Go­ing big: Biotech vets blast off with Ar­rakis on a new jour­ney of RNA ex­plo­ration


Michael Gilman and an ex­pe­ri­enced band of biotech vets are bust­ing out of stealth mode to­day with a start­up that marks the se­r­i­al en­tre­pre­neur’s third launch in 10 years. And he’s com­ing out in style, with a $38 mil­lion A round led by Canaan Part­ners.

Af­ter lit­er­al­ly stum­bling across the com­pa­ny while it was still in the white-board phase of de­vel­op­ment in 2015, Gilman says he was ripe for the chal­lenge and ad­ven­ture. And it’s a big one.

CSO and founder Russ Pet­ter has been hon­ing the bioin­for­mat­ics tools, as­says and chem­i­cal li­braries Ar­rakis Ther­a­peu­tics will need to cre­ate a plat­form tech­nol­o­gy that can be used to de­vel­op small mol­e­cules to in­hib­it dis­ease-caus­ing RNA.

This biotech has a long jour­ney ahead in pre­clin­i­cal work be­fore it can start try­ing this out in hu­mans. But if they’re right, the com­pa­ny will be on their way to work­ing on oral ther­a­pies that would be able to hunt down a host of what had been con­sid­ered un­drug­gable tar­gets — start­ing with an ini­tial aim at neu­rol­o­gy and can­cer.

Most pre­clin­i­cal star­tups like this come out of acad­e­mia. But this is a unique­ly Cam­bridge-based out­fit, bring­ing to­geth­er peo­ple with decades of ex­pe­ri­ence right in the heart of one of the world’s biggest biotech hubs.

“I’ve known Russ for a long time,” says Gilman, whose CV in­ter­sects with the founder’s stint at Bio­gen. “He ran chem­istry when I ran re­search there. He’s been work­ing on this for awhile.”

And Pet­ters isn’t the on­ly oth­er Bio­gen vet on board. Chief Busi­ness Of­fi­cer Daniel Ko­er­w­er and James Bar­soum, SVP of bi­ol­o­gy, al­so trace their ca­reers back to the Cam­bridge, MA gi­ant. Col­lec­tive­ly, they list stints at more than a dozen dif­fer­ent biotech com­pa­nies on their re­sumes.

Gilman crossed paths with Pet­ter in 2015, when the fel­low sci­en­tist was hatch­ing his plans for Ar­rakis — a com­pa­ny named af­ter the plan­et in Frank Her­bert’s Dune — in some shared of­fice space where Gilman had one of his board meet­ings. Pet­ter had had his in­ter­est whet­ted at a con­fer­ence pre­sen­ta­tion on small mol­e­cules and RNA in­ter­ac­tions.

They talked, Bris­tol-My­ers Squibb de­cid­ed it would pay a hand­some price for Gilman’s last com­pa­ny, Pad­lock, and af­ter tak­ing last sum­mer off, they got down to se­ri­ous­ly ex­plor­ing the idea to­geth­er.

The mon­ey fol­lowed the ideas.

Along with Canaan Part­ners, Ad­vent Life Sci­ences, Pfiz­er, Cel­gene, Os­age Uni­ver­si­ty Part­ners, and biotech in­dus­try leader Hen­ri Ter­meer chipped in to the A round. The UK’s Ad­vent and Ter­meer helped seed the ini­tial work. And if Ar­rakis’ team is right, there will be plen­ty of op­por­tu­ni­ties along the way to set up plat­form col­lab­o­ra­tions and part­ner­ships.

Colleen Cuf­faro

“I was very in­trigued from the minute I heard the con­cept,” says Colleen Cuf­faro, a prin­ci­pal at Canaan who’s tak­ing a board spot at Ar­rakis.  “It has enor­mous po­ten­tial, how it opens up a whole new space for un­drug­gable tar­gets on our radar for a long time. I re­al­ly like the ap­proach of us­ing small mol­e­cules that are fun­da­men­tal­ly dif­fer­ent than any oth­er RNA-tar­get­ing ap­proach I’ve seen. The oth­er piece was the team, a re­al­ly proven team, with a track record that is out­stand­ing.”

Gilman isn’t con­cen­trat­ing sole­ly on Ar­rakis. He’s been deeply in­volved with At­las Ven­ture, and they’re plan­ning an­oth­er com­pa­ny launch lat­er in the year. But Gilman isn’t stressed by the idea of helm­ing two ven­tures at once.

At Pad­lock, says Gilman, “I felt like I had ex­tra time on my hands.” Af­ter all, CEOs of star­tups of­ten “spend a lot of time wait­ing around for stuff to hap­pen.” And if you have the kind of team he’s work­ing with at Ar­rakis, it’s not a full-time job.

This is not Gilman’s first biotech rodeo. He knows first hand that a ven­ture-backed start­up of­ten lands in oth­er hands, as hap­pened with Stromedix and Pad­lock. This time around though, he would like to take it all out much fur­ther.

“You can nev­er rule it out,” he says, “but this is a com­pa­ny I would like to see built for the long haul. I tru­ly be­lieve we’ll have the ca­pa­bil­i­ty to crank out new drugs oth­er peo­ple haven’t been able to make.”

Gilman’s a long­time be­liev­er in the adage that in biotech, you should go big or go home.

Once again, he’s go­ing big.

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We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

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Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

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Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

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The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.

Lisa M. DeAngelis, MSKCC

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His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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