On November 9, 2023 Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, reported financial results for the third quarter ended September 30, 2023, and highlighted recent Company progress (Press release, Omega Therapeutics, NOV 9, 2023, View Source [SID1234637406]).

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"The promising initial data from our ongoing MYCHELANGELO I trial establish clinical proof for our pioneering OMEGA platform and support the potential of epigenomic controllers, our programmable mRNA candidates, as a new class of therapeutics," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "We are thrilled to have clearly demonstrated the ability to site-specifically target and controllably modulate the expression of MYC in all eight patients evaluated in the initial two dose cohorts of the trial. MYC, considered a "holy grail" oncogene, is just the first of many broadly-implicated targets in oncology where an epigenomic controller may provide therapeutic value. Moreover, these data highlight the promise of precision epigenomic control to address a broad range of diseases."

Mr. Karande added, "In parallel, we have made tremendous progress advancing our pipeline, with recent and upcoming presentations of new preclinical data demonstrating the breadth and versatility of our platform capabilities at scientific and medical meetings this fall. We look forward to building on this growing body of data and delivering a new class of medicines to patients."

Recent Highlights and Key Anticipated Milestones

Development Pipeline and Platform

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Reported promising preliminary safety, tolerability, pharmacokinetic and translational data from the ongoing MYCHELANGELO I clinical trial evaluating OTX-2002 (data cut-off date of September 18, 2023):
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Data from the initial two dose level cohorts (n=8) from the monotherapy dose escalation portion of the Phase 1/2 study evaluating OTX-2002 in patients with hepatocellular carcinoma (HCC) and other solid tumors associated with the c-MYC (MYC) gene showed that all eight patients treated with OTX-2002 achieved

highly specific on-target genomic engagement, intended epigenetic state change and rapid, robust and durable downregulation of MYC expression.
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OTX-2002 was generally well tolerated at both dose levels, with no dose-limiting toxicities. Consistent dose-dependent pharmacokinetics were observed, and no drug accumulation was observed following repeat doses.
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These data represent the first-known clinical observation of pre-transcriptional gene modulation using a programmable epigenomic mRNA candidate and establish clinical proof-of-platform.
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Based on these encouraging data, OTX-2002 continues to advance in monotherapy dose escalation and the Company is actively evaluating patients with HCC in Cohort 3. Omega expects to report updated clinical data from monotherapy dose escalation in the first half of 2024.
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The Company is planning to initiate expansion cohorts in monotherapy and in combination settings with standard of care agents mid-2024.
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Presented new preclinical data supporting the potential of OTX-2101 for the treatment of non-small cell lung cancer (NSCLC) at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper):
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The combination of a MYC-targeting epigenomic controller with immune checkpoint or EGFR inhibitors significantly enhanced anti-tumor activity in multiple preclinical models of NSCLC.
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Omega continues to advance OTX-2101 in Investigational New Drug (IND)-enabling studies.
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Presented preclinical proof-of-concept data for CXCL 1-8 program at the International mRNA Health Conference:
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New preclinical data demonstrate the ability of the Company’s CXCL 1-8-targeting epigenomic controller to multiplex and specifically modulate gene expression of the multigenic locus in multiple models of inflammatory disease.
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Omega has completed lead optimization activities for this program, including demonstration of in vivo proof-of-concept efficacy. The CXCL 1-8-targeting epigenomic controller has potential in multiple inflammatory indications including neutrophilic asthma, acute respiratory distress syndrome (including COVID-19-related), dermatological and rheumatological indications, and oncology, representing a potential franchise opportunity.

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Announced upcoming presentation of preclinical proof-of-concept data on liver inflammation and fibrosis epigenomic controller programs at the American Association for the Study of Liver Diseases (AASLD) annual meeting: Omega will present new preclinical data from its hepatology programs in two posters at AASLD’s "The Liver Meeting" annual meeting, being held November 10-14, 2023, in Boston, Massachusetts.
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One poster will highlight a CXCL 9-11-targeting epigenomic controller candidate for the treatment autoimmune hepatitis (AIH).
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A second poster will highlight new in vitro and in vivo data demonstrating the ability of an investigational epigenomic controller to durably upregulate HNF4α expression to drive liver regeneration in preclinical models of fibrosis.

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Continued to advance and expand OMEGA platform capabilities:
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The Company continues to advance multiple epigenomic controller programs through discovery and lead optimization, and to further progress and

characterize its internal formulation and lipid nanoparticle (LNP) delivery technologies for the delivery of programmable mRNA therapeutics to the lung and other tissues.
Corporate

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Further strengthened Board of Directors: In August, Omega announced the appointment of Chris Schade as Chairman of the Board. In addition, Michelle C. Werner, CEO of Alltrna and CEO-Partner at Flagship Pioneering, joined the Board, bringing with her over 20 years of biopharma experience spanning commercial and R&D responsibilities.
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Named to BioSpace’s Best Places to Work 2024 report in small employer category: The annual report lists 60 U.S. operating employers that are recognized as the most sought-after in the industry by the life sciences community. Recognition by BioSpace is determined by input from company employees as well as voting from more than 3,000 life sciences professionals.

Third Quarter 2023 Financial Results

As of September 30, 2023, the Company had cash, cash equivalents and marketable securities totaling $89.3 million.

Research and development (R&D) expenses for the third quarter of 2023 were $16.5 million, compared to $20.7 million for the third quarter of 2022. The $4.2 million decrease in R&D expenses was primarily driven by decreases in external manufacturing costs and study costs, partially offset by an increase in facilities expense and, to a lesser extent, increases in personnel-related expenses and clinical development costs.

General and administrative (G&A) expenses for the third quarter of 2023 were $7.9 million, compared to $5.2 million for the third quarter of 2022. The $2.7 million increase in G&A expenses was primarily driven by an increase in facilities expense and, to a lesser extent, increases in professional and consulting fees, partially offset by a decrease in personnel-related expenses.

Net loss for the third quarter of 2023 was $22.2 million, compared to $25.8 million for the third quarter of 2022, driven predominantly by a decrease in R&D expenses.