Malcolm Weir, Sosei vice chairman

Glax­o­SmithK­line joins So­sei's who's-who list of clients with glob­al li­cens­ing pact for IBD small mol­e­cules

Big drug­mak­ers are on the hunt for nov­el ther­a­peu­tics in a range of ul­tra-com­pet­i­tive ther­a­peu­tic ar­eas. Japan’s So­sei Hep­tares has emerged in re­cent years as a sought-af­ter part­ner for Big Phar­mas seek­ing nov­el neu­ro can­di­dates — but now Glax­o­SmithK­line wants to try the pre­clin­i­cal R&D ex­pert’s work in in­flam­ma­to­ry bow­el dis­ease.

GSK has tapped So­sei Hep­tares for dis­cov­ery work tar­get­ing IBD and oth­er gas­troin­testi­nal im­mune dis­or­ders with small-mol­e­cule ag­o­nists for GPR35, an or­phan G pro­tein-cou­pled re­cep­tor (GPCR) with a ge­net­ic link to those con­di­tions, the com­pa­nies said Sun­day.

As part of the deal, So­sei will lead pre­clin­i­cal R&D on ag­o­nists iden­ti­fied by its struc­ture-based drug de­sign plat­form and pro­pri­etary StaR tech­nol­o­gy, and will hand clin­i­cal work, man­u­fac­tur­ing and com­mer­cial­iza­tion to GSK. The British drug gi­ant will hold glob­al com­mer­cial li­cens­ing rights to any win­ning can­di­dates and will hand an ini­tial $44 mil­lion for near-term de­vel­op­ment mile­stones and re­search fund­ing. So­sei will al­so be due up to $437 mil­lion pend­ing a se­ries of de­vel­op­ment, reg­u­la­to­ry and com­mer­cial­iza­tion mile­stones.

“Us­ing ge­net­ics to pri­ori­tise po­ten­tial treat­ments for a chal­leng­ing im­mune dis­or­der like IBD ex­em­pli­fies the ap­proach we are tak­ing at GSK to de­vel­op nov­el trans­for­ma­tive med­i­cines,” John Lep­ore, GSK’s se­nior VP of re­search, said in a re­lease. “So­sei Hep­tares is a world leader with an es­tab­lished track record in GPCR drug dis­cov­ery, so we are con­fi­dent in our po­ten­tial to joint­ly iden­ti­fy high qual­i­ty clin­i­cal can­di­dates to de­liv­er im­por­tant new med­i­cines for the mil­lions of pa­tients who are wait­ing.”

It’s a big-name pick­up for So­sei, which has made its name in li­cens­ing out ear­ly-stage GPR tar­get­ing can­di­dates to ma­jor drug­mak­ers, most no­tably Ab­b­Vie and Roche’s Genen­tech. Mal­colm Weir, So­sei’s vice chair­man, tout­ed GSK’s ex­ist­ing ex­per­tise in IBD and ex­ten­sive clin­i­cal ex­pe­ri­ence as a ma­jor draw for So­sei’s li­cens­ing strat­e­gy.

“In our minds, (this deal) was very dri­ven by a group that we can work with and col­lab­o­rate ef­fec­tive­ly,” Weir said. “But, it’s a project that still needs to work it­self out in the clin­ic.”

Weir pre­dict­ed a clutch of So­sei can­di­dates would see the clin­ic in the “near-term” fu­ture but de­clined to guess on ex­act­ly when that would be. The Japan­ese com­pa­ny has made its name iden­ti­fy­ing GPCR can­di­dates, most­ly spe­cial­iz­ing in neu­ro­science, Weir said. While IBD is a most­ly new en­deav­or, So­sei thinks there’s po­ten­tial in that ther­a­peu­tic area.

“What we did was looked at a fair num­ber of nov­el tar­gets and there was ev­i­dence of ge­net­ic as­so­ci­a­tions with dis­eases like ul­cer­a­tive col­i­tis and oth­er dis­or­ders,” Weir said. “We were very struck by that and struck by the promise of that.”

