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Automation Strategies to Enable Scale-up of CAR-T Therapies
CAR-T therapies continue to show tremendous clinical potential; however, manufacturing remains challenging due to the lack of control and automation, as well as process and product characterization during development and manufacturing stages. In this article, Dr. Qasim Rafiq of UCL discusses the importance of establishing standardized, scalable manufacturing and shares insights from his development of an automated CAR-T expansion process.
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Are we clos­er than we think to per­son­al­ized ge­net­ic med­i­cine?
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Max Gelman
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Happy Monday and thanks for starting off your week with Endpoints. With the third quarter well underway, we’re prepping our coverage for all the big earnings reports due out soon. Be sure to subscribe to Endpoints Pharma to get the latest on that — you can sign up here.

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Max Gelman
Senior Editor, Endpoints News
@MaxGelman
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Are we clos­er than we think to per­son­al­ized ge­net­ic med­i­cine?
by Metagenomi

When gene edit­ing ex­plod­ed on­to the scene over three decades ago, it brought pre­vi­ous­ly in­con­ceiv­able dis­ease treat­ment and po­ten­tial­ly cu­ra­tive ther­a­pies in­to view. To­day, gene edit­ing re­mains one of the most grip­ping top­ics in bio­phar­ma — and a re­cent wave of part­ner­ships may move the in­dus­try even clos­er to broad, cu­ra­tive treat­ment for ge­net­ic dis­ease.

Dis­cov­er­ies across the nat­ur­al en­vi­ron­ment de­riv­ing in vi­vo and ex vi­vo biotech­nolo­gies have ush­ered a flood­gate of de­vel­op­ment pos­si­bil­i­ties. With gi­ants like Bay­er, Mod­er­na, Ver­tex and oth­ers sig­nal­ing that gene edit­ing will be a key dri­ver of their fu­ture pipelines, how will the in­dus­try lever­age this new fron­tier of ge­nom­ic tech­nol­o­gy?

A pan­el spon­sored by Metageno­mi and mod­er­at­ed by End­points News at the 2022 BIO In­ter­na­tion­al Con­ven­tion con­vened an in­te­grat­ed group of thought lead­ers, lead­ing sci­en­tists and in­vestors to dis­cuss the best prac­tices to de­vel­op safe, ef­fi­ca­cious gene ther­a­pies for the. Here we cap­ture in­sights from each per­spec­tive and pan­elist.

Ex­pand­ing the gene edit­ing tool­box through metage­nomics

Most ge­net­ic mod­i­fi­ca­tion tri­als to­day em­ploy the rev­o­lu­tion­ary CRISPR-Cas9 method of DNA cut­ting, whose cre­ators were award­ed the No­bel Prize in chem­istry just two years ago. While CRISPR-Cas9 tech­niques may pro­vide a chance to treat some gene-re­lat­ed dis­eases, more in­no­va­tion is need­ed to un­lock the full po­ten­tial of gene edit­ing. With that goal in mind, Metageno­mi is lead­ing a new wave of star­tups look­ing to cre­ate pow­er­ful next-gen­er­a­tion gene edit­ing sys­tems that can en­able and ac­cel­er­ate the dis­cov­ery and de­vel­op­ment of cus­tomized gene edit­ing ther­a­pies across a wider range of ge­net­ic dis­eases and tis­sues.

Backed by $300 mil­lion, Metageno­mi is comb­ing the world’s nat­ur­al mi­cro­bial en­vi­ron­ment to dis­cov­er break­through gene edit­ing sys­tems ca­pa­ble of edit­ing DNA more pre­cise­ly than cur­rent tech­nol­o­gy can. The sci­ence be­hind the com­pa­ny is metage­nomics, which ex­am­ines the ge­nealog­i­cal com­po­si­tion of mi­crobes from rich, nat­ur­al en­vi­ron­ments such as hot springs, wet­lands and salt flats. It’s a sci­ence that was pi­o­neered by Metageno­mi’s CEO and founder, Bri­an C. Thomas, who spent two decades ded­i­cat­ed to sci­en­tif­ic re­search at UC Berke­ley.

“Us­ing metage­nomics, we’re tap­ping in­to four bil­lion years of mi­cro­bial evo­lu­tion, and the an­swers are there,” said Thomas. “We haven’t had to spend sig­nif­i­cant amounts of time in the lab en­gi­neer­ing these en­zymes in or­der to at­tain the high lev­els of ac­tiv­i­ty that they’re able to hit, be­cause they’ve been op­ti­mized nat­u­ral­ly.”

