Slide 1: Improved Developmental Outcomes with Early Initiation of Cerliponase Alfa Treatment in Children with CLN2 Disease
Improved Developmental Outcomes with Early Initiation of Cerliponase Alfa Treatment in Children with CLN2 Disease Angela Schulz1,2, Emily de los Reyes3, Paul Gissen4, Nicola Specchio5, Kathleen Delaney6, Jessica Cohen Pfeffer6, Pascal Reisewitz6 1University Medical Center Hamburg-Eppendorf, Hamburg,
Slide 2: Conflicts of Interest Disclosures
Conflicts of Interest Disclosures • Angela Schulz has received consulting fees from Regenxbio Inc. and Neurogene 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 disease caused by deficient TPP1 enzyme activity1–3 • Affected children are functionally normal until seizures and delayed language acquisition emerge at 2 to 4 years of age, followed by rapid, progressive decline in motor, cognitive, language, a
Slide 4: Methods
Methods 190-203 study design • Open-label, multicenter, international trial of cerliponase alfa for approximately 3 years (144 weeks) • Cerliponase alfa dose was age-adjusted for children <2 years Key inclusion criteria Key exclusion criteria • Diagnosis of CLN2 disease as determined • Other inherit
Slide 5: 190-203 Study: Baseline Characteristics and Disposition
190-203 Study: Baseline Characteristics and Disposition N=14 Treated 14 (100) Completed treatment 13 (93) Disposition, n (%) Completed study 13 (93) Discontinued studya 1 (7) Male 6 (43) Sex, n (%) Female 8 (57) Race, n (%) White 14 (100) Hispanic or Latino 2 (14) Ethnicity, n (%) Not Hispanic or La
Slide 6: 190-203: Primary Efficacy Endpoint and Safety Data
190-203: Primary Efficacy Endpoint and Safety Data Rate of decline in motor-language score Safety Rate of decline in Baseline age Natural history Cerliponase alfa- motor-language Overall controls treated patients n (%) <2 years <3 years ≥3 years score, N=14 (n=29) (n=12) (n=5) (n=9) (n=6) points per
Slide 7: Time to unreversed 2-point decline or score of 0 in motor-language score
Time to unreversed 2-point decline or score of 0 in motor-language score Treated patients <3 years of age at baseline did not experience an unreversed 2-point decline or score of 0 in motor-language score 7 Schulz A, et al. Poster presented at the ILAE International Epilepsy Congress 2023
Slide 8: Denver II Development Scale: Baseline Age-Equivalent Scores
Denver II Development Scale: Baseline Age-Equivalent Scores Baseline Denver II Developmental Scale age-equivalent <2 years ≥2 years scores, months, mean (SD) (n=5) (n=9) Fine motor adaptive 22.2 (6.6) 25.2 (12.3) Gross motor 31.6 (9.8) 33.8 (8.8) Language 25.6 (9.7) 31.3 (10.7) Personal social 30.2
Slide 9: Individual Participant Outcomes for Denver Developmental Performance (1/2)
Individual Participant Outcomes for Denver Developmental Performance (1/2) Fine Motor Adaptive Gross Motor 9 Diagonal line shows intersection between age-equivalent performance and age at assessment
Slide 10: Individual Participant Outcomes for Denver Developmental Performance (2/2)
Individual Participant Outcomes for Denver Developmental Performance (2/2) Language Personal Social 10 Diagonal line shows intersection between age-equivalent performance and age at assessment
Slide 11: Change from baseline in Denver II Developmental Scale Age-Equivalent Scores
Change from baseline in Denver II Developmental Scale Age-Equivalent Scores Denver II Developmental Scale age-equivalent scores, months, mean (SD) <2 years (n=5) ≥2 years (n=9) Baseline Change from baseline Baseline Change from baseline Fine motor adaptive 22.2 (6.6) +41.0 (16.7) 25.2 (12.3) 0.0 (6.
Slide 12: Conclusions
Conclusions These results suggest age-appropriate attainment of developmental milestones in participants <2 years of age at baseline treated with cerliponase alfa Early initiation of cerliponase alfa treatment, prior to typical age of symptom onset, may result in improved developmental outcomes in c
Slide 13: Acknowledgments
Acknowledgments This study was funded by BioMarin Pharmaceutical Inc. Medical writing support was funded by BioMarin Pharmaceutical Inc. and provided by Samuel Devereux, MPharm, of Adelphi Communications Ltd 13