Tony de Fougerolles

A step be­hind its ma­jor ex­o­some chal­lenger, Evox scores $95M round for fi­nal leg of race to the clin­ic

Af­ter years of aca­d­e­m­ic and ear­ly-stage biotech de­vel­op­ment, ex­o­somes fi­nal­ly en­tered the clin­ic last year, when Co­di­ak launched their Phase I/II tri­al in ad­vanced sol­id tu­mors. Now Evox, their chief ri­val in the space, is plot­ting their own path to hu­mans, even if they still have a ways to go.

Evox an­nounced Thurs­day a $95 mil­lion Se­ries C to push their first rare-dis­ease pro­grams in­to the clin­ic in 2022. The round, led by Red­mile Group, will al­so al­low the com­pa­ny to ex­pand in­to new dis­eases and modal­i­ties, in­clud­ing de­liv­er­ing gene ther­a­py and gene edit­ing, CEO Tony de Fougerolles said in an in­ter­view.

Ex­o­somes, tiny bub­bles of fat that func­tion as a postal ser­vice be­tween cells in the body, have be­come a grow­ing fo­cus for drug de­vel­op­ment in re­cent years. Based on stud­ies show­ing that these nanopack­ages can car­ry ge­net­ic ma­te­r­i­al and even pro­teins with­in and across tis­sues, com­pa­nies have tried to turn them in­to de­liv­ery ve­hi­cles for a host of tech­nolo­gies, from old-fash­ioned small mol­e­cules to mR­NA and AAV gene ther­a­py.

Evox, which has now raised over $150 mil­lion in five years, and Co­di­ak, which went pub­lic last year for $83 mil­lion, are the two largest play­ers and as their en­gi­neer­ing ef­forts pro­gressed, they’ve at­tract­ed at­ten­tion from Big Phar­ma and big biotech.

Fougerolles, who helped de­vel­op the first ve­hi­cles for RNA ther­a­pies as one of the first em­ploy­ees at both Al­ny­lam and Mod­er­na, said ex­o­somes have been eas­i­er to en­gi­neer than lipid nanopar­ti­cles — aca­d­e­mics and the body have done a lot of the work for them — but the com­pa­ny has still man­aged to im­prove the amount of pay­load they can de­liv­er by sev­er­al or­ders of mag­ni­tude.

“We’ve en­gi­neered every­thing from a small mol­e­cule to an AAV to an mR­NA, siR­NA, etc. So we kind of have this whole tool­box built out,” he said. “Now we’re in the phase of re­al­ly trans­lat­ing them in­to prod­ucts.”

Take­da and Eli Lil­ly have re­ward­ed Evox ac­cord­ing­ly, each dan­gling over $1 bil­lion for col­lab­o­ra­tions on rare dis­ease and cen­tral ner­vous sys­tem dis­or­ders, re­spec­tive­ly. Co­di­ak has its own deals: with Sarep­ta for a re-dos­ing gene ther­a­py and with Jazz Phar­ma­ceu­ti­cals for can­cer.

Fougerolles said both com­pa­nies have tak­en a broad­ly sim­i­lar ap­proach to en­gi­neer ex­o­somes but far dif­fer­ent ap­proach­es to ap­ply­ing the ex­o­somes they en­gi­neer. Co­di­ak chose im­muno-on­col­o­gy, build­ing out at least six dif­fer­ent can­cer pro­grams. Evox looked at that op­tion and re­ject­ed it as too risky.

Too much is un­known about im­munol­o­gy, and sci­en­tists are still strug­gling to de­vel­op an­i­mal mod­els that trans­late well in­to hu­mans, Fougerolles said. So why pair an untest­ed tech­nol­o­gy in ex­o­somes with high-risk ap­pli­ca­tions?

“Some­one like Co­di­ak has the same ex­o­some plat­form risk, but they’ve al­so now put on ad­di­tion­al risk in terms of, is that the right tar­get in terms of im­muno-on­col­o­gy?” he said.

Evox’s first pro­grams, by con­trast, are in rare dis­or­ders where pa­tients are miss­ing an en­zyme in the urea cy­cle. If their ex­o­some can suc­cess­ful­ly de­liv­er an en­zyme to that pa­tient’s cells, it knows the pa­tient will im­prove and quick­ly. And if the pa­tients don’t, Evox will know what was at fault.

