Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — EPNS 2025
10 slides from Biomarin Pharmaceutical's conference presentation, Dec 10, 2025
Slide 1: Seizures and Movement Disorders in Patients With CLN2 Disease Treated With Cerliponase Alfa
Seizures and Movement Disorders in Patients With CLN2 Disease Treated With Cerliponase Alfa in the Real-World Setting Angela Schulz1, Miriam Nickel1, Christoph Schwering1, Eva Wibbeler1, Lena Marie Westermann1, Luca Hagenah1, Sheila Reddy2, Abigail Hunt2, Matthias Hunger3, Olivia Okoli4, Pascal Reis
Slide 2: Conflicts of interest disclosures
Conflicts of interest disclosures • Angela Schulz has received consulting fees from Neurogene Inc. and Regenxbio Inc. She received honoraria for speaker fees, research grants, and travel support from and was a Principal Investigator in BMN 190 clinical trials for BioMarin Pharmaceutical Inc. 2
Slide 3: Background
Background • CLN2 disease is a rare neurodegenerative disorder caused by deficient TPP1 enzyme activity that typically presents with language delay and/or onset of seizures between 2 and 4 years of age • Onset of symptoms is followed by rapid, progressive decline in motor and language function, wors
Slide 4: Methods
Methods Inclusion criteria • Patients enrolled in the real-world, observational DEM-CHILD database (international NCL patient registry; NCT04613089) • Diagnosis of CLN2 disease confirmed by genetic and enzyme testing • Initiated treatment with cerliponase alfa outside a clinical trial setting • Had
Slide 5: Baseline Characteristics
Baseline Characteristics Baseline characteristic Patients (N=24) Baseline characteristic Patients (N=24) Female, n (%) 14 (58.3) Neurological symptoms, n (%)a Age (months), mean (SD) Seizures 20 (83.3) At diagnosis 53.1 (25.6) Ataxia 18 (75.0) At genetic analysis 55.3 (26.6) Dystonia 5 (20.8) At cer
Slide 6: Frequency of Seizures Over Follow-Up (1/2)
Frequency of Seizures Over Follow-Up (1/2) Primary Generalised Seizures Atonic Seizures Number of seizures in prior 3 months: >5 3–5 1–2 0 Number of seizures in prior 3 months: >5 3–5 1–2 0 100 100 90 90 80 80 70 70 % of patients % of patients 60 60 50 50 40 40 30 30 20 20 10 10 0 0 Baseline 6 12 18
Slide 7: Frequency of Seizures Over Follow-Up (2/2)
Frequency of Seizures Over Follow-Up (2/2) Myoclonic Seizures Anti-Seizure Medication Use (dose increase/new drug addition) Number of seizures in prior 3 months: >5 3–5 1–2 0 100 100 90 90 % of patients with change in 80 80 70 70 prior 3 months % of patients 60 60 50 50 40 40 30 30 20 20 10 10 0 0 B
Slide 8: Incidence and Time to Onset of Myoclonus and Dystonia
Incidence and Time to Onset of Myoclonus and Dystonia Myoclonus Dystonia Proportion of patients Proportion of patients without event without event Number at risk Number at risk Time from treatment initiation (months) Time from treatment initiation (months) Time to first onset of myoclonus N=21 Time
Slide 9: Conclusions
Conclusions This study provides a comprehensive description of the time course of seizures and movement disorders in patients with CLN2 disease receiving cerliponase alfa in a real-world setting Findings support observations from clinical trials suggesting that cerliponase alfa may have benefit in m
Slide 10: Acknowledgments
Acknowledgments This study was funded by BioMarin Pharmaceutical Inc. Medical writing support was funded by BioMarin and provided by Carl Davies, MSc, of Adelphi Communications Ltd 10