The com­pa­ny is rapid­ly build­ing the world’s largest, most di­verse tool­box of both CRISPR and non-CRISPR-based gene edit­ing sys­tems through its pro­pri­etary dis­cov­ery and analy­sis en­gine. This ap­proach re­cov­ers DNA from nat­ur­al sam­ples and us­es ad­vanced AI-based cloud com­put­ing to re­veal nov­el cel­lu­lar ma­chin­ery from pre­vi­ous­ly un­stud­ied or­gan­isms that can be op­ti­mized for ther­a­peu­tic ap­pli­ca­tions. Hav­ing a wider se­lec­tion of mod­u­lar tools en­ables sci­en­tists and health­care providers to tar­get ag­gres­sive can­cers, rare dis­eases and oth­er com­plex­i­ties as they seek to make a dif­fer­ence for pa­tients.

Solv­ing ge­net­ic edit­ing chal­lenges

So far Metageno­mi has iden­ti­fied thou­sands of nov­el en­zymes, many of which are ul­tra-small and dis­play unique char­ac­ter­is­tics for en­hanced gene edit­ing ap­pli­ca­tions. Com­pared to cur­rent edit­ing sys­tems, which are based on a few, large en­zymes with tar­get­ing and de­liv­ery is­sues, Metageno­mi sys­tems of­fer flex­i­bil­i­ty. This broad en­zyme di­ver­si­ty and small­er size en­ables greater ef­fi­cien­cy, speci­fici­ty and genome tar­getabil­i­ty, re­sult­ing in en­hanced pa­tient safe­ty.

“ we start­ed our sur­vey in the nat­ur­al en­vi­ron­ment of these unique nu­cle­as­es, we tried to find char­ac­ter­is­tics that would re­al­ly be ben­e­fi­cial with cur­rent de­liv­ery ca­pa­bil­i­ties,” said Thomas. He not­ed that the com­pa­ny is still ex­plor­ing the bio­chem­i­cal prop­er­ties of its unique nu­cle­as­es, in­clud­ing greater sta­bil­i­ty and re­silience due to their ori­gins in ex­treme en­vi­ron­ments, where pH and tem­per­a­ture, for ex­am­ple, are wild­ly atyp­i­cal.

The com­pa­ny’s tool­box is cur­rent­ly weight­ed to­ward CRISPR-based sys­tems but is ex­pand­ing to in­clude tar­get­ed in­te­gra­tion of large DNA frag­ments, base edit­ing and oth­er types of gene edit­ing tools.

Pro­pelling in vi­vo and ex vi­vo ther­a­pies through part­ner­ships

Metageno­mi’s pipeline fo­cus­es on de­ploy­ing its whol­ly-owned di­verse tool­box in both in vi­vo and ex vi­vo ap­pli­ca­tions. “As we start­ed to de­vel­op these tools from the nat­ur­al en­vi­ron­ment, we re­al­ized they had char­ac­ter­is­tics and prop­er­ties that could make them unique in ei­ther of those set­tings,” not­ed Thomas. “From a busi­ness point of view, we’ve re­al­ly fo­cused on a part­ner­ship strat­e­gy around lever­ag­ing both of these tech­nol­o­gy ap­pli­ca­tions.”  In ad­di­tion to en­abling part­ners, Metageno­mi al­so has a whol­ly-owned pipeline of next-gen­er­a­tion ther­a­pies de­vel­oped us­ing the com­pa­ny’s nov­el tech­nolo­gies.

Mod­er­na re­al­ized a part­ner­ship with Metageno­mi — which was an­nounced last year — could help dif­fer­en­ti­ate it as an in vi­vo mar­ket leader in the fu­ture, in­clud­ing through ge­net­ic sys­tem re­pro­gram­ming. The re­search col­lab­o­ra­tion will com­bine Metageno­mi’s gene edit­ing sys­tems with Mod­er­na’s mR­NA tech­nolo­gies to de­vel­op in­no­v­a­tive in vi­vo gene edit­ing ther­a­peu­tics tar­get­ing var­i­ous ge­net­ic dis­eases.

“When work­ing with part­ners like Metageno­mi, we can ac­tu­al­ly cre­ate the next gen­er­a­tion of func­tion­al ge­nom­ic fac­tors that com­bine mR­NA tech­nol­o­gy and the pow­ers of chain man­u­fac­tur­ing,” ex­plained Er­ic Huang, Mod­er­na Ge­nomics’ chief sci­en­tif­ic of­fi­cer. “That’s our blue-sky am­bi­tion and goal … com­bin­ing that knowhow, the fu­ture tech­nol­o­gy gen­er­a­tion, as well as Mod­er­na’s man­u­fac­tur­ing prowess, to re­al­ly make per­son­al­ized ge­net­ic med­i­cine in­to re­al­i­ty” in years to come.

