Omega Therapeutics, Inc. 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.

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. Based on these encouraging data, OTX-2002 continues to advance in monotherapy dose escalation.

Following the identification of a recommended dose, the Company expects to initiate expansion cohorts in monotherapy and in combination with standard of care therapies.