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Anyone now anything about this company? May 4, 1999 BIOMARIN PHARMACEUTICAL INC (BMRN) S-1 Filing (SEC form S1) BioMarin Pharmaceutical Inc. is a leading developer of carbohydrate enzyme therapies for debilitating, life-threatening, chronic genetic disorders and other diseases and conditions. In October 1998, we completed the primary evaluation for the pivotal clinical trial of our lead enzyme replacement product candidate, BM101, for the treatment of mucopolysaccharidosis-I or MPS- I. Based on the data from that trial, we intend to complete the filing of a BLA with the FDA in the second half of 1999. We have a joint venture with Genzyme Corporation for the worldwide development and commercialization of BM101. MPS-I is a life-threatening genetic disorder caused by the lack of a sufficient quantity of the enzyme (alpha)-L-iduronidase, which affects about 3,400 patients in developed countries, including approximately 1,000 in the United States and Canada. Patients with MPS-I have multiple debilitating symptoms resulting from the buildup of carbohydrates in all tissues in the body. These symptoms include delayed physical and mental growth, enlarged livers and spleens, skeletal and joint deformities, airway obstruction, heart disease and impaired hearing and vision. If untreated, most children diagnosed with MPS-I will die from complications associated with the disease before adulthood. BM101 is a specific form of (alpha)-L-iduronidase that is intended to replace a deficiency of (alpha)-L-iduronidase in MPS-I patients. In October 1998, we completed the primary evaluation period for our pivotal clinical trial for BM101. This clinical trial treated ten patients with MPS-I for a period of six months at five medical centers in the United States. BM101 met the primary endpoints in its pivotal trial, reducing liver or spleen sizes in eight of ten patients and lowering urinary carbohydrate levels in all ten patients. In addition, various secondary endpoints were reached in each of the patients. We received notice from the FDA that our BLA will receive fast track designation for the treatment of the more severe forms of MPS-I, which account for approximately 60% of all cases. The FDA has granted BM101 an orphan drug designation giving us exclusive rights to market BM101 to treat MPS-I for seven years from the date of FDA approval if BM101 is the first (alpha)-L-iduronidase drug to be approved by the FDA for the treatment of MPS-I. In September 1998, we established a joint venture with Genzyme for the worldwide development and commercialization of BM101 for the treatment of MPS- I. Our responsibilities within the joint venture include obtaining U.S. regulatory approvals as well as manufacturing and process development. Genzyme is responsible for obtaining international regulatory approvals, worldwide sales and marketing as well as pricing and reimbursement. We will share expenses and profits from the joint venture equally with Genzyme. Genzyme invested $8.0 million upon signing the agreement and has agreed to purchase $10.0 million of common stock at the initial public offering price in a private placement concurrent with this offering. Genzyme has committed to pay us an additional $12.1 million upon approval of the BLA for BM101. We are also developing enzyme replacement therapies for other life-threatening genetic diseases. There are nine additional MPS disorders caused by single enzyme deficiencies. We are developing BM102 for the treatment of MPS-VI. We received an orphan drug designation for BM102 to treat MPS-VI and intend to file an IND for the use of BM102 to treat MPS-VI in the fourth quarter of 1999. We are also developing carbohydrate enzymes intended to improve burn debridement and act as anti-fungals. Through a wholly-owned subsidiary, we provide carbohydrate analysis to research institutions and commercial laboratories. Our strategy is to: (1) focus on drug candidates with known biology and low technical risk, (2) target products that address life-threatening conditions that may be developed and approved quickly, (3) pursue well-defined, niche markets, (4) develop a direct sales and marketing organization for select markets and (5) enhance enzymatic expertise through our wholly-owned subsidiary, Glyko, Inc. -------------------------------------------------------------------------------- biz.yahoo.com biz.yahoo.com biz.yahoo.com biz.yahoo.com Regards, TOPSTOCK | ||||||||||||
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