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 

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.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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Hal Barron, Endpoints UKBIO20 (Jeff Rumans)

'Al­tos was re­al­ly a once-in-a-life­time op­por­tu­ni­ty': Hal Bar­ron re­flects on his big move

By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.

Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.

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Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Troy Wilson, Kura CEO

FDA lifts par­tial hold on Ku­ra's Phase Ib AML pro­gram as biotech re­dou­bles mit­i­ga­tion ef­forts

Kura Oncology is clear to resume studies for its early-stage leukemia program after the FDA lifted a clinical hold Thursday afternoon.

Regulators had placed the hold on a Phase Ib study of KO-539, an experimental oral treatment for some genetic subsets of acute myeloid leukemia last November after a patient died while taking the drug. Kura expects to begin enrolling patients again imminently, CEO Troy Wilson told Endpoints News.

A Sen­ate bill wants to even an 'un­lev­el play­ing field' for do­mes­tic, for­eign in­spec­tion drop-ins amid back­log

Amid geopolitical tensions between the US and China, two Republican senators are calling for a bill that would aim to strike a balance on domestic and foreign inspection requirements from the FDA.

Sens. Mike Braun (R-IN) and Joni Ernst (R-IA) have penned a bill called the Creating Efficiency in Foreign Inspections Act. It contains a bit of rhetoric, highlighting “communist China” not once, but twice in the release, but states that the goal is to even the playing field between foreign and American manufacturers.

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