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 home run count: The $100M+ mega-round boom in biotech in­spired a $6.7B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMedO

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Ed Engleman (Stanford Blood Center)

Stan­ford star on­col­o­gy sci­en­tist Ed En­gle­man helped cre­ate the im­munother­a­py field. Now he wants to shake up neu­rode­gen­er­a­tion R&D

Over the last generation of drug R&D, Ed Engleman has been a standout scientist.

The Stanford professor co-founded Dendreon and provided the scientific insights needed to develop Provenge into a pioneering — though not particularly marketable — immunotherapy. He’s spurred a slate of startups, assisted by his well-connected perch as a co-founder of Vivo Capital, and took the dendritic cell story into its next chapter at a startup called Bolt.

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Nello Mainolfi (Kymera via YouTube)

Out to re­vive R&D, a resur­gent Sanofi pays $150M cash to part­ner up with a pi­o­neer­ing pro­tein degra­da­tion play­er

Frank Nestle was appointed Sanofi’s global head of immunology and inflammation research therapeutic area just days before dupilumab, the blockbuster-to-be IL-4 antibody, would be accepted for priority review. After four years of consolidating immunology expertise from multiple corners of the Sanofi family and recruiting new talents to build the discovery engine, he’s set eyes on a Phase I-ready program that he believes can grow into a Dupixent-sized franchise.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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Covid-19 roundup: CDC de­bat­ing who should get first avail­able vac­cines; EU in Gilead talks af­ter US gob­bled first remde­sivir dos­es

The federal government has now spent billions of dollars accelerating the development of a Covid-19 vaccine, and yet they’ve remained hush-hush on who, precisely, would actually get inoculated once the first doses are approved and available. Internally, though, they have been debating it.

The CDC and an advisory committee of outside health experts have been working since April to devise a ranking system that would determine who receives a vaccine and when, The New York Times reported. The question of who is first in line for inoculation is important because no matter how many doses developers can make or how quickly they can make them, doses will still come out in batches; 300 million inoculations will not appear overnight.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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