The Metageno­mi team is al­so “ex­cit­ed about the fact that in the ex vi­vo cell ther­a­py space, where the gene edit­ing com­po­nent is a tool to cre­ate a more im­pact­ful and pow­er­ful cell prod­uct, there’s a lot of room for de­vel­op­ment,” shared Thomas.

In fact, the com­pa­ny just an­nounced a part­ner­ship with start­up Affi­ni-T Ther­a­peu­tics to ad­vance next-gen­er­a­tion ex vi­vo T-cell re­cep­tor ther­a­pies — or TCR — for pa­tients with sol­id tu­mors. The work will tar­get core onco­genic dri­vers us­ing Metageno­mi’s pro­pri­etary gene edit­ing sys­tems to pro­mote sus­tained clin­i­cal out­comes for can­cer pa­tients. “Sol­id tu­mors are re­al­ly the next fron­tier in on­col­o­gy,” not­ed Aude Cha­puis, co-founder of Affi­ni-T, as­so­ciate pro­fes­sor at the Fred Hutchin­son Can­cer Cen­ter and a sci­en­tif­ic ad­vi­sor to Metageno­mi. “We’re in­ter­est­ed in TCR tech­nol­o­gy be­cause it is able to tar­get in­tra­cel­lu­lar anti­gens … we can re­al­ly broad­en the num­ber of pro­teins that we’re tar­get­ing,” she said. “Ul­ti­mate­ly, the non-vi­ral gene edit­ing is go­ing to be ex­treme­ly valu­able for the TCR field in par­tic­u­lar.”

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Jay Hagan, Regulus Therapeutics CEO
1
by Max Gelman

Sanofi is scrap­ping a drug from a long­time part­ner.

The Big Phar­ma tells End­points News it has halt­ed de­vel­op­ment of lademirsen, dis­cov­ered by Reg­u­lus Ther­a­peu­tics, af­ter the pro­gram failed a Phase II in­ter­im analy­sis. Sanofi added that the move was not due to safe­ty con­cerns. Lademirsen is de­signed to slow kid­ney func­tion de­cline in pa­tients with Al­port syn­drome.

“While this tri­al did not pro­vide the re­sults we had an­tic­i­pat­ed, our re­search has added val­ue to the sci­en­tif­ic un­der­stand­ing of Al­port Syn­drome, and we re­main com­mit­ted to ad­vanc­ing new ther­a­pies for rare kid­ney dis­eases,” a Sanofi spokesper­son told End­points.

Reg­u­lus in­vestors did not take the news well, with the biotech’s stock RGLS falling rough­ly 20% in ear­ly Mon­day trad­ing, but re­bound­ing to set­tle around a 10% drop. End­points has reached out to Reg­u­lus for com­ment and will up­date ac­cord­ing­ly.

The com­pa­nies first teamed up back in 2010, sign­ing a deal worth $25 mil­lion up­front and up to $750 mil­lion in mile­stones. Their agree­ment cen­tered around mi­croR­NA and at the time was billed as the largest mi­croR­NA pact ever agreed up­on. Sanofi and Reg­u­lus set their sights on fi­bro­sis with four mi­croR­NA tar­gets.

Lademirsen, then known as RG-012, was their lead part­ner­ship pro­gram, and the pair soon ex­pand­ed on the deal in 2012 to in­clude on­col­o­gy. In 2014, Sanofi an­nounced it had ob­tained the op­tion to in-li­cense any of the drugs on which the com­pa­nies col­lab­o­rat­ed, and things were look­ing up for the biotech.

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Endpoints In Focus premium
2
by Jared Whitlock

It’s rare for a com­pa­ny to high­light its bur­geon­ing com­pe­ti­tion. But Il­lu­mi­na did just that in a court fil­ing last month.

The San Diego-based com­pa­ny — which makes DNA se­quencers that pow­er every­thing from con­sumer ge­net­ic tests to pre­ci­sion drug de­vel­op­ment — com­mands about 80 per­cent of the mar­ket. The Fed­er­al Trade Com­mis­sion in 2021 cit­ed Il­lu­mi­na’s mar­ket share in chal­leng­ing the com­pa­ny’s ac­qui­si­tion of Grail, a can­cer test­ing com­pa­ny.

