Bristol-Myers Squibb Co. (BMY) and AstraZeneca (AZN) announced the results from a 12-month sub-group analysis of a National Institutes of Health or NIH, open-label, long-term research study of metreleptin, an investigational agent for the treatment of metabolic disorders associated with inherited or acquired lipodystrophy or LD, a rare disease estimated to affect a few thousand people around the world, often with an early age of onset.
In this analysis, which involved 39 pediatric LD patients less than 18 years of age at the time of study enrollment, investigational metreleptin treatment led to mean reductions in HbA1c (average blood sugar levels over three months) and triglyceride levels, as well as mean reductions in liver function tests (including alanine aminotransferase, or ALT, and aspartate aminotransferase, or AST).
Bristol-Myers stated that the analysis was conducted to examine the effects of investigational metreleptin on select metabolic parameters associated with LD, including HbA1c, triglyceride levels and liver function tests, in pediatric patients enrolled in the NIH study. The four major subtypes of LD - congenital generalized LD, acquired generalized LD, familial partial LD and acquired partial LD - were represented. Patients were stratified to one of two groups: children (=12 years) or adolescents (>12 to <18), and data were analyzed at month 12 of metreleptin treatment, where available (n=27).
In adolescents with LD, metreleptin treatment resulted in statistically significant reductions in elevated HbA1c (mean HbA1c decreased from 9.8±1.8% at baseline to 7.7±1.7% at month 12, for a mean decrease of 2.3±0.4%) and elevated triglycerides (mean triglyceride concentrations decreased from 1378±2024 mg/dL at baseline to 385±446 mg/dL at month 12, for a mean decrease of 44±14%).
According to the company, Elevated liver function tests also decreased (mean ALT decreased from 105±97 U/L at baseline to 59±108 U/L at month 12, for a mean decrease of 46±40%, and mean AST decreased from 87±89 U/L at baseline to 57±118 U/L at month 12, for a mean decrease of 31±38%).
In younger children with LD, confirmed diabetes and hypertriglyceridemia were uncommon; however, mean liver function tests were markedly elevated at baseline and decreased with investigational metreleptin treatment (mean ALT decreased from 193±202 U/L at baseline to 155±274 U/L at month 12, for a mean decrease of 38±47%, and AST decreased from 119±112 U/L at baseline to 90±144 U/L at month 12, for a mean decrease of 30±29%). Reductions in ALT and AST did not reach statistical significance due to the small sample size and limited statistical power, but were clinically meaningful.
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