An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Summary

The Phase 1/2 study (190-201) evaluated the efficacy and safety of a 300 mg dose of BMN 190 administered every other week (qow) to patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study. The rationale for this phase 2 extension study is to provide patients who complete the 190-201 study with the option to continue BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.

Timeline

Start

2015-02

Primary completion

2020-12-10

Completion

2020-12-10

Outcome

Met primary endpoint

registry analysis (other test); BMN 190-202 (300 mg) vs Natural History Comparator; p <0.0001; Hazard Ratio (HR) 0.14 (95% CI 0.06 to 0.33); Cox Proportional Hazards model reads as met primary endpoint NCT02485899 ↗

registry analysis (other test); BMN 190-201/202 (300 mg) vs Natural History Comparator; p <0.0001; Hazard Ratio (HR) 0.01; Cox Proportional Hazards model reads as met primary endpoint NCT02485899 ↗

release “An interim analysis from an ongoing extension study suggests durability of therapeutic effect.” reads as unknown biomarin.com ↗

release “an ongoing open-label extension study treating patients with Brineura ® (cerliponase alfa) continued to show a reduced rate of decline compared to a natural history cohort” reads as met primary endpoint biomarin.com ↗

release “durability of treatment effect in the primary efficacy endpoint where response to treatment was seen in 19 of 23 or 83% of treated patients” reads as met primary endpoint biomarin.com ↗

Publications

• Specchio N, Gissen P, de Los Reyes E, Olaye A, Camp C, Curteis T, Griffiths A, Butt T, Cohen-Pfeffer J, Slasor P, Sisic Z, Jain M, Schulz A. Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data. PLoS One. 2024 May 22;19(5):e0302382. doi: 10.1371/journal.pone.0302382. eCollection 2024.
• Schulz A, Specchio N, de Los Reyes E, Gissen P, Nickel M, Trivisano M, Aylward SC, Chakrapani A, Schwering C, Wibbeler E, Westermann LM, Ballon DJ, Dyke JP, Cherukuri A, Bondade S, Slasor P, Cohen Pfeffer J. Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study. Lancet Neurol. 2024 Jan;23(1):60-70. doi: 10.1016/S1474-4422(23)00384-8.
• Gissen P, Specchio N, Olaye A, Jain M, Butt T, Ghosh W, Ruban-Fell B, Griffiths A, Camp C, Sisic Z, Schwering C, Wibbeler E, Trivisano M, Lee L, Nickel M, Mortensen A, Schulz A. Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2). Orphanet J Rare Dis. 2021 May 12;16(1):217. doi: 10.1186/s13023-021-01829-x.
• Schulz A, Ajayi T, Specchio N, de Los Reyes E, Gissen P, Ballon D, Dyke JP, Cahan H, Slasor P, Jacoby D, Kohlschutter A; CLN2 Study Group. Study of Intraventricular Cerliponase Alfa for CLN2 Disease. N Engl J Med. 2018 May 17;378(20):1898-1907. doi: 10.1056/NEJMoa1712649. Epub 2018 Apr 24.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cerliponase Alfa	Protein / enzyme biologic	300 mg	Intrathecal

Indications