BioMarin Pharmaceutical (BMRN) made progress regarding its pipeline when it commenced dosing in a phase III study (INSPIRE) on its candidate BMN 701 for the treatment of patients suffering from Pompe disease. Pompe disease, a rare lysosomal storage deficiency, has two main forms: infantile onset and late onset.
The single-arm, open-label, switchover study will evaluate patients affected by late onset Pompe disease (an inherited disorder). Patients affected by this form of the disease suffer from muscle weakness and respiratory problems.
The study will find out whether there is an improvement in patients who have previously been treated with Sanofi’s (SNY) Lumizyme (alglucosidase alfa) with respect to measures of respiratory muscle strength and endurance by switching over to BMN 701 therapy. BioMarin also intends to asses the safety effects of such a switch.
The safety and efficacy of the candidate was initially evaluated by BioMarin in a 24-week phase I/II study (n=22). The study assessed treatment naïve patients (aged 13 years and above) affected by late-onset Pompe disease.
Other interesting candidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently undergoing phase III development for the treatment of PKU. The pipeline at BioMarin also includes BMN-673 (a poly polymerase inhibitor). In Oct 2013, the company initiated a phase III study on BMN 673 in germline BRCA (gBRCA) breast cancer. BMN 111, for treating children suffering from achondroplasia, is another interesting pipeline candidate at BioMarin. The successful development and commercialization of the robust pipeline will help drive long-term growth at BioMarin.
Apart from the interesting pipeline, BioMarin has a strong product portfolio with four marketed products – Naglazyme, Kuvan, Aldurazyme, Vimizim and Firdapse.
BioMarin currently carries a Zacks Rank #3 (Hold). Better-ranked healthcare stocks include Alexion Pharmaceuticals (ALXN) and Gilead Sciences (GILD). While Alexion holds a Zacks Rank #2 (Buy), Gilead carries a Zacks Rank #1 (Strong Buy).
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