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.

Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Idor­si­a's brain bleed drug flunks PhI­II tri­al, a decade af­ter pre­vi­ous flop

Idorsia’s long journey with clazosentan came to an abrupt “unexpected result” Monday morning with a Phase III flop.

The Swiss biopharma said the drug did not meet the main goal of the late-stage REACT study, conducted in the US, Canada and Europe since early 2019.

The 409-patient trial tested the intravenous drug’s ability to prevent complications due to delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage (aSAH), in which blood vessels in the brain narrow and blood accumulates around the brain’s surface, which then dials up the pressure on the brain.

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Kenji Yasukawa, Astellas Pharma CEO (Photographer: Akio Kon/Bloomberg via Getty Images)

Astel­las taps chief strat­e­gy of­fi­cer as next CEO to 'go on the ag­gres­sive'

Five years into its big R&D revamp, Astellas says it’s time for a changing of the guard.

Kenji Yasukawa, who took over as president and CEO in 2018, will step down to become chairman of the board in April, making room for Naoki Okamura to take over. Okamura joined the company in 1986 and has served in a variety of finance, business and strategy roles, including most recently as chief strategy officer.

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Clin­i­cal tri­al di­ver­si­ty da­ta show mis­match be­tween en­roll­ment and dis­ease preva­lence, GSK says

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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Goldfinch Bio CEO Tony Johnson (L) and Karuna Therapeutics CEO Bill Meury

Karuna li­cens­es Goldfinch as­sets to com­pete with Boehringer In­gel­heim in neu­ro­science

Karuna Therapeutics is looking to compete with Boehringer Ingelheim on depression and anxiety with a new license to Goldfinch Bio’s assets, starting with $15 million to the shuttered biotech.

Karuna steps into an arena already being tested by Boehringer in multiple Phase II studies — the two are targeting transient receptor potential canonical 4 and 5, or TRPC4/5, which is thought to have a role in neuroscience indications. Goldfinch’s asset went through a Phase II in kidney diseases, but Karuna’s sights are set on mood and anxiety disorders for now.

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