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.”

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

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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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

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AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

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Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

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GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

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Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

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The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

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