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.”

Albert Bourla appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

UP­DAT­ED: Pfiz­er CEO Al­bert Bourla is back in the M&A game, but why is he pay­ing $11.4B for Ar­ray?

Pfiz­er $PFE has cut short its time on the side­lines of bio­phar­ma M&A.

Mon­day morn­ing the phar­ma gi­ant un­veiled an $11.4 bil­lion deal to ac­quire Ar­ray Bio­Phar­ma, beef­ing up its on­col­o­gy work and adding a new re­search hub in Boul­der, Col­orado to its glob­al op­er­a­tions.

At $48 a share, Ar­ray $AR­RY in­vestors will be get­ting a 62% pre­mi­um off the Fri­day close of $29.59.

Pfiz­er, which has strug­gled to gain all the up­side promised in past buy­outs like Medi­va­tion, high­light­ed the ac­qui­si­tion of 2 ap­proved drugs in the deal — Braftovi (en­co­rafenib) and Mek­tovi (binime­tinib).

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In­vestors fret as VBI's hep B vac­cine fails key sec­ondary PhI­II study goal

Sobered by mount­ing costs, Dy­navax $DVAX last month made the de­ci­sion to fo­cus all its re­sources on its 2017-ap­proved he­pati­tis B vac­cine Hep­lisav-B, which ri­vals and su­per­sedes the ef­fi­ca­cy and con­ve­nience pro­file of GSK’s $GSK es­tab­lished En­ger­ix-B. The Cal­i­for­nia-based com­pa­ny will be on the look­out for an­oth­er com­peti­tor — VBI Vac­cines, which on Mon­day un­veiled late-stage da­ta on its hep B vac­cine: Sci-B-Vac.

Image: Shutterstock

Gene ther­a­py R&D deals turn red hot as Big Phar­ma steps up to play

This September will mark the 20th anniversary of the death of Jesse Gelsinger, a young man suffering from X-linked genetic disease of the liver. He was killed in a gene therapy study conducted by Penn’s James Wilson, and the entire field endured a lengthy deep freeze as the field grappled with the safety issues inherent in the work.

Some thought gene therapy R&D would never survive. But it did. And this year marked a landmark approval for Zolgensma, a new gene therapy for spinal muscular atrophy Novartis priced at $2.1 million.

“Gene therapy is the hottest item on the block now. But there was a time when we first got into this trial, where there wasn’t a person in the world who believed that gene therapy would work. We have to remember that,” noted gene therapy investigator Jerry Mendell told SMA News Today.

We’re still right on the pioneering frontier when it comes to getting approvals for gene therapies and launching marketing campaigns with the European green light for bluebird's leading program last Friday underscoring the nascent nature of the field. But gene therapy R&D is booming, and has been for several years now.

The rapid growth of gene therapy clinical development is well known, but we decided to put some numbers on it, to quantify what’s going on. DealForma chief Chris Dokomajilar took a lot over the past 10 years, as the number of deals, R&D partnerships and buyouts steadily gained steam, spiking last year and on track to maintain the surge in 2019.

The upfronts and totals for the dollars on deals so far in 2019 is already close to the 2018 mark, underscoring a new phase of negotiations as the major players step up to gain a piece of the late-stage and commercial action.

Once again, we’re looking at an “overnight” biotech success story, decades in the making.

At some point, that may start to brake the numbers we’re seeing. But for now, as rivals line up to compete for frontline prominence across a range of diseases, the arrows are all pointed north.

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A uni­corn stalks Wall Street in search of IPO cash; CASI Phar­ma in-li­cens­es CD19 ther­a­py from Chi­na’s Ju­ven­tas

→ A herd of up­start biotechs will look to Wall Street for some ma­jor wind­falls this week as a burst of new of­fer­ings con­tin­ues to feed cash in­to the R&D sys­tem. To­day we learned that Bridge­Bio will look to raise in the neigh­bor­hood of $225 mil­lion by of­fer­ing 15 mil­lion shares for $14 to $16 each. And they have a string of joint bookrun­ners: J.P. Mor­gan, Gold­man Sachs, Jef­feries, SVB Leerink, KKR, Piper Jaf­fray, Mizuho Se­cu­ri­ties, BMO Cap­i­tal Mar­kets and Ray­mond James. If suc­cess­ful, Bridge­Bio will emerge with a mar­ket cap of around $1.7 bil­lion. There are 5 biotechs look­ing to IPO this week, in­clud­ing Akero and Pre­vail.

UP­DAT­ED: Sanofi Gen­zyme deserts gene ther­a­py de­vel­op­er Voy­ager Ther­a­peu­tics

While gene ther­a­py com­pa­nies re­joice as the sec­tor gains trac­tion with ap­provals and a flur­ry of M&A ac­tiv­i­ty, one play­er is feel­ing the heat.

Back in 2015, Voy­ager Ther­a­peu­tics joined forces with Sanofi Gen­zyme in a deal worth up to $845 mil­lion ($100 mil­lion up­front + a po­ten­tial $745 mil­lion in mile­stones) to co-de­vel­op gene ther­a­pies for se­vere cen­tral ner­vous sys­tem dis­or­ders. But two years lat­er, the French drug­mak­er re­treat­ed, elect­ing to not pick up the op­tion to work on Voy­ager’s Parkin­son’s dis­ease pro­gram. (Last year, the FDA dis­ap­point­ed Voy­ager, telling the com­pa­ny that it was not open to an ac­cel­er­at­ed fil­ing on the Parkin­son’s drug on the ba­sis of Phase II da­ta — in­stead of re­quir­ing an ad­di­tion­al piv­otal study.)

John Oyler, Founder & CEO of BeiGene, at the US-China Biopharma Innovation and Investment Summit in Shanghai on October 23, 2018; Credit: Endpoints News, PharmCube

UP­DAT­ED: As Bris­tol-My­ers/Cel­gene tie up loose ends, BeiGene pock­ets $150M from PD-1 breakup

As soon as Bristol-Myers Squibb announced its $74 billion buyout for Celgene, BeiGene emerged as a prominent example of a player whose pact with the big biotech could sour, as its PD-1 candidate seems to overlap with Opdivo. After six months of suspense, the partners say they are finally bringing the 2-year-old deal to an amicable end.

BeiGene $BGNE gets $150 million for the termination in addition to full global rights to tislelizumab. In 2017 Celgene had paid $263 million in upfront license fees to develop the PD-1 inhibitor for solid cancers in the US, Europe, Japan and the rest of the world outside Asia. It also threw in a $150 million equity investment in exchange for BeiGene handling its commercial operations — think Abraxane, Revlimid and Vidaza — in China.

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Exterior of the 1 million square foot Discovery Labs in Upper Merion, PA (PR Newswire)

Philadel­phia cham­pi­ons life sci­ences 'co-work­ing,' re­viv­ing for­mer GSK cam­pus in $500M makeover

In a boost to Philadel­phia’s thriv­ing life sci­ences scene, a for­mer Glax­o­SmithK­line cam­pus and a near­by site has been turned in­to what its de­vel­op­er calls “the largest cowork­ing ecosys­tem” for health­care com­pa­nies in the coun­try.

The Dis­cov­ery Labs, a com­pa­ny spawned by MLP Ven­tures, has se­lect­ed two lo­ca­tions in the King of Prus­sia area as the $500 mil­lion test case for its strat­e­gy of ac­quir­ing and con­vert­ing old phar­ma­ceu­ti­cal R&D fa­cil­i­ties world­wide. The sites add up to 1.64 mil­lion square feet.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.