Back in June, Ab­b­Vie tapped So­sei in a very sim­i­lar deal, agree­ing to pay $32 mil­lion up­front for a range of in­flam­ma­to­ry and au­toim­mune dis­eases with po­ten­tial op­tion, de­vel­op­ment and com­mer­cial pay­ments of up to $377 mil­lion. That deal could ex­pand, how­ev­er, if Ab­b­Vie choos­es to ex­e­cute on all four tar­gets in the pact; in that case, the agree­ment could grow in­to the bil­lion-dol­lar range, a So­sei spokesper­son said at the time.

That was ac­tu­al­ly So­sei’s sec­ond deal with Ab­b­Vie af­ter the com­pa­ny signed a deal with Al­ler­gan — which Ab­b­Vie ac­quired in mid-2019 — back in 2016 for a slate of sub­type-se­lec­tive mus­carinic re­cep­tor ag­o­nists. In that deal, So­sei got $125 mil­lion up­front with a $50 mil­lion com­mit­ment to­ward Phase II stud­ies in ad­di­tion to over $3 bil­lion in promised mile­stones.

In Ju­ly 2019, Genen­tech al­so jumped on board with So­sei, agree­ing to front $26 mil­lion in up­front and near-term pay­ments, in ad­di­tion to fu­ture mile­stone pay­ments that may ex­ceed $1 bil­lion, as well as po­ten­tial roy­al­ties. The tar­get­ed ther­a­peu­tic ar­eas weren’t dis­closed in that deal. So­sei has al­so signed pre­vi­ous pacts with British drug­mak­ers As­traZeneca and No­var­tis, among a suite of oth­er Big Phar­mas.

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.

What lured Hal Bar­ron away?; Top FDA minds on ac­cel­er­at­ed ap­proval re­forms; ‘Dead wrong’ Aduhelm ad blitz; 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.

Nothing can really compete with Hal Barron’s departure from GlaxoSmithKline as the news of the week, but we do have plenty of original reporting and analysis from the Endpoints team in this edition. Enjoy and have a nice weekend.

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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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Mer­ck wins le­gal bat­tle over in­sur­ance cov­er­age af­ter ran­somware at­tack

Merck has emerged victorious from a years-long legal battle with insurers over the coverage of more than a billion dollars in losses from the malware NotPetya, with a New Jersey Superior Court judge concluding that the responsibility is on insurers to clarify their policies around cyber attacks.

The pharma giant was one of several victims of a global cyber attack back in 2017 that also hit Danish shipping company Maersk, American food company Mondelēz, French construction giant Saint-Gobain and even the systems monitoring the Chernobyl nuclear power stations, Bloomberg reported back in 2019.

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Crit­ics push back on Alzheimer’s As­so­ci­a­tion ad blitz to get Medicare to change its Aduhelm rul­ing: 'Dead wrong'

The latest Alzheimer’s Association advertising campaign encourages people to fight.

Not against the disease or for more research or treatments, but against the Centers for Medicare and Medicaid Services. More specifically, CMS’ recent reimbursement decision to only pay for Biogen and Eisai’s controversial Alzheimer’s drug Aduhelm for patients in clinical trials.

With CMS’ preliminary decision now in a 30-day comment period, patient advocates’ goal is to convince CMS to reverse its decision with a marketing blitz and public pressure.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Fail­ing to con­firm clin­i­cal ben­e­fit, Gilead pulls 2 ac­cel­er­at­ed ap­proval in­di­ca­tions for can­cer drug

Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.

“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.

Richard Pazdur (via AACR)

Time lim­its on ac­cel­er­at­ed ap­provals? FDA's on­col­o­gy chief Rick Paz­dur eyes po­ten­tial re­forms via in­ter­na­tion­al ap­proach­es

The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.

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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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