Fac­tor Bio­science spins out a new cell ther­a­py play­er with eyes on the clin­ic with­in 2 years

The quest for CAR-T 2.0 is gain­ing an mR­NA play­er, as Cam­bridge, Mass­a­chu­setts-based Fac­tor Bio­science sends a spin­off rac­ing to­ward the clin­ic.

Gre­go­ry Fiore

Fac­tor drew the cur­tains on Exacis Bio­ther­a­peu­tics on Wednes­day morn­ing, with Sol­lis Ther­a­peu­tics co-founder Gre­go­ry Fiore at the helm of a small im­muno-on­col­o­gy fo­cused team built around Fac­tor’s tech­nol­o­gy. The spin­off has the rights to 51 patents and just a bit of seed mon­ey from friends and fam­i­ly to get it go­ing — but Fiore says an IND sub­mis­sion is on the hori­zon.

“We are 18 to 24 months from an IND sub­mis­sion, and we’ve iden­ti­fied our first tar­get, which will be CD19,” Fiore told End­points News. 

The com­pa­ny will be un­veil­ing a CD19-tar­get­ed CAR-T and CAR-NK, Fiore said, with ROR1 as its next tar­get.

The CEO says Exacis’ ap­proach is what dif­fer­en­ti­ates it from oth­ers in the crowd­ed cell ther­a­py field, be­gin­ning with mR­NA tech­nol­o­gy in-li­censed from Fac­tor. The process starts with in­duced pluripo­tent stem cells (iP­SC), which are blood or skin cells that have been en­gi­neered back in­to an em­bry­on­ic-like stem cell state. They’re cre­at­ed with mR­NA re­pro­gram­ming, and then edit­ed to avoid host im­mune sur­veil­lance, add a CAR and en­hance the cells for po­ten­cy against tu­mors.

“That iP­SC is quite a ro­bust cell. It can han­dle a lot of edit­ing and the cells are able to re­cov­er from a lot of edit­ing and ma­nip­u­la­tion,” Fiore said. And the fact that no virus­es or DNA are used “sig­nif­i­cant­ly de­creas­es the re­source re­quire­ment for man­u­fac­tur­ing,” he added lat­er.

The idea of an off-the-shelf CAR-T or CAR-NK ther­a­py — as op­posed to har­vest­ing a pa­tient’s cells, en­gi­neer­ing them in­to a can­cer at­tack ve­hi­cle and rein­ject­ing them — isn’t a new one. Al­lo­gene re­leased a pos­i­tive snap­shot of their off-the-shelf CAR-T pro­gram at AS­CO 2020, and CRISPR Ther­a­peu­tics of­fered a glimpse at their own CAR-T suc­cess in Oc­to­ber al­though it was cloud­ed by the death of a pa­tient giv­en a high dose of the treat­ment.

Dim­itrios Goundis

Exacis’ team of four — in­clud­ing co-founder James Pan and for­mer Max­i­VAX CEO Dim­itrios Goundis as CBO — is shoot­ing for a Se­ries A in the com­ing months to bol­ster its team and pipeline. The com­pa­ny al­so says it’s in talks with sev­er­al po­ten­tial de­vel­op­ment part­ners.

“We are work­ing to­wards a Se­ries A fund­ing to be com­plet­ed in Q1 of 2021, and we’ll use those funds to build out our in­ter­nal team and lab, as well as fur­ther the de­vel­op­ment along the lines of dif­fer­en­ti­a­tion in­to T and NK, ob­tain­ing CARs, re­al­ly putting to­geth­er these tar­get cell types,” Fiore said.

While Fiore stayed mum about the the spe­cif­ic terms of Exacis’ li­cens­ing deal with Fac­tor, he said that Fac­tor has a ma­jor­i­ty own­er­ship in ex­change for the ex­e­cu­tion of the li­cense.

The CEO, who was in­spired to get in­to the field by his fa­ther’s bat­tle with can­cer, said Fac­tor and Exacis’ in­cen­tives were aligned. “There’s plen­ty of op­por­tu­ni­ty to im­prove the pa­tient ex­pe­ri­ence as well as out­comes,” he said.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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Jean-Christophe-Hyvert, Lonza

Lon­za look­ing to build on 'd­if­fer­en­ti­at­ed ad­van­tage' in Covid-19, CD­MO mar­ket­place in 2021

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.

News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.

Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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