Core Viewpoint - Biogen Inc. has initiated the BRAVE study, a global Phase 3 clinical trial to evaluate the efficacy and safety of omaveloxolone in children with Friedreich ataxia (FA) aged 2 to under 16 years, addressing a significant unmet need in the pediatric population [1][2][3]. Group 1: Study Design and Objectives - The BRAVE study will involve approximately 255 children with FA, randomized in a 2:1 ratio to receive either omaveloxolone or placebo for 52 weeks, followed by an open-label extension phase [2][4]. - The primary outcome measure is the change from baseline in the Upright Stability Score (USS), recognized as a sensitive measure of disease progression in children with FA [2][3]. - The study design has been informed by previous research and input from the FA community, with enrollment starting in the U.S. and plans to expand globally [4]. Group 2: Current Product Information - Omaveloxolone, marketed as SKYCLARYS, is currently approved for the treatment of FA in adults and adolescents aged 16 years and older in over 40 countries, including the U.S. and the European Union [1][5]. - The drug has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. FDA, highlighting its significance in treating this rare condition [5]. Group 3: Disease Context - Friedreich ataxia is a rare, genetic, life-shortening neuromuscular disorder, with early symptoms typically appearing in childhood and leading to significant disability [7][8]. - Patients with early onset FA often experience a more aggressive disease progression, underscoring the critical need for effective treatments in the pediatric population [3][7].

Biogen (BIIB) reported first-quarter 2025 adjusted earnings per share (EPS) of $3.02, which missed the Zacks Consensus Estimate of $3.32.Earnings declined 18% year over year on a reported basis, owing to costs related to an upfront payment of $165 million made to Stoke Therapeutics (STOK) . BIIB entered into a collaboration agreement with STOK for the development and commercialization of the latter’s pipeline candidate, zorevunersen for treating Dravet syndrome in February. (Find the latest EPS estimates an ...

Biogen (BIIB) will report first-quarter 2025 results on May 1, before market open. In the last reported quarter, the company's earnings beat expectations by 0.58%. The Zacks Consensus Estimate for first-quarter sales and earnings is pegged at $2.24 billion and $3.34 per share, respectively. (Find the latest earnings estimates and surprises on Zacks Earnings Calendar.)Factors to Consider for BiogenIn the first quarter, lower sales of multiple sclerosis (“MS”) drugs are likely to have been offset by revenues ...