Type 2 diabetes mellitus (T2D) is a major public health concern in Australia and globally. Existing treatments do not directly address a root cause of T2D, namely insulin resistance. This occurs when the body does not respond properly to insulin, leading to high blood glucose levels and ill-health. Many people with T2D progress from tablet treatments to weekly injectables, to needing daily or multiple daily doses of insulin. We have developed an innovative precision small RNA drug for T2D treatment that resolves insulin resistance directly. Our drug candidate, PGP-011, is an antisense oligonucleotide (ASO) designed to block the production of a specific cellular enzyme, a validated target which interferes with insulin signaling. Our data indicate that PGP-011 effectively reduces expression of this target, and lowers glucose levels better than dapagliflozin, one of the current accepted treatments. Thus, PGP-011 holds promise for treating T2D by making the body more responsive to insulin action.
