OMGA - Omega Therapeutics Announces Promising Preliminary Clinical Data for OTX-2002 from Ongoing MYCHELANGELO™ I Trial | Benzinga
All 8 patients treated with OTX-2002 in initial two cohorts achieved highly specific on-target genomic engagement, intended epigenetic state change and robust downregulation in expression of c-MYC, a historically ‘undruggable' target
First-known clinical observation of pre-transcriptional gene modulation using a programmable epigenomic mRNA candidate
Clinical proof-of-platform established; potential applicability across a broad range of diseases
Company to host webcast today at 8:00 a.m. ET
CAMBRIDGE, Mass., Sept. 26, 2023 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (NASDAQ:OMGA) ("Omega"), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced encouraging preliminary safety, tolerability, pharmacokinetic and translational data from the initial two dose level cohorts (n=8) from Part 1 of its ongoing Phase 1/2 MYCHELANGELO™ I study evaluating OTX-2002 in patients with hepatocellular carcinoma (HCC) and other solid tumors associated with the c-MYC (MYC) gene. OTX-2002, the Company's lead development candidate, is designed to pre-transcriptionally downregulate MYC, a master oncogene implicated in more than 50% of all cancers and approximately 70% of HCC cases.
"We believe these promising data represent a landmark moment for Omega that supports the potential of our approach and marks a new era of therapeutic development utilizing programmable mRNA candidates for controlled epigenomic modulation," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "For the first time ever in the clinical setting, we have site-specifically targeted and controllably modulated the expression of the MYC oncogene, one of the most promising targets in oncology that has proven difficult to successfully drug by other modalities. We are excited to continue advancing OTX-2002 as we aim to deliver a new class of medicines for patients in need."
"We are thrilled to see that all eight patients evaluated at these initial low doses demonstrated clear evidence of on-target epigenetic changes and correlated rapid, robust and durable decreases in MYC mRNA expression levels," added Thomas McCauley, Ph.D., Chief Scientific Officer of Omega Therapeutics. "These early clinical data are consistent with our preclinical experiments, giving us confidence that our approach has the potential to translate to anti-tumor activity and clinical benefit. Coupled with encouraging safety and predictable pharmacokinetics, we believe that OTX-2002 holds transformative potential for patients living with HCC."
MYCHELANGELO I (NCT05497453) is an ongoing Phase 1/2 open label trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of OTX-2002 as a monotherapy (Part 1) and in combination with standard of care therapies (Part 2) in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene. These preliminary data cover the first two dose cohorts from the monotherapy dose escalation portion of the trial, which is currently being conducted at clinical sites across the United States and Asia. Patients were treated intravenously with either 0.02 mg/kg (n=4) or 0.05 mg/kg (n=4) of OTX-2002 once every two weeks. Changes in MYC DNA methylation and mRNA levels were analyzed through measurements of cell-free DNA and exosomal mRNA, respectively. As of the data cut-off date of September 18, 2023, one HCC patient in the 0.05 mg/kg dose level cohort remained on treatment.
Key Highlights:
Translational:
- Highly specific on-target engagement and intended epigenetic changes at the target genomic loci were observed for all eight patients across both dose levels, as evidenced by a robust increase in cell-free DNA MYC methylation signal following administration ...