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Last $146.27 USD
Change Today +1.71 / 1.18%
Volume 1.1M
BMRN On Other Exchanges
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As of 8:10 PM 07/31/15 All times are local (Market data is delayed by at least 15 minutes).

biomarin pharmaceutical inc (BMRN) Key Developments

BioMarin Pharmaceutical Announces Positive Results from Phase II ABRAZO Study

BioMarin Pharmaceutical Inc. announced positive results from the Phase II ABRAZO study with its poly ADP-ribose polymerase, or PARP, inhibitor, talazoparib, for the treatment of patients with deleterious germline BRCA 1 or BRCA 2 mutations and locally advanced and/or metastatic breast cancer. The company announced that the ABRAZO Phase II trial has met the study's protocol-specified threshold for documented tumor reduction (using the RECIST response rate criteria) in order to warrant expanding enrollment in the study from 70 to 140 patients. The ABRAZO study includes two cohorts of patients with BRCA mutated metastatic breast cancer. The first cohort consists of patients who have initially responded to a platinum-containing regimen then progressed, while the second cohort consists of patients who have received more than two prior chemotherapy regimens for metastatic disease. The protocol-specified expansion criteria requires that a minimum of five responses per cohort, of up to 35 patients, be observed in order to expand the study. The minimum of 5 responses was seen prior to full enrollment in each cohort. The ABRAZO study is the first study treating BRCA breast cancer patients with a PARP inhibitor monotherapy that has demonstrated activity in patients who are in a salvage setting defined as having failed at least two prior chemotherapy regimens for metastatic disease. In addition, this is the first reported data showing tumor reduction from a PARP inhibitor in BRCA breast cancer patients previously treated with a platinum regimen. The trial, now targeting enrollment of a total of 140 patients, is expected to be fully enrolled in the first quarter of 2016 with results expected by year end 2016. EMBRACA is a Phase III, open-label, 2:1 randomized trial to compare the safety and efficacy of oral PARP inhibitor talazoparib versus protocol-specific physician's choice in patients who have locally advanced and/or metastatic breast cancer with germline BRCA mutations. Prior guidance had been for enrollment of 430 patients by year end 2015. Since study initiation, a newly completed review of published data suggests that the median progression-free survival (PFS) is lower than originally estimated for the control arm in this patient population, and that fewer than the originally estimated 430 patients may need to be enrolled in order to achieve the targeted hazard ratio. This is a Phase II, 2-Stage, 2-Cohort Study of oral PARP inhibitor talazoparib (BMN 673) in patients with locally advanced and/or metastatic breast cancer with germline BRCA mutations. Subjects will be assigned to either Cohort 1 or 2 based on prior chemotherapy for metastatic disease: Cohort 1 include subjects who have previously responded (PR or CR) to a platinum-containing regimen for metastatic disease with disease progression > 8 weeks following the last dose of platinum; and Cohort 2 include subjects who have received more than two prior chemotherapy regimens for metastatic disease and no prior platinum therapy for metastatic disease. This is a Phase III, open-label, 2:1 randomized trial to compare the safety and efficacy of oral PARP inhibitor talazoparib (BMN 673) versus protocol-specific physician's choice in patients who have locally advanced and/or metastatic breast cancer with germline BRCA mutations. The primary objective of the study is to compare progression-free survival (PFS) of subjects treated with talazoparib (BMN 673) as a monotherapy relative to those treated with protocol-specific physician's choice. The secondary objectives are to evaluate objective response rate (ORR) and overall survival (OS). Exploratory objectives are to evaluate duration of response (DOR) and health-related quality of life.

BioMarin Pharmaceutical Inc.(NasdaqGS:BMRN) added to NASDAQ-100 Index

BioMarin Pharmaceutical Inc. has been added to NASDAQ-100 Index.

Biomarin Provides Program Update for Talazoparib in Metastatic Breast Cancer

BioMarin Pharmaceutical Inc. announced an update on the ABRAZO Phase 2 study of its poly ADP-ribose polymerase (PARP) inhibitor, talazoparib for the treatment of patients with deleterious germline BRCA 1 or BRCA 2 mutations and locally advanced and/or metastatic breast cancer. The company announced that the ABRAZO Phase 2 trial has met the study's protocol-specified threshold for documented tumor reduction (using the RECIST response rate criteria) in order to warrant expanding enrollment in the study from 70 to 140 patients. The ABRAZO study includes two cohorts of patients with BRCA mutated metastatic breast cancer. The first cohort consists of patients who have initially responded to a platinum-containing regimen then progressed, while the second cohort consists of patients who have received more than two prior chemotherapy regimens for metastatic disease. The protocol-specified expansion criteria requires that a minimum of five responses per cohort, of up to 35 patients, be observed in order to expand the study. The minimum of 5 responses was seen prior to full enrollment in each cohort. The ABRAZO study is the first study treating BRCA breast cancer patients with a PARP inhibitor monotherapy that has demonstrated activity in patients who are in a salvage setting defined as having failed at least two prior chemotherapy regimens for metastatic disease. In addition, this is the first reported data showing tumor reduction from a PARP inhibitor in BRCA breast cancer patients previously treated with a platinum regimen. The trial, now targeting enrollment of a total of 140 patients, is expected to be fully enrolled in the first quarter of 2016 with results expected by year end 2016. These interim results of the ABRAZO study are planned to be presented at an upcoming medical meeting in 2016. The company also updated guidance for completion of enrollment of the pivotal EMBRACA study, which the company now estimates to be in the first half of 2016. EMBRACA is a Phase 3, open-label, 2:1 randomized trial to compare the safety and efficacy of oral PARP inhibitor talazoparib versus protocol-specific physician's choice in patients who have locally advanced and/or metastatic breast cancer with germline BRCA mutations. Prior guidance had been for enrollment of 430 patients by year end 2015. Since study initiation, a newly completed review of published data suggests that the median progression-free survival (PFS) is lower than originally estimated for the control arm in this patient population, and that fewer than the originally estimated 430 patients may need to be enrolled in order to achieve the targeted hazard ratio.

BioMarin Pharmaceutical Inc. Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee Act (PDUFA) goal date for a decision is December 27, 2015. The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. In the FDA's filing communication, the Agency informed the company that it is currently planning to hold an advisory committee meeting to discuss the application. No date has been set for this meeting. Drisapersen previously has been granted Orphan and Fast Track status, as well as Breakthrough Therapy designations by the FDA. The U.S. filing is based on three randomized placebo-controlled trials and two long-term open-label studies of more than 300 patients in which some boys have been treated for more than three years. Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the subset of DMD amenable to single exon skipping. Drisapersen induces the skipping of dystrophin exon 51, otentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. In the U.S., it is estimated there are approximately 2,000 patients who would be candidates for drisapersen.

BioMarin Pharmaceutical Inc. Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA's standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016. Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the large subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births. In Europe, it is estimated there are 23,000 boys and young men with Duchenne Muscular Dystrophy, and approximately 3,000 of those would be candidates for drisapersen. In BioMarin's commercial territories, approximately 85% of Duchenne patients are located outside of the United States, including Western Europe, Middle East, Eastern Europe, Latin America and Japan. It is estimated that DMD affects approximately 90,000 patients in BioMarin's commercial territories. Duchenne Muscular Dystrophy: Duchenne muscular dystrophy is a severely debilitating childhood neuromuscular disease caused by mutations in the dystrophin gene. This results in the absence or defect of the dystrophin protein, which is important in connecting the cytoskeleton of muscle fibers to the extracellular matrix. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12 years. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure. Because the Duchenne gene is found on the X-chromosome, it primarily affects boys.

 

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