Retrotope, based in Los Altos, Calif., is developing a new category of patented, chemically stabilized compounds—D-PUFAs—aimed at preventing lipid peroxidation in mitochondrial and cellular membranes. Its lead compound, RT001, is an orally available D-PUFA designed to incorporate into membranes and protect against disease-induced excess lipid peroxidation. RT001 is being tested in clinical trials for Friedreich’s ataxia and Infantile Neuroaxonal Dystrophy, with expanded access studies underway in ALS, PSP Parkinsonism, GM1, late-onset Tay‑Sachs, Huntington’s disease and other disorders. The company has completed a blinded, randomized Phase 1/2 in Friedreich’s ataxia with post hoc statistical signals and has an ongoing pivotal trial in Infantile Neuroaxonal Dystrophy; a pivotal Friedreich’s ataxia trial was expected to start in Q3 2019. Retrotope reports over 250 patient‑months of drug safety exposure across clinical trial subjects and expanded access patients. The company is positioning its D-PUFA platform as a potential disease‑modifying approach for multiple neurodegenerative and retinopathy indications where lipid peroxidation is implicated.

Total raised

$20M

Funding rounds

1

Latest round

Series C

Latest activity

Jul 2019