Last $69.62 USD
Change Today -1.18 / -1.67%
Volume 764.7K
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As of 5:20 PM 08/28/14 All times are local (Market data is delayed by at least 15 minutes).
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Company Description

Contact Info

770 Lindaro Street

San Rafael, CA 94901

United States

Phone: 415-506-6700

Fax: 415-382-7889

art disease. VIMIZIM was granted marketing approval in the U.S. in February 2014. The company began marketing VIMIZIM in the U.S. using its own existing sales force and commercial organization and it completed its first commercial sale in the U.S. Products in Clinical Development PEG PAL: PEG PAL is an investigational enzyme substitution therapy that the company is developing as a subcutaneous injection for the treatment of PKU. A Phase 3 clinical trial of PEG PAL was initiated in 2013. BMN 673: BMN 673 is a PARP inhibitor, a class of molecules that has shown clinical activity against cancers involving defects in DNA repair that the company is investigating for the treatment of certain cancers. BMN 701: BMN 701 is a novel fusion of acid alpha glucosidase (GAA) with a peptide derived from insulin-like growth factor 2. BMN 111: BMN 111 is a peptide therapeutic in development for the treatment of achondroplasia. In January 2014, the company announced the initiation of a Phase 2 clinical trial for BMN 111 for the treatment of children with achondroplasia. BMN 190: BMN 190 is a recombinant human tripeptidyl peptidase 1 for the treatment of patients with CLN2, a form of Batten disease. In 2013, the company announced the initiation of a Phase 1/2 study for BMN 190. Sales and Marketing The company has established a commercial organization, including a sales force, to support its product lines directly in the U.S., Europe, South America, and certain other markets. Customers The company’s Naglazyme, Kuvan, and Firdapse customers include various specialty pharmacies and end-users, such as hospitals and foreign government agencies, which act as retailers. It also sells Naglazyme to its authorized European distributors and to certain larger pharmaceutical wholesalers. Genzyme Corporation is its sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third-parties. Patents and Proprietary Rights The number of the company’s issued patents stands at approximately 297, including approximately 64 patents issued by the U.S. Patent and Trademark Office (the USPTO). Furthermore, its portfolio of pending patent applications totals approximately 302 applications, including approximately 42 pending U.S. applications. With respect to Naglazyme, the company has 11 issued patents, including 3 U.S. patents. Claims cover its ultrapure N-acetylgalactosamine-4-sulfatase compositions of Naglazyme, methods of treating deficiencies of N-acetylgalactosamine-4-sulfatase, including MPS VI, methods of producing and purifying such ultrapure N-acetylgalactosamine-4-sulfatase compositions. These patents would expire between 2021 and 2023 (methods of detecting). With respect to Kuvan and BH4, the company owns, co-owns, or has licensed various patents and pending patent applications that relate to formulations and forms of its drug substance, methods of use for various indications under development and dosing regimens. The company has rights to 75 issued patents, including 13 issued U.S. patents with claims to a stable tablet formulation of BH4, methods of treating PKU using a once daily dosing regimen, methods of administration of Kuvan with food, crystalline forms of BH4, and methods of producing BH4. These patents would expire between 2024 and 2029. The company has rights to 33 issued patents, including 6 U.S. patents, related to Aldurazyme. These patents cover its ultra-pure alpha-L-iduronidase composition of Aldurazyme, methods of treating deficiencies of alpha-L-iduronidase by administering pharmaceutical compositions comprising such ultra-pure alpha-L-iduronidase, a method of purifying such ultra-pure alpha-L-iduronidase and the use of compositions of ultra-pure biologically active fragments of alpha-L-iduronidase. These patents would expire in 2019 and 2020. The company has rights to 11 issued patents, including 5 issued U.S. patents with claims to compositions of purified recombinant N-acetylgalactosamine-6-sulfate sulfatase (VIMIZIM) methods of treating Morquio Syndrome and sulfatase-modifying factor I (SUMF1) polypeptides and nucleic acids used in the manufacture of VIMIZ

 

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