Sucampo Pharmaceuticals, Inc. (Sucampo), a global biopharmaceutical company focused on innovative research, discovery, development and commercialization of proprietary drugs based on prostones, announced the positive top-line results of two recently completed clinical trials, a phase 1a trial of cobiprostone and a phase 1a trial of SPI-3608. The studies tested the tolerability and pharmacokinetic profiles versus placebo of the two compounds, both of which were demonstrated to be generally well-tolerated in the tested populations.

Cobiprostone is being investigated for the prevention and/or treatment of oral mucositis. The phase 1a clinical trial for the investigational oral spray formulation of cobiprostone demonstrated that the drug is generally well-tolerated in healthy volunteers. A phase 1b trial is expected to begin in the fourth quarter of 2013.

"Oral mucositis is an extremely painful and often debilitating side effect of radiation therapy and chemotherapy in cancer patients, with limited prescription treatments available," said Ryuji Ueno, chairman, chief executive officer, and chief scientific officer of Sucampo. "In oral mucositis, with the breakdown of the mucosal barrier function, ulcers form as surface cells die. Our pre-clinical data indicate that cobiprostone induces mucosal barrier repair and prevents mucosal barrier damage, and the fact that it was well-tolerated in this study is extremely encouraging. Even though we have not yet evaluated efficacy in patients, we are looking forward to advancing cobiprostone into Phase 1b later this year."

The single-center, randomized, double-blind, placebo-controlled, dose-escalation study conducted in Japan was designed to assess tolerability and pharmacokinetics of a single dose of cobiprostone oral spray versus placebo in healthy adults aged 21-50 years. Approximately 48 individual subjects were analyzed, 36 of whom were treated with cobiprostone.

The study consisted of six escalating-dose steps and one repeat-dose step. In total, six doses of cobiprostone were tested. No serious or severe adverse events (AEs) were reported and the most frequently observed AE was mild diarrhoea.

Sucampo also announced the positive top-line results of SPI-3608 in a phase 1a single-oral-dose study that demonstrated the drug to be generally well tolerated in healthy volunteers. SPI-3608 is being investigated for the treatment of mild to moderate lumbar spinal stenosis (LSS) and may be investigated for additional indications in the future. The next phase of development for SPI-3608 is expected to begin in the first quarter of 2014.

"Spinal stenosis is a common degenerative disease, caused by the narrowing of and diminished blood flow to the spinal cord. There are an estimated 400,000 Americans suffering from the pain associated with this disease1, which can include numbness and muscle weakness in the lower extremities," said Dr. Ueno. "Our pre-clinical data on SPI-3608 indicate that the compound is capable of the treatment of spinal stenosis by increasing spinal cord blood flow. This is an underserved market because there are currently limited options to treat lumbar spinal stenosis, and we are excited to continue investigating SPI-3608 to address this unmet need."

The clinical pharmacology study conducted in Japan assessed the tolerability and pharmacokinetics of SPI-3608 after a single oral administration in healthy men aged 20 to 37 years. Approximately 48 individuals were analyzed in this single-center, randomized, placebo-controlled, double-blind, dose-escalation study, 36 of which were treated with SPI-3608. In total, six doses of SPI-3608 were tested.

No serious or severe AEs were observed and the most frequently observed AE was mild vomiting. These findings showed that SPI-3608 is generally well-tolerated at all doses tested in the study.

Cobiprostone is an investigational prostone compound under development by Sucampo as a potential treatment for oral mucositis. Sucampo holds worldwide, exclusive rights to cobiprostone.

SPI-3608 is an investigational prostone compound under development by Sucampo as a potential oral treatment for mild to moderate lumbar spinal stenosis and may be investigated for additional indications in the future. Sucampo holds worldwide, exclusive rights to SPI-3608. Sucampo is also developing SPI-017, an IV formulation for the treatment of severe lumbar spinal stenosis, which is currently in phase 2a clinical trials and is expected to conclude in Q4 2013.