NephroDI Therapeutics (NephroDI), a leading near-clinical stage pharmaceutical company specializing in kidney disorders, proudly announces a significant Series A investment from life sciences venture capital firm Sound Bioventures. This latest infusion of capital, in tandem with a previously established strategic corporate partnership, is set to advance the development of NDI-5001, a groundbreaking small molecule AMPK activator designed to target the kidney. NDI-5001 aims to become the first-in-class treatment for Congenital Nephrogenic Diabetes Insipidus (NDI) and is expected to enter clinical trials later this year.
Congenital NDI, a rare genetic condition linked to the X chromosome, manifests from birth, causing individuals to produce copious amounts of dilute urine daily due to their kidneys’ inability to concentrate urine. Affected individuals often consume up to 20 liters of water per day to avoid severe dehydration, significantly impacting their quality of life, including sleep, education, travel, and employment.
In addition to congenital cases, NDI can be drug-induced, particularly by lithium used in neuropsychiatric treatments. Current off-label and dietary interventions for NDI are largely ineffective, leading to high discontinuation rates due to minimal clinical benefits and significant side effects. Unlike other AMPK activators under development for Type 2 diabetes, NDI-5001 does not affect blood glucose levels, as demonstrated in pre-clinical studies. This innovative drug has received Orphan Drug Designation and Rare Pediatric Disease designation, qualifying it for the Priority Review Voucher from the U.S. FDA’s Office of Orphan Products Development.
Rachael Hagan, CEO of NephroDI, expressed her enthusiasm: “These children and their families are in dire need of effective therapies to maintain their health. We are thrilled to welcome Sound Bioventures as an investor and board member. Their expertise will be instrumental in advancing NDI-5001 through the initial clinical phase towards proof of concept, positioning us for rapid entry into pivotal trials for this promising pediatric therapy.”
Jeff Sands, MD, Chief Medical Officer of NephroDI, highlighted the collaborative efforts: “NephroDI has cultivated close relationships with leading adult and pediatric NDI experts and the NDI Foundation. We all share a common goal of enhancing therapeutic options for NDI patients. I am optimistic about NDI-5001’s potential to significantly improve the quality of life for these patients, particularly children.”
Mike Parnell, President of the NDI Foundation, added, “Our mission is to support the NDI community through education, research, and the development of treatments and cures. We are excited to support NephroDI’s efforts to bring innovative treatments to the NDI community.”