But in re­sponse to the FTC, Il­lu­mi­na in the le­gal brief con­tend­ed it’s not a mo­nop­o­list by point­ing to ri­vals, in­clud­ing com­pa­nies that re­cent­ly re­leased DNA se­quencers and a Chi­nese be­he­moth that will soon en­ter the US mar­ket. The FTC has ar­gued that se­quenc­ing re­mains a tough mar­ket to crack and po­ten­tial com­pe­ti­tion will take time to scale.

Il­lu­mi­na has si­mul­ta­ne­ous­ly played up ri­vals in hopes of fend­ing off an­titrust reg­u­la­tors while spend­ing heav­i­ly on in­ter­nal pro­grams aimed at com­peti­tors. Re­searchers and clin­i­cians sud­den­ly have more se­quencer op­tions, with more due to ar­rive soon, for pur­pos­es like find­ing the ge­net­ic ba­sis of life-threat­en­ing dis­eases.

“It’s fun­ny to have a com­pa­ny com­ing out and say­ing some­thing to the ef­fect of, ‘No, we’re not ac­tu­al­ly that great,’” said Kei­th Ro­bi­son, a com­pu­ta­tion­al bi­ol­o­gist who blogs about the ge­nomics in­dus­try.

Ob­servers say Il­lu­mi­na’s nov­el strat­e­gy of tout­ing se­quenc­ing com­peti­tors serves to not on­ly se­cure Grail, but tap a can­cer test­ing mar­ket that could be worth as much as $50 bil­lion by 2035. Il­lu­mi­na de­clined an in­ter­view re­quest.

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Baisong Mei, incoming Editas VP and CMO
3
by Aayushi Pratap

Cam­bridge-based genome edit­ing com­pa­ny Ed­i­tas Med­i­cine has ap­point­ed Baisong Mei as its new VP and chief med­ical of­fi­cer, the com­pa­ny an­nounced Mon­day.

The new hire comes five months af­ter Ed­i­tas fired its for­mer CMO Lisa Michaels just 15 months af­ter her join­ing. It is un­clear why she was let go.

In April, the com­pa­ny got its new CEO, Gilmore O’Neill, its third CEO in the last three years. The new hires are part of a re­build­ing of the C-suite.

“I am very ex­cit­ed to join Ed­i­tas,” Mei told End­points News. “Ed­i­tas has great tech­nol­o­gy and sci­ence in gene edit­ing and ther­a­py and have a great po­ten­tial to treat pa­tients with un­met needs.”

Mei has over 20 years of ex­pe­ri­ence in the biotech and phar­ma­ceu­ti­cal in­dus­try. He pre­vi­ous­ly served as the se­nior glob­al project head for rare dis­eases and rare blood dis­or­ders at Sanofi for near­ly six years where he led the clin­i­cal de­vel­op­ment pipeline in sev­er­al rare dis­eases in hema­tol­ogy, neu­rol­o­gy and nephrol­o­gy.

Pri­or to that, he was at Bio­gen for al­most sev­en years where he worked on he­mo­phil­ia and he­mo­glo­binopa­thy. Some of his ma­jor achieve­ments in­clude bring­ing nov­el med­i­cines through clin­i­cal de­vel­op­ment and glob­al reg­u­la­to­ry ap­proval, in­clud­ing Al­pro­lix and Eloc­tate, med­i­cines for he­mo­phil­ia B.

“First thing I need to do is learn more about what is go­ing on at Ed­i­tas,” Mei added. Ed­i­tas, found­ed in 2013, was born out of the sci­ence of CRISPR and gene edit­ing lead­ers Feng Zhang, George Church, J. Kei­th Joung and David Liu. The com­pa­ny us­es gene edit­ing tools such as CRISPR/Cas9 and CRISPR/Cas12a to build a pipeline of treat­ments for dis­eases. While Ed­i­tas was the first to test gene-edit­ing ther­a­py in hu­mans, In­tel­lia Ther­a­peu­tics, its com­peti­tor, be­came the first biotech to an­nounce re­sults of genome edit­ing in hu­mans.

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Thomas Cannell, Sesen Bio CEO
4
by Lei Lei Wu

Fol­low­ing an FDA re­jec­tion last year, Sesen Bio an­nounced this morn­ing that it will be paus­ing de­vel­op­ment on its lead blad­der can­cer drug.

With the pause, Sesen can save mon­ey while it looks for al­ter­na­tives, the Cam­bridge, MA-based biotech said in a press re­lease. It not­ed that it would be look­ing for a part­ner for its blad­der can­cer drug Vicineum.

