Sanford Burnham Prebys announces start of Phase 2 clinical trial of DS-1211 in individuals with PseudoXanthoma Elasticum

A Phase 2 clinical trial has started of DS-1211 in individuals with Pseudoxanthoma Elasticum (PXE), a rare multisystem genetic disease that causes calcium deposits in soft tissue resulting in considerable morbidity. DS-1211 is a potential first-in-class small molecule developed through a research collaboration between Daiichi Sankyo and Sanford Burnham Prebys.

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“Calcification is an essential, tightly regulated process in skeleto-dental tissues,” says José Luis Millán, Ph.D., a professor at Sanford Burnham Prebys. “But when the process goes awry and occurs in ‘soft’ tissues—known as ectopic calcification—it can be extremely dangerous. There’s an unmet medical need for treatments to prevent ectopic calcification, and this drug could help meet that need.” 

PXE affects approximately one in 25,000 people. It causes tissues in the body to improperly accumulate minerals, which leads to changes in skin texture, problems with vision, and cardiovascular disease in the later stages. PXE is a progressive disease without a cure, which means that for most people, the disease only tends to get worse.

“The foundational science for DS-1211 comes from the pioneering research of Dr. Millan, whose work led to a molecular and cellular understanding of how physiological mineralization is controlled,” says Michael Jackson, Ph.D., senior vice president of drug discovery and development at the Institute’s Conrad Prebys Center for Chemical Genomics (Prebys Center).

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