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NephroDI Therapeutics is developing a novel AMPK activator as a first-in-class potential therapy for Nephrogenic Diabetes Insipidus (NDI).

The genesis of this technology began with Drs. Jeff Sands and Janet Klein, recognized clinical and research leaders in kidney diseases. While NDI patients are now living longer lives with fewer cognitive deficits, the quality of their lives remains diminished. Drs. Sand and Klein were investigating the role of AMPK activators as a promising potential therapy to reduce urine volume in NDI patients.  Current AMPK activators, FDA approved for or being developed to treat the unrelated condition of diabetes mellitus, showed encouraging preclinical data in animal models. But the need for high doses and the risk of hypoglycemia, (i.e., dangerously low blood sugar levels), prevent their long-term use in NDI patients. Concurrently, Drs. Khanna and Pillarisetti were developing a novel class of AMPK activators for the treatment of type 2 diabetes mellitus. A mutual colleague introduced the two teams of Investigators. Both teams sensed an opportunity to address the high unmet need in children affected by NDI and decided to work together to investigate potential of the approach. The proof-of-concept studies done on rodent models of NDI with select AMPK activators, which lacked activity in controlling blood sugar levels, yielded very encouraging results. The Team received grant support for their early efficacy studies from the Georgia Research Alliance (GRA) and Wallace H. Coulter Foundation’s Coulter Fund led by Rachael Hagan, the managing director of the fund. The scientific results and need amongst these children proved so compelling, that the four investigators and Ms. Hagan, who left the Coulter Fund to manage the Company as CEO, started a new biotech company, NephroDI Therapeutics, Inc.