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. 

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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Hal Barron, Endpoints UKBIO20 (Jeff Rumans)

'Al­tos was re­al­ly a once-in-a-life­time op­por­tu­ni­ty': Hal Bar­ron re­flects on his big move

By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.

Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.

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Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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Troy Wilson, Kura CEO

FDA lifts par­tial hold on Ku­ra's Phase Ib AML pro­gram as biotech re­dou­bles mit­i­ga­tion ef­forts

Kura Oncology is clear to resume studies for its early-stage leukemia program after the FDA lifted a clinical hold Thursday afternoon.

Regulators had placed the hold on a Phase Ib study of KO-539, an experimental oral treatment for some genetic subsets of acute myeloid leukemia last November after a patient died while taking the drug. Kura expects to begin enrolling patients again imminently, CEO Troy Wilson told Endpoints News.