Mighty Therapeutics has dosed the first patient in 4TAZPower, its global Phase IV confirmatory study assessing Forzinity (elamipretide) injection in those aged at least five years with Barth syndrome.
The double-blind, randomised, parallel-group, placebo-controlled study aims to further assess the efficacy, safety and pharmacokinetics of once-daily elamipretide in this population.
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Elamipretide previously received accelerated approval from the US Food and Drug Administration (FDA) for use in individuals with Barth syndrome weighing at least 30kg.
This approval was based on the knee extensor muscle strength improvement, an intermediate endpoint.
The 4TAZPower trial aims to provide additional data to confirm clinical benefit and better understand the long-term safety profile of the treatment.
Barth syndrome is described as a severe and progressive mitochondrial disorder, and according to Mighty Therapeutics, has no geographical boundaries.
Mighty Therapeutics CEO Reenie McCarthy said: “We are committed to supporting and potentially expanding access to elamipretide for individuals living with Barth syndrome in the US and, we hope, globally. Barth syndrome is a devastating progressive disease that knows no borders.
“Timely dosing of the first individual in our 4TAZPower study is a key step toward meeting our post-marketing commitments to the FDA. As importantly, this milestone underscores our commitment to broaden participation opportunities to the global patient community.
“Patients remain at the forefront of our mission, so we are celebrating this step toward what we hope will be global access to the only therapy currently approved for this ultra-rare, life-threatening mitochondrial disease.”
The 4TAZPower study will follow participants with genetically confirmed Barth syndrome over a period of 72 weeks to assess both efficacy and safety.
Mighty Therapeutics is further developing elamipretide for other conditions involving mitochondrial dysfunction, including polymerase gamma-related mitochondrial disease and dry age-related macular degeneration.
