BBP-561

BBP-561 is a topical small molecule inhibitor of kallikrein 5 (KLK5) and kallikrein 7 (KLK7) being developed by BridgeBio Pharma for the treatment of Netherton syndrome. Netherton syndrome is a rare and severe autosomal recessive genetic skin disorder caused by mutations in the SPINK5 gene, which leads to a deficiency of the serine protease inhibitor LEKTI. This deficiency results in the hyperactivity of KLK5 and KLK7 proteases, which drive premature skin desquamation, chronic inflammation, and barrier dysfunction. BBP-561 is designed as a topical therapy to locally inhibit these proteases, aiming to restore the balance of protease activity in the skin and alleviate the symptoms of the disease.