Mighty Therapeutics, a commercial-stage biotechnology company pioneering a new class of medicines that directly target mitochondrial dysfunction in rare and age-related diseases, has dosed the first patient in its global phase 4 study to confirm the efficacy, safety and pharmacokinetics of once-daily elamipretide in individuals at least five years of age living with Barth syndrome.
Forzinity (elamipretide) injection received accelerated approval from the US Food and Drug Administration (FDA) for individuals with Barth syndrome weighing at least 30kg, based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. The largest clinical development effort in Barth syndrome to date, the 4TAZPower study, has been designed to further confirm the clinical benefit and expand the understanding of the long-term safety of elamipretide for individuals living with Barth syndrome.
The 4TAZPower study is a randomised, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once-daily subcutaneous injection of elamipretide in participants with genetically confirmed Barth syndrome for 72 weeks.
