Noubar Afeyan, Flagship CEO (Victor Boyko/Getty Images for Aurora Humanitarian Initiative)

Flag­ship start­up Omega takes lead 'epige­nom­ic con­troller' can­di­date clos­er to the clin­ic with new $126M round

In its quest to drug the un­drug­gable, Flag­ship Pi­o­neer­ing start­up Omega Ther­a­peu­tics is chas­ing a moon­shot with “epige­nom­ic con­trollers” to get all of the ben­e­fits of gene edit­ing with­out the ac­tu­al edit­ing. That’s a tall task to craft a new class of ther­a­peu­tics, but in­vestors are cer­tain­ly show­ing in­ter­est.

Omega plans to ad­vance its lead can­di­date for the c-myc onco­gene in­to hu­man test­ing and es­tab­lish a man­u­fac­tur­ing foot­print with pro­ceeds from a $126 mil­lion Se­ries C the biotech closed Tues­day.

Omega, which launched in 2019, is us­ing its drug dis­cov­ery plat­form to tar­get what it calls in­su­lat­ed ge­nom­ic do­mains (IGDs), paired DNA sites with a pro­tein binder that up- or down­reg­u­late gene ex­pres­sion in lo­cal­ized “zip codes,” Omega said. By tar­get­ing epige­nomics, Omega be­lieves it can mod­u­late gene ex­pres­sion in a hy­per­tar­get­ed way with­out hav­ing to add or delete nu­cleotides in pa­tients’ ge­net­ic code.

It’s an am­bi­tious plan but one that keeps earn­ing in­vestors’ in­ter­est for its promise at a nov­el class of ther­a­peu­tics as well as an­oth­er shot on goal at “un­drug­gable” tar­gets. So far, Omega has raked in $210 mil­lion in fundrais­ing with Flag­ship lead­ing the way on the newest round. It’s joined by In­vus, Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, and funds and ac­counts man­aged by Black­Rock, Cowen, Point72, Lo­gos Cap­i­tal, Mi­rae As­set Cap­i­tal and oth­ers.

Omega’s im­me­di­ate next step is tak­ing lead can­di­date OTX-2002, what it calls an “Omega con­troller,” in­to a Phase I proof-of-con­cept study against c-myc, a “mas­ter” onco­gene that crops up in a high per­cent­age of tu­mor types. The drug is in IND-en­abling stud­ies, and Omega isn’t yet ready to say when that fil­ing will come, Flag­ship CEO and Omega co-founder Noubar Afeyan told End­points News. One of the oth­er aims of the round is to ad­vance and even­tu­al­ly un­veil even more can­di­dates for tri­al, and Afeyan said OTX-2002 would like­ly be filed when at least one oth­er can­di­date is al­so on the road to the clin­ic.

Mean­while, Omega is plan­ning to use some of the round’s pro­ceeds to es­tab­lish a man­u­fac­tur­ing foot­print that Afeyan called “quite mod­est” in scope de­spite the po­ten­tial for mul­ti­ple can­di­dates in the com­ing year. Un­like gene ther­a­py or gene edit­ing which re­quire the cul­ti­va­tion of live virus­es or com­plex bi­o­log­ics, Omega’s con­trollers uti­lize mR­NA and lipid nanopar­ti­cle tech­nol­o­gy that Flag­ship com­pa­nies — in­clud­ing Mod­er­na — have pi­o­neered else­where, Afeyan said. That depth of ex­pe­ri­ence and the “pro­gram­ma­ble” na­ture of Omega’s drugs means the biotech can plug and play with its man­u­fac­tur­ing ap­proach and scale to com­mer­cial de­mand with rel­a­tive ease.

“Giv­en the po­ten­cy of that ap­proach, the ac­tu­al vol­umes you need for dis­ease like can­cer or liv­er dis­eases is rel­a­tive­ly mod­est,” Afeyan said. “So what the com­pa­ny needs in the next two to three years is quite mod­est, but it’s re­al­ly im­por­tant that we own and con­trol it be­cause in these kinds of com­pa­nies, a big part of the suc­cess de­pends on the pre­dictabil­i­ty of sup­ply of clin­i­cal ma­te­ri­als. That’s a big part of our val­ue propo­si­tion.”

Ed­i­tor’s Note: This sto­ry was up­dat­ed to cor­rect an er­ror. Omega closed a $126 mil­lion Se­ries C. 

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Steffen Schuster, ITM CEO

Ra­dio­phar­ma re­mains hot as Ger­many's ITM rais­es $109M to ad­vance neu­roen­docrine can­cer pro­gram

The world of radiopharmaceuticals has been heating up over the last few years, and Thursday saw another company focused on the field pull in a new nine-figure raise.

Germany’s ITM, or Isotopen Technologien München, scored a $109 million round of loan financing to push forward its precision oncology pipeline and fund late-stage development for its lead program. As part of the agreement, the loan will convert to shares in the event of future financial or corporate transactions, ITM said.