Company Overview

Retrotope is a clinical-stage biopharmaceutical company focused on the development of first-in-class therapies for degenerative diseases ranging from orphan neurodegenerative indications to large market degenerative conditions.

Our scientific team possesses industry-leading knowledge of the natural lipid peroxidation (LPO) process and the role that LPO plays in triggering disease . Based on this unique expertise, we have created a proprietary drug discovery platform that utilizes an elegant chemistry-based solution to create novel, disease-modifying drugs designed to combat the oxidative stress and cellular degeneration that arise from LPO. These first-in-class new chemical entities (NCEs) are isotopically stabilized synthetic versions of polyunsaturated fatty acids (PUFAs) capable of downregulating the LPO process.

Our lead development candidate, RT001, is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) that is in development for 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 the equivalent of more than 1000 patient exposure months.

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) that is currently advancing toward the clinic for the treatment of dry age-related macular degeneration (AMD), including geographic atrophy.