Ru­bius lands $120M bo­nan­za to fund a ground­break­ing pipeline ef­fort on a new drug plat­form

The peo­ple at Ru­bius Ther­a­peu­tics think they’re on to a game-chang­ing new plat­form for drug R&D, one that can spawn a wave of trans­for­ma­tion­al new med­i­cines. And they now have one of the biggest ven­ture cap­i­tal rounds in drug R&D this year to put their dreams to the test in a line­up of new pro­grams now head­ed for the clin­ic.

Seed­ed by Flag­ship Pi­o­neer­ing and ini­tial­ly backed with a $25 mil­lion A round in late 2015, Ru­bius to­day is un­cork­ing a $120 mil­lion in­vest­ment “to move a wave of prod­ucts in­to the clin­ic next year,” says Ru­bius Pres­i­dent Tor­ben Straight Nis­sen. Af­ter dou­bling the staff to 40 over the last 9 months, Ru­bius will now shoot for 100 over the next year.

Tak­ing a page from Mod­er­na — a Flag­ship start­up that at­tract­ed hun­dreds of mil­lions of dol­lars ear­ly on to fi­nance an­oth­er shot at a close­ly-watched break­through ef­fort — Ru­bius isn’t look­ing to prove what they can do with just one or two pro­grams. The team wants to lay out a full pipeline, prefer­ably with a cou­ple of ma­jor league part­ners com­ing in ear­ly to help lever­age the kind of fi­nan­cial fire­pow­er need­ed to ex­e­cute on what they be­lieve is a game-chang­er in biotech.

“We’re mov­ing for­ward on dif­fer­ent in­di­ca­tions and dif­fer­ent fea­tures that test the tech­nol­o­gy,” Straight Nis­sen tells me. The most ad­vanced are en­zyme re­place­ments ther­a­pies and new treat­ments for sol­id tu­mors and hema­to­log­i­cal ma­lig­nan­cies.

“We’ve pro­ject­ed that we’ll be in the clin­ic in 2018,” he says, adding that that is as spe­cif­ic as he wants to get on that top­ic right now.

The red-cell ther­a­py tech they have is de­signed to iso­late hematopoi­et­ic stem cells from O neg­a­tive donors and use them as the build­ing blocks for new ther­a­pies, ge­net­i­cal­ly en­gi­neer­ing them in­to red blood drugs that can ex­press a mul­ti­tude of pro­teins on the cells while they still have a nu­cle­us, mod­u­lat­ing them for pro­tein ex­pres­sion and then shed­ding the nu­cle­us as they switch on the ther­a­peu­tic qual­i­ties of the cell.

Es­sen­tial­ly, it’s a sci­en­tif­ic hi­jack­ing, tak­ing cells and turn­ing them in­to a new fleet of ther­a­pies.

David Ep­stein

The key chal­lenge right now, says Straight Nis­sen, is pick­ing the first wave for the pipeline from a large num­ber of po­ten­tial tar­gets. But this kind of al­lo­gene­ic ap­proach sug­gests a va­ri­ety of av­enues, in­clud­ing find­ing a new way to de­vel­op an off-the-shelf mech­a­nism to re­cruit a T cell at­tack on can­cer cells, hit­ting the tu­mor mi­croen­vi­ron­ment — ground ze­ro in the can­cer field — in a way that avoids un­con­trolled ex­pan­sion of T cells with po­ten­tial­ly cat­a­stroph­ic re­ac­tions.

Noubar Afeyan

Ru­bius in many ways rep­re­sents the kind of biotech gam­ble that Flag­ship wants to be­come known for. It draws on some in­spi­ra­tion from a mar­quee sci­en­tist — in this case MIT’s Har­vey Lodish — while build­ing on the tech­nol­o­gy in their Flag­ship Ven­ture­Labs and em­ploy­ing a big team led by in­dus­try pro­fes­sion­als to prove it works. In this case lead­ers in­clude Straight Nis­sen, who joined Ru­bius from Pfiz­er, as well as No­var­tis vet­er­an David Ep­stein, a new ex­ec­u­tive part­ner at Flag­ship who is tak­ing on the chair­man’s role.

Flag­ship is pro­vid­ing the cash, along with some silent in­sti­tu­tion­al part­ners. And you can bet they’re talk­ing to some ma­jor play­ers to see who would like to col­lab­o­rate on the cause.

The biotech is “fol­low­ing in the foot­steps of Flag­ship’s fam­i­ly of suc­cess­ful mul­ti­prod­uct plat­form com­pa­nies,” says Flag­ship CEO and Ru­bius co-founder Noubar Afeyan. And they have the cash now to find out just how far that path might lead.

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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