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12:00 AM
Jun 29, 2009
 |  BioCentury  |  Product Development

Mineralizing Success

Enobia Pharma Inc. is seeking to improve on an earlier attempt to develop an enzyme replacement therapy for hypophosphatasia by using a fusion protein that combines the missing enzyme with a bone-targeting polyacidic sequence. Early results from a pair of studies of its ENB-0040 suggest that both infants and adults benefit from the treatment, and now the company plans to conduct additional Phase II trials this summer before entering pivotal testing.

In addition to determining the size and shape of its next study, the company eventually will have to decide between making itself a takeout target for one of the established players in the ERT space or going at it alone. The latter is a possibility given the limited marketing infrastructure required for targeting a worldwide population of little more than 2,000 patients.

Hypophosphatasia is an ultra-orphan disease in which a loss-of-function mutation results in defective TNSALP (tissue non-specific alkaline phosphatase), an enzyme that is responsible for mineralizing bone. The clinical presentation of the disease can range from stillbirth or early death to respiratory problems, motor deficits and...

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