In Au­gust, the FDA re­ject­ed Vicineum — which the biotech as­cribed to man­u­fac­tur­ing is­sues and the FDA ask­ing for more da­ta. The biotech said its de­ci­sion for paus­ing de­vel­op­ment came af­ter as­sess­ing costs for an ad­di­tion­al Phase III tri­al for the drug in blad­der can­cer.

In a state­ment, Sesen Bio CEO and pres­i­dent Thomas Can­nell not­ed that the biotech has had four meet­ings with the FDA since the re­jec­tion. He added:

We have al­so re­cent­ly ob­served an evo­lu­tion of the cur­rent treat­ment par­a­digm in NMIBC, with sub­stan­tial up­take of in­trav­es­i­cal chemother­a­py (monother­a­py and com­bi­na­tion ther­a­py) dur­ing the on­go­ing BCG short­age. In as­sess­ing the im­pact of the reg­u­la­to­ry and com­mer­cial land­scape, we have made the de­ci­sion to pause the clin­i­cal de­vel­op­ment of Vicineum.

In a sep­a­rate SEC fil­ing, the biotech al­so said that it is sell­ing its line of IL-6 an­ti­bod­ies to Roche, which it orig­i­nal­ly out-li­censed to the Swiss phar­ma in 2016, when it was still known as Eleven Bio. In the orig­i­nal deal, the biotech had li­censed the an­ti­bod­ies, in­clud­ing one known as EBI-031 for which the FDA had cleared for clin­i­cal tri­als, for an up­front fee of $7.5 mil­lion.

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Bahija Jallal, Immunocore CEO
5
by Paul Schloesser

Look­ing to se­cure a fresh raise to in­vig­o­rate R&D, Im­muno­core is putting to­geth­er a PIPE.

The for­mer Medi­gene spin­out, six months af­ter the FDA ap­proved its TCR drug tebenta­fusp, put out word Mon­day that it's sell­ing just over 3.7 mil­lion shares of com­pa­ny stock at $37.50 each. This is go­ing to cer­tain in­vestors as a PIPE fi­nanc­ing for a to­tal of $140 mil­lion.

Shares of IM­CR stayed con­sis­tent in pre­mar­ket trad­ing, on­ly slid­ing down half of 1% af­ter clos­ing at $43.25 a share Fri­day af­ter­noon.

Dif­fer­ent in­vestors that jumped in on the round in­clud­ed RTW In­vest­ments, LP, Rock Springs Cap­i­tal, and Gen­er­al At­lantic. The fi­nanc­ing is slat­ed to close some­time Wednes­day, Im­muno­core said in a state­ment.

The news comes amidst a grow­ing trend of biotechs seek­ing pri­vate place­ments as bears con­tin­ue to close the mar­ket's win­dow for fol­low-on rounds. Ear­li­er this month, a tri­umvi­rate of com­pa­nies — An­nex­on, Proven­tion Bio and Nurix Ther­a­peu­tics — put to­geth­er pri­vate place­ments in the span of 24 hours, and Faseem Has­nain's Gos­samer Bio chan­neled its ef­forts in­to a place­ment of its own last week.

CEO Bahi­ja Jal­lal tells End­points News that ac­cel­er­at­ing the com­pa­ny's pipeline is some­thing that is "al­ways on our radar," and that the fo­cus will be on de­vel­op­ing can­di­dates for more in­di­ca­tions in can­cer, in­clud­ing in sol­id tu­mors and in in­fec­tious dis­eases. So far, the biotech has two can­di­dates in in­fec­tious dis­ease: one for HIV and one for HBV.

"We're very hap­py with where we are," the CEO said.

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Chris Schelling, Acer Therapeutics CEO
6
by Amber Tong

Less than a month af­ter the FDA re­ject­ed Ac­er Ther­a­peu­tics’ treat­ment for urea cy­cle dis­or­ders be­cause its con­tract pack­ag­ing man­u­fac­tur­er wasn’t ready for an in­spec­tion, the biotech said it’s sort­ed things out.

The biotech has re­filed an NDA for AC­ER-001, it an­nounced Mon­day morn­ing, and be­lieves the re­sub­mis­sion ad­dress­es all the con­cerns raised in the com­plete re­sponse let­ter.

In a state­ment, founder and CEO Chris Schelling praised his team’s “out­stand­ing job” re­sub­mit­ting the NDA one month af­ter the CRL.

“Our third-par­ty con­tract man­u­fac­tur­ing part­ner has been in­cred­i­bly re­spon­sive and has con­firmed that it is ready for in­spec­tion,” he added. “Our man­u­fac­tur­ing part­ner is re­gard­ed as a glob­al leader in clin­i­cal sup­ply chain and com­mer­cial pack­ag­ing ser­vices with more than 70 years of ex­pe­ri­ence.”

