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.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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AbCellera founder and CEO Carl Hansen (L) and Rallybio CEO Martin Mackay

Rally­bio, Ab­Cellera form new part­ner­ship around an­ti­bod­ies for rare dis­ease

Two biotechs that have been working heavily on different stages of antibody candidate development over the past several years are looking to work together to find potential candidates for rare diseases.

Canadian-based AbCellera and Connecticut-based Rallybio have entered a strategic partnership to find, develop and commercialize antibodies primarily for rare diseases. The multi-year, multi-target deal will seek to combine AbCellera’s antibody “discovery engine” with Rallybio’s expertise in rare diseases. However, the dollar amount for the deal was not disclosed.