Diabetes Insipidus, Nephrogenic

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 www.nephrodi.com. NephroDI Media Contact: Josh Knackert jknackert@nephrodi.com 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 Forward-looking Statements This press release contains certain “forward-looking statements” concerning the development of NephroDI Therapeutics’ products, the potential benefits and attributes of those products, and the company’s expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions, and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. NephroDI Therapeutics undertakes no obligation to update any forward-looking statements for any reason. Press Release URL: News | NephroDI Therapeutics

NephroDI Therapeutics’ NDI-5001 is being developed to treat X-linked congenital nephrogenic diabetes insipidus, a rare genetic kidney condition. Credit: crystal light / Shutterstock.com. NephroDI Therapeutics has signed a deal with the McQuade Center for Strategic Research and Development (MSRD), a member of the global Otsuka group of pharmaceutical companies which is based in Princeton, US, for the development of NDI-5001 to treat nephrogenic diabetes insipidus (NDI). NephroDI and MSRD will clinically develop the former’s small molecule AMPK activator, NDI-5001, as a potential therapy to treat X-linked congenital NDI, a rare genetic kidney condition. MSRD will have the option to expand the partnership following the trials, or on reaching specified milestones. NephroDI Therapeutics CEO Rachael Hagan stated: “X-linked NDI paediatric patients and their caregivers currently navigate a lifetime of intense symptom management. There are minimally effective treatments currently available for NDI that do little to significantly improve the quality of life for patients and their families. “The NDI, renal and nephrology experts now collaborating across NephroDI, MSRD, and Otsuka are uniquely positioned to embark on the next phase of development for a potential novel NDI treatment. “This team is driven by the opportunity to bring a first-in-class treatment to these patients and their families.” The company stated that the later trials are expected to include affected patients. This will provide a chance for early evidence of target engagement through an increase in urine-concentrating ability. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Antidiuretics are drugs used to control water balance in individuals suffering from diabetes insipidus (DI). Antidiuretic drug consist of antidiuretic hormone (ADH) and use of thiazide base diuretics. Antidiuretic hormone is commonly known as arginine vasopressin. Antidiuretic hormone is secreted by the posterior pituitary gland. The rate of secretion of antidiuretic hormone is controlled by osmoreceptors present in the hypothalamus. Increase in contraction of plasma fluid leads to physiological stimuli for antidiuretic hormone release. Antidiuretics bind with receptor cells present in the kidney and increase water reabsorption by stimulating aquaporins present in membranes of kidney tubules. These aquaporins transport solute free water from the tubules to the blood by decreasing the osmolality of plasma cells. As the concentration of plasma cells increases, the rate of antidiuretic hormone release increases in the blood which increases reabsorption of water. This increases water retention and urine concentration. Deficiency of antidiuretic hormones causes two types of diabetes insipidus: cranial diabetes insipidus and nephrogenic diabetes insipidus. The symptoms of diabetes insipidus are increase in water intake to compensate the loss of water. In very rare situations, diabetes insipidus can develop during pregnancy and lead to gastrointestinal diabetes insipidus. Cranial diabetes occurs when there is not enough antidiuretic hormone present in the body to control urine production. It is the most common type of diabetes insipidus which occurs when the pituitary gland or the hypothalamus does not function properly. Nephrogenic diabetes insipidus occurs when the kidneys fail to function properly in the presence of antidiuretic hormone. It is caused due to kidney malfunction. In order to treat diabetes insipidus, desmopressin and vasopressin are used which replicate the function of antidiuretic hormone function. The most common medications used for the treatment of nephrogenic diabetes insipidus are thiazide diuretics, which help to reduce the amount of urine produced by the kidney. The inherited form of nephrogenic diabetes insipidus is due to mutation in AVPR gene, in very rare cases approximately 10% of inherited diabetes insipidus results from mutation in aquaporin 2 gene. The acquired form of nephrogenic diabetes insipidus can lead to cyst formation in the kidney, high calcium levels in blood, and lead to kidney infections. The prevalence rate of diabetes insipidus in the U.S. is about 1 in 6,666, while the prevalence rate of diabetes insipidus among general population is approximately 1 in 25,000 individuals. Major drivers of the antidiuretics market are growing awareness, rising prevalence of diabetes, increasing geriatric population, increase in obesity in the young generation, and government initiatives to promote research related to the study of antidiuretics and its use to control diabetes insipidus. High cost associated with the drug, stringent regulatory scenarios in drug development, risk factors associated with antidiuretics such as excessive bleeding, light headedness, and hematoma during collection of blood sample from individuals on diuretics are likely to restrain the antidiuretics market during the forecast period. Geographically, the global antidiuretics market has been segmented into five key regions: North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America leads the global antidiuretics market largely because of increasing population, rising number of diabetic population, and growing research activity. North America was closely followed by Europe and Asia Pacific. Asia Pacific is an emerging market for antidiuretics primarily because of increasing health care expenditure in the region and rising demand. Key players in the global antidiuretics market are Pfizer, Novartis AG, F Hoffmann-La Roche, bioMérieux, Merck & Co., Sanofi, Cayman Chemical, and JHP Pharmaceuticals LLC, and others. More Trending Reports by Transparency Market Research: Gestational Diabetes Treatment Market: Gestational diabetes can be defined as a type of diabetes that affects pregnant women. It is usually a temporary condition which develops during pregnancy and subsides after the birth of the baby. However, there are possibilities that diabetes may return in the later stages of life as type II diabetes mellitus. Non-insulin Anti-diabetes Drugs Market: Diabetes is a metabolic disease, wherein the body is either unable to secret insulin (type 1 diabetes) or is unable to utilize the generated insulin (type 2 diabetes). This elevated sugar level leads to various symptoms such as increased thirst & appetite, weight loss, frequent urination, fatigue, cuts and bruises that do not heal, numbness and tingling in hands and feet. If untreated, diabetes leads to various acute and chronic life-threatening complications. About Us Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals. Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. 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