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Bob Nelsen (Lyell)

As bear mar­ket con­tin­ues to beat down biotech, ARCH clos­es a $3B ear­ly-stage fund

One of the biggest names in biotech investing has a whole lot of new money to spend.

ARCH Venture Partners closed its 12th venture fund early Wednesday morning, the firm said, bringing in almost $3 billion to invest in early-stage biotechs. The move comes about a year and a half after ARCH announced its previous fund, for almost $2 billion back in January 2021.

In a statement, ARCH managing director and co-founder Bob Nelsen appeared to brush off concerns about the broader market troubles, alluding to the downturn that’s seen several biotechs downsize and the XBI fall back to almost pre-pandemic levels.

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Peter Marks (Jim Lo Scalzo/Pool via AP Images)

FDA's VRB­PAC votes in fa­vor of adapt­ing the Covid-19 vac­cine to the lat­est Omi­cron vari­ant

The FDA’s Vaccine and Related Biological Products Advisory Committee on Tuesday gave the thumbs up — by a vote of 19-2 — that the FDA should require an Omicron-related component in this next season’s booster dose for Covid-19, which both Pfizer/BioNTech and Moderna are hard at work on.

And while neither booster will likely be ready to go with adequate supplies for all American adults by the beginning of the next school year, the situation is still complex and fluid, with CBER Director Peter Marks telling the committee that it’ll take companies at least three months to ready their supplies for this expected next wave.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Sanofi to cut in­sulin prices for unin­sured from $99 to $35, match­ing the in­sulin cap com­ing through Con­gress

As the House-passed bill to cap the monthly price of insulin at $35 nationwide makes its way for a Senate vote soon, Sanofi announced Wednesday morning that beginning next month it will cut the monthly price of its insulins for uninsured Americans to $35, down from $99 previously.

The announcement from Sanofi, which allows the uninsured to buy one or multiple Sanofi insulins (Lantus, Insulin Glargine U-100, Toujeo, Admelog, and Apidra) at $35 for a 30-day supply effective July 1, follows House passage (232-193) of the monthly cap in March, with just 12 Republicans voting in favor of the measure.

Shehnaaz Suliman, ReCode Therapeutics CEO (Photo by Jennifer Leahy)

Pfiz­er, Sanofi-backed LNP out­fit goes back to the well and draws $120M for its trek to the clin­ic

A preclinical biotech touting a five-lipid drug delivery platform is looking to break out of its preclinical mold, and it just secured a sizable raise to do just that.

ReCode Therapeutics reported Wednesday morning that Leaps by Bayer and Matrix Capital Management affiliate AyurMaya co-led a Series B extension round, adding $120 million to the biotech’s previous Series B haul of $80 million. The biotech has been backed by several players in Big Pharma, notably Pfizer and Sanofi from its original Series B close last fall. And in this extension — featuring all new investors, CEO Shehnaaz Suliman tells Endpoints News — Amgen’s VC arm jumped on board.

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Eliot Charles, MiroBio executive chairman

Ox­ford spin­out nabs al­most $100M in new cash to test PD-1 in au­toim­mune dis­eases and hunt for a CEO

After about 15 years in an Oxford lab and three more years in the upstart world following a 2019 spinout, MiroBio is ready to enter UK clinical trials and, en route to the clinic, the biotech has picked up $97 million in Series B funds.

The £80 million financing round kicked off in earnest last September and includes a transatlantic consortium — led by Medicxi — ready to bankroll that first clinical trial, beginning “imminently,” as well as the debut study of a second program thereafter, executive chair Eliot Charles, an SR One venture partner, told Endpoints News.

Hank Safferstein, Generian CEO

Astel­las sub­sidiary to part­ner with Pitts­burgh up­start in search for 'un­drug­gable' pro­teins

As Astellas continues its drive to build out its gene therapy portfolio and capabilities, a subsidiary of the Japanese pharma company has entered into a collaboration with a little-known Pittsburgh biotech.

Astellas-owned Mitobridge and Generian Pharmaceuticals announced on Wednesday that they will work together in a new deal for “undruggable” protein targets. Generian will net an undisclosed upfront payment and could get up to $180 million in milestones, should anything from its platform prove successful, as well as single-digit royalties on global net sales.