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.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.