US-based biotechnology firm BioMarin Pharmaceutical (BMRN) has dosed the first patient in the Phase I/II study for BMN 190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) designed to treat patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a neurodegenerative disorder.
The Phase I/II trial is an open-label, dose-escalation study and its primary objectives are to assess the safety and tolerability of BMN 190, as well as its effectiveness using an NCL-2-specific rating scale score compared to natural history data after 48 weeks of treatment.
The trial's secondary objectives are to assess the impact of treatment on brain atrophy compared with NCL-2 natural history after 48 weeks of treatment, as well as to characterise pharmacokinetics and immunogenicity.
Twenty two subjects will be enroled in the trial, which will be carried out at around ten clinical sites for the treatment duration of 48 weeks.
BioMarin executive vice-president and chief medical officer Hank Fuchs said: "This programme is representative of the company's core competency of developing life-altering enzyme replacement therapies for serious unmet medical needs.
"We are inspired and motivated by the patient and physician community and encouraged by the pharmacological activity demonstrated in preclinical models."
Neuronal ceroid lipofuscinoses, a form of Batten disease, is a type of neurodegenerative disorder that results from excessive accumulation of lipopigments (lipofuscin) in the body's tissues.
According to the company, this is the first instance where a patient with Batten disease has been treated with an enzyme replacement therapy in a clinical trial setting.
The company believes the clinical trial could help create a new viable option for people suffering with Batten disease, a neurodegenerative disease of childhood.
BioMarin develops and markets new biopharmaceuticals for serious diseases and medical conditions and its product portfolio includes four approved products and several clinical and pre-clinical product candidates.