NOVATO, Calif. , Sept. 26, 2012 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the completion of a Phase 1 study for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia. The company expects to initiate a Phase 2 trial in mid-2013.
The Phase 1 study was a two-part, double-blind, placebo-controlled study in healthy adult males. Part 1 examined a series of single subcutaneous doses and Part 2 included ten days of either fixed dosing or dose escalation. The primary objective of the study was to evaluate safety, tolerability and pharmacokinetics (PK) of single and multiple doses of BMN-111 in 48 healthy adult volunteers.
BMN-111 was generally well-tolerated. Mild, transient, self-limited hypotension was observed. The majority of these cases were asymptomatic, and only observed upon assumption of an upright posture following recumbence. No dose-limiting toxicities were identified outside of these cardiovascular findings. Systemic exposure to BMN-111 was similar at these doses to what has been observed to cause growth in healthy and disease model animals. All adverse events were of mild severity.
"We have identified a safe starting dose for treatment of achondroplastic children," stated
Based on the results of the Phase 1 study, the design of a Phase 2 proof-of-concept and dose finding study is underway. The goal of the program is to assess growth velocity as well as medical complications of achondroplasia, including non-growth endpoints. To better understand the nature of the disease, a study to collect consistent baseline growth measurements in children with achondroplasia was initiated in the second quarter of 2012. Participants in this study will be considered for enrollment in the Phase 2 study.
Achondroplasia is the most common form of human dwarfism and is characterized by failure of normal conversion of cartilage into bone. It is caused by an autosomal dominant activating mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of bone growth. Eighty percent of cases are the result of a spontaneous mutation, and ninety-eight percent of those cases have a G380R mutation. Clinical manifestations of the disease include short stature, cervico-medullary compression, sleep apnea, bowed legs, frontal bossing and mid-face hypoplasia, permanent sway of the lower back, spinal stenosis, recurrent ear infections and obesity.
The rate of incidence of achondroplasia is one in 15,000 to one in 40,000 live births, with approximately 18,000 to 24,000 patients in the U.S. and
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® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with
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Eugenia Shen BioMarin Pharmaceutical Inc.(415) 506-6570 Media Bob Purcell BioMarin Pharmaceutical Inc.(415) 506-3267