Businessweek

Protalix BioTherapeutics, Inc., a biopharmaceutical company, focuses on the development and commercialization of recombinant therapeutic proteins based on proprietary ProCellEx protein expression system (ProCellEx). Using ProCellEx system, the company is in the process of developing a pipeline of proprietary and biosimilar versions of recombinant therapeutic proteins based on plant cell-based expression technology that target large, established pharmaceutical markets and that rely upon known biological mechanisms of action. Its initial commercial focus has been on complex therapeutic proteins, including proteins for the treatment of genetic disorders, such as Gaucher disease and Fabry disease. The company’s wholly-owned subsidiary and sole operating unit, Protalix Ltd. (Protalix), focuses on the expression of recombinant therapeutic proteins in plant cells. Product Candidate, Taliglucerase Alfa Taliglucerase alfa, the company’s proprietary product candidate, is a plant cell expressed recombinant glucocerebrosidase enzyme (GCD) for the treatment of Gaucher disease. The U.S. Food and Drug Administration (FDA) has granted taliglucerase alfa a Prescription Drug User Fee Act (PDUFA) in May 2012. In 2010, Pfizer Inc. (Pfizer), the company’s commercialization partner, filed, with the company’s cooperation, a Marketing Authorization Application (MAA) for taliglucerase alfa with the European Medicines Agency (EMA). In addition, marketing applications have been submitted for taliglucerase alfa with each of the Israeli Ministry of Health (Israeli MOH), the National Sanitary Vigilance Agency (ANVISA), an agency of the Ministry of Health of Brazil, and the Australian Therapeutic Goods Administration (Australian TGA). In 2009, Protalix and Pfizer entered into a license and supply agreement, pursuant to which Pfizer was granted a worldwide license to develop and commercialize taliglucerase alfa. In 2011, the company completed a nine-month, worldwide, multi-center, open-label, switch-over clinical study evaluating the safety and efficacy of switching Gaucher patients treated with Cerezyme, which is produced by Genzyme Corporation (Genzyme) (which was acquired by Sanofi-Aventis (Sanofi) in April 2011), with taliglucerase alfa. Product Pipeline The company also develops a product pipeline using ProCellEx protein expression system. Its product pipeline includes PRX-102, a therapeutic protein candidate for the treatment of Fabry disease, a rare, genetic lysosomal disorder in humans; PRX-105, a plant cell expressed pegylated recombinant acetylcholinesterase product candidate for biodefense and other indications; PRX-106, or pr-antiTNF, a plant cell expressed recombinant fusion protein made from the soluble form of the human TNF receptor (TNFR) and an antibody portion, which is being developed as a treatment of certain immune diseases, such as rheumatoid arthritis, juvenile idiopathic arthritis, spondylitis, psoriatic arthritis and plaque psoriasis; an orally-administered glucocerebrosidase enzyme for the treatment of Gaucher disease patients utilizing oral delivery of the recombinant enzyme produced within carrot cells; and two additional undisclosed therapeutic proteins, both of which are being evaluated in animal studies. Strategic Collaborations The company has a collaboration and licensing agreement with Teva Pharmaceutical Industries Ltd. (Teva) for the development and manufacture of two proteins to be identified by Teva and the company using its ProCellEx protein expression system. In 2006, Protalix entered into a research and license agreement with the Yeda Research and Development Company Limited (Yeda), the technology transfer arm of the Weizmann Institute of Science. Under the terms of the agreement, Yeda agreed to use its technology to design a glucocerebrosidase (GCD) for the treatment of Gaucher disease that can be expressed using ProCellEx protein expression system. Strategy The company intends to become an integrated biopharmaceutical company focused on the development and commercialization of proprietary and biosimilar or generic versions of recombinant therapeutic proteins. It also intends to obtain regulatory approval for Taliglucerase Alfa for the Treatment of Gaucher Disease and develop a pipeline of Biosimilar Versions of Recombinant Therapeutic Proteins. Intellectual Property As of December 31, 2011, the company holds, or has license rights to, 31 patents and 81 pending patent applications with respect to various compositions, methods of production and methods of use relating to ProCellEx protein expression system and proprietary product pipeline. As of December 31, 2011, the company also holds, with a third party, one joint patent and one joint patent application, and licensed rights to six patents and seven patent applications. Competition The company faces competition from companies with approved treatments of Gaucher disease, including Genzyme and Actelion Ltd. It also faces competition from companies with approved enzyme treatments of Fabry disease, including Genzyme, Shire plc and Amicus Therapeutics, Inc. History Protalix BioTherapeutics, Inc. was founded in 1993.