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 

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; 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.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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Mi­rati's KRAS drug looks like the fa­vorite in colon can­cer with new da­ta, putting the pres­sure square on Am­gen

With Amgen already providing proof-of-concept for KRAS inhibitors with its sotorasib, Mirati Therapeutics is piecing together a follow-up effort in lung cancer with data it thinks are superior. But in colon cancer, where solo sotorasib has turned in a dud, Mirati may now have a strong case for superiority.

Mirati’s adagrasib, dosed solo or in combination with chemotherapy cetuximab, showed response rates grater than sotorasib solo  and as part of combination study in a similar patient population also revealed this week at #ESMO21. Mirati’s data were presented as part of a cohort update from the Phase II KRYSTAL-1 study testing adagrasib in a range of solid tumors harboring the KRAS-G12C mutation.

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

The best of the rest: High­lights from the be­low-the-fold pre­sen­ta­tions at #ES­MO21

This year’s ESMO Congress has had a major focus on Big Pharma drugs — most notably candidates from Merck and AstraZeneca — but there have also been updates from smaller biotechs with data looking to challenge the big-name drugmakers.

Today, we’re highlighting some of the data releases that flew under the radar at #ESMO21 — whether from early-stage drugs looking to make a mark or older stalwarts with interesting follow-up data.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

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Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.