On August 4, 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 financial results for the second quarter ended June 30, 2022, and highlighted recent Company progress (Press release, Omega Therapeutics, AUG 4, 2022, View Source [SID1234617576]).
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"This has been an exciting second quarter for Omega, in which we were thrilled to receive FDA clearance of our first IND application for OTX-2002, representing the first ever Omega Epigenomic ControllerTM, a new class of programmable mRNA therapeutics. This is a critical milestone for Omega as we enter our next phase of growth and reflects our pioneering work to realize the potential of epigenomic programming," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "Additionally, we were also pleased to share exciting, new supportive preclinical data, both from our lead program OTX-2002 in hepatocellular carcinoma, as well as from another program in our pipeline focused on non-small cell lung cancer, a potential future indication. We look forward to continuing this momentum as we enter the clinic in the second half of this year and further exploring the broad ranging capabilities of our novel platform in additional therapeutic areas."
Recent Business Highlights
Development Pipeline and Platform
Received FDA Clearance of Investigational New Drug (IND) application for OTX-2002, the First Omega Epigenomic ControllerTM (OEC), for MYC driven Hepatocellular Carcinoma (HCC): OTX-2002 is a novel, engineered, and programmable mRNA therapeutic designed to downregulate c-Myc (MYC) expression pre-transcriptionally through epigenetic modulation while potentially overcoming MYC autoregulation. This represents the first ever epigenomic controller, a new class of programmable mRNA therapeutics, to receive IND clearance.
On Track to Launch a Phase 1/2 Clinical Trial Under the MYCHELANGELOTM Clinical Trial Program in HCC patients in the 2H’22: The study consists of Part 1 (OTX-2002 as monotherapy) and Part 2 (OTX-2002 combined with standards of care in HCC). The Phase 1/2 trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a monotherapy and in combination with standard of care therapies in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene. The study is expected to enroll approximately 190 patients at clinical trial sites in the United States, Asia, and Europe and the Company expects to dose the first patient during the fourth quarter of 2022.
Part 1: This dose escalation portion of the trial will evaluate OTX-2002 monotherapy in patients with relapsed or refractory HCC and other solid tumors. Investigators will utilize a 3+3 design to identify the following primary endpoints of any dose limiting toxicities (DLTs), maximum tolerated dose (MTD), incidence of treatment emergent adverse events (TEAEs), and the recommended dose for expansion (RDE). There will then be a monotherapy expansion for patients with relapsed or refractory HCC who have received at least 1 prior line of systemic anticancer treatment and are without available subsequent standards of care. These patients will receive the RDE of OTX-2002. The primary endpoints of the monotherapy expansion will be overall response rate (ORR) and duration of response (DOR).
Part 2: This portion of the trial will evaluate OTX-2002 in combination with standard of care therapies and will consist of safety run-in and expansion cohorts. During the safety run-in, patients with relapsed or refractory HCC will receive OTX-2002 at the selected dose in combination with one of three current standard of care therapies. These combination partners will be one of two types of tyrosine kinase inhibitors (TKIs) or an immune checkpoint inhibitor (ICI). The primary endpoints of the safety run-in will be DLT, MTD, and incidence of TEAEs. Once the combination therapies have been determined to be tolerable in the safety run-in, patients with relapsed or refractory HCC will be enrolled into the expansion cohorts for each of the combination therapies, with the primary endpoints of ORR and DOR.
New OTX-2002 Preclinical Data Presented at Three Major Medical Meetings Show Robust In Vivo Efficacy and In Vitro Loss of Cancer Cell Viability:
Non-Human Primate (NHP) Data for OTX-2002 in HCC Presented at ESMO (Free ESMO Whitepaper)-GI 2022: Results showed that treatment with OTX-2002 resulted in robust in vivo efficacy in xenograft tumor models and successfully achieved pre-transcriptional downregulation of hepatocyte MYC gene expression in NHPs. These data support the clinical potential of OTX-2002 in combination with existing standard of care therapies for HCC, including ICIs. The ESMO (Free ESMO Whitepaper)-GI poster presentation is available here.
In Vivo Data for OTX-2002 in HCC Presented at AACR (Free AACR Whitepaper) 2022: Results demonstrated that OTX-2002 suppresses MYC gene expression resulting in a loss of cancer cell viability in vitro and a reduction in tumor growth in in vivo xenograft models. These data support the potential of Omega’s platform to engineer programmable mRNA therapeutics that successfully regulate gene expression. The AACR (Free AACR Whitepaper) poster presentation is available here.
In Vivo OEC Data in Models of Non-Small Cell Lung Cancer (NSCLC) Presented at ASGCT (Free ASGCT Whitepaper) 2022: Results demonstrated that regulation of MYC gene expression via epigenomic programming resulted in decreased viability of cancer cells in vitro and reduced tumor burden in in vivo xenograft models. These data support the potential of OECs to precisely and durably regulate gene expression and their potential as a future treatment for NSCLC. The ASGCT (Free ASGCT Whitepaper) poster presentation is available here.
Additional OEC Development: Beyond HCC and NSCLC, the Company is advancing multiple programs through preclinical studies spanning oncology, multigenic diseases including immunology, regenerative medicine, and select monogenic diseases.
OMEGA Epigenomic ProgrammingTM Platform: Omega is creating a new generation of programmable mRNA medicines that are designed to control the fundamental epigenetic processes to correct the root cause of disease by restoring aberrant gene expression to normal levels without altering native nucleic acid sequences. Omega has developed a highly rational and deterministic approach to drug design that enables the Company to rapidly develop and optimize novel OECs with high target specificity to durably tune the expression of single or multiple genes. Omega is advancing multiple preclinical development programs spanning oncology, multigenic diseases including immunology, regenerative medicine, and select monogenic diseases.
Corporate
Joshua Reed Appointed Chief Financial Officer. Mr. Reed brings over 25 years of successful and diverse corporate and financial operations experience including capital raising, business development, and investor relations.
Second Quarter 2022 Financial Results
As of June 30, 2022, the Company had cash, cash equivalents and marketable securities totaling $173.7 million.
Research and development (R&D) expenses for the second quarter of 2022 were $19.4 million, compared to $11.2 million for the second quarter of 2021. The $8.2 million increase in R&D expense was primarily driven by an increase in personnel-related expenses, external manufacturing costs, and study costs in support of the advancement of our programs
General and administrative (G&A) expenses for the second quarter of 2022 were $6.2 million, compared to $3.6 million for the second quarter of 2021. The $2.6 million increase in G&A expense was primarily driven by an increase in personnel-related expenses to support business growth.
Net loss for the second quarter of 2022 was $25.9 million, compared to $15.4 million for the second quarter of 2021, driven predominantly by increased R&D and G&A expenses to support the Company’s growth and operations as a public company.