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.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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