BioMarin Pharmaceutical Inc. (BMRN - Free Report) recently submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK for BMN-190. BMN-190 is being developed for the treatment of patients suffering from neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of batten disease.
BioMarin intends to begin enrolling for a phase I/II study on BMN-190 in mid-2013. BioMarin is expected to enrol around 22 subjects globally for the study. The subjects will be evaluated for 48 weeks.
The study will evaluate the safety and tolerability of BMN-190 in NCL-2 patients. The study will also evaluate the effectiveness of BMN-190 using an NCL-2-specific rating scale score in comparison to natural history data.
Meanwhile, BioMarin recently submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for Vimizim (BMN-110, elosulfase alfa) for the treatment of patients suffering from mucopolysaccharidosis Type IVA (MPS IVA) or morquio A syndrome. BioMarin also intends to seek approval for Vimizim in the European Union (EU) for the same indication towards the end of this month.
Vimizim is the most important pipeline candidate at BioMarin. The company expects Vimizim candidate to be approved by the end of 2013. We believe that the company can swing to profitability in the event of Vimizim gaining approval.
We note that BioMarin has several data read-outs and trial initiations lined up this year. We expect investor focus to stay on these results.
BioMarin, a biopharma stock, currently carries a Zacks Rank #3 (Hold). However other biopharma stocks such as Athersys Inc. (ATHX - Free Report) , Cytokinetics Inc. (CYTK - Free Report) and Array BioPharma Inc. (ARRY - Free Report) currently look better positioned. While Athersys and Cytokinetics carry a Zacks Rank #1 (Strong Buy), Array carries a Zacks Rank #2 (Buy).