Pharma Focus Asia

NephroDI Therapeutics Receives Investment from Sound Bioventures to Hasten Development of NDI-5001 for Pediatric Orphan Kidney Disease

Friday, June 21, 2024

NephroDI Therapeutics (NephroDI), a pharmaceutical company in the advanced stages of development focusing on kidney disorders, has received a Series A investment from Sound Bioventures, a life sciences venture capital firm. This funding, along with a previously announced strategic corporate partnership, will support the advancement of NDI-5001, a proprietary small molecule AMPK activator designed to target kidney function. The primary aim is to develop it as a potential first-of-its-kind treatment for Congenital Nephrogenic Diabetes Insipidus (NDI).

Congenital NDI is a rare genetic condition that manifests from birth, characterized by the kidneys' inability to concentrate urine, leading to excessive daily urine production. Individuals affected by this condition often need to consume large amounts of water—up to 20 liters per day—to prevent severe dehydration. Beyond the medical challenges, managing this condition significantly impacts daily life, including sleep patterns, educational pursuits, travel plans, and career choices.

NDI can also be induced by certain medications, such as lithium used in the treatment of neuropsychiatric disorders, affecting a subset of patients with rare diseases.

Current therapies and dietary adjustments have proven largely ineffective, causing many patients to discontinue treatment due to minimal efficacy and significant side effects. Importantly, unlike other AMPK activators under development for Type 2 diabetes, NDI-5001 does not affect blood glucose levels, as demonstrated in pre-clinical studies. The compound has received Orphan Drug Designation and Rare Pediatric Disease designation from the U.S. Food & Drug Administration, making it eligible for a Priority Review Voucher.

Rachael Hagan, CEO of NephroDI, stressed the urgent need for improved therapies for patients and their families, welcoming Sound Bioventures' investment and board participation. This collaboration aims to leverage Sound Bioventures' expertise to advance NDI-5001 through initial clinical trials to establish proof of concept. The ultimate goal is to progress swiftly to pivotal trials focusing on pediatric patients, potentially transforming the treatment landscape for NDI.

NephroDI, highlighted collaborative efforts with leading specialists in adult and pediatric NDI and the NDI Foundation. He expressed optimism about NDI-5001's potential to significantly improve quality of life, especially for children affected by NDI.

Mike Parnell, President of the NDI Foundation, expressed support for NephroDI's mission to advance NDI treatments, emphasizing their commitment to education, research, and patient care within the NDI community.

In summary, NephroDI is positioned to make substantial progress with NDI-5001, driven by strategic partnerships and focused investment, aiming to address critical unmet needs in the treatment of NDI.



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