BioMarin Pharmaceutical Inc. has entered into a strategic business partnership with Daiichi Suntory Pharma Co., Ltd., for Phenoptin (sapropterin hydrochloride), an investigational oral enzyme cofactor for the treatment of the genetic disease phenylketonuria (PKU). This partnership provides BioMarin extensive preclinical and clinical data on 6R- BH4 (the active ingredient in Phenoptin) and access to commercial grade 6R- BH4. These assets will enable BioMarin to begin a phase 2 clinical trial of Phenoptin in PKU patients by the end of 2004, the company announced here.

According to terms of the agreement, BioMarin obtains exclusive rights to preclinical and clinical data on 6R-BH4, which was filed in its Investigational New Drug (IND) application with the US FDA in August 2004, and exclusive access to commercial grade 6R-BH4 manufactured by Daiichi Suntory for use in treating PKU. In exchange for the exclusive rights to these assets in developing a therapeutic for PKU, BioMarin will pay Daiichi Suntory approval milestones and a royalty on sales of Phenoptin outside of Japan, and Daiichi Suntory will retain rights to market the product for PKU in Japan. Daiichi Suntory currently manufactures its own formulation of 6R-BH4, which is approved in Japan to treat the genetic disease, BH4-deficiency. With 6R-BH4 supplied by Daiichi Suntory, BioMarin is positioned to produce Phenoptin at substantially lower cost compared to other sources of 6R-BH4, the official statement said.

Emil Kakkis, senior VP of Business Operations at BioMarin commented, "Daiichi Suntory's pioneering work in developing and studying 6R-BH4 was critical in defining its potential as viable treatment option for PKU. This strategic partnership leverages BioMarin's clinical and product development capabilities in genetic diseases and Daiichi Suntory's manufacturing expertise, ability to produce commercial grade 6R-BH4 and extensive preclinical and clinical data set. Both companies are excited about working together to bring the first FDA approved therapeutic to PKU patients."