BioMarin's BMN-401 fails key endpoint in ENPP1 deficiency Phase 3 trial

An experimental therapy, BMN 401, was tested in a Phase 3 study for ENPP1 deficiency, a rare disease. The drug missed one of its two co-primary endpoints, a result that regulators consider critical and that has raised doubts about its approval prospects.

The treatment did raise levels of a vital molecule that is deficient in patients, but this biochemical improvement did not translate into better skeletal health, the second co-primary goal. No positive trends were seen in any of the secondary endpoints reported.

BioMarin said it is actively evaluating the data to decide on next steps and will present detailed results at a future medical meeting. The miss is a setback for the company’s plan to add new revenue sources as it continues a strategic overhaul that has included staff layoffs and cost cuts.