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NephroDI Therapeutics Announces Investment by Sound Bioventures to Accelerate Development of NDI-5001 as a Potential First-in-Class Therapy for Pediatric Orphan Kidney Disease

  • 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.

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

The press releases on this page 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.

NephroDI Therapeutics Announces Four New Leadership Team Appointments, Including Dr. Jeff Sands as Chief Medical Officer

Sep. 8, 2023 Seattle, WA – NephroDI Therapeutics (NephroDI), a pharmaceutical company focusing on concentration disorders of the kidney, is thrilled to announce the appointment of four key members of its executive team.

Jeff M. Sands, MD, a NephroDI co-founder, has been appointed the organization’s Chief Medical Officer. Dr. Sands’ research focuses on the molecular physiology of the kidney, the urine concentrating mechanism, and the translation of these basic research findings into novel therapies for Nephrogenic Diabetes Insipidus (NDI).

Numerous national and international awards (including the American Society of Nephrology’s Homer W. Smith Award), chair/leadership roles, and more than 175 peer-reviewed manuscripts highlight his more than 35-year career in renal research and nephrology patient care. Dr. Sands will leverage his comprehensive knowledge of NDI physiology and patient experience to help develop NephroDI’s asset, NDI-5001, a potential therapy for X-linked congenital NDI.

Additionally, NephroDI welcomes to their leadership team Janet Klein, PhD as Director of Biology, Dennis “Mike” Rose as the VP of Operations, and Josh Knackert as Director of Scientific Operations.

Janet will draw upon over 30 years’ experience, including over 100 peer-reviewed manuscripts on urea transporters and urine concentrating defects. She is a co-founder of NephroDI and will manage the research and development efforts for the organization.

Mike will build upon thirty-two years of successful enterprise program and project leadership experience in the healthcare industry to lead enterprise operations. Josh brings to bear experiences as a life science researcher, biotech consultant, and lifelong congenital NDI patient to his work overseeing the enterprise scientific and clinical strategies.

“With these four phenomenal appointments, NephroDI continues to establish the medical, scientific, and operational foundations essential to develop our potential NDI therapy,” said Rachael Hagan, CEO of NephroDI. “Dr. Sands represents one of the preeminent minds in kidney concentration and NDI. The organization could not hope for a more ideal Chief Medical Officer.”

“I am honored by the opportunity to transition this potential therapy into the clinic, with potentially meaningful impacts for NDI patients and their families,” said Dr. Sands. “I am excited to continue my rewarding research and clinical work on NDI in the unique and immensely important role as NephroDI’s Chief Medical Officer.”

NephroDI Therapeutics Enters Agreement to Develop a First-in-Class Treatment for a Genetic, Rare Disease of the Kidneys

June 22, 2023 Seattle, WA – NephroDI Therapeutics (NephroDI), a pharmaceutical company focusing on concentration disorders of the kidney, announces today it has entered an agreement with The McQuade Center for Strategic Research and Development, LLC (MSRD), a member of the global Otsuka family of pharmaceutical companies which identifies and supports early-stage opportunities that can change the landscape of treatments for renal disorders and mental illness.

MSRD at Otsuka in the U.S. and NephroDI will collaborate with respect to the clinical development of NephroDI’s asset, NDI-5001, a novel kidney-targeted small molecule AMPK activator, as a potential therapy for X-linked congenital Nephrogenic Diabetes Insipidus (NDI). NDI is a rare genetic kidney condition that affects individuals from birth, causing them to produce up to 20 liters of urine per day.

Under the terms of the agreement, NephroDI and MSRD will exclusively develop NDI-5001 through completion of certain clinical studies with the opportunity for MSRD to elect to expand the collaboration after those studies or upon the occurrence of certain triggering events. Later studies are anticipated to include affected individuals, providing an opportunity for early evidence of target engagement through increases in urine concentrating ability.  

“X-linked NDI pediatric 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 quality of life for patients and their families,” said Rachael Hagan, CEO of NephroDI. “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.”