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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.