Reg­u­la­tors now have 14 days to de­cide whether to ac­cept the re­filed NDA.

Shares AC­ER climbed around 6% in the wake of the news to $1.62 but were far from re­cu­per­at­ing the loss from the re­jec­tion.

When it dis­closed the re­jec­tion, Ac­er not­ed that while the FDA re­quest­ed “ad­di­tion­al ex­ist­ing non­clin­i­cal in­for­ma­tion to be pro­vid­ed in the re­sub­mis­sion of the NDA, it was “not an ap­prov­abil­i­ty is­sue.”

De­vel­oped in part­ner­ship with Re­lief Ther­a­peu­tics, AC­ER-001 is an oral for­mu­la­tion of sodi­um phenyl­bu­tyrate de­signed to min­i­mize the un­pleas­ant taste that Ac­er said has led to com­pli­ance prob­lems with ex­ist­ing drugs con­tain­ing the com­pound and ap­proved to treat urea cy­cle dis­or­ders. It is al­so be­ing de­vel­oped for maple syrup urine dis­ease.

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Bob Azelby, Eliem Therapeutics CEO
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by Kyle LaHucik

In the drug R&D world, plans are not set in stone, as Eliem Ther­a­peu­tics ex­hib­it­ed Mon­day with shifts to its de­pres­sion strat­e­gy.

The Seat­tle-UK biotech orig­i­nal­ly want­ed to start two mid-stage de­pres­sion tri­als this year but will have to de­lay one — in ma­jor de­pres­sive dis­or­der — un­til next year and in­def­i­nite­ly post­pone the oth­er in per­i­menopausal de­pres­sion. Back in April, Eliem de­layed the launch­es of both Phase IIa tri­als be­cause the biotech was cu­ri­ous as to why the ex­po­sure was low­er than ex­pect­ed in a Phase Ib study, which tried to prove out the con­cepts of ETX-155 in pho­to­sen­si­tive epilep­sy.

Now, with re­sults from an ini­tial re­view, the biotech thinks cer­tain parts of the CMC are “most like­ly” to blame for the re­duced ex­po­sure lev­els. The com­pa­ny thinks there might have been dif­fer­ences be­tween the batch­es of drug used in Phase I tri­als and the Phase Ib PSE study.

With that in­fo in hand, Eliem will take the GABAa pos­i­tive al­losteric mod­u­la­tor through an­oth­er Phase I phar­ma­co­ki­net­ic tri­al us­ing the drug batch­es that were used in the PSE study. The biotech plans to find out which dose will pro­vide a sim­i­lar ex­po­sure to the 60 mg dose used in the 14-day re­peat dose healthy vol­un­teer study pre­vi­ous­ly con­duct­ed.

Re­sults from that tri­al are slat­ed for the fourth quar­ter, and then a ran­dom­ized, place­bo-con­trolled Phase IIa in MDD pa­tients will kick off the first quar­ter of the new year. The mid-stage tri­al will test the drug as a four-week treat­ment, with topline da­ta to come through in mid-2024.

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Emma Walmsley, GSK CEO (GSK Investor Day 5)
8

What a dif­fer­ence one re­al­ly good Phase III read­out and a cou­ple of late-stage buy­outs can make.

GSK CEO Em­ma Walm­s­ley has been try­ing, hard, to build some ex­cite­ment around the pipeline. And with the re­cent pos­i­tive out­come for their RSV vac­cine, she’s fi­nal­ly earn­ing some mar­ket re­spect on that score. And with the big con­sumer split to­day, with the birth of Ha­le­on, you can ex­pect plen­ty of buzz about the need for an­oth­er M&A deal to po­si­tion the com­pa­ny.

Reuters helped set the stage for that with some num­bers crunch­ing around New GSK, which will stake its fu­ture on drug R&D. Ha­le­on sailed away with a heavy load of debt, re­ports the wire ser­vice, New GSK will be left “with bor­row­ing of less than 1 times 2023 EBIT­DA, ac­cord­ing to Break­ingviews cal­cu­la­tions.”

They’ll al­so have a sol­id HIV busi­ness at Vi­iV and a block­buster shin­gles vac­cine Shin­grix that has enor­mous po­ten­tial, dri­ving ex­pec­ta­tions of sales growth. And if the R&D team un­der Tony Wood — with Hal Bar­ron’s de­par­ture from the top post in re­search — can’t add some more ex­cite­ment about block­busters-to-be, then the BD team will need to step in again.

