Carol Robinson, Professor Dame Carol Robinson Research Group

Drug dis­cov­ery in HD: Ox­ford spin­of­f's mass spec­trom­e­try ap­proach scores fresh fund­ing

The tech­nol­o­gy used to de­tect ex­plo­sives at air­ports — mass spec­trom­e­try — is be­ing pi­lot­ed as an en­gine for drug dis­cov­ery.

Mass spec­trom­e­try is a tool de­signed to mea­sure with pro­found ac­cu­ra­cy the mass of a sin­gle mol­e­cule. Typ­i­cal­ly, mass spec­trom­e­ters can be used to iden­ti­fy un­known com­pounds, to quan­ti­fy known com­pounds, and to de­ter­mine the struc­ture and chem­i­cal prop­er­ties of mol­e­cules.

Us­ing this in­for­ma­tion to de­vel­op drugs, ver­sus us­ing tra­di­tion­al cell-based as­says is an ap­proach pi­o­neered by Car­ol Robin­son, the first fe­male pro­fes­sor of chem­istry at the Uni­ver­si­ty of Ox­ford. Along­side her for­mer stu­dents, Robin­son found­ed OMass Ther­a­peu­tics to move it for­ward in the field of mem­brane tar­gets, in­clud­ing GPCRs.

On Mon­day, the Ox­ford spin­off un­veiled £27.5 mil­lion in ad­di­tion­al Se­ries A fi­nanc­ing from Syn­cona, Ox­ford Sci­ences In­no­va­tion (OSI), and the Uni­ver­si­ty of Ox­ford, bring­ing its to­tal haul to £41.5 mil­lion.

“It’s drug dis­cov­ery in high de­f­i­n­i­tion…it’s all about re­veal­ing the pic­ture with high­er res­o­lu­tion. And it’s about high con­tent da­ta. The rea­son mass spec­trom­e­try is in­ter­est­ing in drug dis­cov­ery is that the con­tent that you get from the da­ta is much high­er res­o­lu­tion than through a cell-based as­say,” OMass chief Ros Dee­gan told End­points News.

“So in a cell-based as­say, you’re ef­fec­tive­ly an­swer­ing on­ly the ques­tion that you asked, where­as, in na­tive mass spec­trom­e­try, it’s a bio­phys­i­cal ap­proach.”

The ma­jor­i­ty of ex­ist­ing ther­a­peu­tics tar­get mem­brane pro­tein — ac­ces­si­ble on the sur­face of cells — to mod­i­fy cel­lu­lar sig­nal­ing. In con­trast to tra­di­tion­al cell-based as­says, the mass spec­trom­e­try ap­proach of­fers ‘high de­f­i­n­i­tion’ in­sight in­to un­der­stand­ing mem­brane pro­tein bi­ol­o­gy, Dee­gan not­ed.

The plat­form pro­vides ac­cess to dif­fer­ent chem­i­cal start­ing points be­cause bio­phys­i­cal ap­proach­es tend to point to­ward dif­fer­ent chem­istry — as they are not bi­ased to spe­cif­ic types of chem­istry through the use of cell-based as­says, she said. “Cell-based as­says tend to put you in lipophilic, quite of­ten large lipophilic com­pounds.”

If the ap­proach is shown to work, it could serve to short­en drug dis­cov­ery time­frames.

“Ul­ti­mate­ly the com­pa­ny aims to use high de­f­i­n­i­tion tech­nolo­gies to ef­fi­cient­ly ac­cess can­di­dates with a greater PoS (prob­a­bil­i­ty of suc­cess) in high-val­ue ar­eas, there­fore sig­nif­i­cant­ly short­en­ing the drug dis­cov­ery time­line for small mol­e­cules,” said Lach­lan MacK­in­non, prin­ci­pal at OSI in an email to End­points.

OMass in­tends to not just iden­ti­fy mol­e­cules of in­ter­est but to take them through to the mar­ket them­selves. It has three pro­grams at the mo­ment — al­though they are far from be­ing ready for the clin­ic.

The cap­i­tal in­jec­tion will give OMass — which is fo­cus­ing on im­munol­o­gy and ge­net­i­cal­ly de­fined dis­eases out­side of im­munol­o­gy — just over two years of run­way and will be used to shep­herd its lead pro­gram in­to pre­clin­i­cal de­vel­op­ment.

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.

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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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.

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.

Stephen Squinto, Gennao Bio chairman

Scoop: Stephen Squin­to’s Yale spin­out is rais­ing a Se­ries B for ge­net­ic med­i­cines

The nucleic acid therapeutics at Stephen Squinto’s Yale-originated biotech are en route to a $40 million R&D payday.

After a Series A of that size in the spring of 2021, Hopewell, NJ-based Gennao Bio is closing on a round of the same amount, Endpoints News has learned. A Series B is in the works, according to an investor’s post on LinkedIn and as confirmed by a spokesperson. An SEC filing on Thursday outlines $15 million in equity sold thus far in the round.

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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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