UPDATE 1-BioMarin's genetic disease therapy shows mixed results in late-stage study

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

BMRN

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- BioMarin Pharmaceutical BMRN.O said on Monday its experimental treatment for a rare genetic condition met one of the two main goals in a late-stage study.

The company was testing its enzyme replacement therapy, BMN 401, in children aged 1 to 12 years with ENPP1 deficiency.

ENPP1 deficiency results in a decrease in plasma inorganic pyrophosphate, which can cause damage to blood vessels, soft tissues and bones.

BioMarin said the therapy met a main goal of significant increases in plasma pyrophosphate through 52 weeks, compared with conventional therapy.

However, it did not show an improvement in a measure of the treatment impact in children with rickets, which causes weak bones, the company said.