BioMarin’s genetic disease therapy shows mixed results in late-stage study

By Sneha S K and Kunal Das

May 18 (Reuters) – BioMarin Pharmaceutical 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 called BMN 401 in children aged 1 to 12 years with ENPP1 deficiency, a rare, lifelong genetic condition.

The condition is caused by changes in the ENPP1 gene that result in a decrease in plasma inorganic pyrophosphate, leading ‌to ​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.

Shares of the company were down 2.3% at $50.62.

“We interpret the result as ⁠a meaningful clinical failure for BMN 401,” ⁠H.C. Wainwright analyst Mitchell Kapoor said.

Children with ENPP1 ​deficiency typically develop a type of rickets that may cause pain and ​difficulty with movement.

“We are disappointed that the significant increases ‌in plasma PPi observed with BMN 401 did not translate into meaningful clinical improvements for children with ENPP1 deficiency,” said Greg Friberg, chief research and development officer at BioMarin.

BioMarin also said it saw no ⁠meaningful improvement in rickets severity, which reflects bone weakness and deformities, or in growth, including height and weight.

The company said it is evaluating ⁠the data to determine ‌next steps.

The result “materially lowers the probability that BMN ⁠401 becomes a meaningful near-term commercial asset,” Kapoor ​said.

He ‌added that it “increases pressure on BioMarin to deliver ​elsewhere, including ⁠Voxzogo expansion in hypochondroplasia, BMN 333’s long-acting CNP program, and additional business development.”

Currently there are no approved treatments for ENPP1 deficiency, and current care focuses on managing symptoms such as bone deformities, pain and movement issues.

(Reporting by Sneha S K and Kunal Das in Bengaluru; Editing ​by Shreya Biswas)