Andrew Schiermeier, Intellia COO

Black­stone throws $250M be­hind In­tel­lia-Cellex quest to com­bine CRISPR and con­trol­lable CAR-Ts

So here’s how Black­stone is spend­ing its $4.6 bil­lion bio­phar­ma pot.

The pri­vate eq­ui­ty firm an­nounced Tues­day they were team­ing with the CRISPR biotech In­tel­lia and the lit­tle-known Ger­man CAR-T start­up GEMoaB to launch a new — and so far un­named — CAR-T com­pa­ny. Black­stone, the sole in­vestor, will pour $250 mil­lion in­to the joint ven­ture and take a third own­er­ship. In­tel­lia and Cellex, GEMoaB’s par­ent com­pa­ny, will each take an­oth­er third.

The com­pa­ny will fo­cus on build­ing off-the-shelf CAR-Ts that can be ma­nip­u­lat­ed and con­trolled even af­ter they’re ad­min­is­tered to pa­tients — two of the ma­jor goals that oth­er CRISPR and cell ther­a­py com­pa­nies are now try­ing to at­tain.

“This launch rep­re­sents the first of many steps to ad­dress­ing the var­i­ous lim­i­ta­tions that cur­rent­ly ex­ist in the cell ther­a­py space,” In­tel­lia COO An­drew Schier­meier, who will be­come pres­i­dent and CEO of the new com­pa­ny, said in a state­ment.

Olivi­er Brandi­court

Olivi­er Brandi­court, the for­mer Sanofi CEO who joined Black­stone as an ad­vi­sor this year, will chair the board.

The new com­pa­ny will start out with two pro­grams al­ready in the clin­ic. A sub­sidiary of Cellex, GEMoaB al­ready put two con­trol­lable CAR-Ts in Phase I: one for leukemia and one for prostate can­cer.

Both pro­grams re­ly on GEMoaB’s con­trol­lable CAR-T tech­nol­o­gy. In­stead of arm­ing T cells with re­cep­tors that bind to pro­teins on can­cer cells, they put on re­cep­tors that bind to a “tar­get­ing mod­ule.” When the mod­ules are in­fused in­to a pa­tient, they bind to a pro­tein on can­cer cells and the CAR-Ts bind to the mod­ules. They then, hope­ful­ly, de­stroy the can­cer cells. If the mod­ules are ever tak­en away, GemOAB says — for ex­am­ple, be­cause the pa­tients are start­ing to show ma­jor side ef­fects — the CAR-Ts stop work­ing with­in four hours.

The ap­proach, they say, al­lows them to go af­ter tar­gets that would prove tox­ic un­der tra­di­tion­al CAR-T ap­proach­es. That in­cludes CD123, a promis­ing leukemia tar­get that oth­er de­vel­op­ers avoid­ed be­cause it al­so sits on healthy cells. Carl June’s Tmu­ni­ty Ther­a­peu­tics saw two deaths last month go­ing af­ter the same tar­get, PS­MA, that GEMoaB is pur­su­ing in can­cer.

It’s a sim­i­lar ap­proach to the one Ar­cel­lx is now pur­su­ing pre­clin­i­cal­ly and the one now-de­funct Unum pur­sued dis­as­trous­ly be­fore its col­lapse. But GEMoaB dis­tin­guished it­self with proof-of-con­cept hu­man da­ta at ASH last month.

Those pro­grams both re­ly on a pa­tients’ own cells, but the new com­pa­ny will al­so look to de­vel­op off-the-shelf ther­a­pies. There are few de­tails yet for the rest of the com­pa­ny’s ap­proach, ex­cept that it could al­so cov­er au­toim­mune dis­eases as well as can­cer. The new com­pa­ny will co-de­vel­op a CAR-T for can­cer with In­tel­lia and has an op­tion to co-de­vel­op a sec­ond one for a dif­fer­ent in­di­ca­tion.

In­tel­lia has been large­ly tight-lipped around its can­cer pipeline so far, dis­clos­ing on­ly one of sev­er­al ther­a­pies they’re de­vel­op­ing. But the deal comes at a time when more CRISPR com­pa­nies are push­ing in­to CAR-T. Large­ly, they’ve used CRISPR to en­gi­neer cells that can per­sist longer or that can be giv­en off-the-shelf.

Af­ter mak­ing progress on their sick­le cell ther­a­pies, CRISPR Ther­a­peu­tics’ next ma­jor batch of da­ta will come from a pair of can­cer cell ther­a­pies. In March, Cari­bou Bio­sciences raised $115 mil­lion as they pushed to de­vel­op CAR-Ts for the first time.

For Black­stone, the deal rep­re­sents one of their largest in­vest­ments in a start­up since they closed their $4.6 bil­lion fund last year, al­though they do not have a per­fect score­card on their pre­vi­ous bets. Fer­ring, a gene ther­a­py de­vel­op­er they gave $400 mil­lion, im­plod­ed this year.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Stéphane Bancel, AP Images

Mod­er­na takes on a low-risk pact with CAR-T play­er Au­to­lus for mR­NA-based can­cer drugs

Moderna’s Covid-19 vaccine has transformed the once-backwater biotech into one of the most highly valued drugmakers in the world in the span of a year. But what does the future hold for Moderna’s star turn? A small-scale discovery pact could offer a clue.

Moderna will hold exclusive rights to four mRNA-based immuno-oncology candidates using proprietary binding tech from Autolus, a biotech best known for its work on “off-the-shelf” CAR-T therapies, the partners said Monday.

UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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Ipsen con­tin­ues its shop­ping spree with a $1B-plus deal for Ex­i­cure's next-gen oligonu­cleotides

Ipsen has been on a deal-making spree the last few weeks, shelling out more than a billion dollars in two separate deals to work on a mid-stage levodopa-induced dyskinesia (LID) candidate and a preclinical BAX inhibitor in several cancers. But on Monday, the company inked its largest collaboration deal yet.

Ipsen is putting down $20 million upfront and up to $1 billion in biobucks for exclusive options to two of Exicure’s discovery-stage spherical nucleic acid (SNA) treatments for Huntington’s disease and Angelman syndrome.

Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; and more

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As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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