RARE Daily

June 21, 2024

Rare Daily Staff

NephroDI Therapeutics raised $2 million from Sound Bioventures that will be used to advance NDI-5001, a proprietary small molecule AMPK activator that targets the kidney, initially as a potential first-in-class treatment of congenital nephrogenic diabetes insipidus.

The funding follows a partnership NephroDI struck with The McQuade Center for Strategic Research and Development, a member of Otsuka Pharmaceutical, to advance the drug.

Congenital nephrogenic diabetes insipidus (NDI) is a rare X-linked genetic condition that affects individuals from birth, in which they produce significant amounts of dilute urine daily because their kidneys lack the ability to concentrate urine. This can require affected individuals to drink up to 20 liters of water per day to prevent life threatening dehydration. In addition to the clinical problems caused by this disease, the need for water and frequent urination dramatically interferes with sleep, school, travel and choices of employment, among other aspects of quality of life. NDI can also be induced as an unwanted side effect of various drugs including lithium usage in neuropsychiatric patients.

Current off-label therapies and diet-based interventions are ineffective, and patients often discontinue them for lack of clinical benefit and associated high burden of side effects. Diabetes insipidus is unrelated to diabetes mellitus and NDI-5001 does not affect blood glucose levels in pre-clinical studies, in contrast to other AMPK activators being developed for Type 2 diabetes. NDI-5001 has received Orphan Drug designation and was granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration.

NephroDI plans to start clinical trials of NDI-5001 this year.

“NephroDI has sought advice and formed close relationships with other physicians that are adult and pediatric experts in NDI, and the NDI Foundation,” said Jeff Sands, chief medical officer of NephroDI. “All parties share in the common goal of advancing therapeutic options for patients suffering with NDI. I am highly optimistic about the potential of NDI-5001 to improve the quality of life in this patient population, especially in children.”

Photo: Jeff Sands, chief medical officer of NephroDI

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