By Randall Osborne

With its enzyme-replacement therapy in a pivotal trial, BioMarin Pharmaceutical Inc. raised $11.5 million in an ongoing private placement.

Privately held BioMarin, of Novato, Calif., has agreed to form a joint venture with Genzyme Corp., of Cambridge, Mass., to commercialize BioMarin's lead product, L-iduronidase, a recombinant enzyme. (See BioWorld Today, June 4, 1998, p. 1.)

The enzyme is designed to treat mucopolysaccharidosis (MPS I), a genetic disorder of which the best known and most severe form is Hurler syndrome. BioMarin and Genzyme expect to duplicate the success of Genzyme's Cerezyme (recombinant glucocerebrosidase), an enzyme to treat Gaucher's disease and Ceredase, the natural version derived from human placenta.

MPS I's effects include enlargement of the liver and spleen, skeletal deformity, vision impairment, stunted growth, hearing loss and fluid on the brain. The FDA granted orphan drug designation to BioMarin's therapy for the disease last year.

A six-month "pivotal Phase I study" of the recombinant enzyme for MPS I began earlier this year, and is expected to conclude by the end of this month, although the company intends to keep the trial going in order to get longer-term data.

That study is said to be the equivalent of Phase I, II and III trials rolled into one, since it is unblinded and sick children are being treated.

BioMarin, 38 percent owned by Glyko Biomedical Ltd., also of Novato, is focused on developing carbohydrate-acting enzyme therapeutics — using rapid cloning and Glyko's protein expression technology — and pure natural protein enzymes by recombinant means.

Glyko invested $1 million in the placement. In January, BioMarin raised $10 million in a private placement of stock, and has raised $22 million to date.

Major investors in the latest round of financing included BB BioVentures LP, managed by Cambridge, Mass.-based MPM Asset Management; Danske & Co., represented by BankInvest, of Copenhagen, Denmark; and LaMont Asset Management SA, of Zug, Switzerland. *