDA had cleared its application to start a clinical trial for its allogeneic, or “off-the-shelf,” CAR-T cell therapy targeting the antigen CD19 for patients with B-cell lymphomas and leukemias. Sana said its therapy, dubbed SC291, was designed to evade the immune system, which could help cell therapy produce a more durable response in patients, a concern that has followed such off-the-shelf therapies that use donor cells as opposed to a patient’s own cells.

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Ying Huang, Legend CEO

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.

Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.