Suck­ing up tal­ent out of No­var­tis, a boom­ing Ru­bius adds an­oth­er $100 mil­lion mega-round to the kit­ty

Just 8 months af­ter putting the fin­ish­ing touch­es to a $120 mil­lion round, Flag­ship start­up Ru­bius Ther­a­peu­tics is back at the mon­ey well, draw­ing up an­oth­er big mega-round of $100 mil­lion.

Tor­ben Straight Nis­sen

This time, with more than $220 mil­lion to the good since jump­ing off the start­ing line, Ru­bius de­scribes the raise as a “crossover round.” So does that mean that there’s an S-1 un­der con­struc­tion?

Not ex­act­ly.

“We’re con­sid­er­ing all op­tions,” says CEO Tor­ben Straight Nis­sen. As for an IPO, that’s one of sev­er­al routes that could be open to the com­pa­ny — when the time is right.

Un­usu­al­ly, the biotech is not spelling out where the cash is com­ing from. All that the CEO is will­ing to say is that the syn­di­cate in­cludes “some of the largest mu­tu­al funds…as well as oth­er ma­jor in­vestors.”

A few years ago it would have been a vir­tu­al mis­sion im­pos­si­ble to gath­er more than $200 mil­lion for a biotech that has yet to file its first IND. But Flag­ship chief Noubar Afeyan has been one of the lead­ing pro­po­nents of a move to find new plat­form com­pa­nies with big po­ten­tial for some game-chang­ing tech­nol­o­gy and then com­ing up with the big mon­ey need­ed to build a full pipeline of ther­a­pies, rather than one or two pi­lot projects to demon­strate their po­ten­tial.

Noubar Afeyan

It’s a high risk, high re­ward strat­e­gy that has been pur­sued ag­gres­sive­ly by Mod­er­na, an­oth­er Flag­ship com­pa­ny which just added $500 mil­lion on a $7 bil­lion val­u­a­tion — well ahead of piv­otal da­ta.

What­ev­er lim­it there may be to in­vestor ap­petite for biotech risk, we aren’t see­ing it yet. In the past 72 hours we’ve tracked more than $650 mil­lion in new in­vest­ments in the field, with the mon­ey scat­tered in Cam­bridge/Boston, the Bay Area, Mary­land and New York. The $250 mil­lion raised for a new As­traZeneca spin­out came from three new Chi­nese in­vestors, who are burst­ing on the scene with more cash for qual­i­fied syn­di­cates. And this is the sec­ond $100 mil­lion raise this week.

The R&D plan at Ru­bius is to push ahead with some lead en­zyme re­place­ment ther­a­pies be­ing spawned on a plat­form that seeks to de­vel­op a brand new class of cell ther­a­pies.

The big idea at Ru­bius is that re­searchers can take red blood cells — de­signed by na­ture to trans­port oxy­gen — hi­jack them through ge­net­ic en­gi­neer­ing tech and get them to car­ry pro­teins need­ed to fight var­i­ous dis­eases. It’s an off-the-shelf ap­proach, rather than one per­son­al­ized for each pa­tient.

De­vel­op­ing a new class of med­i­cine, though, calls ear­ly on for some pricey man­u­fac­tur­ing ca­pac­i­ty. And af­ter es­tab­lish­ing a sup­ply line with CMOs, Ru­bius is do­ing some cal­cu­la­tions on how it should con­struct its own in-house process.

“Clear­ly,” says the CEO, “one of the ex­plo­rations is build or buy.” And they don’t have to stay too close to home in Cam­bridge, MA. Oth­er lo­ca­tions in or around Boston and the rest of Mass­a­chu­setts could serve.

The staff, mean­while, has al­ready grown to 67, which in­cludes adding a man­u­fac­tur­ing chief. Spencer Fisk jumped to Ru­bius just a few days ago, join­ing what seems to be a steady mi­gra­tion out of No­var­tis’ cell and gene ther­a­py op­er­a­tion. He was the glob­al head of Cell and Gene Tech­ni­cal De­vel­op­ment and Man­u­fac­tur­ing. Fisk took the place of Karen Walk­er, who left for Seat­tle Ge­net­ics last spring. Walk­er had, in turn, be­come one of three key man­agers in the cell unit at No­var­tis, which was thor­ough­ly re­or­ga­nized about two years ago, when Us­man “Oz” Azam left the helm to run a cell ther­a­py start­up.

It’s a small world in the cell ther­a­py field. Now Fisk is work­ing at a biotech where for­mer No­var­tis can­cer chief David Ep­stein is ex­ec­u­tive chair­man.

Burst­ing at the seams, Ru­bius is al­so now mov­ing in­to 45,000 square feet in new digs at 399 Bin­ney. By this sum­mer, he adds, the staff should be up to around 100 — no small af­fair.

Im­age: 399 Bin­ney Street (Alexan­dria Re­al Es­tate Eq­ui­ties)

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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