Genen­tech dives in­to mR­NA, bet­ting $310M on BioN­Tech's per­son­al­ized can­cer vac­cine tech

Genen­tech is tap­ping in­to the promise of mes­sen­ger RNA for build­ing per­son­al­ized can­cer vac­cines. The gi­ant Roche sub­sidiary is part­ner­ing with Ger­many’s BioN­Tech on its in­di­vid­u­al­ized can­cer ther­a­pies, promis­ing $310 mil­lion in near-term pay­outs to col­lab­o­rate on a com­bo with its new­ly ap­proved check­point in­hibitor Tecen­triq (ate­zolizum­ab).

James Sabry, Genen­tech

In this deal, BioN­Tech will con­tribute its per­son­al­ized can­cer vac­cine plat­form spot­light­ing unique neoanti­gens that can be iden­ti­fied in a can­cer pa­tient’s tu­mors, a trendy new tar­get of drug de­vel­op­ers like Grit­stone, Mod­er­na and Neon Ther­a­peu­tics. Phase I stud­ies are slat­ed to be­gin next year.

BioN­Tech’s work is fo­cused on cre­at­ing syn­thet­ic mR­NAs de­signed to pro­duce ther­a­peu­tic pro­teins, turn­ing a pa­tient’s cells in­to drug fac­to­ries. Batch­es of code spur cells to cre­ate a ther­a­peu­tic pro­tein. And the part­ners are look­ing to kick up a broad as well as spe­cif­ic im­mune re­sponse to fight can­cer. In this case, the tech starts with se­quenc­ing a pa­tient’s genome for their tu­mor, then en­cod­ing the neoanti­gens for that par­tic­u­lar tu­mor in­to a mes­sage de­liv­ered by mR­NA as a vac­cine. Den­drit­ic cells de­code the in­for­ma­tion, and use it to mark tu­mor cells for de­struc­tion by the im­mune sys­tem.

These new per­son­al­ized can­cer vac­cines promise to help ex­pand on the ef­fec­tive­ness of check­point in­hibitors, which dis­man­tle hur­dles set up by can­cer cells, and hope to suc­ceed where the first wave of off-the-shelf can­cer vac­cines large­ly failed.

BioN­Tech gets rights to co-com­mer­cial­ize new ther­a­pies in the US and cer­tain Eu­ro­pean mar­kets, in­clud­ing Ger­many. And it will al­so co-fund the up­com­ing R&D work.

BioN­Tech COO Sean Marett

The deal an­nounce­ment is char­ac­ter­is­ti­cal­ly light on fi­nan­cial de­tails. But in light of the up­front and near-term cash in the deal, that sum is like­ly to be no­table.

Found­ed in 2008, Mainz, Ger­many-based BioN­Tech has been large­ly fund­ed by the Strüng­mann group, a fam­i­ly firm led by Ger­man bil­lion­aires Thomas and An­dreas Strüng­mann, iden­ti­cal twins who have been fu­el­ing a va­ri­ety of biotechs in Ger­many.

The com­pa­ny is keep­ing qui­et about the mile­stones in the deal and isn’t talk­ing about prospec­tive time­lines on the work with Genen­tech, anx­ious to keep some of its cards close to its vest.

“Im­muno-on­col­o­gy is un­be­liev­ably com­pet­i­tive,” says BioN­Tech COO Sean Marett, and that re­quires some cir­cum­spec­tion on the de­tails.

The com­pa­ny, though, laid out a sig­nif­i­cant piece of the on­col­o­gy puz­zle it’s been work­ing on in two pa­pers pub­lished last year and then last June in Na­ture. The first out­lined its work with se­quenc­ing tu­mors for neoepi­topes and in the sec­ond pa­per in­ves­ti­ga­tors out­lined how they used RNA-lipoplex­es to pre­cise­ly tar­get den­drit­ic cells, over­com­ing a key hur­dle by en­cod­ing shared tu­mor anti­gens and get­ting the T cell re­sponse they were look­ing for.

BioN­Tech has com­plet­ed one Phase I study in pa­tients, and Marett isn’t re­luc­tant to claim the lead in the field, not­ing that some U.S. biotechs start­ed af­ter the first pub­li­ca­tion in 2015. The Ger­man biotech now has a staff of 500, even larg­er than Mod­er­na’s 460, and it’s build­ing its sec­ond man­u­fac­tur­ing fa­cil­i­ty in Mainz to back up the ex­pand­ing clin­i­cal plans.

Next steps in­clude tak­ing a close look at how BioN­Tech will re­al­ize its plans, in­clud­ing co-fund­ing the work with Genen­tech, ex­pand­ing man­u­fac­tur­ing, push­ing part­nered and in-house pro­grams, with a pos­si­ble IPO on the ta­ble as one re­al pos­si­bil­i­ty.

Get­ting a pow­er­house U.S. part­ner like Genen­tech in their cor­ner moves BioN­Tech clos­er to the day it can ef­fec­tive­ly start sell­ing the ther­a­pies they’re now test­ing in the clin­ic.

“We’ve got a lot of el­e­ments in the com­pa­ny that we need,” says Marett. “The thing we were miss­ing, this abil­i­ty to be able to not on­ly man­u­fac­ture but al­so com­mer­cial­ize. That’s some­thing that this deal will al­low us to do.”

The pact marks the lat­est in a string of Big Phar­ma tie-ups with the Ger­man biotech. Sanofi signed on in a $1.5 bil­lion deal last spring. Both Mod­er­na and Cure­Vac are al­so el­bow­ing in­to the mR­NA field, with big mon­ey deals of their own. And Sanofi fol­lowed up af­ter a sep­a­rate deal BioN­Tech struck with Eli Lil­ly, which has its own plans for the on­col­o­gy field.

Com­bos are the fu­ture of can­cer drugs, and BioN­Tech is acute­ly aware of the po­ten­tial the al­liance of­fers with com­bin­ing its per­son­al­ized vac­cines with Roche’s re­cent­ly ap­proved PD-L1 check­point Tecen­triq, or ate­zolizum­ab. So is Roche/Genen­tech.

“Un­like any med­i­cine we have ever de­vel­oped, vir­tu­al­ly all can­cer pa­tients may po­ten­tial­ly ben­e­fit from a cus­tom-built can­cer vac­cine,” said James Sabry, M.D., Ph.D., Se­nior Vice Pres­i­dent and Glob­al Head of Genen­tech Part­ner­ing, in pre­pared text. “By col­lab­o­rat­ing with BioN­Tech on this cut­ting edge ap­proach, we hope to tru­ly ad­vance can­cer treat­ments by us­ing a com­mon mol­e­c­u­lar back­bone – mR­NA – that is unique­ly tai­lored to an in­di­vid­ual pa­tient.”

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

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Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

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Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

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An­oth­er Cipla site lands a Form 483 over clean­ing is­sues and QC con­trols

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

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That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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