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.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; 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.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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