UCB presents latest research advancements at European Pediatric Neurology Society Congress

• 15 scientific abstracts, including three oral presentations demonstrate UCB’s continued commitment to leading research aimed at improving treatment outcomes for people living with neurological conditions.
• Data include the use of FINTEPLA^®▼ (fenfluramine)¹ in Dravet syndrome and Lennox-Gastaut syndrome, and BRIVIACT^® (brivaracetam)² in focal-onset seizures.  
• There is also a focus on investigational therapy with pyrimidine nucleos(t)ide therapy and the current care landscape in people living with thymidine kinase 2 deficiency, an ultra-rare, life-threatening, genetic mitochondrial disease with no currently approved treatment options.^3,4,5,6,7,8

Brussels, Belgium –July 8, 2025 – 7:00AM CET– UCB, a global biopharmaceutical company, today announced it will present 15 abstracts from its targeted neurology portfolio at the European Pediatric Neurology Society (EPNS) Congress, Munich, Germany, July 8-12, 2025. The data will focus on thymidine kinase 2 deficiency (TK2D) and epilepsies, including rare developmental and epileptic encephalopathies (DEEs) such as Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS).

Donatello Crocetta, Chief Medical Officer and Head of Global Medical Affairs, UCB, commented: “At UCB we are driven by our commitment to people living with severe diseases and where there is a high unmet need. Together, with a shared passion and purpose, we aim to redefine what is possible in the future of neurology - ensuring that no one facing a severe neurological condition is left behind. We look forward to the discussions at the congress and sharing our latest epilepsy and TK2d data with the medical community.”

Highlights of data to be presented at EPNS:

• Developmental and Epileptic Encephalopathies (DEEs): 
• DS: include evaluations of preliminary real-world data on non-seizure benefits of fenfluramine treatment in pediatric and adult patients,^9,10  and antiseizure medication regimen adjustments after fenfluramine initiation in adult and pediatric patients who participated in the European Early Access Program.¹¹ 
• LGS: final safety and effectiveness of fenfluramine data from the open-label extension (OLE) study,¹² as well as two post-hoc analyses looking at the onset and duration of aerse events following fenfluramine treatment initiation,¹³ and the efficacy trajectory¹⁴ of fenfluramine based on data from patients who transitioned from placebo during randomized controlled trials to fenfluramine in the OLE study.
• DS and LGS: data include pharmacokinetics and clinical outcomes of fenfluramine in combination with cannabidiol based on results from a Phase 1 study.¹⁵ Data from a retrospective claims analysis that describes persistence of fenfluramine (FFA) and cannabidiol (CBD) in LGS and DS patients in Germany and their use based on age, gender, prior anti-seizure medication use, and dosage.¹⁶
• Epilepsy:
• Focal-onset seizures with/without focal to bilateral tonic-clonic seizures: real-world prospective, non-interventional study evaluated 12-month effectiveness and tolerability of adjunctive brivaracetam (BRV) pediatric and adult patients with focal-onset seizures with no previous BRV treatment experience across mid-European countries, focusing on retention rates, responder rates, and drug-related treatment-emergent adverse events of patients in routine clinical practice.¹⁷ 
• TK2D: 
• Disease course: findings from the largest international TK2d dataset evaluating the disease course in untreated people living with TK2d, who were aged ≤ 12 years at TK2d symptom onset.^18,19
• Survival analysis and functional outcomes: data demonstrates improvement in survival and functional outcomes with respect to regain of lost developmental motor milestones and decrease need of ventilatory support associated with treatment with doxecitine and doxribtimine* - an investigational pyrimidine nucleos(t)ide therapy - in patients with an age of TK2d symptom onset ≤12 years.^20,21
• Genomics: The Genomics England National Genomic Research Library (NGRL) and UK Biobank were used to investigate the genetic, phenotypic and clinical landscape of TK2d.²²
• Quality of life: An analysis of a TK2d patient perspectives study captured patients’ experiences of TK2d and highlights the debilitating physical impacts and severe psychological strain associated with living with TK2d.²³

Symposium
EPNS will feature one UCB-supported symposium:

• Title: The Clinician and Family Unified Efforts: Joint Approach on Optimising Outcomes in Lennox-Gastaut Syndrome 
• Date: Wednesday, 9th July 2025, 13:00–14:00 (CEST)
• Overview: The upcoming symposium will spotlight the latest advances and ongoing challenges in managing LGS, with experts sharing practical insights on improving diagnosis, optimizing treatment strategies, and enhancing daily living for patients and their families.

UCB presentations during the European Pediatric Neurology Society (EPNS) Congress Annual Meeting

*Doxecitine and doxribtimine is currently under regulatory review by US and EU regulatory authorities. The safety and efficacy have not been established, and doxecitine and doxribtimine has not been approved by the US Food and Drug Administration (FDA) nor the European Medicines Agency (EMA) and by any regulatory authority worldwide.