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BioMarin Announces Fourth Quarter And Full Year 2012 Financial Results

1Q 2013: Results for Phase 1/2 trial for BMN-701 for Pompe disease

1Q 2013: Clinical trial application filing for BMN-190 for LINCL (Batten disease)

2Q 2013: File market authorization application for Vimizim (GALNS) for MPS IVA in the EU

2Q 2013: Initiation of Phase 3 trial for PEG-PAL for PKU

2Q 2013: Presentation on Phase 1/2 BMN-673 solid tumor data at ASCO meeting

Mid 2013: Initiation of Phase 2 trial for BMN-111 for achondroplasia

4Q 2013: Potential initiation of Phase 3 trial for BMN-673 for solid tumors

4Q 2013: Potential FDA approval of Vimizim for MPS IVA

4Q 2013: Potential initiation of Phase 2/3 trial, a key component of the pivotal program for BMN-701 for Pompe disease

Research and Development Programs

BioMarin continues to make significant investments in research and development to ensure a strong and profitable pipeline for the company. The current pipeline includes programs in various stages of development that focus on treating a range of rare and serious unmet medical needs.     

Advanced Clinical Programs
  • Vimizim for MPS IVA: The company is on track to begin submitting market authorization application filings by the end of the first quarter of 2013. The three ancillary studies are ongoing: a clinical trial for patients under five years of age (fully enrolled), a cardiopulmonary study to more completely document the health benefits of Vimizim (fully enrolled), and a study in patients with limited ambulation (enrolling). 

Mid-Stage Clinical Programs
  • PEG-PAL for PKU: The company plans to initiate a pivotal Phase 3 study in the second quarter of 2013, following a recent end of Phase 2 meeting with the FDA. The Phase 3 design includes (1) an open-label study to evaluate safety and blood Phe levels in naïve patients and (2) a randomized controlled study of the Phase 2 extension study patients to evaluate blood Phe levels and neurocognitive endpoints. The FDA indicated the possibility for an accelerated approval based on demonstrating sustained reduction in Phe levels, though full approval may require demonstration of neurocognitive improvement.  
  • Kuvan Outcomes Study: The company recently reported top-line results of a randomized, placebo-controlled, 13-week Kuvan outcomes study. The primary endpoint of the study was an attention deficit hyperactivity rating scale (ADHD-RS), a commonly used test to evaluate symptoms of inattentiveness and hyperactivity. Kuvan improved the ADHD-RS (p=0.085), driven by a statistically significant change in the inattention component of the score (p=0.036). The company plans to discuss the submission of this data with the FDA for possible inclusion in the Kuvan label.

Early-Stage Clinical Programs
  • BMN-701 for Pompe Disease: Top-line results for the Phase 1/2 trial and a program go/no go decision are expected at the end of the first quarter of 2013. The company recently determined that if it proceeds with development of BMN-701, it will utilize a new cell line. If the decision is made to continue development,  the company will be prepared to start a Phase 2/3 trial, a key component of the pivotal program for BMN-701 with the new cell line in the fourth quarter of 2013.  
  • BMN-673 (PARP inhibitor): An update on the Phase 1/2 study in solid tumors is expected in the second quarter of 2013 at the ASCO Annual Meeting in June 2013. A Phase 3 trial in advanced or recurrent solid tumors could start enrolling by the end of 2013.  
  • BMN-111 for Achondroplasia: The company plans to initiate a Phase 2 trial in patients in mid 2013.  The primary objective of the clinical proof of concept study in pediatric patients will be to evaluate the safety and tolerability of daily subcutaneous (sc) injections of BMN-111 administered for six months. Secondary objectives of the study will be to assess changes in annualized growth velocity, changes in absolute growth and changes in body proportions. Other exploratory objectives will also be assessed.  
  • BMN-190 for LINCL (Batten disease): BioMarin expects to file for its first clinical study of BMN-190 in the first quarter of 2013. Pre-CTA/IND meetings have already been held with MHRA BfArM, and FDA. BioMarin expects to begin enrolling the study mid year 2013.

Preclinical Programs
  • Other early stage programs: BioMarin is working on multiple additional early development opportunities, including two new lead optimization programs gained through the acquisition of Zacharon Pharmaceuticals: inhibition of heparan sulfate synthesis for MPS III and inhibition of ganglioside synthesis for diseases such as Tay Sachs and Sandhoff. The company also announced today the licensing of a Factor VIII gene therapy research program for hemophilia A from University College London and St. Jude Children's Research Hospital.

Non-GAAP Financial Information and Reconciliation

The results for the three months and year ended December 31, 2012 and December 31, 2011 are all determined in accordance with GAAP except for non-GAAP adjusted EBITDA which is determined on a non-GAAP basis. As used in this release, non-GAAP adjusted EBITDA is based on GAAP earnings before interest, taxes, depreciation and amortization (EBITDA) and further adjusted to also exclude certain non-cash stock compensation expense, non-cash contingent consideration expense and certain other nonrecurring material items (non-GAAP adjusted EBITDA). 

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