Soros, Gates back medtech's move in­to a 'so­cial en­ter­prise'; Ag­ing an­ti-re­jec­tion drug earns fresh ap­proval

George Soros and Bill Gates are team­ing up to buy out a British di­ag­nos­tics com­pa­ny.

The pair and their foun­da­tions will spend about $41 mil­lion to trans­form Mo­log­ic in­to a “So­cial En­ter­prise” aimed at ex­pand­ing ac­cess to state-of-the-art med­ical tech­nol­o­gy, the com­pa­ny an­nounced Mon­day. Launched in 2003, Mo­log­ic comes from the fa­ther-and-son team of Mark and Paul Davis, the lat­ter of whom helped cre­ate one of the first at-home preg­nan­cy tests.

“Mo­log­ic’s tran­si­tion in­to a so­cial en­ter­prise is a de­lib­er­ate, log­i­cal and nat­ur­al step for a com­pa­ny fo­cused on de­liv­er­ing af­ford­able di­ag­nos­tics and biotech­nol­o­gy to places that have been left un­der­served by the re­lent­less pur­suit of prof­i­teer­ing,” Mark Davis, who al­so serves as CEO, said in a state­ment. — Max Gel­man

An­ti-re­jec­tion drug gets FDA OK for lung trans­plants

An an­ti-re­jec­tion drug pre­vi­ous­ly used to pre­vent or­gan re­jec­tion in pa­tients with liv­er, kid­ney and heart trans­plants has been ap­proved by the FDA for lung trans­plant pa­tients as well on Fri­day.

Pro­graf was ap­proved for use in com­bi­na­tion with oth­er im­muno­sup­pres­sant drugs. The ap­proval marks the first and on­ly for a drug to pre­vent re­jec­tion of a lung trans­plant.

Ap­proval was grant­ed based on a non-in­ter­ven­tion­al, fit-for-pur­pose study us­ing re­al-world da­ta. Da­ta were col­lect­ed on all US lung trans­plants, and da­ta from ran­dom­ized con­trolled tri­als of Pro­graf in oth­er set­tings pro­vid­ed con­fir­ma­to­ry ev­i­dence, the FDA said.

“A dra­mat­ic im­prove­ment in out­comes was ob­served among lung trans­plant pa­tients re­ceiv­ing Pro­graf as part of their im­muno­sup­pres­sion med­ica­tions com­pared to the well-doc­u­ment­ed nat­ur­al his­to­ry of a trans­plant­ed drug with no or min­i­mal im­muno­sup­pres­sive ther­a­py,” the press re­lease from the FDA said.  — Josh Sul­li­van

As­traZeneca pneu­mo­nia can­di­date tar­get­ed in li­cens­ing deal

Aridis Phar­ma­ceu­ti­cals will ex­clu­sive­ly li­cense As­traZeneca’s late-stage pneu­mo­nia an­ti­body can­di­date su­vra­tox­um­ab.

Su­vra­tox­um­ab, which re­cent­ly fin­ished a Phase II tri­al, will com­ple­ment Aridis’ own Phase III pneu­mo­nia pro­gram for AR-301. Both can­di­dates tar­get the S. au­reus strain.

As part of the deal, As­traZeneca will be­come a share­hold­er in Aridis and will re­tain fu­ture “first-to-ne­go­ti­ate” rights for li­cens­ing the pro­gram.

In that Phase II test, su­vra­tox­um­ab re­duced the rel­a­tive risk of pneu­mo­nia by 32% in 196 pa­tients with a 47% re­duc­tion in the over-65 pop­u­la­tion. The drug was al­so as­so­ci­at­ed with a sub­stan­tial re­duc­tion in du­ra­tion of care need­ed at the ICU and hos­pi­tal. — Kyle Blanken­ship

Ra­tio­nal de­sign for pro­tein degra­da­tion? Aus­tri­an deep learn­ing start­up takes a stab

For all the promis­es of PRO­TACs and mol­e­c­u­lar glues as a class, there re­mains lots of room for im­prove­ment on how in­di­vid­ual pro­tein de­graders are dis­cov­ered.

Christo­pher Trum­mer

That’s ac­cord­ing to Christo­pher Trum­mer, a for­mer bioin­for­mat­ics con­sul­tant who no­ticed, while do­ing con­tract re­search for a va­ri­ety of bio­phar­ma com­pa­nies, that there was re­al­ly no ra­tio­nal ap­proach to de­sign­ing mol­e­cules that can grab tar­gets and tag them for dis­pos­al. Iden­ti­fy­ing the right ones was al­ways a tri­al and er­ror af­fair.

“Typ­i­cal­ly if you do some brute force meth­ods, high through­put screen­ing — even if you part­ner with [what are con­sid­ered] the cheap­est CROs, right — you would end up in at least half a mil­lion or a mil­lion for screen­ing, in the PRO­TAC area, maybe 2,000, 3,000 com­pounds, some­thing like that,” he said. “So it is very very cost­ly.”

