Our lead development candidate, RT001, is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) that is in development for a range of orphan neurodegenerative diseases, including infantile neuroaxonal dystrophy (INAD), Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and progressive supranuclear palsy (PSP). To date, RT001 has been safely administered orally on a daily basis to more than 100 patients, spanning more than 1,000 patient months.

The compound is currently being evaluated in clinical trials across several neurodegenerative diseases with multiple data readouts expected by the end of 2021. These include a potentially pivotal Phase 2/3 trial in INAD, a pivotal Phase 2/3 study in FA and a Phase 2 trial in ALS.

RT001 has received a variety of key regulatory designations from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In the U.S., these include rare pediatric disease designation for INAD and FA, Fast Track designation for FA, and orphan drug designation for FA, PSP, and PLA2G6-associated neurodegeneration, which includes INAD. Additionally, in Europe, RT001 has received orphan drug designation for the treatment of INAD.

Our pipeline also includes RT011, an isotopically stabilized, synthetic docosahexaenoic acid (DHA). DHA is the dominant PUFA in human retinal phospholipids and its degradation by the LPO process plays a key role in retinal cell degeneration. As such, RT011 represents a potentially interesting therapeutic candidate for addressing degenerative retinal diseases such as dry age-related macular degeneration (AMD). Retrotope is currently advancing RT011 toward the clinic for the treatment of dry AMD with an investigation new drug (IND) application expected to be filed by the end of 2021.