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.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Stymied by the pan­dem­ic, Im­munomedic­s' new CEO bows out, tak­ing a mil­lion bucks plus perks as he heads out the vir­tu­al ex­it

Just a little more than a month since taking over as the latest CEO to helm Immunomedics, $IMMU Harout Semerjian is exiting the company after being confronted by “logistical” obstacles thrown up by the pandemic that made it impossible for him to move from London to carry out the job. And he’s getting a little over a million dollars in cash plus perks to grease the skids on the way out.

Word of the changeup arrived right after the market closed Wednesday.

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