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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Michael Rome (Foresite)

In search of 'house­hold health­care brands of the fu­ture,' Fore­site Cap­i­tal rais­es $969M to sa­ti­ate a tech-heavy ap­petite

Back in April 2018, just before Foresite Capital unveiled its $668 million Fund IV and a strategy to focus on tech-driven life science bets, one of its portfolio companies quietly made an announcement.

Fount Therapeutics, a drug discovery outfit backed by Foresite and Eshelman Ventures, had raised $22 million in Series A cash to hatch several fledgling spinouts. “The first ‘NewCo,’ Kinnate, will be focused on developing precision oncology treatments,” read a press release.

Masayoshi Son, SoftBank CEO (glen photo/Shutterstock)

Japan's Soft­Bank plots bil­lions in biotech in­vest­ments in move that could keep the val­u­a­tion flood ris­ing — re­port

The valuation crazy train in biotech continues to roll into the new year with more than a dozen companies taking a chance on Nasdaq and money flowing in from all sides. Now, a Japanese institutional investor is reportedly weighing an entry into the market in a big way — will it keep the bitcoin-esque flood rising?

Already a part-time investor in biotech, SoftBank could drop billions of dollars into the industry as part of helmsman Masayoshi Son’s plan to spend around $80 billion of the firm’s own assets, according to a report from Bloomberg citing people familiar with the plan.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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