An Ox­ford spin­out rais­es $44M as it looks to res­ur­rect an old Roche an­ti­body in pur­suit of a rare dis­ease

Three months ago, a small and lit­tle-known Ox­ford Uni­ver­si­ty spin­out called Celleron an­nounced they would try to res­ur­rect an an­ti­body Roche re­tired back in 2018. Now that spin­out is spin­ning out its own new com­pa­ny and rais­ing cash from a high-pro­file in­vestor to push that an­ti­body through the clin­ic.

Francesco De Ru­ber­tis

Backed with €37 mil­lion in Se­ries A cash, Syn­Ox Ther­a­peu­tics will look to de­vel­op the an­ti­body, known as emac­tuzum­ab, for tenosyn­ovial gi­ant cell tu­mours (TGCT), a rare dis­ease where se­ries of be­nign tu­mors be­gin to grow around the joints and ten­dons. Health­Cap and Medicxi led the round and were joined by For­bion and Gimv.

Medicxi part­ner Francesco De Ru­ber­tis tout­ed the drug as an all-but-proven as­set for this dis­ease.

“We are ex­cit­ed to sup­port Syn­Ox in its con­tin­ued de­vel­op­ment of emac­tuzum­ab, an agent which has demon­strat­ed pro­found ef­fi­ca­cy in­over 60 TGCT pa­tients treat­ed to date,” he said.

John Reed Sanofi

A fa­vorite of John Reed’s back be­fore he left for Sanofi, emac­tuzum­ab was one of sev­er­al an­ti­bod­ies in de­vel­op­ment across phar­ma meant to block CSF1R. The re­cep­tor is found on in­nate im­mune cells called macrophages that swarm around tu­mors and, counter-in­tu­itive­ly, can help the tu­mor evade the rest of the im­mune sys­tem and grow. They test­ed it in com­bi­na­tion with their PD-1 in­hibitor Tecen­triq on the the­o­ry that it could help T cells en­ter the tu­mor’s mi­cro-en­vi­ron­ment and en­hance the check­point block­er’s ef­fi­ca­cy.

Al­though the drug showed re­sults as a monother­a­py, com­bin­ing it with Tecen­triq didn’t ap­pear to of­fer any ben­e­fit over Tecen­triq alone and Roche shelved it as part of a sweep out of the pipeline.

The drug, though, al­ways had ap­plic­a­bil­i­ty in at least one rare dis­ease out­side can­cer: TGCT. The dis­ease oc­curs when cells over­pro­duce CSF1R, mak­ing it a clear tar­get when com­pa­nies be­gan de­vel­op­ing mol­e­cules that in­hib­it the re­cep­tor. Al­though Roche chose not to pur­sue late-stage tri­als, they pub­lished a study in The Lancet that showed 24 out of 28 TGCT pa­tients re­spond­ed, with two pa­tients hav­ing a com­plete re­sponse.

Nick La Thangue

Syn­Ox said they could pur­sue oth­er in­di­ca­tions, but their main job will be to bring it through late-stage test­ing, try­ing to find the path of least re­sis­tance through the FDA.

“Syn­Ox will be fo­cused on gen­er­at­ing all nec­es­sary da­ta to reg­is­ter this new ther­a­py in a time- and cost-ef­fi­cient way,” Nick La Thangue, a pro­fes­sor of can­cer bi­ol­o­gy and CEO of the new com­pa­ny, said in a state­ment.

Robert Clarke (Kinaset)

Ki­naset launch­es with $40M and a JAK in­hibitor from Vec­tura's old pipeline

Kinaset Therapeutics is joining the search for a better severe asthma treatment, picking up where Vectura left off when it decided to clear house last year.

UK-based Vectura — which took a big hit when its most advanced candidate flopped in a Phase III asthma trial back in 2018 — recently shifted to a CDMO model, offloading all of its R&D programs. Robert Clarke, who’s worked on inhalable therapeutics for 21-plus years, had close contacts at the company and took a look at what they were offering. After doing some research, he was attracted by VR475, a pan-JAK inhibitor.

Scoop: Google’s GV spear­heads the Spot­light syn­di­cate — back­ing an up­start biotech aimed at ‘de­moc­ra­tiz­ing’ gene edit­ing

CRISPR had no sooner started to shake the very foundations of drug development before its limitations began to loom large. Gene editing could change the world — if only you could get around the hurdles that threatened to trip up every program.

So it’s only natural to see CRISPR 2.0 taking shape before the pioneers can get the lead therapies through development. And who better than Google’s GV venture arm to take the lead spot in a small syndicate backing some scientists with their own unique twist on a solution?

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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FDA gives Rhythm the green light for set­melan­otide, a drug aimed at re­duc­ing obe­si­ty in cer­tain ge­net­ic dis­or­ders

A little over a year after completing successful pivotal trials, Rhythm Pharmaceuticals $RYTM has its first drug approval on its hands.

The Boston-based biotech announced Friday that the FDA gave setmelanotide the thumbs-up for three rare genetic disorders that result in obesity in patients six and older. It’s the agency’s first such approval, Rhythm said, with the indicated deficiencies being the POMC, PCSK1 and LEPR genes. Rhythm will market the drug as Imcivree, and plans to have it on the shelves in the first quarter of 2021.

Pascal Soriot (Getty)

As­traZeneca CEO So­ri­ot plans new study to test that con­tro­ver­sial 90% ef­fi­ca­cy fig­ure, wait­ing for US da­ta be­fore go­ing to FDA

Pascal Soriot spent the long Thanksgiving weekend digging AstraZeneca out of a hole, promising to put an end to the questions around its interim Phase III vaccine data by conducting a new study while going to regulators with a large part of what it already has.

AstraZeneca and its partners at Oxford had initially touted high-level results from two studies conducted in the UK and Brazil as positive. But the enthusiasm was soon shadowed by confusion as observers probed into how the highest, 90% efficacy was seen in a dosing regimen given to a small group of volunteers due to an error. Among a larger cohort given the intended shots, the vaccine was only 62% effective, a rate that would’ve been respectable had Pfizer/BioNTech and Moderna not posted efficacy rates of 94%, 95% for their mRNA candidates. And many weren’t sure what to make of the average 70% number that AstraZeneca ran in headlines.

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Stephané Bancel (Endpoints at JPM20)

Mod­er­na cal­i­brates fi­nal Covid-19 vac­cine ef­fi­ca­cy at 94.1% — and to­day it's gun­ning for the EUA

Nearly a year ago, as the coronavirus emerged in China, the NIH and four major companies bet on an unproven genetic technology as the best tool for developing a vaccine to stem the outbreak. Today, a second such vaccine is heading to the FDA.

Moderna said Monday that they will request an emergency use authorization from the FDA after a final analysis showed their mRNA vaccine was 94.1% effective at preventing symptomatic Covid-19. The data confirm the results from an interim analysis and matches efficacy Pfizer and BioNTech showed in a Phase III study, setting the biotech up to potentially nab one of the first two Covid-19 vaccine OKs.

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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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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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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.