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.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 128,900+ biopharma pros reading Endpoints daily — and it's free.

A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 128,900+ biopharma pros reading Endpoints daily — and it's free.

Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 128,900+ biopharma pros reading Endpoints daily — and it's free.

Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 128,900+ biopharma pros reading Endpoints daily — and it's free.

CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 128,900+ biopharma pros reading Endpoints daily — and it's free.

‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.