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NephroDI Therapeutics. Lack of concentration is our focus.

NephroDI Therapeutics is a pharmaceutical company that focuses on concentration disorders of the kidney. The initial clinical indication for its orally administered lead small molecule is for Nephrogenic Diabetes Insipidus in children, a pediatric orphan indication.

Nephrogenic Diabetes Insipidus (NDI) is a disease where patients produce extremely large amounts of dilute urine resulting from an inability of the kidney to respond to vasopressin. Congenital NDI in the pediatric population results primarily from mutations in the type 2 vasopressin receptor, which is located on the X chromosome. Congenital NDI has a profound impact on children. Since these children can produce up to 20 L of urine per day, they must drink 20 L of water per day to avoid dehydration. Children who suffer multiple episodes of severe dehydration often end up with mental retardation.

NDI should not be confused with diabetes mellitus (i.e. sugar diabetes). Despite the similar name, the two conditions are completely different. NDI does not involve disorders of blood glucose control.

Congenital NDI manifests at birth and is a life-long condition with a normal life expectancy. It is considered an orphan condition in the US and Europe: the best estimate is that for every 1 million males born in the US, four are likely to have X-linked congenital NDI.

Currently there is no effective therapy for NDI. Current management requires patients to drink as much fluid as they excrete to prevent dehydration. Patients are also prescribed a thiazide diuretic and must maintain a extremely low sodium diet (0.5 g/day). While often onerous, especially the ultra-low sodium diet, these measures are minimally effective. They may also be prescribed non-steroidal anti- inflammatory drugs or NSAIDs, but these can cause additional kidney damage with chronic use. In an attempt to maintain a semblance of a normal lifestyle, NDI patients can succumb to the temptation to void less frequently. This behavior can result in an enlarged bladder, which can cause an obstructive nephropathy and lead to kidney failure. There is a compelling clinical need for an effective therapy. A therapy that significantly reduces urine output would be a tremendous benefit to children suffering from NDI.

Rachael Hagan, M.Sc.
  • President & Chief Executive Officer
  • RHagan@NephroDI.com

Rachael Hagan is a recognized leader with a demonstrated track record of commercializing early-stage technology out of universities. Ms. Hagan is experienced in promoting emerging technologies and businesses to investors with a strong track record of identifying, planning, and implementing strategic partnerships.

Ms. Hagan formerly served as the Managing Director of the Coulter Translational Partnership Fund (CTPF), an evergreen fund providing $1.5M a year for investments at both Emory University/Georgia Tech and previously at the University of Washington. Beyond providing capital, she evolved the Coulter Fund into a team-based approach that provided a rich resource for advancing translational projects to clinical adoption.

Ms Hagan managed, conceived and implemented investment processes and procedures that brought business discipline to academic environments. She served as interim management on all investments. She actively participated with innovators and co-investors on operational, funding, growth and exit strategies culminating in the formation of more than 72 start-up companies, twenty-three (38) of which raised over $200M in total funding and introduced new products to the market. Ten (12) products were licensed directly to established medical device companies.

Ms. Hagan has provided expert advice on medical technology investment to early-stage funds and angel investors, including Keiretsu Forum Northwest, Wings, Seraph Capital Network, Johns Hopkins University, Seattle Children’s Hospital, Imperial College London, and University of Calgary. Ms. Hagan has an M.Sc. in Medical Regulatory Affairs and was a lecturer at the University of Washington’s School of Pharmacy Master program for Medical Regulatory Affairs. She has advised, coached, and collaborate with numerous entrepreneurs to develop business models, identify and pitch to potential investors, structure funding, and grow the resulting emerging businesses.

Jeff M. Sands, M.D.
  • Founder
  • Juha P. Kokko Professor of Medicine and Physiology
  • Renal Division Director, Emory University

Dr. Jeff M. Sands is the Juha P. Kokko Professor of Medicine and Physiology, and Director of the Renal Division at Emory University School of Medicine. Dr. Sands is a graduate of Harvard College and Boston University School of Medicine. He trained in Medicine at the University of Chicago and the NIH’s National Heart, Lung, and Blood Institute (NHLBI). Following research fellowship training in the Laboratory of Kidney and Electrolyte Metabolism, NHLBI, NIH, he proceeded to a clinical nephrology fellowship at Emory. Dr. Sands joined the Emory faculty as an Assistant Professor in 1989, was promoted to Associate Professor in 1993, and to Professor 1998. He accepted the Kokko Professorship and Division Director position at Emory in 2002. He served as Executive Vice-Chair of Medicine from 2009-2015 and as Associate Dean for Clinical and Translational Research from 2006- 2010. Dr. Sands served as Editor-in- Chief of the American Journal of Physiology – Renal from 2001- 2007, as a councilor of the American Physiological Society from 2003-2006, as chair of the 2004 American Society of Nephrology Program Committee, and as a member of the NIDDK Board of Scientific Councilors from 2008-2013. Dr. Sands was elected 91 st President of the American Physiological Society in 2017 and will serve from 2018-2019. He is a member of the American Association of Physicians, the American Society for Clinical Investigation, and the American Clinical and Climatological Association. Dr. Sands has served on study sections for the NIH, AHA, and NKF. Dr. Sands’ research focuses on urea transporters, aquaporins, and the urine concentrating mechanism. He has had NIH R01 funding continuously since 1989 and is the PI on the Emory Renal Division’s T32 NIH Training Grant and an R25 to train undergraduate students in renal research. He is considered a Key Opinion Leader for nephrogenic diabetes insipidus by the National Organization of Rare Diseases.

