Progressive Supranuclear Palsy (PSP)

Progressive supranuclear palsy (PSP) is a progressive neurodegenerative disease that results in impaired balance, difficulty with eye movement, speech, and swallowing, rigid muscle tone and ultimately, cognitive impairment.  The condition, which typically strikes individuals between the ages of 60 and 70, is associated with deterioration and death of specific cells within brain responsible for balance/coordination, walking, speech, swallowing, eye movement and cognition.  PSP affects approximately five-to-six people out of every 100,000 and is commonly misdiagnosed as other neurodegenerative diseases such as Parkinson’s disease and Alzheimer’s disease.  There are currently no effective treatments for PSP, though some symptoms of the disease can be managed therapeutically.

Retrotope is currently developing RT001, its lead clinical candidate, for the treatment of PSP.  The company is planning to initiate a Phase 2 of RT001 in PSP during the mid-2021.  To date, RT011 has been administered to three PSP patients through an expanded access program, with all having received treatment for more than 27 months.  For all three of these patients, disease progression significantly slowed or reversed at 12 months and 24 months according to standard severity scales used in the measurement of the disease.

RT001 has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of PSP.

Additional patient and healthcare provider resources on PSP are available in the Patient Resources section.