UCB announces positive phase III results for rozanolixizumab in generalized myasthenia gravis.

The trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful change from baseline in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at Day 43. All secondary endpoints were also met with statistical significance. Overall rozanolixizumab was well tolerated and no new safety signals were identified.

The safety and efficacy of rozanolixizumab have not been established, and it is not approved for use in any indication by any regulatory authority worldwide. The final Phase III data from the study and additional details will be presented at a forthcoming medical meeting in 2022. Based on these results, UCB anticipates regulatory filings in the U.S., European Union and Japan from Q3 2022.

Alongside rozanolixizumab, UCB is also investigating whether its developmental medicine zilucoplan, a peptide inhibitor of complement component 5 (C5 inhibitor), could deliver patient value to people living with gMG. Preliminary results from the company’s RAISE study are expected in the coming weeks. The safety and efficacy of zilucoplan have not been established, and it is not approved for use in any indication by any regulatory authority worldwide.

About the rozanolixizumab MycarinG study ; The MycarinG study (NCT03971422) is a multi-center, Phase III, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of rozanolixizumab in adult patients with gMG, with an open-label extension. The primary endpoint for the MycarinG study is change in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) score, an eight-item patient-reported scale developed to assess MG symptoms and their effects on daily activities. Secondary endpoints include response rates, changes in the Myasthenia Gravis composite (MGC) score, the Quantitative MG (QMG) score, patient-reported outcomes and adverse events (AEs).