Cel­lec­tis signs $760M mile­stone deal with NK up­start; Im­muno­core brings on As­traZeneca vet to shep­herd bis­pe­cif­ic launch plans

Af­ter near­ly two decades as of­ten one of the on­ly com­pa­nies de­vel­op­ing off-the-shelf CAR-Ts, Cel­lec­tis has in re­cent years racked up big-name part­ner­ships, in­clud­ing with Arie Bellde­grun’s Al­lo­gene and French gi­ant Servi­er.

Now they’ve teamed with a lit­tle known start­up that has yet to an­nounce a Se­ries A: New York-based Cy­tovia Ther­a­peu­tics. The two com­pa­nies an­nounced on Tues­day a deal to de­vel­op uni­ver­sal gene-edit­ed nat­ur­al killer cells and CAR-NK cells for sol­id tu­mors and oth­er can­cers.

Cy­tovia will be re­ly­ing on tech­nol­o­gy Cel­lec­tis has built over the last 20 years as they look to build their own nat­ur­al killer cell plat­form. To make their uni­ver­sal CAR-Ts, Cel­lec­tis takes T cells and ma­nip­u­lates them with TAL­ENs, a gene edit­ing sys­tem that de­vel­oped be­fore the ad­vent of CRISPR and still has ad­van­tages in some ar­eas.

An­dré Chouli­ka

Cy­tovia will give Cel­lec­tis $15 mil­lion in cash or eq­ui­ty, with $760 mil­lion in mile­stones, to de­vel­op TAL­ENs that they can use to en­gi­neer iP­SC stem cells in­to nat­ur­al killer cell ther­a­pies. Cel­lec­tis CEO An­dré Chouli­ka said the deal was a chance to ex­pand their gene edit­ing tech­nol­o­gy in­to a new do­main.

“Down the road, this col­lab­o­ra­tion should al­low for NK cell ther­a­pies to be made avail­able to can­cer pa­tients, which is very much in line with Cel­lec­tis’ mis­sion to pro­vide life-sav­ing prod­uct can­di­dates to ad­dress un­met pa­tient needs in this field,” he said in a state­ment.

Daniel Te­per, a long­time in­vestor with Phar­mD, launched Cy­tovia in 2019, a few months be­fore re­searchers at MD An­der­son would show some of the first strong ev­i­dence that CAR-NKs could work in hu­mans. Like T cells, NK cells have the abil­i­ty to rec­og­nize and de­stroy can­cer­ous cells. Cru­cial­ly, though, they don’t trig­ger im­mune re­ac­tions when trans­ferred be­tween pa­tients, mak­ing them a po­ten­tial plat­form for off-the-shelf cell ther­a­py.

Most NK de­vel­op­ers, such as Celu­lar­i­ty, de­rive their NK cells from donor um­bil­i­cal cord blood. Cy­tovia will in­stead try to de­vel­op them in the lab from stem cells, re­ly­ing on col­lab­o­ra­tions with the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co, the New York Stem Cell In­sti­tute and now Cel­lec­tis. They are al­so de­vel­op­ing a set of an­ti­bod­ies de­signed to bridge a pa­tient’s own NK cells to their tu­mors.

The first two pro­grams are set to be in the clin­ic lat­er this year, with two more fol­low­ing in 2022. — Ja­son Mast

Im­muno­core brings on an As­traZeneca vet as bis­pe­cif­ic launch nears

As it pre­pares for the po­ten­tial com­mer­cial launch of tebenta­fusp, its I/O T cell re­cep­tor bis­pe­cif­ic, UK-based Im­muno­core has tapped an As­traZeneca vet­er­an to help shep­herd the process.

Im­muno­core has added Ralph Tor­bay as its head of com­mer­cial, hop­ing to lean on his Big Phar­ma ex­per­tise. Dur­ing a four-year stint at As­traZeneca, Tor­bay helped launch three on­col­o­gy meds, Im­muno­core said, and helped de­vel­op the drug­mak­er’s hema­tol­ogy port­fo­lio.

