SAN RAFAEL, CA, USA I April 1, 2013 I BioMarin Pharmaceutical Inc. (BMRN) announced today that it has submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN-190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease. The company expects to start enrolling patients in a Phase 1/2 trial in mid-2013.
“The Batten disease program embodies our core mission at BioMarin — to develop life-altering therapies for ultra-orphan diseases with a significant and serious unmet medical need,” said Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer of BioMarin. “We hope to leverage our expertise in enzyme replacement therapy development to advance this program and bring a therapeutic option to patients suffering from NCL-2. We have demonstrated encouraging pharmacological activity in preclinical models and hope to see this benefit translated into human subjects.”
The Phase 1/2 study is an open-label, dose-escalation study in patients with NCL-2. The primary objectives are to evaluate the safety and tolerability of BMN-190 and to evaluate effectiveness using an NCL-2-specific rating scale score in comparison with natural history data. Secondary objectives are to evaluate the impact of treatment on brain atrophy in comparison with NCL-2 natural history, and to characterize the pharmacokinetics and immunogenicity. The study will enroll approximately 22 subjects worldwide for a treatment duration of 48 weeks.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse(TM) (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include BMN-110 (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia, and BMN-190, a recombinant human tripeptidyl peptidase 1 (rhTPP1), which is currently in Phase 1/2 development for the treatment of neuronal ceroid lipofuscinosis Type 2 disease. For additional information, please visit www.BMRN.com. Information on BioMarin’s website is not incorporated by reference into this press release.
SOURCE: BioMarin Pharmaceutical
Post Views: 41
SAN RAFAEL, CA, USA I April 1, 2013 I BioMarin Pharmaceutical Inc. (BMRN) announced today that it has submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN-190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease. The company expects to start enrolling patients in a Phase 1/2 trial in mid-2013.
“The Batten disease program embodies our core mission at BioMarin — to develop life-altering therapies for ultra-orphan diseases with a significant and serious unmet medical need,” said Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer of BioMarin. “We hope to leverage our expertise in enzyme replacement therapy development to advance this program and bring a therapeutic option to patients suffering from NCL-2. We have demonstrated encouraging pharmacological activity in preclinical models and hope to see this benefit translated into human subjects.”
The Phase 1/2 study is an open-label, dose-escalation study in patients with NCL-2. The primary objectives are to evaluate the safety and tolerability of BMN-190 and to evaluate effectiveness using an NCL-2-specific rating scale score in comparison with natural history data. Secondary objectives are to evaluate the impact of treatment on brain atrophy in comparison with NCL-2 natural history, and to characterize the pharmacokinetics and immunogenicity. The study will enroll approximately 22 subjects worldwide for a treatment duration of 48 weeks.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse(TM) (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include BMN-110 (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia, and BMN-190, a recombinant human tripeptidyl peptidase 1 (rhTPP1), which is currently in Phase 1/2 development for the treatment of neuronal ceroid lipofuscinosis Type 2 disease. For additional information, please visit www.BMRN.com. Information on BioMarin’s website is not incorporated by reference into this press release.
SOURCE: BioMarin Pharmaceutical
Post Views: 41
