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. 

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; 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.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.

Skin tu­mors in mice force Pro­tag­o­nist to halt lead pro­gram, crush­ing stock

Protagonist Therapeutics just can’t catch a break.

Six months after the Newark, CA-based biotech unveiled grand plans to launch its lead candidate for blood disorders into a Phase III trial, the FDA has slapped the program with a clinical hold. The halt — which applies to all trials involving the candidate, rusfertide — comes after skin tumors were discovered in mice treated with the drug, according to Protagonist.

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Chi­nese biotech Ever­est signs $550M+ li­cens­ing deal for BTK in­hibitors on heels of Covid-19 pact

Everest Medicines is on a roll with two licensing deals in one week.

The Shanghai-based biotech has paid Sinovent and SinoMab $12 million upfront for the rights to a BTK inhibitor for renal diseases, the company announced Thursday. The deal comes just days after Everest came away with rights to a Covid-19 vaccine in China, Taiwan, Singapore, Thailand and Indonesia.

Everest will pay Sinovent and SinoMab up to $549 million in milestone payments and royalties. The agreement includes tech transfer of Sinovent and SinoMab’s manufacturing process for the candidate, named XNW1011.