Diabetes Insipidus, Nephrogenic

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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When Chris Garabedian set out early last year to raise money for a second Perceptive Advisors fund focused on early-stage biotech startups, it took just a matter of months to draw interest from investors willing to commit more than $1 billion total. The fund could have closed with that much money, maybe more, said Garabedian, the portfolio manager. But it ended up with a little more than half of that, which he described as “the right size.” Garabedian acknowledges the large sums raised by large venture capital firms, many of them pumping money into platform technologies with potential for a broad range of applications. Then he points out that that’s exactly where Perceptive Xontogeny Ventures (PXV) doesn’t want to be. Rather than looking at platforms, Garabedian said PXV is scouting products. He’s looking for biotech companies that have an asset showing a promising signal and a path to reach clinical testing. “We’re very product focused,” he said. “We’re not trying to do next-generation CRISPR, which requires a couple of hundred million dollars before a candidate gets to the clinic.” On Wednesday, Perceptive Advisors announced its second fund to invest in biotech startups with promising potential products. PXV II closed with $515 million in financial commitments. The new fund, which will invest in 10 to 12 startups, comes about 18 months after the firm unveiled PXV I, which totaled $210 million. The new Perceptive fund starts with lessons learned from the first fund. Garabedian said that PXV I was targeting up to a dozen investments in drug companies raising Series A rounds ranging from $15 million to $25 million—enough to get them through late-preclinical research, perhaps through early clinical testing in healthy volunteers. However, Garabedian said that founders wanted to go for bigger Series A financings to advance their technologies to proof-of-concept testing. The larger rounds meant that PXV I didn’t have much left over to deploy in Series B rounds. The larger fund size of PXV II is an acknowledgement that the Series A rounds may be larger, ranging from $25 million to $40 million. The fund size also affords the flexibility to participate in Series B financings. PXV II is not starting from scratch. Based on the outreach and the diligence done for the first fund, Garabedian and his team already have connections and a pipeline of potential investments. Some of those startups could come from Xontogeny, a biotech accelerator that Garabedian also runs. Launched in 2017, Xontogeny provides seed financing and other support to these startups, some of which are led by first-time founders from academia. Garabedian, a former executive at Gilead Sciences, Celgene, and Sarepta Therapeutics, mentors those companies. Though Xontogeny and PXV operate separately, there are connections between them. Xontogeny’s first investor was Perceptive Advisors. Fred Callori, Xontogeny’s senior vice president of corporate development, and Ben Askew, the accelerator’s head of R&D, are partners in PXV Fund II. Also, startups in the accelerator may be considered for Series A investment from the PXV fund. Two Xontogeny graduates secured PXV I Series A funding and went on to exit events earlier this year. Quellis Biosciences was acquired by Catabasis Pharmaceuticals in late January; days later, Landos Biopharma priced its IPO. Most of PXV I’s investments were in drug developers, though the fund did invest in one medical device company and one contract development and manufacturing organization. PXV II will continue the first fund’s drug focus, but Garabedian said that the life sciences industry is changing and innovation can also be found in drug discovery tools. He added that companies developing artificial intelligence and machine learning technologies offer additional investment opportunities, along with startups working to digitize health care and bring treatment into the home. Separate from the close of the second PXV fund, Xontogeny announced five new portfolio companies that have joined the accelerator. They are: • Nephraegis Therapeutics is developing potential first-in-class drug for renal disease. The company’s lead program, NPH-022, is for the prevention of acute kidney injury following surgical procedures, such as heart valve replacement, abdominal surgery, and liver transplant. The drug, a next-generation epoxyeicosatraenoic acid (EET) analog, is being developed in both intravenous and oral formulations. • NephroDI Therapeutics is also focusing on kidney disorders. The company’s lead compound, NDI-5033, is an experimental treatment for Nephrogenic Diabetes Insipidus, a rare, congenital disease in which the kidneys do not respond to the anti-diuretic hormone vasopressin, leading patients to produce extremely large amounts of dilute urine. The small molecule is an oral AMPK activator. • Peroxitech is developing a treatment for acute lung injury. The company’s lead program is a peptide that works through a novel pathway to eliminate molecules that build up in the lung leading to respiratory failure. The startup is a University of Pennsylvania spinout. • Shifa Biomedical aims to treat dyslipidemia, which is a high level of lipids in the blood. The company is developing an oral small molecule intended to block PCSK9, an enzyme that plays a role in cholesterol metabolism. The biotech’s drug, P-21, has data showing it could provide an alternative to currently available PCSK9 inhibitors, antibodies that are injectable drugs. The technology was developed by the Malvern, Pennsylvania-based biotech with National Institute of Health grant funding. • Tellus Therapeutics, based on research from Duke University, is developing a treatment for babies born at risk of brain injury. The company’s lead program, TT-20, is made from compounds found in breast milk that have been shown to repair brain damage in animal models. The treatment will be delivered on top of parenteral nutrition given to infants in neonatal intensive care units. Photo by Perceptive Advisors