NephroDI Therapeutics’ NDI-5001 is being developed to treat X-linked congenital nephrogenic diabetes insipidus, a rare genetic kidney condition. Credit: crystal light /

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.”

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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.

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