Omega Therapeutics presents preclinical data on first-in-class epigenomic controller OTX-2002 as a potential treatment for hepatocellular carcinoma at the 2022 AACR Annual Meeting

  • OTX-2002 suppresses c-Myc gene expression, leading to loss of cancer cell viability in vitro and reduces tumor growth in live xenograft models
  • Data supports the potential of the OMEGA Epigenomic Programming™ Platform to design programmable epigenetic mRNA therapies that successfully regulate gene expression
  • Strong Data Supports Investigational New Drug Application Filing in H1 2022 and Positions OTX-2002 Program for Further Development

CAMBRIDGE, Mass., April 8, 2022 /PRNewswire/ — Omega Therapeutics (NASDAQ: OMGA) (Omega), a development-stage biotechnology company pioneering the first systematic approach to using mRNA therapies as a new class of programmable epigenetic drugs by leveraging its OMEGA Epigenomic platform Programming™, will present preclinical data highlighting the potential of its Omega Epigenomic Controller™ master controller, OTX-2002, to regulate overexpression of the c-Myc (MYC) oncogene in models of hepatocellular carcinoma (HCC) in a presentation by poster at the American Association for Cancer Research (AACR ) Annual Meeting 2022, taking place at New Orleans, Louisiana, April 8-13, 2022.

“Despite its pivotal role in a wide range of cancers, MYC has remained unusable to date,” said Thomas McCauley, Ph.D., Chief Scientific Officer of Omega Therapeutics. “However, we believe that targeting the MYC gene pre-transcriptionally into its isolated genomic domain (IGD) and epigenetic tuning it using our epigenomic controller could overcome challenges that have limited previous technologies, including small molecules, antisense oligos and siRNAs.We believe that these data strongly support the ability of OTX-2002 to tune and restore MYC expression to a normal range and demonstrate the broader potential of our epigenomic programming platform. to fight previously incurable diseases and look forward to filing an Investigational New Drug Application in the first half of this year.”

Main conclusions

  • A single dose of OTX-2002 induces long-lasting changes in the epigenetic profile of the MYC gene
  • OTX-2002 reduced MYC mRNA expression and protein levels over approximately 2 weeks in in vitro
  • Downregulation of MYC in several HCC cell lines resulted in significant loss of viability of MYC-dependent cancer cells while sparing normal cells
  • In mouse HCC xenograft models, OTX-2002 significantly reduced tumor growth and was well tolerated

Cumulatively, these data support the filing of an Investigational New Drug Application with the United States Food and Drug Administration for the clinical development of OTX-2002 in the first half of 2022.

The poster can be viewed on Omega’s website at

About OTX-2002

OTX-2002 is a first-in-class Omega Epigenomic Controller™ being developed for the treatment of hepatocellular carcinoma (HCC). OTX-2002 is designed to modulate the levels of c-MYC (MYC) expression using targeted mRNA-expressed proteins to mediate epigenetic regulation while potentially overcoming MYC autoregulation. The MYC oncogene is associated with aggressive disease in approximately 70% of patients with HCC. Omega is currently evaluating OTX-2002 in Enabling Investigational New Drug (IND) studies.

About Omega Therapeutics

Omega Therapeutics, founded by Flagship Pioneering, is a development-stage biotechnology company pioneering the first systematic approach to using mRNA therapies as a new class of programmable epigenetic drugs. The Company’s OMEGA Epigenomic Programming™ platform harnesses the power of epigenetics, the mechanism that controls gene expression and every aspect of an organism’s life, from genesis, growth and differentiation cell to cell death. Using a suite of technologies, coupled with Omega’s systematic, rational and integrative drug design process, the OMEGA deterministic platform enables control of fundamental epigenetic processes to correct the root cause of disease by bringing back aberrant gene expression to normal range without altering native nucleic genes. acid sequences. Omega’s modular and programmable mRNA epigenetic drugs, Omega Epigenomic Controllers™, target specific epigenomic loci in isolated genomic domains, EpiZips™, among thousands of unique, genome-wide mapped and validated DNA sequences, with high specificity to sustainably adjust single sequences or multiple genes to treat and cure disease with Precision Genomic Control™. Omega is currently advancing a wide range of development candidates spanning a range of disease areas, including oncology, regenerative medicine, multigenic diseases including immunology, and certain single gene diseases.

For more information, visit omegatherapeutics.comor follow us on Twitter and LinkedIn

Forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release that do not relate to historical facts should be considered forward-looking statements, including, without limitation, statements regarding our expectations regarding the potential of our product candidates, including our lead OEC candidate OTX-2002; and our plans to present preclinical data on OTX-2002 and file an Investigational New Drug Application for it in the first half of 2022. These statements are not promises or guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, that that: the new technology upon which our product candidates are based makes it difficult to predict the time and cost of preclinical and clinical development and subsequently obtain regulatory approval, if at all; the substantial development and regulatory risks associated with epigenomic controller machines due to the novel and unprecedented nature of this new class of drugs; our limited operating history; the occurrence of material losses and the fact that we expect to continue to incur additional material losses for the foreseeable future; our need for substantial additional funding; our investments in research and development efforts that further improve the OMEGA platform, and their impact on our results; uncertainty regarding preclinical development, particularly for a new class of drugs such as epigenomic controllers; that our product candidates may be associated with serious adverse events, adverse side effects, or have other properties that could halt their regulatory development, prevent their regulatory approval, limit their commercial potential, or result in material adverse consequences; the impact of increased manufacturing demand for mRNA and LNP-based vaccines to treat COVID-19 on our development plans; difficulties in manufacturing the new technology upon which our OEC candidates are based; our ability to adapt to rapid and significant technological changes; our reliance on third parties to manufacture materials; our ability to successfully acquire and establish our own manufacturing facilities and infrastructure; our dependence on a limited number of suppliers for the lipid excipients used in our product candidates; our ability to advance our product candidates into clinical development; and our ability to obtain, maintain, enforce and adequately protect our intellectual property rights. These and other important factors discussed under “Risk Factors” in our Annual Report on Form 10-K for the period ended the 31st of December, 2021, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. These forward-looking statements represent management’s estimates as of the date of this press release. Although we may choose to update these forward-looking statements at some time in the future, we disclaim any obligation to do so, even if subsequent events change our views.

Media contact:
Jason Braco
LifeSci Communications
[email protected]

Investor contacts:
Kevin Murphy/Brendan Burns
Slang Partners
[email protected]

SOURCE Omega Therapeutics

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