Shire spins out mR­NA group to RaNA, which is hatch­ing plans to tack­le cys­tic fi­bro­sis in the clin­ic

Over the last few years, the ex­ec­u­tive crew at Shire has on a few rare oc­ca­sions spot­light­ed their work in mR­NA, tout­ing its po­ten­tial for adding to its fore­cast of bil­lions in new rev­enue. A lit­tle more than two years ago, the re­search arm of the Cys­tic Fi­bro­sis Foun­da­tion con­tributed $15 mil­lion to the ven­ture. But now, with their big merg­er with Bax­al­ta still be­ing worked through, Shire is lat­er­al­ing their mR­NA team — more than a dozen staffers and two key pro­grams — to RaNA Ther­a­peu­tics in ex­change for a chunk of eq­ui­ty.

The Cam­bridge, MA-based RaNA gets two pre­clin­i­cal pro­grams that are poised to en­ter the clin­ic in the near fu­ture, with a shot at launch­ing clin­i­cal pro­grams on cys­tic fi­bro­sis and urea cy­cle dis­or­ders in H1 2018. And RaNA CEO Ron Re­naud is clear­ly pumped about adding a new plat­form to the RNAi com­pa­ny at a time that he is halfway through rais­ing a “sub­stan­tial” amount of cash to back the next stage of de­vel­op­ment work at the biotech.

“What I was most in­trigued by,” Re­naud tells me, “is that this ini­tia­tive was un­der­way in 2008. This is some­thing they have been work­ing on for awhile, build­ing IP, work­ing through all those de­vel­op­ment is­sues to where it is to­day. Hav­ing this new plat­form opens up a vast amount of tar­get space for us.”

Get­ting the group will in­crease RaNA’s head count from 48 to more than 60, says the CEO, giv­ing it a makeover that will fun­da­men­tal­ly al­ter its pro­file.

Re­naud de­clined to say for now how much eq­ui­ty Shire is get­ting, or how much he plans to raise.

For its part, a Shire spokesper­son says the grow­ing com­pa­ny spun out the plat­form as part of an on­go­ing as­sess­ment of what works best in­side, or out­side, Shire.

“From a big pic­ture per­spec­tive,” she adds, “pe­ri­od­i­cal­ly eval­u­at­ing and dis­con­tin­u­ing pro­grams is nec­es­sary to main­tain a ful­ly op­ti­mized pipeline where achiev­ing in­no­va­tion/main­tain­ing fo­cus are the goals….Shire will stay close to RaNA and the MRT Plat­form as Shire re­ceives an eq­ui­ty stake in RaNA and is el­i­gi­ble for fu­ture mile­stones and roy­al­ties on prod­ucts de­vel­oped with the tech­nol­o­gy.”

In a short pe­ri­od, RaNA will jump in­to a po­si­tion where it can start mak­ing a mark in a field that has at­tract­ed in­tense in­ter­est. The near­by biotech Mod­er­na has earned the li­on’s share of the at­ten­tion, and $1.9 bil­lion in back­ing, for their mR­NA work — us­ing mes­sen­ger RNA tech­nol­o­gy to smug­gle in in­struc­tions for cells to make spe­cif­ic ther­a­peu­tic pro­teins. Mod­er­na plans to un­veil more of its pipeline work next week at the big JP Mor­gan con­fab, but it’s still in ear­ly-stage de­vel­op­ment with its lead ef­forts fo­cused on new vac­cines. Cure­Vac in Ger­many has more ad­vanced work on a prostate can­cer vac­cine.

It is ex­treme­ly risky work with lit­tle by way of sol­id clin­i­cal da­ta to prove that you can ac­tu­al­ly do what’s in­tend­ed. On the oth­er hand, RaNA now is close enough be­hind the lead­ers that it can po­si­tion it­self in key fields just as the sec­tor is ei­ther tak­ing off, or count­ing ca­su­al­ties.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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As Covid-19 shifts the world's at­ten­tion to biotech, Noubar Afeyan's Flag­ship builds $3.4B fund to fu­el new in­ven­tions. Here's the plan

A little more than a year ago, Flagship Pioneering rolled out a monster fund with $1.1 billion in it to bankroll the platform companies they were creating inside their own labs. But it turns out, that was just the prelude to a much, much larger raise, as both current investors — who’ve been reaping the rewards of some booming biotech stocks — join in with new investors betting on more in the years to come.

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Stephen Hahn (AP Images)

UP­DAT­ED: Ex-FDA com­mish Stephen Hahn joins Flag­ship, a ven­ture group that spawned Covid-19 vac­cine mak­er Mod­er­na

That revolving door between the FDA and industry is spinning even faster than usual.

Former FDA commissioner Stephen Hahn is joining Flagship Pioneering, the venture outfit that founded Moderna — which raced its way to an FDA EUA for a Covid-19 vaccine that is making billions of dollars– as the new CMO of its Preemptive Medicine and Health Security initiative.

Flagship confirmed the hire — first reported in The Washington Post — in a statement out late Monday.

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Patrizia Cavazzoni, CDER

FDA’s Cavaz­zoni calls for ad­comms to ‘get back to the sub­stance’

While her comments were recorded prior to the FDA’s recent approval of Biogen’s controversial Alzheimer’s drug, CDER Director Patrizia Cavazzoni presciently called for substantiating reforms to the advisory committee process at the agency.

Short on examples, Cavazzoni said at a BIO event aired on Monday that some recent adcomms show “how they can be swayed by emotion in the face of hard facts,” but they need to “get back to the fundamentals, which is listening to thoughtful input from experts in response to thoughtful questions that we ask them.”

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Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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Paul Burton, incoming Moderna CMO (J&J/file photo)

Look­ing be­yond the pan­dem­ic, Mod­er­na ap­points J&J vet­er­an Paul Bur­ton as new CMO

Moderna is turning to one of its Covid-19 vaccine competitors to fill its open CMO slot, but this time, it’s not the vaccine experience they’re after.

Paul Burton, who’s spent 16 years at J&J, most recently as chief global medical affairs officer of Janssen, will take over as Moderna CMO on July 6.

With an eye toward a future beyond the pandemic, the mRNA biotech went with Burton, who earned his MD and PhD degrees in London, as he offers experience on a range of therapeutic areas, as well as work as a cardiothoracic surgeon and leading tech projects with Apple.

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Geoffrey Porges, SVB Leerink

Top an­a­lyst maps a rocky road for Ver­tex — un­less they adopt a $10B M&A cam­paign to save the pipeline

After repeatedly poking Vertex $VRTX execs with pointed criticism of their R&D strategy, top SVB Leerink analyst Geoffrey Porges is now turning up the heat to a full-scale roasting.

In a note out early Monday morning, Porges spotlights the impact of Vertex’s recent follow-up failure on AATD — their treatment hit the endpoint but missed on commercial prospects — rewriting his recommended scenario for CEO Reshma Kewalramani as she grapples with the setback.

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75M vac­cine dos­es wast­ed: FDA spells out Emer­gent fa­cil­i­ty de­fi­cien­cies at length in new memo

The FDA is offering a fuller account of what went wrong at Emergent BioSolutions’ Covid-19 manufacturing facility in Baltimore, where the cross contamination of J&J and AstraZeneca vaccines led to the discarding of about 75 million vaccine doses.

CBER director Peter Marks released a memo on Saturday with new specifics, making clear up front that no vaccine manufactured at this plant has been distributed for use in the US yet.

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