UCB Showcases Groundbreaking Neurology Research at AAN 2025 Meeting

UCB, a global leader in biopharmaceuticals, is excited to present 24 pioneering abstracts from its expansive neurology portfolio at the upcoming American Academy of Neurology (AAN) meeting, scheduled for April 5-9, 2025, in San Diego, California. This significant occasion will spotlight UCB's unwavering focus on enhancing the treatment and care for individuals grappling with severe neurological and neuromuscular disorders, particularly focusing on rare conditions like Dravet syndrome and Lennox-Gastaut syndrome, as well as generalized myasthenia gravis (gMG) and thymidine kinase 2 deficiency (TK2d).

The research highlights include extensive analyses that delve into the long-term efficacy and safety of existing therapies for rare epilepsies, with a special emphasis on fenfluramine for treating Lennox-Gastaut syndrome (LGS). Notably, the final safety and effectiveness data from a significant open-label extension study involving 247 participants will be unveiled, offering a comprehensive overview of how patients respond over time. In addition to that, intriguing insights derived from a Phase 1 study assessing the safety and pharmacokinetics of fenfluramine combined with cannabidiol in individuals with dual diagnoses of Dravet syndrome (DS) or LGS will also be presented.

UCB's commitment extends beyond epilepsy treatment. Their presentations encompass critical advancements related to generalized myasthenia gravis, including notable findings from studies on two novel therapies: RYSTIGGO® (rozanolixizumab-noli) and ZILBRYSQ® (zilucoplan). A significant portion of the research includes an open-label extension study focusing on the self-administration of rozanolixizumab, showcasing the safety and efficacy of a more patient-centered approach to treatment. Furthermore, data from a Phase 3 study assessing zilucoplan's impact on specific outcome scores and patient satisfaction will shed light on this treatment option's effectiveness.

A Deep Dive into Rare Epilepsies

UCB's focus on rare epilepsies is underscored by data on multiple existing treatments and investigational therapies. Research indicates that approximately 1%-3% of patients with Dravet syndrome experience prolonged seizures, emphasizing the need for targeted therapies that cater to their specific challenges. Current studies highlighting the association between sleep apnea and increased mortality risk in children with severe forms of epilepsy will also gain attention, aiming to raise awareness and provide actionable insights for clinical practice.

The Journey of Generalized Myasthenia Gravis

UCB's submissions further aim to enlighten healthcare professionals regarding the evolving landscape of gMG diagnostics and therapeutic options. With a focus on patient outcomes, new international registry initiatives aim to connect clinical data with patient-reported outcomes, thereby enriching the understanding of living with gMG. Such advancements are crucial for designing future treatment protocols tailored towards optimizing individual patient management.

Commitment to Innovation and Education

Emphasizing its commitment to advancing clinical education, UCB will also host the inaugural Rare Disease Connect in Neurology Annual Summit at the AAN event. This exclusive forum will bring together healthcare providers to discuss the pressing needs of the gMG community, allowing for tailored education and conversations centered around treatment innovations. Moreover, UCB will sponsor key symposia focusing on the critical role of early identification and innovative management strategies, particularly in relation to developmental and epileptic encephalopathies.

UCB remains dedicated to filling gaps in healthcare delivery for those affected by severe neurological disorders. Their pursuit of meaningful changes in treatment landscapes exemplifies a proactive approach to empowering patients. Each presentation at AAN not only demonstrates UCB’s commitment to scientific advancement but also shines a light on the urgent and ongoing needs within the neurological community.