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.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Stephen Hahn, FDA commissioner (AP Images)

Stephen Hahn: FDA will make some changes amid Covid-19 per­ma­nent

The FDA will look to permanently implement some of the processes and policies adopted in its response to the Covid-19 pandemic, FDA commissioner Stephen Hahn said during a virtual briefing hosted by the Alliance for a Stronger FDA on Monday.

Hahn’s statements during the briefing closely mirrored statements he made in a pre-recorded video FDA uploaded to YouTube on Friday.

“As this pandemic has evolved, it was clear to all of us that some FDA processes needed to be adjusted to accommodate the urgency of the pandemic and I think the entire FDA team has now seen first-hand that we need to take a critical look at some of our processes and policies,” Hahn said.