Reuters help­ful­ly even posts a sug­gest­ed nat­ur­al tar­get for New GSK: their part­ner Spring­Works, which has been roughed up this year on Wall Street. Shares are off more than 50% since the start of the year, leav­ing a $1.5 bil­lion tar­get well with­in Walm­s­ley’s reach. And that is af­ter a pos­i­tive Phase III tri­al for their part­nered drug nirogace­s­tat in soft tis­sue tu­mors.

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9
by Amber Tong

For the past sev­en years, biotech spin­outs from the Uni­ver­si­ty of Ox­ford have of­ten tapped in­to a re­li­able source of in­vest­ments from Ox­ford Sci­ence En­ter­pris­es, the com­pa­ny tasked with found­ing, fund­ing and build­ing star­tups based on “ex­cep­tion­al sci­ence” flow­ing out of Ox­ford.

And the pool has got­ten $300 mil­lion (£250 mil­lion) deep­er as Ox­ford Sci­ence En­ter­pris­es bags a new raise that puts its tal­ly over $1 bil­lion (£850 mil­lion).

Life sci­ences isn’t the on­ly fo­cus of Ox­ford Sci­ence En­ter­pris­es — there’s al­so health tech and deep tech vy­ing for at­ten­tion — but drug de­vel­op­ers may take par­tic­u­lar de­light in its plans to de­ploy “in­creas­ing­ly large amounts of cap­i­tal in lat­er-stage fund­ing rounds.”

With port­fo­lio com­pa­nies ma­tur­ing and get­ting clos­er to promised break­throughs in can­cer, heart fail­ure and in­fec­tious dis­eases along­side cli­mate change, food se­cu­ri­ty and quan­tum com­put­ing, such “scale-up cap­i­tal” will be in­creas­ing­ly cru­cial, said CEO Alex­is Dor­mandy.

"This fundraise comes at an ex­cit­ing and piv­otal time for OSE,” he said in a state­ment. “Over the next few years, we ex­pect these com­pa­nies will con­tin­ue to make im­por­tant progress; with our on­go­ing ef­forts, and the sup­port of our share­hold­ers, we look for­ward to help­ing them de­liv­er im­pact and re­turns.”

Ox­ford Sci­ence En­ter­pris­es likes to tout its found­ing in­vestor role in Vac­citech, the vac­cine de­vel­op­er that played a con­sid­er­able role in As­traZeneca’s Covid-19 shot.

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10
by Aayushi Pratap

French com­pa­ny En­terome, which works with the mi­cro­bio­me, has scored $40 mil­lion in a wide-rang­ing deal with its long-time Swiss in­vestor Nestlé Health Sci­ence to co-de­vel­op an oral pill for food al­ler­gies that's on the cusp of en­ter­ing the clin­ic.

“To­day, we be­lieve that we have reached a lev­el where we un­der­stand what the bac­te­ria liv­ing in the hu­man gut are able to pro­duce,” said Pierre Be­lichard, CEO of En­terome.

Study­ing hu­man gut bac­te­ria, En­terome has dis­cov­ered a pro­tein that mim­ics a nat­u­ral­ly pro­duced hu­man hor­mone called In­ter­leukin- 10 (IL-10), known to fight food al­ler­gies and con­di­tions such as in­flam­ma­to­ry bow­el dis­ease.

“IL-10 has been seen for a long time as a mag­ic bul­let able to de­crease in­flam­ma­tion in a lot of dif­fer­ent or­gans in the body,” Be­lichard added. Re­duced in­flam­ma­tion re­duces the im­pact of food al­ler­gies, he said.

The two com­pa­nies will work on a com­pound dubbed EB1010, which con­tains the new­ly dis­cov­ered pro­tein.

Nestlé is al­so bet­ting that there's more where that came from. Lever­ag­ing what En­terome calls En­doMim­ics, the two part­ners will keep look­ing for com­pounds that act like hu­man hor­mones or cy­tokines. Down the road, they al­so hope to cre­ate a new class called Aller­Mim­ics: anti­gens pro­duced by mi­cro­bio­me that mim­ic al­ler­gens such as peanuts.

To kick off this al­liance, En­terome will re­ceive a cash up­front as well as eq­ui­ty from Nestlé. The biotech will be re­spon­si­ble for lead­ing drug dis­cov­ery ac­tiv­i­ties and bear re­lat­ed costs up to the in­ves­ti­ga­tion­al new drug ap­pli­ca­tion. In the fu­ture, Nestlé, which hold around 17% stakes in En­terome, could shell out ad­di­tion­al clin­i­cal and sales pay­ments for each li­censed ther­a­peu­tic can­di­date plus roy­al­ties on net sales.

