BioMarin Pharmaceutical, Inc. (BMRN) recently announced that it has begun an early stage trial of its pipeline candidate BMN 673 (PARP Inhibitor) for the treatment of patients suffering from advanced hematological malignancies whose commonest forms are various types of leukemia, lymphoma and myelodysplastic syndrome.
The trial is a two-arm, open-label, dose escalation study (n~80) in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL). The objective of the trial is to determine the maximum tolerated dose of daily oral BMN 673 to assess the safety, tolerability, preliminary efficacy and pharmacodynamics activity of BMN 673 and determine the pharmacokinetic profile of the candidate. Patients will be enrolled in the study at many sites in the US and UK.
BioMarin acquired BMN 673 from Lead Therapeutics Inc. in February 2010. BioMarin is also conducting a mid-stage trial of BMN 673 in patients with advanced solid tumors. The trial is expected to enroll about 70 patients above the age of 18 with advanced solid tumors. The primary objective of the trial is to determine the maximum tolerated dose of daily oral BMN 673. The secondary objectives include assessing the safety, tolerability, preliminary efficacy and pharmacodynamics activity of BMN 673, and determining the pharmacokinetic profile.
Our Recommendation
We currently have a Neutral recommendation on BioMarin. The stock carries a Zacks #3 Rank (short-term “Hold” rating).
We are encouraged by BioMarin’s better-than-expected performance in the first quarter of 2011. Moreover, the company has a robust pipeline with multiple events lined up. The successful development and commercialization of pipeline candidates would help drive long-term growth at BioMarin. However, the disappointing initial sales ramp of Firdapse, launched in the EU in April 2010, concerns us. Moreover, we expect cash burn to increase owing to the company’s heavy investments in pipeline development.
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