RARE Daily

Xontogeny Seeds NephroDI to Develop a Treatment for Nephrogenic Diabetes Insipidus

May 12, 2021

NephroDI Therapeutics received seed investment from investment firm and incubator Xontogeny to advance its lead therapeutic for the X-linked/congenital form of nephrogenic diabetes insipidus through preclinical development.

Photo: Rachael Hagan, president and CEO of NephroDI

X-linked nephrogenic diabetes insipidus (NDI) affects one in 250,000 males in the United States and is characterized by profoundly excessive urination of up to 20L (5 gallons) per day and frequent complications.

Congenital NDI manifests at birth and is a life-long condition with a normal life expectancy. NDI patients produce extremely large amounts of dilute urine resulting from an inability of the kidney to respond to vasopressin. Congenital NDI results primarily from mutations in the vasopressin 2 receptor, which is located on the X chromosome. Congenital NDI has a profound impact on children. If hydration is not maintained, it can cause mental retardation and even chronic kidney disease by middle school age. The current standard of care for NDI is centered around hydrating with up to 80 glasses of water per day, low sodium diet, diuretics, and non-steroidal anti-inflammatory drugs or NSAIDs, which can cause additional kidney damage with chronic use.

NephroDI’s lead product, NDI-5033 is a unique adenosine monophosphate activated kinase (AMPK) activator that can stimulate water reabsorption without causing hypoglycemia, decreasing overall urine output. NDI-5033 is being developed for the congenital form of NDI and is positioned to become a much-needed alternative to current management measures.

Rachael Hagan, president and CEO of NephroDI, said that Xontogeny’s support, investment, and industry experience, will be especially important to its goal to bring NDI-5033 into clinical trials.

Author: Rare Daily Staff

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