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.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.