KP2051

KP2051 is a small molecule drug candidate being developed by K Pharma, Inc. for the treatment of Nasu-Hakola Disease (NHD), also known as polycystic lipomembranous osteodysplasia with sclerosing leukoencephalopathy (PLOSL). NHD is a rare, fatal autosomal recessive disorder characterized by systemic bone cysts and early-onset progressive dementia, often associated with mutations in the TREM2 or TYROBP genes. KP2051 was identified through K Pharma's drug repositioning strategy, which utilizes iPSC-based disease modeling to screen existing compounds for new therapeutic applications. As of 2024, the program is in the preclinical stage, with the company focused on constructing evaluation systems for compound screening and efficacy validation.