Flamel Technologies’ second clinical study in healthy volunteers using its proprietary Micropump technology applied to sodium oxybate has achieved the objective of one single dose before bedtime for patients suffering from narcolepsy. This confirms the results of a previous, first-in-man study.
The current dosing regimen for the standard of care, Xyrem (sodium oxybate), in the United States is two equal, divided doses: the first dose at bedtime and the second dose 2.5 to 4 hours later. The elimination of the second dose for narcolepsy patients would not only provide more convenience, but may improve the benefit sodium oxybate provides as there will be no disruption to nighttime sleep, Flamel says. The potential for additional benefits, including improved safety, will be studied.
The trial was designed as a two-arm study with 12 patients in each arm evaluating two different formulations of Micropump sodium oxybate at a nightly dose of 4.5 g, 6 g, and 7.5 g. Each subject consumed a standard meal 2 hours prior to dosing. Subjects were instructed to maintain a consistent mealtime and dosing schedule throughout the study. One subject dropped out of the study prior to the completion of the 7.5 g dosing portion for reasons unrelated to drug. The data for both formulations at the 4.5 g and 6 g doses were consistent with the data seen in the previous study, which showed:
- Onset of action similar to Xyrem
- Cmax lower than Xyrem
- Mean blood concentration (ug/ml) at hours 7 and 8 similar to Xyrem
The data at the 7.5 g dose for both formulations were consistent with expectations given the data generated at the lower doses. While both formulations were successful, Flamel has chosen to move forward with the optimal formulation.
To date, Micropump sodium oxybate has been tested in 40 healthy subjects across three doses among three different formulations with no safety or tolerability issues.
Flamel plans to meet with the US Food and Drug Administration (FDA) before the middle of 2015. Based on current expectations, the company plans to begin registration studies prior to the end of 2015.