BioMarin Pharmaceutical Inc has received positive results for the phase-I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to initiate a pivotal phase-III trial in the fourth quarter of 2010.

Endurance improvements with GALNS were consistent with, and in some cases, better than those observed in pivotal studies of approved enzyme replacement therapies. Clinically meaningful improvements in two measures of endurance (6-minute walk distance and 3-minute stair climb) were achieved at both 24 weeks and 36 weeks as compared to baseline. (See Table 1 below). Clinically meaningful improvements in two measures of pulmonary function (forced vital capacity and maximum voluntary ventilation) were achieved at 36 weeks as compared to baseline. (See Table 1 below). Keratan sulfate levels decreased shortly after the initiation of treatment and fell further as the study progressed. (See Table 1 below). The frequency and severity of infusion reactions were comparable to those observed with Naglazyme and Aldurazyme.

"We are encouraged that clinically significant improvements in endurance and pulmonary function can be detected with GALNS treatment even in a relatively short study of a heterogeneous population. This gives us confidence that we can design a robust 24 to 36 week phase-III clinical study with a measure of endurance as the primary endpoint and several additional supportive endpoints. We will be working to reach an agreement with regulatory authorities on a powerful and large phase-III trial protocol that minimizes regulatory risk and captures the maximum amount of clinical benefit expeditiously," said Hank Fuchs, chief medical officer of BioMarin. "We are on track to initiate a phase-III registration-enabling program in the fourth quarter of 2010."

Dr Chris Hendriksz, clinical lead for Inherited Metabolic Disorders and director for Lysosomal Storage Disorders at Birmingham Children's Hospital in Birmingham, UK added, "The data from the phase-I/II trial are extremely encouraging as they clearly demonstrate that GALNS has an important, positive effect. The data supports my long-held observation that this disorder is much more than only a bone disease. The greatest misconception about Morquio is that it is a skeletal dysplasia only disease and that there is very little to gain from therapy. Patients with Morquio look forward to breathing easier, playing with their friends outside and living a more normal life. Patients experienced benefits that were quite remarkable and evidently rather quickly. This is an exciting milestone for the MPS IV community as BioMarin's programme moves one step closer to providing the first much-needed and possibly life-changing treatment option for Morquio patients."

Jean-Jacques Bienaime, chief executive officer of BioMarin added, "Assuming the GALNS clinical program is successful, we are very optimistic about the commercial potential of the product. There are over 600 MPS IV patients identified to date which is more than the number of patients on commercial Naglazyme therapy. Since there is no neurocognitive component to the disease and no competing treatment, we expect that all patients may be candidates for therapy. The large identified patient base also provides an accessible pool from which to draw patients for the phase-III trial. GALNS is not only our most advanced clinical program, it also has the potential to be our largest and most profitable product because of the manufacturing and commercial infrastructure that is already in place."

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.