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.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Bat­tle-test­ed biotech ex­ecs turn to an ex­pe­ri­enced in­dus­try god­fa­ther for some board­room guid­ance

Little Sling Therapeutics out of Ann Arbor, MI may be just a few days out of the starting gate from the announcement of its $35 million launch round, but execs are positioning themselves within clear sight of a key clinical finish line. And they’re revving up with an experienced player to help guide them on the way.

The biotech is putting out word this morning that Faheem Hasnain has agreed to godfather the drug developer as chairman of the board, adding a measured dash of seasoning at a point when most biotechs are still struggling with the embryo stage.

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