Thomas McCauley, PhD, on Proof-of Concept With In Vivo Epigenomic Editing


The chief scientific officer at Omega Therapeutics discussed how the MYCHELANGEO-1 trial may validate the company’s OEC platform.

“There's a tremendous amount of excitement, both for this particular product and for HCC. But the other key piece for us too, is that, as a platform company, all of the data we’ll generate translationally from this in terms of target engagement and pharmacodynamic activity, in our view, is a real read through for the entire platform. It would be the first in vivo human epigenetic gene modulation in this way, and so, if we succeed, thisreads through really to the entire platform, so it's even more momentous for us in that in that respect.”

Omega Therapeutics recently announced promising data from the phase 1/2 MYCHELANGELO-1 trial (NCT05497453) of its MYC-targeted Omega Epigenomic Controller (OEC) therapy OTX-2002. The preliminary data demonstrated specific, on-target genomic engagement and proof-of-concept in changing the epigenetic state and downregulating expression of c-MYC in patients with hepatocellular carcinoma (HCC) and other solid tumors associated with the c-MYC (MYC) gene.

The data were from 8 patients, 4 each receiving 0.02 mg/kg or 0.05 mg/kg of OTX-2002 monotherapy intravenously every 2 weeks in 2 cohorts. The therapy was well tolerated with no dose-limiting toxicities (DLTs) and the maximum tolerated dose was not reached. Most adverse events (AEs) were grade 1 or 2 (87%) and the most common treatment-related AEs were infusion-related reactions (26%). The second part of the study will assess OTX-2002 in combination therapy.

CGTLive spoke with Thomas McCauley, PhD, chief scientific officer, Omega Therapeutics, to learn more about the company’s OEC technology and how the MYCHELANGELO-1 trial is generating the first-in-human proof-of-concept data for the platform in general. He shared his belief in the potential of in vivo epigenomic therapies and how this technology sets the company apart.

McCauley T. MYCHELANGELO™ I preliminary data overview. Presentation. September 26, 2023.
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