We are developing odiparcil for the treatment of several subtypes of MPS, a group of rare and progressive genetic disorders which manifest early in life
Odiparcil, an orally-available small molecule that can potentially decrease the lysosomal accumulation of chondroitin sulfate (CS) and dermatan sulfate (DS) in patients with certain MPS subtypes (MPS I, II, IVa, VI and VII).
Odiparcil changes the way DS and CS are synthesized, thereby facilitating the production of soluble DS and CS glycosaminoglycans (GAGs), which can be excreted in the urine, rather than accumulating in cells.
We believe odiparcil’s mechanism of action is relevant to a number of tissues in which GAGs accumulate and which are poorly addressed with limited efficacy by the current standard of care, which is enzyme replacement therapy (ERT). Unlike ERT, odiparcil is a small molecule that is well distributed in the body, even in tissues that are poorly vascularized or protected by a barrier such as the eyes.
In the recent iMProveS trial odiparcil demonstrated a dose of dependent increase of GAGs excretion via urine in MPS VI patients. At the same time, patients treated with odiparcil experienced improvements in organs such as the heart, lungs and eyes. For more information on the headline results of the iMProveS study please click here.