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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Per­cep­tive's fourth — yes, fourth — SPAC jumps to Nas­daq as the blank check tree con­tin­ues to ripen

The biotech SPAC boom has gone almost hand-in-hand with the industry’s IPO gold rush, and this week saw more blank check companies hop aboard the train.

Leading the way is Perceptive Advisors’ fourth SPAC, appropriately named Arya Sciences Acquisition IV, which priced Friday morning after raising $130 million. And on top of that, new Ziopharm executive chair James Huang is launching his own SPAC with MSD Partners and Panacea Venture, filing S-1 paperwork Thursday with plans to raise $200 million.

CEO Fred Aslan (Artiva)

NK cell ther­a­py play­er Arti­va makes some more noise, pulling in $120M Se­ries B less than a month af­ter Mer­ck deal

Not even one month after Big Pharma took notice of Artiva when Merck signed a collaboration worth nearly $2 billion in milestones, the off-the-shelf NK cell biotech already has its next big fundraise.

Artiva returns from the venture well Friday with a $120 million Series B round, money they will use to get their first program into the clinic and to file INDs for another two candidates. The raise marks the latest development in a rapidly expanding footprint for Artiva, which, in addition to the Merck deal last month, has now raised almost $200 million since its Series A last June.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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