Sucampo Pharmaceuticals has reported positive results from its Phase IIa study evaluating safety and efficacy of an intravenously (IV) administered compound of the company's proprietary ion channel activator programme in patients with lumbar spinal stenosis.
In this proof of concept study, patients who received the ion channel activator have experienced a statistically significant improvement in pain, as determined by improvements in the visual analogue scale score, compared with placebo.
Although the extent of improvement was not statistically significant, improvements in the primary and other efficacy measures have been observed, including pain-associated quality of life measures for patients treated with the ion channel activator.
In the placebo-controlled, dose-escalation study, the ion channel activator was well-tolerated and was not associated with serious adverse events. In addition, worsening of any of the efficacy measures in the active group was not recorded.
Sucampo Pharmaceuticals chairman and CEO Dr Ryuji Ueno said these findings suggest that the company's proprietary ion channel activator could have the ability to target and treat several aspects of lumbar spinal stenosis.
"We are pleased that these preliminary data show that this compound has the potential to improve pain, one of the major aspects of LSS, in a patient population with the condition. Based on the positive findings from this study, we will embark on an additional Phase 2a study in 2014 to identify the appropriate endpoints and dosing regimen for the compound," Ueno said.
"We are encouraged that we can move forward to the next phase of clinical development for our ion channel activator, and we are committed to pursuing it in LSS, an underserved market with limited treatment options."
The randomised, double-blind study has enrolled 51 hospitalised patients between the ages of 20 and 75 with a confirmed diagnosis of lumbar spinal stenosis and these patients were administered 30 or 60mcg of the ion channel activator or placebo twice daily for two weeks.
Common adverse events observed in the study are headache, diarrhoea, injection site inflammation and feeling cold.
Data from the Phase I study demonstrated a favourable safety profile for the IV formulation of this ion channel activator.
The IV compound of Sucampo's proprietary ion channel activator programme is an investigational prostone compound in development as a potential treatment for severe lumbar spinal stenosis. Sucampo holds worldwide, exclusive rights for this compound.
Apart from this compound, Sucampo is also developing an oral ion channel activator for potential treatment in mild to moderate lumbar spinal stenosis, among other indications under investigation.
The company expects to begin Phase Ib study of this oral compound in early 2014.
Image: MRI of the lumbar spine showing spinal stenosis. Photo: courtesy of A E Francis.