Tor­bay led the glob­al launch for lung can­cer med Imfinzi as well as Tagris­so, an­oth­er lung can­cer drug tar­get­ing EGFR. While serv­ing as glob­al head of hema­tol­ogy at the com­pa­ny, Tor­bay al­so helped build the com­mer­cial in­fra­struc­ture for Calquence.

Pri­or to As­traZeneca, Tor­bay led “sev­er­al high-pro­file pipeline in­te­gra­tions and prod­uct launch­es” with No­var­tis On­col­o­gy.

“The im­pres­sive qual­i­ty of da­ta al­ready pub­lished for tebenta­fusp clear­ly demon­strates its po­ten­tial as a new break­through treat­ment for metasta­t­ic uveal melanoma pa­tients, an un­der­served group where new ther­a­pies are ur­gent­ly need­ed,” Tor­bay said in a state­ment. “This is a tes­ta­ment to the ex­cel­lence of Im­muno­core’s pi­o­neer­ing sci­ence and un­der­lines the po­ten­tial of our bis­pe­cif­ic im­munother­a­pies across a range of in­di­ca­tions.”

In tan­dem with Tor­bay’s hir­ing, Im­muno­core will al­so add Roy Herb­st, a di­rec­tor at the Yale Can­cer Cen­ter, to its board of di­rec­tors. Herb­st pre­vi­ous­ly served on Im­muno­core’s sci­en­tif­ic ad­vi­so­ry board. — Kyle Blanken­ship

Ite­ri­on rais­es $17M Se­ries B for sol­id tu­mors

Ite­ri­on has spent the past few years try­ing to drug β-catenin, a com­mon onco­gene that drug de­vel­op­ers have long strug­gled to hit, be­cause it lacks an easy bind­ing site. They will now have some ex­tra cash to do so, rais­ing $17 mil­lion in a Se­ries B led by Lu­mi­ra Ven­tures.

Ite­ri­on will use the cash to ad­vance their drug, tega­vivint, in tri­als for a hand­ful of can­cers, in­clud­ing desmoid tu­mors, AML and non-small cell lung can­cer.

Rather than go af­ter β-catenin di­rect­ly, Ite­ri­on tries to block TBL1, a pro­tein β-catenin en­gages to dri­ve tu­mor growth. — Ja­son Mast

Be­yond Air wins $2M+ grant for ni­tric ox­ide treat­ment

Be­yond Air has some new grant mon­ey to play with.

The Gar­den City, NY-based com­pa­ny an­nounced Tues­day it has re­ceived a $2.17 mil­lion grant from the Cys­tic Fi­bro­sis Foun­da­tion to ad­vance de­vel­op­ment for an in­haled ni­tric ox­ide treat­ment for NTM pul­monary dis­ease. Funds will be di­rect­ed to­ward a pi­lot study of a high con­cen­tra­tion for­mu­la­tion of the pro­gram.

“We are grate­ful to re­ceive fund­ing sup­port from the Cys­tic Fi­bro­sis Foun­da­tion,” CEO Steve Lisi said in a state­ment. “To date, our NTM pro­gram has pro­duced da­ta from four com­pas­sion­ate use pa­tients and nine pa­tients from a pre­vi­ous pi­lot study.”

The new study is a 12-week, sin­gle-arm tri­al in Aus­tralia ex­pect­ed to en­roll about 20 CF or non-CF bronchiec­ta­sis pa­tients with cer­tain types of lung in­fec­tions. Be­yond Air said it ex­pects to re­port in­ter­im da­ta around the mid­dle of 2021, which will be fol­lowed by topline da­ta rough­ly six months lat­er. — Max Gel­man

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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Mi­to­chon­dria biotech re­ceives Morn­ing­side buy­out of­fer af­ter PhII fail; On­col­o­gy mi­cro­cap with­draws IPO plans

Not even two months after reporting a Phase II fail for its lead drug candidate, Stealth BioTherapeutics is now being eyed for a potential acquisition, with one firm wanting to take it private.

Stealth reported Monday that Morningside Venture Investments sent Stealth’s board of directors a non-binding preliminary proposal to acquire all outstanding shares not yet owned by Morningside for 2.6 cents per share and 31.3 cents per ADS.