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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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