NephroDI Therapeutics Announces Investment by Sound Bioventures to Accelerate Development of NDI-5001 as a Potential First-in-Class Therapy for Pediatric Orphan Kidney Disease

  • New funding to take NDI-5001 into and through clinical proof of concept in Congenital Nephrogenic Diabetes Insipidus (NDI)
  • New investment complements previously announced strategic corporate partnership

June 20, 2024 Seattle, WA – NephroDI Therapeutics (NephroDI), a near-clinical stage pharmaceutical company focusing on kidney disorders, announces a Series A investment from life sciences VC Sound Bioventures. together with a previously announced strategic corporate partnership, the capital 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 (NDI). NDI-5001 is projected to enter the clinic later this year.

Congenital 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. Several of these drug-induced conditions are also Orphan Disease populations.

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 making it eligible for the Priority Review Voucher by the Office of Orphan Products Development at the U.S. Food & Drug Administration.

“These children and their families are in desperate need for new and better therapies that assist in keeping them healthy,” said Rachael Hagan, CEO of NephroDI. “We are excited that Sound Bioventures has joined NephroDI as an investor and member of the Board. The expertise that NephroDI gains via this partnership will help propel NDI-5001 during the initial clinical phase towards proof of concept. This will position the Company to rapidly enter pivotal trials in pediatric patients with this potentially first-in-class NDI therapy.”

“NephroDI has sought advice and formed close relationships with other physicians that are adult and pediatric experts in NDI, and the NDI Foundation,” stated Jeff Sands, MD, 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.”

Mike Parnell, President of the NDI Foundation, added, “It is the Foundation’s mission to serve the entire NDI community: patients and their families, researchers, physicians and other health care providers by supporting education, research, treatment and cures for Nephrogenic Diabetes Insipidus. We look forward to communicating and facilitating the efforts by NephroDI to bring new treatments to the NDI community.”

About NephroDI Therapeutics

NephroDI Therapeutics, Inc. is a pharmaceutical company headquartered in Seattle, Washington, focused on developing and commercializing drugs to treat concentration disorders of the kidney. The company’s lead program is NDI-5001, a novel kidney-targeted small molecule AMPK activator being developed as a potential first-in-class treatment of Congenital Nephrogenic Diabetes Insipidus. For more information, please visit the company website at

NephroDI Media Contact:

Josh Knackert

About Sound Bioventures

Sound Bioventures is a specialist life sciences venture capital fund investing in about-to-be clinical or clinical stage private companies in Europe and the USA developing specialty therapeutics in areas of significant clinical unmet need. The founding team has an active, hands-on approach and brings considerable operational and VC investment experience, including working together on over 40 investments over the past 15 years. 

Sound Bioventures operates from key locations in Washington DC (USA), Copenhagen (Denmark), and Stockholm (Sweden).

For more information, see our website Sound Bioventures


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