Af­ter build­ing a rep as an ar­chi­tect of ex­per­i­ments, Rx­Cel­er­ate plants its flag in Amer­i­ca

When XO1 was bought out by J&J a cou­ple of years ago, the seed in­vestors at In­dex, now Medicxi, made it Ex­hib­it A for their busi­ness mod­el on cre­at­ing a port­fo­lio of as­set-based biotechs.

What start­ed off with a bit of sci­en­tif­ic sleuthing by a pair of re­searchers at Cam­bridge Uni­ver­si­ty — Trevor Baglin and Jim Hunt­ing­ton — led to an an­ti­body pro­gram for a drug dubbed ichor­cum­ab that promised to po­ten­tial­ly act as an an­ti­co­ag­u­lant, with­out the in­her­ent risk of se­vere bleed­ing that in­evitably haunts the class.

Patrick Ver­heyen, who was run­ning the J&J In­no­va­tion of­fice in Lon­don at the time, called this deal a prime ex­am­ple of the way the phar­ma gi­ant was able to reel in top sci­en­tif­ic pro­grams around the world.

But be­tween the chance dis­cov­ery based on an odd med­ical case dat­ing back to 2008 and J&J’s ac­qui­si­tion, XO1, with­out build­ing a staff or do­ing all the nor­mal things as­so­ci­at­ed with biotech star­tups, had to push through a crit­i­cal pre­clin­i­cal study that could prove it had some­thing of re­al val­ue.

Richard Ma­son

“What we re­al­ly need­ed to do in XO1,” XO1 for­mer chief and sole full time staffer Richard Ma­son tells me, “was to show this an­ti­body we cre­at­ed was an an­ti­co­ag­u­lant that didn’t in­crease bleed­ing risk.”

That’s nev­er been done in any sim­i­lar de­vel­op­ment pro­gram be­fore, by any­one.

To do that, Ma­son re­lied on a group in Cam­bridge, UK called Rx­Cel­er­ate. Co-found­ed by David Grainger, a Medicxi part­ner who al­so act­ed as chief sci­en­tif­ic of­fi­cer for XO1, the lab staff didn’t just run a pre­clin­i­cal study un­der con­tract. They built the study from the ground up, ques­tion­ing every step along the way in a field be­set by ques­tions of le­git­i­ma­cy and in­tegri­ty.

Says Ma­son: “They are the ar­chi­tects of the ex­per­i­ment.”

XO1’s ex­pe­ri­ence un­der­scores one of the hard truths about drug dis­cov­ery projects. The lit­er­a­ture is of­ten mis­lead­ing, the mod­els in use may be sus­pect, some­times some of the hard­ware de­scribed doesn’t even ex­ist, says Ma­son. And all of that was on dis­play in their ground­work on an­ti­co­ag­u­la­tion.

A sci­en­tist by train­ing, Grainger and his col­leagues at Medicxi have carved out a rep­u­ta­tion for com­bin­ing sci­en­tif­ic rig­or in ear­ly-stage re­search with a rep for carv­ing the hard costs of do­ing a start­up down to the bare es­sen­tials — in­clud­ing the cre­ative use of out­sourc­ing to keep their star­tups vir­tu­al.

Now Rx­Cel­er­ate has opened up a Boston/Cam­bridge of­fice to in­tro­duce their work un­der US EVP Lau­ra Hamil­ton, the for­mer BD chief at Mass­Bio.

David Grainger

“We are not a CRO,” Grainger tells me lev­el­ly. So I asked for a look at one case to ex­plain what they do. And that led me to Ma­son, who didn’t just do a deal with J&J — a few months lat­er he took Ver­heyen’s job run­ning J&J In­no­va­tion in Lon­don. (Ver­heyen was pro­mot­ed to run BD for all of J&J. Hunt­ing­ton, mean­while, caught the se­r­i­al en­tre­pre­neur bug and has since launched a se­ries of biotech up­starts out of his Cam­bridge lab.)

The way Rx­Cel­er­ate works, Ma­son says, “it starts with the ba­sic sci­ence and takes noth­ing for grant­ed.”

Jill Reck­less — the CEO at Rx­Cel­er­ate, who left with a group of re­searchers at Cam­bridge to launch the lab at Rx­Cel­er­ate with Grainger — start­ed by re­view­ing the lit­er­a­ture on throm­bo­sis and bleed­ing mod­els.

“We found that many of those pa­pers had some prob­lems with them,” says Ma­son. Then there was the rat tail clip mod­el used to mea­sure bleed­ing in ro­dents.

“We found sub­stan­tial prob­lems with the way that was done in the lit­er­a­ture,” adds Ma­son. “Prob­lems with re­pro­ducibil­i­ty.”

Rather than repli­cate bad sci­ence, Rx­Cel­er­ate cre­at­ed new mod­el ex­per­i­ments where nec­es­sary, and dou­bled down with larg­er an­i­mal mod­els to demon­strate po­ten­tial in hu­mans.

“What we are try­ing to of­fer is the abil­i­ty to out­source the think­ing be­hind drug de­vel­o­ment,” says Grainger. CROs are great for de­fined tasks, he adds, but when it comes to do­ing the ar­chi­tec­ture: “CROs don’t do that very well.”

Jill Reck­less

It’s the kind of work that ap­peals to any­one in a cash con­strained en­vi­ron­ment, says Reck­less, whether that’s a biotech start­up or a ma­jor bio­phar­ma, work­ing on a bud­get to see where it has as­sets of re­al val­ue to pur­sue. It’s all about stay­ing fo­cused while re­main­ing skep­ti­cal about what’s come be­fore.

“By not do­ing the things that don’t need to be done,” says Grainger,  “that’s where the bulk of the cost sav­ings come.”

How’s J&J’s team do­ing with XO1’s drug, now dubbed “9375”?

So far so good, says Ma­son, who vis­it­ed with the group in charge at J&J as they look for a next-gen­er­a­tion an­tithrom­bin to fol­low Xarel­to. They’re through Phase I and ex­am­in­ing next steps for Phase II.

Says Ma­son: “Stay tuned.”

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

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 128,800+ 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.

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 128,800+ 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 128,800+ 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 128,800+ biopharma pros reading Endpoints daily — and it's free.

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 128,800+ 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.