UCB Announces Positive Phase 3 Results for Rozanolixizumab in Generalized Myasthenia Gravis

UCB announced positive topline results from the Phase 3 MycarinG study1 evaluating rozanolixizumab, a subcutaneously (SC) infused monoclonal antibody targeting the neonatal Fc receptor (FcRn), versus placebo in adults with generalized myasthenia gravis (gMG).

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 3 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. 

"Today's encouraging findings from the MycarinG study show the potential of rozanolixizumab in the treatment of myasthenia gravis, and further reinforce the suggestion that FcRn inhibition may be a promising approach for this disease," explained Professor Vera Bril, MycarinG study Lead Investigator, Professor of Medicine (Neurology), University of Toronto, and Director of the Neuromuscular Section, Division of Neurology, University of Toronto and University Health Network, Toronto, Canada.

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.

"For the many thousands of people living with myasthenia gravis around the world, current treatment options can be very limited," said Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America (MGFA). "Given that this disease causes a wide range of symptoms, some of which can require urgent intervention or hospitalization, there is a critical need for new treatment options that could address the unmet needs of patients living with myasthenia gravis." 

UCB's portfolio approach of two medicines with different but potentially complimentary mechanisms of action creates a unique opportunity for UCB to deliver choice, flexibility and impact to patients and healthcare professionals, giving them options best suited to their individual needs.