RXBio, a microRNA (miRNA) based drug development company, is ramping up its global clinical trials after confirming the efficacy of its drug candidate for metabolic dysfunction-associated steatohepatitis (MASH) in human organoid experiments.
On Tuesday, RXBio announced promising results from their collaboration with cellArtgen, a specialized organoid testing firm. Their candidate, miRNA-10a, showed significant therapeutic effects in key indicators including reduced fat accumulation, inhibited fibrosis, and decreased inflammation when tested on liver organoids derived from human-induced pluripotent stem cells (iPSCs).
The study directly compared RXBio’s candidate against Resmetirom (brand name Rezidipra), currently the only U.S. Food and Drug Administration (FDA) approved treatment for MASH, which served as the control group.
Analysis of triglyceride (TG) levels, a key marker of fat accumulation, revealed that the miRNA-10a treatment group achieved a 50.4% reduction in TG concentration compared to the control. This outperformed the Resmetirom group, which showed a 17.02% reduction.
Further fluorescent imaging using confocal microscopy confirmed that organoids treated with miRNA-10a exhibited both smaller and fewer fat cells.
MASH, a condition where fatty liver progresses to inflammation and fibrosis, can potentially lead to cirrhosis and liver cancer. This makes it a hotbed for new drug development.
The study also demonstrated improvements in liver fibrosis and inflammation. Pro-collagen I alpha 1, a crucial fibrosis protein, decreased by 53% in the miRNA-10a group, surpassing the 37% reduction seen with Resmetirom. Similarly, the inflammatory protein IL-6 showed a 51% decrease with miRNA-10a, compared to a 28% reduction with Resmetirom.
RXBio emphasized that these evaluations, conducted on 3D liver organoids created from human cells, provide strong evidence for potential clinical applications. Human iPSC-derived organoids closely mimic the microenvironment of actual patient liver tissues, offering a highly reliable model for drug evaluation.
RXBio Chief Executive Officer (CEO) Song Myung-seok stated that the collaboration with cellArtgen has confirmed that our lead candidate outperforms Resmetirom, the only approved MASH treatment globally, in fat reduction, anti-fibrotic, and anti-inflammatory efficacy. Armed with this data, it’s now accelerating discussions to advance the MASH drug pipeline into clinical trials both in the U.S. and internationally.