Not so crazy now: Biotech’s most close­ly watched uni­corn just pro­duced its first clear snap­shot of hu­man ef­fi­ca­cy da­ta

The ex­ec­u­tive team at Mod­er­na raised a cheer to­day af­ter pub­lish­ing their first ear­ly snap­shot of hu­man ef­fi­ca­cy da­ta that demon­strate their mes­sen­ger RNA tech works — at least on the first try.

Stéphane Ban­cel

The biotech test­ed their H10N8 flu vac­cine on a small group of 31 sub­jects, look­ing at their re­sponse in two dif­fer­ent mea­sures. All demon­strat­ed a suf­fi­cient im­mune re­sponse to fight off the virus in the first mea­sure, and all but 3 in the sec­ond, for a to­tal of 23 who re­ceived the vac­cine.  None of the 8 sub­jects who re­ceived a place­bo re­spond­ed.

Typ­i­cal­ly, ev­i­dence of Phase I suc­cess in a vac­cine study — which fol­lowed a num­ber of an­i­mal stud­ies — wouldn’t at­tract much at­ten­tion for your av­er­age biotech. But there’s noth­ing av­er­age about Mod­er­na. CEO Stéphane Ban­cel and his team have at­tract­ed $1.9 bil­lion to cre­ate a com­pa­ny that’s build­ing a pipeline around mes­sen­ger RNA, one of a hand­ful of play­ers look­ing to coax cells to pro­duce pro­teins to fight dis­ease. And that’s a mas­sive amount for a com­pa­ny that just showed hu­man proof of con­cept da­ta.

This vac­cine is not point­ed to­ward com­mer­cial­iza­tion, Ban­cel told me Thurs­day af­ter­noon. It’s a demon­stra­tion project. The goal was to start out with “low bi­ol­o­gy risk where we knew where the goal post was be­fore we start­ed the tri­al.”

Mod­er­na’s board has al­ready de­cid­ed not to seek an IPO this year, and Ban­cel adds that there’s no de­fin­i­tive plans set for 2018 ei­ther. What he would like to do, he says, is add new pieces to the pipeline — new ver­ti­cals to the three he’s al­ready out­lined — adding more da­ta on the po­ten­tial mR­NA has to cre­ate a new path­way on drug de­vel­op­ment for a wide ar­ray of dis­eases. Then, have some­thing sub­stan­tial that proves what they can do, he’ll set out to con­vince in­vestors.

The huge amount of cash raised to date and Mod­er­na’s plans to prove the prin­ci­ples be­hind its sci­ence — fo­cus­ing on vac­cines as the most like­ly ini­tial demon­stra­tion pro­gram that could prove that mR­NA can safe­ly work — has al­so trig­gered more than a lit­tle schaden­freude for a com­pa­ny that is of­ten billed as mys­te­ri­ous and lav­ish­ly fund­ed. STAT picked up on that, re­port­ing a bat­tery of most­ly anony­mous crit­i­cism about its tough work en­vi­ron­ment and lofty claims, lay­ing in­to the biotech at every turn.

It is still ear­ly days at Mod­er­na, which has to prove that it can de­vel­op mR­NA drugs for some of the more lu­cra­tive ther­a­pies than the fron­trun­ning vac­cines. That will take some time. But Mod­er­na’s been do­ing much more to out­line what pro­grams it has in the pipeline, and when it can dis­cuss more about the da­ta.

“I’m cau­tious­ly op­ti­mistic this could be a re­al­ly im­por­tant ad­di­tion to our vac­cine ar­se­nal,” Bar­ney Gra­ham, deputy di­rec­tor of the Vac­cine Re­search Cen­ter at the Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases, told the Wall Street Jour­nal. He added that RNA is un­sta­ble, leav­ing him ea­ger to see more ma­ture da­ta on how this works.

Ban­cel and his fel­low ex­ecs at Mod­er­na may have a long way to go, but they’re con­fi­dent that they took a big step along the jour­ney to­day. From now on, it’s all about clin­i­cal da­ta at Mod­er­na.

H10 mR­NA Im­muno­genic­i­ty in Hu­mans | Mol­e­c­u­lar Ther­a­py

Said Tal Zaks, Chief Med­ical Of­fi­cer of Mod­er­na:

The da­ta “pro­vide im­por­tant val­i­da­tion of our core mR­NA plat­form, as we con­tin­ue to ad­vance our de­vel­op­ment pipeline, tack­ling more com­plex vac­cines in­clud­ing per­son­al­ized can­cer vac­cines, and mov­ing our mR­NA ther­a­peu­tics in­to clin­i­cal de­vel­op­ment.”

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,100+ biopharma pros reading Endpoints daily — and it's free.

Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.