Monday, November 12, 2007

Biotechs find promise in rare disease drugs

By Ryan McBride

A Watertown drug developer has plans to enter human clinical trials with a compound that could vastly alter how patients are treated for a rare neurodegenerative disorder.

EnVivo Pharmaceuticals Inc. wants to enter Phase 1 clinical trials next year with a so-called histone deacetylase inhibitor (HDACi) to treat Huntington's disease, said Kees Been, chief executive of the company, last week. Though just one of several compounds in the firm's pipeline, Been said, the HDACi would be the first of its kind to treat Huntington's.

The inhibitor compound is intended to block an enzyme that prevents DNA in brain cells from expressing certain proteins important to healthy cellular function in patients with Huntington's and other diseases of the central nervous system, Been said.

"This will be big," said Been, who noted the multibillion-dollar annual market for drugs to treat neurological diseases. "That will be exciting for the entire industry."

Huntington's disease, with about 250,000 Americans who either have or are at risk of developing the disorder, causes a loss of cognitive ability and motor control due to a degeneration of brain cells. There are no effective treatments for the disease, which often results in death, according to the Huntington's Disease Society of America, a New York-based nonprofit.

But EnVivo isn't the only biotechnology firm in Massachusetts with designs to treat neurological diseases with HDACi compounds.

Waltham-based Repligen Corp. in April licensed HDACi technology from nonprofit medical researcher The Scripps Research Institute, of La Jolla, Calif. The firm reports it plans to first develop the compounds to treat a rare neurological disorder called Friedreich's ataxia, but notes its technology would also be useful to treat Huntington's disease and other disorders.

On a national level, New Jersey-based drug giant Merck & Co. Inc. markets such an inhibitor in a product called Zolinza to treat a rare form of skin cancer, according to the U.S. Food and Drug Administration, which approved the drug last year. The drug was the first HDACi compound to garner FDA approval. Merck did not release exact sales figures for Zolinza in its third-quarter report last month.

At EnVivo, Been said he expects the firm's HDACi compound to draw significant interest from large drug companies. Firm executives say they believe the compound is the first oral HDACi drug able to break the blood-brain barrier, an important feature for treating diseases of the central nervous system. The drug is being developed in partnership with Montreal-based biotech MethylGene Inc.