On November 2, 2022 Omega Therapeutics, Inc. ("Omega"), a clinical-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OTX-2002, the company’s first-in-class epigenomic controller engineered to downregulate c-Myc (MYC), for the treatment of hepatocellular carcinoma (HCC) (Press release, Omega Therapeutics, NOV 2, 2022, View Source [SID1234622844]).

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The company recently announced that the first patient had been dosed in its Phase 1/2 MYCHELANGELO I clinical trial investigating 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.

"HCC is a devastating illness that often develops resistance to current standard of care therapeutics. The FDA’s decision to grant orphan drug designation for OTX-2002 underscores the need for novel therapies to address HCC and the potential of epigenomic programming to transform the treatment landscape," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "We look forward to continuing to work with clinical investigators, patients and the FDA as we advance our MYCHELANGELO clinical program and evaluate the potential of OTX-2002 to bring a new treatment option to the liver cancer patient community."

About Orphan Drug Designation
The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC) is a leading cause of cancer deaths worldwide and represents an unmet clinical need with few therapeutic options. Tyrosine kinase inhibitors (TKIs) have been used as a systemic therapy for HCC, but patients frequently develop resistance with oncogenic MYC identified as a correlating prognostic factor. The MYC oncogene is associated with aggressive disease in up to 70% of patients with HCC.

About OTX-2002
OTX-2002 is a first-in-class Omega Epigenomic Controller in development for the treatment of hepatocellular carcinoma (HCC). OTX-2002 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. 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. OTX-2002 is currently being evaluated in the Phase 1/2 MYCHELANGELO I trial in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene; visit clinicaltrials.gov (NCT05497453) for more details.