To­geth­er with his friend Jakob Ho­hen­berg­er, who has a back­ground in tech, Trum­mer be­gan ex­plor­ing a so­lu­tion steeped in deep learn­ing. The re­sult was Celeris Ther­a­peu­tics, whose Celeris One soft­ware promis­es to pre­dict pro­tein-pro­tein in­ter­ac­tions and dock­ing specif­i­cal­ly for ap­pli­ca­tions in tar­get­ed pro­tein in­ter­ac­tions.

Jakob Ho­hen­berg­er

It was enough to draw in­quiries from Big Phar­ma and biotech com­pa­nies, Trum­mer said, one of which has al­ready agreed to a part­ner­ship.

The team of 15 is cur­rent­ly based in Aus­tria. While Celeris plans to keep a pres­ence here — and grow even big­ger by build­ing a wet lab lat­er this year — Trum­mer, the CEO, plans to move to Sil­i­con Val­ley soon to be clos­er to in­vestors as well as sci­en­tists and po­ten­tial part­ners.

True to its Eu­ro­pean roots, though, Celeris has kept its fund­ing mod­est. Af­ter APEX Med­ical brought their pre-seed fund­ing to €1.6 mil­lion ($1.89 mil­lion), the biotech is tar­get­ing €5 mil­lion for the seed round.

“We gen­uine­ly be­lieve that these ma­chine learn­ing based sim­u­la­tions will stream­line the way ear­ly stage drug de­vel­op­ment is per­formed and even­tu­al­ly will ben­e­fit all over the world,” Gor­don Eu­ller, gen­er­al part­ner at APEX, wrote in an email.

The goal, ul­ti­mate­ly, is to op­er­ate like the cash-rich AI play­er Ex­sci­en­tia, which is lend­ing its tech­nol­o­gy to clients but al­so pur­su­ing in-house dis­cov­ery and de­vel­op­ment. — Am­ber Tong

Opi­oid al­ter­na­tive starts PhII tri­al in bunionec­to­my and ab­domino­plas­ty surgery

Ver­tex has kicked off a Phase II proof-of-con­cept study in acute pain af­ter bunionec­to­my surgery, the com­pa­ny an­nounced Mon­day. It will start a Phase II study of pain fol­low­ing ab­domino­plas­ty surgery in the com­ing weeks.

Car­men Boz­ic

The ran­dom­ized, dou­ble-blind­ed, place­bo-con­trolled stud­ies will eval­u­ate its se­lec­tive NaV1.8 in­hibitor VX-548, and in­clude a hy­drocodone bitar­trate/ac­eta­minophen ref­er­ence arm. The small mol­e­cule has shown to re­duce neu­ro­path­ic pain and mus­cu­loskele­tal pain in pre­vi­ous clin­i­cal tri­als. If Ver­tex hits on VX-548, it could pro­vide a po­tent, non-ad­dic­tive al­ter­na­tive to opi­oids.

“NaV1.8 is a ge­net­i­cal­ly and phar­ma­co­log­i­cal­ly val­i­dat­ed tar­get and we are ex­cit­ed about the po­ten­tial for VX-548 as a new class of ef­fec­tive pain treat­ments with­out the lim­i­ta­tions of cur­rent ther­a­pies, in­clud­ing the ad­dic­tive po­ten­tial of opi­oids,” EVP Car­men Boz­ic said in a press re­lease.

Re­sults from the study are ex­pect­ed by Q1 of 2022. — Josh Sul­li­van

French biotech PEP-Ther­a­py ex­pands Se­ries A round

A few months af­ter clos­ing its $3.4 mil­lion Se­ries A round, Paris-based PEP-Ther­a­py has in­vestors reach­ing a lit­tle deep­er in­to their wal­lets.

PEP-Ther­a­py has reeled in an­oth­er $3 mil­lion in a Se­ries A ex­pan­sion, bring­ing its to­tal haul to $6.4 mil­lion. The com­pa­ny’s work­ing on what it calls Cell Pen­e­trat­ing & In­ter­fer­ing Pep­tide (CP&IP) tech­nol­o­gy, de­signed to pen­e­trate cells and specif­i­cal­ly block rel­e­vant in­tra­cel­lu­lar pro­tein-pro­tein in­ter­ac­tions. Its lead can­di­date PEP-010 is cur­rent­ly in a Phase Ia/b tri­al for ad­vanced sol­id tu­mors.

“We are de­light­ed to have com­plet­ed this fi­nanc­ing round via an at­trac­tive bal­ance of di­lu­tive and non-di­lu­tive funds from new high qual­i­ty and di­ver­si­fied in­vestors who will bring ex­per­tise and new in­sights to sup­port our de­vel­op­ment,” CEO An­toine Pre­stat said in a state­ment. — Nicole De­Feud­is 

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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WIB22: Lead­ing NK cell re­searcher re­flects on roots in Iran, the UK and Texas

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

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WIB22: Chas­ing af­ter ever-evolv­ing sci­ence takes a drug hunter across the pond

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Like many scientists, Fiona Marshall would tell you that she loved the natural world growing up — going to look at crabs running around the beach near her childhood home, pondering about the tides. But one thing about biology, in particular, stood out: It was constantly changing, and changing very quickly.

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