On October 12, 2022 Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a clinical-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programming platform, reported that it has selected OTX-2101 as the next Omega Epigenomic Controller (OEC) development candidate to advance into Investigational New Drug (IND)-enabling studies for the treatment of non-small cell lung cancer (NSCLC) (Press release, Omega Therapeutics, OCT 12, 2022, View Source [SID1234621977]).

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Omega scientists rationally engineered OTX-2101 to control the expression of the c-Myc (MYC) oncogene, a historically undruggable target in NSCLC. MYC is a master transcription factor that regulates cell proliferation, differentiation and apoptosis and plays a significant role in more than 50% of all human cancers. Genetic analysis has revealed that MYC overexpression is present in approximately 60% of NSCLC1. Preclinical data presented at the 2022 American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting showed that OTX-2101 potently down-regulates MYC in multiple NSCLC cell lines. OTX-2101 effectively reduced tumor growth in vivo and was well tolerated in murine xenograft models, further supporting its clinical potential. IND-enabling activities for OTX-2101 are underway.

"NSCLC accounts for nearly 25% of cancer deaths worldwide and despite its high prevalence, treatment options are limited. Our approach to epigenomic programming has the potential to address NSCLC by targeting a key oncogene implicated in a broad segment of the patient population. In preclinical studies, OTX-2101 has demonstrated clear anticancer activity supporting its clinical potential and our overall approach to targeting MYC," said Thomas McCauley, Ph.D., Chief Scientific Officer of Omega Therapeutics. "We look forward to advancing OTX-2101 through IND-enabling studies and to continuing to leverage the power of our platform to realize the promise of epigenetics to treat disease."

The OTX-2101 clinical development program will utilize a lung tissue-targeting lipid nanoparticle (LNP) technology exclusively licensed from Nitto Denko Corporation ("Nitto"). This represents the first option exercised by the Company as part of an existing arrangement that provides Omega the option to exclusively license Nitto’s LNP technology for therapeutic development across multiple targets and tissue types.

"This milestone for OTX-2101, which comes on the heels of the MYCHELANGELO I trial initiation for OTX-2002 for the treatment of hepatocellular carcinoma, highlights the power of our OMEGA Epigenomic Programming platform to engineer candidate mRNA therapeutics customized to the biology of the disease," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "Our data-driven platform enables us to rapidly design novel medicines addressing the root cause of disease and tailor our delivery strategy to target the relevant cells and tissues. By leveraging both our internal development efforts and strategic external partnerships, we are able to accelerate clinical development of our OECs, with the goal of bringing innovative new therapies to patients sooner. We are excited to continue to deliver strong execution of our strategy, meet committed milestones, and establish a deep pipeline of promising candidates to treat a broad range of indications."

About OTX-2101
OTX-2101 is a first-in-class Omega Epigenomic Controller in development for the treatment of non-small cell lung cancer (NSCLC). OTX-2101 is an mRNA therapeutic delivered via lipid nanoparticles (LNPs) and is designed to downregulate MYC expression pre-transcriptionally through epigenetic modulation while potentially overcoming MYC autoregulation. Genetic analysis conducted by others has revealed that MYC overexpression is present in approximately 60% of NSCLC1. Omega is currently evaluating OTX-2101 in Investigational New Drug (IND)-enabling studies.