Janet D. Klein, Ph.D.
  • Founder

Dr. Janet Klein, is a recognized leading expert on the cellular and molecular mechanisms that contribute to changes in water and solute homeostasis that occur in systemic and kidney diseases. Dr. Klein is an Associate Professor at Emory University. She has been in the Renal Division since 1993 in several capacities and works closely with Dr. Sands. For the past twelve years she has been investigating various transport mechanisms in the kidney.

Dr Klein is a member of the Editorial Board of the American Journal of Physiology, and is the Associate Editor of Renal Frontiers in Physiology. Among her honors, she has been an invited speaker at the Nephrogenic Diabetes Insipidus Foundation 2008 Global Conference and the 2015 United States National Institutes (NIH) of Health Renal Pathophysiology Workshop. She is a grant reviewer for the American Heart Association, United States Veterans Administration, NIH and National Kidney Research Fund of the United Kingdom. Her society memberships include American Society of Nephrology, the American Physiological Society and the American Heart Association where she has chaired or co-chaired several committees. Dr. Klein is a frequent reviewer for the Journal of the American Society of Nephrology, the Journal of Biological Chemistry, European Journal of Pharmacology and The American Journal of Physiology Heart, Integrative Physiology and Cell journals.

Dr. Klein received her B.S. in Biochemistry from the University of Scranton and M.S. and Ph.D. in Chemistry from New Mexico State University with a major in Biochemistry and a minor in Analytical Chemistry. She has held postdoctoral positions at the Terre Haute Center for Medical Education, the American Red Cross, and at Emory University working with Dr. Lawrence Philips in the Division of Endocrinology.

Dr. Klein is the author on a total of over one hundred peer reviewed papers, review articles and book chapters. She is a co-inventor on two pending patents.

Ish Khanna, Ph.D.
  • Founder

Dr. Khanna has over twenty eight years of pharmaceutical industry experience with proven record of success in advancing drug molecules from “concept to clinic and market” He has held Technical and Executive Leadership positions in pharmaceutical companies (Pfizer, Pharmacia, Searle, Dr. Reddy’s Labs, NeuroPn Therapeutics). Dr. Khanna is experienced in building balanced discovery portfolio comprising First in Class, Best in Class and Drug Repositioning. Dr. Khanna is well-versed with emerging trends in global pharmaceutical industry and is skilled fostering research collaborations with partners across the globe. Dr. Khanna’s areas of expertise include medicinal chemistry, PK/PD correlations of drug molecules, IP strategies, and lead optimization strategies for novel drugs across therapeutics. He is a co-inventor and co-author of over 110 patents and peer reviewed science publications. Dr. Khanna is co-inventor of celecoxib and multiple clinical agents across therapeutics.

Ram Pillarisetti, Ph.D.
  • Founder

Dr. Pillarisetti has over 23 years of experience in research in Academia and Industry. Most recently he was cofounder of NeuroPn Therapeutics and head of research at Kareus Therapeutics. Prior to that he was the Vice President of Research at Reddy US Therapeutics and Dr Reddy’s Laboratories (DRL). At DRL he headed the biology department and was managing activities from discovery to clinic. He has successfully brought several drug molecules from concept to clinic. Key accomplishments include three candidates in Phase 1/2 and several in preclinical development. He has been conducting research in the cardiovascular and metabolic diseases for over 20 years. Prior to Reddy US Therapeutics/Dr Reddy Laboratories, Dr. Pillarisetti had held faculty appointments at Columbia University and the North Shore- Long Island Jewish Health System in New York, where he established a peptide discovery program (therapeutic angiogenesis and cancer) for Angion Biotech. His areas of expertise include Inflammation, Pain and Metabolic diseases. He is the author of over 60 research papers and review articles and inventor on several issued patents. Dr. Pillarisetti had been an investigator of the American Heart association (1996-99) and a member of the American Diabetes association and a fellow of the American Heart Association.