RGX-202 is a small molecule that suppresses gastrointestinal cancer growth by inhibiting a novel cancer metabolism pathway involved in supplying energy to cancer cells.
Mechanism of Action

Creatine metabolism has been implicated in colon cancer progression and metastatic colonization of the liver. Cancer cells upregulate and release creatine kinase-B (CKB) into the extracellular space, where it generates the high energy metabolite phosphocreatine.
Phosphocreatine is imported via the creatine transporter SLC6a8, the target of RGX-202 that is over-expressed in several prevalent cancer types, including gastrointestinal cancers.
Intracellular phosphocreatine can be converted to ATP to fuel the survival of cancer cells as they proliferate and spread. Consistent with this finding, genetic depletion of SLC6a8 in colon and pancreatic cancer cell lines significantly reduced cancer growth in animal studies.1
Pipeline
RGX-104
RGX-104 is a novel oral small molecule immunotherapy in development for the treatment of solid tumors, including drug-resistant malignancies.
Pipeline
RGX-019
RGX-019 is a monoclonal antibody that targets key pathway that drives tumor progression and metastasis of several cancer types, including triple-negative breast cancer.
Reference:
1) Loo JM et al. Cell. 2015 Jan 29;160(3):393-406
2) Kurth I et al. Proceedings of the American Association for Cancer Research Annual Meeting 2018; 2018 Apr 14-18; Chicago, Illinois (IL)