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11
by Paul Schloesser

Ap­pli­ca­tions are now open for Mer­ck KGaA's newest col­lab­o­ra­tion pro­gram fo­cused on star­tups lo­cat­ed in Asia.

The Ger­man phar­ma an­nounced Thurs­day, spar­ing a few de­tails, that it was launch­ing a new pro­gram in Asia called Up­tune, which "aims to gen­er­ate col­lab­o­ra­tion op­por­tu­ni­ties with ear­ly-stage in­no­v­a­tive com­pa­nies."

The plan, ac­cord­ing to Mer­ck KGaA, will sup­port and give some fi­nanc­ing to cer­tain com­pa­nies in the health­care and life sci­ence space, plus elec­tron­ics and smart man­u­fac­tur­ing. It did em­pha­size it will look for com­pa­nies with a fo­cus on dig­i­tal health and "in­no­v­a­tive tech­nolo­gies/ma­te­ri­als for semi­con­duc­tor and dis­play."

On top of those em­phases, Mer­ck KGaA not­ed it was al­so look­ing at com­pa­nies work­ing in man­u­fac­tur­ing, uti­liz­ing cel­lu­lar, mol­e­c­u­lar- and im­muno-as­says or cell and gene ther­a­py tools, en­gag­ing in man­u­fac­tur­ing spe­cial­ties such as sup­ply chain in­no­va­tion, da­ta man­age­ment, sim­u­la­tion and an­a­lyt­ics; and fi­nal­ly, "chem­istry and ma­te­ri­als for life sci­ence."

As part of the ap­pli­ca­tion process, Mer­ck KGaA said it is look­ing to part­ner with up to five com­pa­nies and grant up to €100,000 in fi­nan­cial as­sis­tance, plus men­tor­ing and coach­ing. The goal for these star­tups is to de­vel­op proof-of-con­cepts and pos­si­bly test what they come up with — and if they do well, it could end up in a part­ner­ship with Mer­ck KGaA.

The Mer­ck Up­tune start­up col­lab­o­ra­tion pro­gram is tak­ing ap­pli­cants through Sept. 4, start­ing the pro­gram of­fi­cial­ly some­time in mid-No­vem­ber.

The com­pa­ny did not re­spond to a re­quest for com­ment from End­points News.

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News Briefing: Quick hits from the biopharma web
12
by Kyle LaHucik, Tyler Patchen

The FDA ap­proved an oral liq­uid for­mu­la­tion of the drug zon­isamide, orig­i­nal­ly made by Eton Phar­ma­ceu­ti­cals and cur­rent­ly owned by Azu­ri­ty Phar­ma­ceu­ti­cals.

The nod gives Azu­ri­ty the abil­i­ty to mar­ket Zon­isade as an ad­junc­tive ther­a­py for treat­ing par­tial seizures in adults and kids 16 years and old­er who have epilep­sy. The green light comes af­ter three dou­ble-blind and place­bo-con­trolled clin­i­cal stud­ies of the drug, which is ad­min­is­tered once or twice a day. Zon­isamide was orig­i­nal­ly ap­proved in the ear­ly 2000s as a cap­sule for treat­ing par­tial epilep­tic seizures and has been mar­ket­ed by a va­ri­ety of com­pa­nies, in­clud­ing the for­mer Irish biotech Elan Phar­ma­ceu­ti­cals, Japan­ese drug­mak­er Ei­sai and Sum­it­o­mo Dainip­pon. In 2010, Elan and Ei­sai agreed to pay $214.5 mil­lion to re­solve al­le­ga­tions of off-la­bel mar­ket­ing of Zone­gran.

Eton sold the oral liq­uid for­mu­la­tion of the drug to Azu­ri­ty as part of a Feb­ru­ary 2021 deal, worth up to $45 mil­lion in pay­ments, in­clud­ing $5 mil­lion when the drug is launched.

"This is now the eighth prod­uct ap­proval that our team has con­tributed to, and we are ex­cit­ed for Azu­ri­ty to bring the prod­uct to pa­tients. The pro­ceeds from the launch mile­stone will be used to fur­ther grow our rare dis­ease port­fo­lio,” Eton CEO Sean Bryn­jelsen said in a state­ment. For Azu­ri­ty, the nod adds to a long list of FDA-ap­proved prod­ucts across car­dio­vas­cu­lar, CNS, en­docrinol­o­gy and or­phan dis­eases. — Kyle LaHu­cik

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