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.

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; 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.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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Mol­e­c­u­lar Di­ag­nos­tics Can Trans­form Can­cer Care. Let’s Make It Hap­pen.

Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States.

Mod­er­na es­tab­lish­es pub­lic health-fo­cused char­i­ty; FDA ap­proves As­traZeneca di­a­betes drug for pe­di­atric use

To help promote public health and healthcare in underserved areas of the world, Moderna will establish a charity with a $50 million endowment.

The Cambridge, MA-based company announced the board of directors’ approval Thursday. The foundation will focus on “charitable, scientific and educational endeavors” with an emphasis on promoting public health and the access to healthcare, the press release said. The foundation will start operations once its status as a 501(c)(3) is approved.