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.”

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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In­vestors could emerge from Neil Wood­ford de­ba­cle with £1B loss, in­ter­nal analy­sis re­veals

When Link Fund Solutions announced that it is closing Woodford Equity Income Fund permanently and kicking out Neil Woodford, it was implied that investors probably won’t get back everything they entrusted to the fund manager. But nobody knew just how much they would lose.

An internal analysis commissioned by Link suggested that the collective loss could amount to £1 billion — out of a fund last valued at £3.1 billion — Citywire has revealed.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.