About ZY-001
(Disodium Etidronate)
A First-in-Class, Disease-Modifying
Therapy for Brain Calcifications
ZY-001 (disodium etidronate) is the first CNS-targeted, potentially disease-modifying therapy being developed for Sturge-Weber Syndrome, Tuberous Sclerosis Complex, and other rare disorders driven by intracranial calcifications.
A Novel Approach to “Brain Stones”
Intracranial calcifications, or “brain stones,” are calcium-phosphate deposits that disrupt normal neuronal signaling and blood flow. This disruption is a key driver of seizures, cognitive decline, and behavioral symptoms.
ZY-001 is designed to directly address this root cause by chelating (binding) excess calcium and promoting the gradual normalization of tissue mineral balance—something no approved CNS therapy does today.
It is a modern, modified-release formulation of disodium etidronate, a proven calcium-binding compound originally marketed as Didronel® for bone disorders.
A First-in-Class Opportunity
ZY-001 represents a first-in-class opportunity to transform care in rare epileptic and neurodegenerative diseases caused by pathological calcium buildup in the brain.
Why It Matters
A Critical Unmet Need
No approved disease-modifying therapy currently exists for SWS or related brain calcification disorders.
Targeting the Driver
More than 80% of SWS patients experience seizures linked to intracranial calcifications.
A New Mechanism
ZY-001 is designed to directly address the root cause of the disease rather than only managing its symptoms.