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In The News

BioMarin Trial Begins Batten Disease Battle

An experimental drug from BioMarin Pharmaceutical Inc. will take on a deadly rare childhood disease that currently has no treatment.

The San Rafael-based drug-development company (NASDAQ: BMRN) said Tuesday during its annual R&D Day for investors that it will dose the first patient Friday in a Phase I/II trial of a drug designed to treat children with a type of Batten disease.

The 48-week study ultimately will enroll 22 patients in 10 sites worldwide, starting with a 4-year-old girl in Germany this week. Patients will receive BioMarin's experimental enzyme replacement therapy, called BMN-190, directly into the brain.

Batten is a collection of nine or 10 neurodegenerative genetic diseases that make it impossible for neurons to properly recycle in the brain. It robs children of their vision, speech and muscle control and typically kills them by the age of 12.

BioMarin is targeting a type of Batten called neuronal ceroid lipofuscinosis type 2, or Jansky-Bielschowsky disease, which begins to show itself between the ages of 2 and 4 through seizures and loss of some speech and other skills the child had already learned.

BioMarin CEO Jean-Jacques Bienaimé in January said that BMN-190 won't reverse damage already done by Batten disease, but in dogs it showed that it dramatically slowed progression of the disease.

Batten disease affects 400 to 600 children worldwide.

Although Batten is among the rarest diseases, patient advocates are active and were vocal with their criticism of StemCells Inc. (NASDAQ: STEM) when the Newark company in 2011 pulled out of a Phase Ib trial because it said it couldn't find enough patients in earlier stages of the disease.

Patient awareness of treatments could help BioMarin enroll its trial, Dr. David Jacoby, senior medical group director at BioMarin, said during an R&D Day presentation. The company is working with a German research group and with Weill Cornell Medical College in shaping its study.

"The Batten disease group is aware, educated and trained," Jacoby said in response to a question about the trial's enrollment. "I think they're mobilized."

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