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Find Clinical Drug Pipeline Developments & Deals by Retrotope
At 24 weeks, data demonstrated that patients treated with RT001 (linoleic Acid) experienced less worsening (a 3.3-point reduction from baseline) in ALSFRS-R score as compared to greater worsening (4.6-point reduction from baseline) for the placebo group.
Lead Product(s):
Di-deuterated Linoleic Acid Ester
RT001 to impact the progression of INAD based on the compound’s demonstrated ability to down-regulate lipid peroxidation used in patients with infantile neuroaxonal dystrophy.
Comapny's first D-PUFA drug candidate in development, RT001, has shown robust safety in over 50 patient years of oral dosing, and we expect RT011 to have a similar safety profile.
Retrotope filed this IND for a PSP Phase 2/3 trial after 3 patients treated with RT001 in PSP Expanded Access protocols showed encouraging results after more than a year on drug.
The company stated that, RT001 has a synergistic downstream benefit in the pathophysiology of PSP. This orphan designation encourages clinical trials and rapid review of data from trials to treat PSP.
With this trial, Retrotope expands its pipeline of indications for RT001 which is currently being studied in a pivotal trial in Infantile Neuroaxonal Dystrophy, which is fully enrolled.