Biogen has commenced dosing in the Phase III BRAVE study to assess omaveloxolone in children of two to less than 16 years of age with Friedreich ataxia (FA).
The trial aims to assess the safety, pharmacodynamics, efficacy, and pharmacokinetics of the therapy in nearly 255 subjects, including non-ambulatory and ambulatory.
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Part one is a double-blind, 52-week, randomised, placebo-controlled trial, with a primary outcome measure of changes from baseline in Upright Stability Score (USS). The USS is a critical subscale of the modified FA rating scale (mFARS), recognised by the FA community for its sensitivity in measuring disease progression in children.
Subjects in the trial will be randomised in a 2:1 ratio and given either the therapy or a placebo once daily.
Following the initial 52-week period, they will have the opportunity to enter an open-label extension (OLE), which forms part two, for up to 104 weeks, where all subjects will be given the therapy to assess its long-term effects.
According to the company, the design of the study has been informed by insights from prior studies and consultations with investigators, the FA community, and medical experts.
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By GlobalDataEnrolment has started in the US, with plans to open additional trial sites worldwide, pending discussions with local regulators and ethics committees.
Biogen neuromuscular development unit head Stephanie Fradette said: “Building on the work of Reata, we have been urgently advancing the paediatric development plan for omaveloxolone and are thrilled that the Phase III BRAVE study has now begun.
“We are immensely grateful for the input from the entire FA community that has helped shape the design of this important study.”
Marketed as Skyclarys, omaveloxolone is approved for the FA treatment in adults and adolescents aged 16 and older in over 40 nations, including the European Union and the US.
It has secured fast track, rare paediatric disease, and orphan drug designations from the US Food and Drug Administration (FDA), as well as orphan drug status by the European Commission.
In March 2025, Biogen began dosing in the Phase III TRANSCEND trial of felzartamab in adult kidney transplant recipients with late antibody-mediated rejection (AMR).
