Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 190 · 2 trials · 5 indications
Motor and Language are each 0-3 point subscales in which 3 represents best function and 0 represents loss of function. Thus, the 0-6 point ML score was used as the primary mode of evaluation of loss of function. In 190-201, the primary endpoint was a responder endpoint: unreversed ML 2-point decline or score of 0 at Week 48 compared to the 50% rate expected in natural history using the 1-sample binomial test. For 190-202, the primary endpoint was revised to assess response over the full duration of follow-up. It is not clear, given the variable follow-up much greater than 48 weeks, what response rate is expected in natural history. For this reason, the assessment of the primary endpoint is based on comparing to natural history data and using the Kaplan-Meier method and Cox proportional hazards model with covariate adjustment (i.e., baseline ML score and age as continuous covariates, and genotype \[common alleles\] and sex as categorical covariates).
Motor and Language are each 0-3 point subscales in which 3 represents best function and 0 represents loss of function. Thus, the 0-6 point ML score was used as the primary mode of evaluation of loss of function. In 190-201, the primary endpoint was a responder endpoint: unreversed ML 2-point decline or score of 0 at Week 48 compared to the 50% rate expected in natural history using the 1-sample binomial test. For 190-202, the primary endpoint was revised to assess response over the full duration of follow-up. It is not clear, given the variable follow-up much greater than 48 weeks, what response rate is expected in natural history. For this reason, the assessment of the primary endpoint is based on comparing to natural history data and using the Kaplan-Meier method and Cox proportional hazards model with covariate adjustment (baseline ML score, age, genotype \[common alleles\], and sex).
The progression of ceroid lipofuscinosis (CLN2) disease was assessed using adapted motor and language domains of the Hamburg rating scale (ML scale score). Motor and Language are each 0 - 3 point subscales in which 3 represents best function and 0 represents loss of function. The sum of the motor and language scores (ML score, 0-6 points) was used to evaluate the loss of function.
| Arm | Type | Description |
|---|---|---|
| BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1) | EXPERIMENTAL | All 190-202 study subjects administered BMN 190 300 mg by continuous Intracerebroventricular (ICV) infusion at a rate of 2.5 mL/hour for approximately 4 hours) every 14 days. |
| BMN190 | EXPERIMENTAL | recombinant human tripeptidyl peptidase-1 (rhTPP1/cerliponase alfa) |
| Name | Type | Description |
|---|---|---|
| BMN 190 | BIOLOGICAL | 300 mg Intracerebroventricular (ICV) infusion administered every other week for up to 240 weeks |
| Intracerebroventricular (ICV) access device | DEVICE | Surgical implantation of an MRI compatible ICV access device in the lateral ventricle of the right hemisphere is required for administration of study drug. |
Inclusion Criteria: * Must have completed 48 weeks in Study 190-201. * Is willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written info...
BMN 190 is an investigational monoclonal antibody being developed for Jansky-Bielschowsky Disease, also known as Batten Disease or Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2 Disease). It is administered intracerebroventricularly and is currently in Phase 1 clinical development.
BMN 190 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is conducting clinical trials for this investigational therapy in patients with CLN2 Disease.
BMN 190 is in Phase 1 clinical development. Two Phase 1 trials have been completed, including a dose-escalation study and a long-term extension study. The drug is investigational and has not been approved by regulatory authorities.
BMN 190 has been studied in two completed Phase 1 trials. NCT01907087 was a Phase 1/2 open-label dose-escalation study evaluating safety, tolerability, pharmacokinetics, and efficacy in patients with CLN2 Disease. NCT02485899 was an extension study assessing long-term efficacy and safety.
BMN 190 is the investigational name for the drug also known as cerliponase alfa. It is a monoclonal antibody developed by BioMarin Pharmaceutical Inc. for the treatment of CLN2 Disease, a form of Batten disease.