"The initiation of another pivotal trial this year is a major milestone for PTC," said
The Phase 3 MOVE-FA trial is an 18-month parallel arm, placebo-controlled study evaluating vatiquinone versus placebo in approximately 110 children and young adults with FA. The primary endpoint is the change from baseline in the modified Friedreich ataxia rating scale (mFARS), with key secondary endpoints assessing ambulation and activities of daily living. This endpoint strategy was developed in consultation with both the FDA and
"FARA and the FA community are grateful to
About Vatiquinone (PTC743)
Vatiquinone is an investigational small molecule that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress and inflammation response pathways that underpin neurological disease pathology. Vatiquinone has been evaluated in a number of clinical studies in which it has been demonstrated to have an impact on mortality risk and a number of neurological and neuromuscular disease symptoms. In more than 500 patients with duration of exposure up to 10 years, vatiquinone has demonstrated a favorable safety profile.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.
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1 National Organization for Rare Disorders (NORD) (2020). Friedreich's Ataxia. https://rarediseases.org/rare-diseases/friedreichs-ataxia/. Accessed