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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.