Bristol-Myers, AstraZeneca report positive data for rare metabolic disease drug

Thirteen years after launching an uncontrolled study of metreleptin for rare cases of lipodystrophy, a subgroup analysis concluded that the experimental drug significantly reduced average blood sugar levels in a pediatric arm with 39 patients under the age of 18. The treatment, under review at the FDA, was acquired by Bristol-Myers Squibb ($BMY) as part of its $5.3 billion acquisition of Amylin last year and swiftly partnered with AstraZeneca ($AZN) in an extension of their 2007 diabetes pact.

Patients with lipodystrophy suffer from a shortage of the human hormone leptin, triggered by the loss of fat tissue under the skin. 

"Metabolic disorders resulting from lipodystrophy can develop in childhood and adolescence and are exacerbated over time," said Rebecca Brown, assistant clinical investigator of diabetes, endocrinology, and obesity branch at the National Institute of Diabetes and Digestive and Kidney Diseases. "This new analysis supports the continued study of investigational metreleptin as a potential treatment option for pediatric patients with lipodystrophy."

Triglyceride levels also declined while mean liver function tests, elevated at baseline, declined with treatment as well.

The NIH trial is a long-running test, but this latest round of subgroup data was obtained over a 12-month period. Metreleptin is an analogue of leptin--meaning it's comparable to the hormone--and has orphan drug status in the U.S. and Europe.

- here's the release
- read the Dow Jones report

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