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.

David Meek (Photographer: Marlene Awaad/Bloomberg via Getty Images)

Scoop: Fer­ring, Black­stone’s $570M gene ther­a­py ven­ture has im­plod­ed af­ter CMC is­sues stymied a once high-pro­file quest

Back in late 2019, FerGene looked golden. It had a gene therapy for bladder cancer — spun out of Ferring — at the FDA, with a priority review and breakthrough status indicating that great things awaited on the near horizon.

Ferring backed up the venture with $170 million, with Nick Galakatos’ Blackstone Life Sciences chipping in another $400 million with great fanfare to get the company running. David Meek jumped from the CEO job at Ipsen to run it all, and a little over a year ago much of the executive team was assembled to bring it all home.

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The End­points 20 un­der 40, biotech's new nor­mal, Stéphane Bancel's one re­gret, 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.

What a week! We were proud to present a slate of special reports that showcase the breadth and depth Endpoints is striving to deliver, and it’s truly a team effort. None of this can happen without our readers — so thank you for your support.

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Eli Lil­ly blazes new trail for RET in­hibitor, gin­ning up a re­sponse in 9 sol­id tu­mor types in ear­ly da­ta

Eli Lilly was first to market with its RET fusion-inhibitor Retevmo in July, staking its claim in the rare tumor indication. But Roche wasn’t far on its heels with an approval of its own for RET inhibitor Gavreto, and now Lilly is looking to get the leg up on its closest competitor.

Lilly’s Retevmo posted a 47% objective response rate with responses in nine unique RET fusion-positive tumor types, according to early data from the Phase I/II LIBRETTO-001 unveiled Sunday at the virtual AACR meeting.

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Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

The CHIC in­ter­view: Stéphane Ban­cel on Mod­er­na's overnight trans­for­ma­tion, the new time­line on vac­cine de­vel­op­ment and his one re­gret in 2020

The China Healthcare Investment Conference — better known as CHIC — took place last week in Shanghai at the Ritz-Carlton Pudong. Endpoints partnered with CHIC this year in an effort to highlight the global nature of drug development, and John Carroll conducted two virtual “fireside chats” that was broadcast to attendees. We’ll be sharing both with the Endpoints Premium audience, and first up is a one-on-one interview with Moderna’s Stéphane Bancel.

John Carroll has been covering Moderna and Bancel since the very beginning. He started their conversation by noting it was the first time he’s taken a drug from a company he’s covered from its inception. They go on to discuss how Moderna is being transformed by cash-flow earlier than expected, Bancel’s one big regret in 2020, and much more.

Their conversation is below, and we hope you enjoy.

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iTeos busts out ear­ly hu­man da­ta for an­ti-TIG­IT an­ti­body. Is it enough to match up with Roche, Mer­ck?

The TIGIT protein has turned into an arms race for Big Pharma, with Merck and Roche looking to add their own candidates to checkpoint inhibitor combos. A smaller player in that race, iTeos Therapeutics, is one step behind those big names and is rolling out early data that looks pretty par for the course.

iTeos’ anti-TIGIT immune receptor antibody EOS-448 posted one confirmed partial response and churned up no surprising safety signals in the dose-escalation, monotherapy portion of a Phase I/IIa study in patients with advanced solid tumors, according to data presented Saturday at the virtual AACR annual meeting.

As­traZeneca un­veils next-gen PARP in­hibitor, look­ing to fol­low up on suc­cess of first-gen suc­cess sto­ry Lyn­parza

AstraZeneca’s PARP inhibitor Lynparza has raced out to blockbuster sales on the premise of killing tumor cells by inhibiting their ability to adequately heal. But Lynparza isn’t a perfect molecule, and the British drugmaker is now rolling out a potential successor drug it hopes can achieve similar efficacy without the notorious side effects.

AstraZeneca rolled out early data on its PARP1-selective, next-gen inhibitor AZD5305 at the virtual AACR meeting Saturday, hoping to highlight the drug’s chances at following up — and improving — the science behind blockbuster drug Lynparza.

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The End­points 100 sur­vey: CEOs changed the way biotechs op­er­ate dur­ing the pan­dem­ic. Things will nev­er be the same again

With the US at the front of the line for new Covid-19 vaccines, the biotech industry is looking at the near-term end of the pandemic — or at least a more manageable easing of the rules established during the lockdown. But only a relative handful of CEOs expect things to return to the way they were in a pre-pandemic era. Most are planning to go forward with a whole new set of guidelines for staffers — and flexibility lies at the heart of what’s ahead as people are cleared to go back to the office.

We surveyed our group of Endpoints 100 executives, mostly CEOs, to take the industry’s pulse. This was the latest in close to 5 years of surveys. And several common themes were clear.

First, after a rip-roaring spin on Wall Street as a wave of new IPOs delivered fresh billions to drug hunters, there’s a  record level of excitement about asset valuations and access to capital. The vast majority are expanding this year with new hires. And a remarkable 88% of the 82 executives who took the survey say they won’t be returning to the business-as-usual style they were accustomed to in a pre-pandemic world.

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EMA finds link be­tween ‘very rare’ but some­times fa­tal blood clots and As­traZeneca vac­cine

The EMA said Wednesday that very rare but sometimes fatal blood clots should be listed as a side effect for AstraZeneca’s Covid-19 vaccine.

The EMA’s safety committee said the change to the vaccine label is part of an in-depth review of 62 cases of cerebral venous sinus thrombosis (CVST) and 24 cases of splanchnic vein thrombosis reported in the EU drug safety database as of March 22. Eighteen of the cases were fatal.

Anthony Sun, Zentalis CEO (Zentalis)

Zen­tal­is keeps the heat on As­traZeneca with ear­ly WEE1 da­ta show­ing a group of 'ex­cep­tion­al' re­spon­ders

Long a target for drugmakers, the DNA damage repair pathway has picked up some new steam in recent years with Big Pharma placing its bets. One of the leading candidates there is a WEE1 enzyme inhibitor from AstraZeneca, and now a small biotech player is looking to play catch up.

Zentalis’ WEE1 inhibitor, ZN-c3, posted partial responses across a slate of tumor types with a tolerable safety profile as monotherapy for solid tumor patients who are either treatment-resistant or have no established standard of care, according to interim Phase I data presented as a late breaker Saturday at the virtual AACR annual meeting.