Joël de Beer, Anjarium Biosciences CSO

Pfiz­er backs a small Swiss up­start's at­tempt to dis­cov­er a non-vi­ral gene ther­a­py

Joël de Beer made it just a few months in­to his PhD pro­gram at ETH Zurich be­fore he dropped out to join the hunt for the holy grail of gene ther­a­py — a non-vi­ral de­liv­ery mech­a­nism.

Now that he’s raised $61 mil­lion from some big-name back­ers — in­clud­ing Pfiz­er’s ven­ture arm — to aid that quest, the pres­sure is on.

An­jar­i­um Bio­sciences un­cloaked Thurs­day morn­ing af­ter four years in stealth mode, sport­ing a Se­ries A round co-led by Abing­worth and Gimv. The team of five has set up shop in a pic­turesque vil­lage about 40 min­utes out­side of Zurich to solve the cur­rent chal­lenges of AAV us­ing DNA-based gene vec­tors.

“Our am­bi­tion now is to re­al­ly paint the fu­ture of the field our­selves,” de Beer, An­jar­i­um’s founder and CSO, told End­points News. 

One of the ma­jor ad­van­tages of An­jar­i­um’s ap­proach is that DNA-based vec­tors, un­like AAV, don’t trig­ger an im­mune re­sponse. With AAV, some pa­tients have a nat­ur­al im­mu­ni­ty to the vi­ral vec­tors, or, once they’re dosed with it, they mount an im­mune re­sponse, mak­ing it dif­fi­cult to re-dose.

“We don’t have any vi­ral com­po­nents in it, and hence the pre-ex­ist­ing im­mu­ni­ty, or maybe an ac­quired im­mu­ni­ty, is some­thing that we are not ex­pect­ing,” de Beer told End­points News. “Mean­ing that you could start to have in­di­vid­u­al­ized dos­ing, de­pend­ing on, let’s say, (the) age of a pa­tient, (or) their lifestyle.”

The abil­i­ty to re-dose could al­low sci­en­tists to treat some peo­ple with a low­er dose to start, po­ten­tial­ly avoid­ing some of the safe­ty is­sues in­volved with giv­ing high­er dos­es. There’s al­so the po­ten­tial for an in­creased pay­load ca­pac­i­ty and more tar­get­ed de­liv­ery, de Beer ex­plained.

An­jar­i­um’s de­liv­ery plat­form in­cludes what the com­pa­ny calls its Hy­bri­do­some tech, which com­bines a lipid nanopar­ti­cle and an ex­tra­cel­lu­lar vesi­cle.

“Of­ten­times when you’re try­ing to tack­le very com­plex prob­lems, see what Moth­er Na­ture has giv­en you as a so­lu­tion … and try to mim­ic that,” de Beer said. “So that’s where the ex­tra­cel­lu­lar vesi­cle com­po­nent comes in­to play, where we can hope­ful­ly bank on 3 bil­lion years of evo­lu­tion to re­al­ly im­prove that de­liv­ery, which you can then … fur­ther en­gi­neer in view of that syn­thet­ic bi­ol­o­gy rev­o­lu­tion, which is go­ing on right now.”

An­jar­i­um is far from alone in the search for gene ther­a­py 2.0. Just a few months ago, Glax­o­SmithK­line vet Bri­an McVeigh launched Code Bio­ther­a­peu­tics with $10 mil­lion in seed fund­ing to de­vel­op a syn­thet­ic DNA-based vec­tor. Gen­er­a­tion Bio closed a $230 mil­lion IPO last June to fund its pre­clin­i­cal push for a non-vi­ral gene ther­a­py to treat dis­eases of the liv­er and reti­na. And Michael Ehlers is lead­ing a new ef­fort over at Ap­ple Tree Ven­tures.

“The bright­est minds in the world are try­ing to tack­le ex­act­ly that is­sue of not be­ing able to ap­ply such med­i­cine twice, hav­ing to get it right the first time,” de Beer said.

The Se­ries A round, which al­so saw par­tic­i­pa­tion from Omega Funds and Sur­vey­or Cap­i­tal, will be used to ex­pand An­jar­i­um’s team and progress sev­er­al pro­grams to­ward the clin­ic. De Beer de­clined to re­veal the com­pa­ny’s tar­gets just yet, adding that they won’t be in the clin­ic for at least an­oth­er two years.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.