NephroDI Therapeutics, a pharmaceutical company specializing in kidney disorders, has announced a Series A investment from Sound Bioventures, a venture capital firm. This new funding, combined with a previously established strategic corporate partnership, will support the progression of
NDI-5001, a unique small molecule
AMPK activator designed to treat
Congenital Nephrogenic Diabetes Insipidus (NDI). NDI-5001 is set to begin clinical trials later this year.
Congenital NDI is an uncommon X-linked genetic disorder that affects individuals from birth, causing them to produce large volumes of dilute urine daily. Those affected may need to drink up to 20 liters of water daily to avoid severe
dehydration. This condition greatly impacts various aspects of life, such as sleep, education, travel, and employment. Additionally, NDI can be induced by certain medications, including
lithium used in neuropsychiatric treatments, which also fall under Orphan Disease categories.
Current treatments for NDI, including off-label therapies and diet-based interventions, often prove ineffective, leading many patients to abandon them due to minimal benefits and significant side effects. Unlike
diabetes mellitus,
diabetes insipidus does not involve blood glucose levels, and pre-clinical studies have shown that NDI-5001 does not influence blood glucose levels, distinguishing it from other AMPK activators meant for
Type 2 diabetes. NDI-5001 has been granted Orphan Drug Designation and
Rare Pediatric Disease designation by the U.S. Food & Drug Administration, making it eligible for a Priority Review Voucher.
NephroDI's CEO, Rachael Hagan, emphasized the urgent need for improved therapies for children and their families dealing with NDI. She expressed enthusiasm for Sound Bioventures' investment and the strategic expertise they bring to the Board. This partnership aims to drive NDI-5001 through its initial clinical phase towards proof of concept, ultimately positioning the company for pivotal trials in pediatric patients.
Jeff Sands, MD, Chief Medical Officer of NephroDI, highlighted the company's collaboration with medical experts and the NDI Foundation. He expressed optimism about NDI-5001's potential to enhance the quality of life for NDI patients, especially children.
Mike Parnell, President of the NDI Foundation, reiterated the Foundation's commitment to supporting the entire NDI community, including patients, families, researchers, physicians, and healthcare providers. He expressed the Foundation's eagerness to assist NephroDI in developing new treatments for NDI.
NephroDI Therapeutics, headquartered in Seattle, Washington, focuses on developing and commercializing drugs to treat
kidney concentration disorders. Its lead program, NDI-5001, is a novel kidney-targeted small molecule AMPK activator being developed as a potential first-in-class treatment for Congenital Nephrogenic Diabetes Insipidus.
Sound Bioventures is a venture capital fund specializing in life sciences, investing in nearly clinical or clinical-stage private companies in Europe and the USA. The fund focuses on specialty therapeutics addressing significant unmet clinical needs, with an experienced team actively involved in the operational and investment process.
In conclusion, NephroDI Therapeutics' new funding from Sound Bioventures represents a significant step towards advancing NDI-5001 through clinical trials. This partnership underscores the urgent need for effective treatments for Congenital Nephrogenic Diabetes Insipidus and highlights the potential impact of NDI-5001 on improving patients' lives.
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