Retrotope Announces First Dosing of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD)
Retrotope announced today that it has begun enrollment of the first patients and has started dosing of infants with the ultra-rare neurodegenerative disease, Infantile Neuroaxonal Dystrophy (INAD). The U.S. Food and Drug Administration (FDA) has granted the company approval to conduct this trial as an open-label Phase 2/3 clinical trial in 15-20 patients. Due to the paucity of publicly available natural history of the progression of the disease, Retrotope is currently also enrolling a prospective natural history study in a matched group of INAD patients who are not participating in the trial. RT001 is the first-in-class of a new category of drugs called D-PUFAs (deuterated polyunsaturated fatty acids), which are designed to protect against free radical damage resulting in cell death that is a hallmark of numerous neurodegenerative diseases including INAD.