Biotech uni­corn Mod­er­na gam­bles $110M on a ground­break­ing mR­NA man­u­fac­tur­ing fa­cil­i­ty, blue­prints plant #2

Start­ing in a few weeks, Mod­er­na will be­gin work on the biggest biotech man­u­fac­tur­ing fa­cil­i­ty in the world ded­i­cat­ed to ear­ly stage R&D, pro­duc­ing its mes­sen­ger RNA drugs for every­thing from tox work through Phase II stud­ies. And with the first crit­i­cal round of clin­i­cal da­ta due to ar­rive in H1 2017, Mod­er­na CEO Stephane Ban­cel says the biotech is al­ready blue­print­ing an­oth­er fa­cil­i­ty that may well dwarf the first $110 mil­lion man­u­fac­tur­ing project.

Ban­cel and his ex­ec­u­tive crew have land­ed a site for their first 200,000-square-foot fa­cil­i­ty in Nor­wood, MA, not far from the biotech’s head­quar­ters in Cam­bridge. And char­ac­ter­is­ti­cal­ly, they’re mov­ing as fast as they can to get the dirt fly­ing.

Mod­er­na is think­ing big about every as­pect of drug de­vel­op­ment. That starts with rais­ing cash: Now close to $2 bil­lion. Staff: 460 and grow­ing fast. And pipeline projects: 11. There are al­so four spin­out ven­tures work­ing on per­son­al­ized can­cer vac­cines and on­col­o­gy, in­fec­tious dis­eases and rare ail­ments.

The score on hu­man tri­al out­comes? Ze­ro so far, but that will soon change.

This new fa­cil­i­ty will even­tu­al­ly be able to make up to 100 mR­NA lots an­nu­al­ly, a num­ber that won’t mean much to any­one not schooled in biotech man­u­fac­tur­ing. But the ex­pla­na­tion pro­vides some in­sights in­to what Mod­er­na has set out to achieve.

In the first dozen pro­grams to make it to the clin­ic, ex­plains Ban­cel, in­ves­ti­ga­tors can mix mR­NA in a sin­gle vial, or lot. But in the very near fu­ture, they’ll need sev­er­al dif­fer­ent anti­gens in the same vial, or sev­er­al lots. For one up­com­ing vac­cine study for a par­tic­u­lar­ly tough virus that Mod­er­na plans to tack­le, it will take sev­en.

And af­ter Phase II, what then?

Ban­cel says he is al­ready look­ing on build­ing out the man­u­fac­tur­ing Mod­er­na will need for Phase III and com­mer­cial quan­ti­ties, a project that could con­sume any­where from $200 mil­lion to $400 mil­lion. Once the first clin­i­cal da­ta starts to come in, he says, some fast-mov­ing projects could move in­to Phase III in less than a year. And that will spur more ex­pan­sion.

Mod­er­na has been work­ing with a va­ri­ety of groups, in­clud­ing a team from GE, to ramp up new tech­nol­o­gy to make the man­u­fac­tur­ing work more ef­fi­cient, with­out get­ting tripped up by de­con­t­a­m­i­na­tion pit­falls.

“We try to use a lot of equip­ment that can be reused,” says the CEO, “so that you can go much faster.”

These man­u­fac­tur­ing projects in the Boston area have be­come a plum catch in the eco­nom­ic de­vel­op­ment field. And for one ob­vi­ous rea­son: More jobs. The plan at Mod­er­na now is to shift 100 of their work­ers to the new site once it be­comes op­er­a­tional in ear­ly 2018. And they’ll add 100-plus em­ploy­ees once it’s in full swing.

The gam­ble, and it’s as big as Mod­er­na’s goals, is that they can build a glob­al op­er­a­tion from scratch, with a full suite of tech­nolo­gies and fa­cil­i­ties that can keep pace with its am­bi­tions — and, they hope, with­out any com­pa­ny-end­ing blowups along the way.

Mod­er­na’s been able to stay on track to that goal, gain­ing big in­vestors like As­traZeneca along the way who have bought in to the vi­sion. And un­like vir­tu­al­ly every oth­er biotech in ex­is­tence, they built up their ex­ten­sive in­fra­struc­ture with­out a sin­gle shred of hu­man clin­i­cal da­ta to prove that their drugs ac­tu­al­ly work in peo­ple by spurring pa­tients’ cells to pro­duce drugs.

The da­ta, though, are com­ing. And Mod­er­na be­lieves it can quick­ly start to rack up a wide range of tri­al re­sults, lay­ing out a smor­gas­bord of da­ta rather than plug along on 2-3 lead proof-of-con­cept pro­grams.

Mod­er­na is al­so work­ing to stay out front of mR­NA com­peti­tors like BioN­Tech, which co­in­ci­den­tal­ly just struck a ma­jor col­lab­o­ra­tion pact with Genen­tech. What start­ed out as a biotech race has quick­ly swelled in­to a show­down that in­cludes some of the world’s biggest drug de­vel­op­ers. Wa­gers rarely get this big, this ear­ly.

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.

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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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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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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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.

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After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

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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

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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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