ML-016

ML-016 is a first-in-class, experimental drug being developed for advanced cancers with lung and/or liver involvement, including metastatic disease. It is comprised of an amino acid polymer conjugated to doxorubicin, delivered in a formulation that includes platelet-like bio-erodible mesoporous silicon elements. This unique delivery system is designed to target the vascular endothelium of blood vessels within the tumor microenvironment. Once delivered, the amino acid-drug conjugate is released and forms exosome-like vesicles (“exosomoids”), which are intended to be preferentially taken up by cancer cells—including those resistant to conventional therapy. The exosomoids are transported to late-stage endosomes and lysosomes, where increased acidity releases the cytotoxic agent (doxorubicin) into the cell nucleus. In preclinical studies, this mechanism led to long-term survival in 50% of laboratory animals with otherwise uniformly lethal metastatic burden[1]. ML-016 is the lead product of BrYet’s “Transport Oncophysics” platform, which uses physics-based principles to overcome biological barriers in tumor targeting[5][7]. The drug is currently in Phase 1/2 clinical trials for advanced solid tumors with lung and/or liver involvement, with enrollment expected to begin in early 2026[1][8].

Other names

iNPG-pDox