Structured around addressing concentration disorders of the kidney, NephroDI Therapeutics is focused initially on clinical indication for its orally administered lead small molecule is for Nephrogenic Diabetes Insipidus (NDI) in children: a pediatric orphan indication. NDI is a disease where patients produce extremely large amounts of dilute urine resulting due to an inability of the kidney to respond to vasopressin. Congenital NDI in the pediatric population results primarily from mutations in the type 2 vasopressin receptor -- located on X chromosome -- and has a profound impact on children. With these children producing up to 20 L of urine per day, to avoid dehydration., they must drink an equivalent amount of water per day. Children suffering multiple episodes of severe dehydration often suffer mental retardation. Congenital NDI manifests at birth. A life-long condition, patients often have a normal life expectancy. Considered an orphan condition in US and Europe, best estimates are that for every One million males born in the US, FOUR are likely to have X-linked congenital NDI. With no effective NDI therapy, current management necessitates afflicted patients to drink as much fluid as they excrete to prevent dehydration. Currently available control measures are only minimally effective and other behavioral changes eg reduced voiding may lead to kidney failure
