BioMarin, Serono Form Strategic Alliance for Phenoptin and Phenylase

BioMarin Pharmaceutical and Serono have formed a strategic alliance for the further development and commercialization of two BioMarin product candidates, Phenoptin (sapropterin HCl) and Phenylase (phenylalanine ammonia lyase).

Both products have shown potential in the treatment of phenylketonuria (PKU), and there is preliminary clinical evidence that suggests that the active ingredient in Phenoptin, a synthetic form of the naturally occurring enzyme cofactor 6R-BH4, may also be useful in the treatment of other serious diseases, including diabetes and cardiovascular diseases.

By the terms of their agreement, Serono acquires exclusive rights to market the products in all territories outside the U.S. and Japan. BioMarin retains exclusive rights to market the products in the U.S. Serono will make an upfront payment of $25 million to BioMarin, and will make additional milestone payments of up to $232 million based on the successful development and registration of both products in multiple indications, of which $45 million are associated specifically with Phenoptin in PKU.

Serono will also pay BioMarin undisclosed royalties on its net sales of the products. The companies will share equally all development costs following successful completion of Phase II trials for each product candidate in each indication.