Pipeline Progress at BioMarin - Analyst Blog


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BioMarin Pharmaceutical Inc. ( BMRN ) announced that it has started dosing for a phase II study (n=24) on BMN 111, an analog of C-type Natriuretic Peptide (CNP), for the treatment of children suffering from achondroplasia. The news positively impacted BioMarin's shares.

The phase II open-label, sequential cohort, dose-escalation study will primarily evaluate the safety and tolerability of daily subcutaneous doses of BMN 111 in children (5-14 years) suffering from achondroplasia.

We remind investors that during its second quarter 2013 conference call on Jul 25, 2013, BioMarin stated that the U.S. Food and Drug Administration (FDA) placed a partial clinical hold on the development of BMN 111. The FDA had asked for additional clinical pharmacokinetic and safety data in children suffering from achondroplasia.

However, in Sep 2013, BioMarin announced that the clinical hold on the candidate has been lifted by the U.S. regulatory body following the submission of modified phase II protocol and withdrawal of the previous one. The regulatory authorities in the U.S. as well as in Europe agreed that a phase II study for the indication can be initiated without additional data.

We are encouraged by the successful study initiation after the clinical hold. However, further objections from regulatory authorities related to BMN 111 may put the candidate's future in jeopardy.

In a separate press release the company announced that it has initiated Investigational New Drug (IND)-enabling studies on BMN 270 for the treatment of hemophilia A. BioMarin intends to start clinical studies on the hemophilia A candidate early next year. We are encouraged by the company's progress with its pipeline so far.

BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks include Affymetrix Inc. ( AFFX ), Actelion Ltd. ( ALIOF ) and Gentium ( GENT ). All these stocks hold a Zacks Rank #1 (Strong Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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