Sucampo Reports Results from Phase 2a Study of Intravenous Ion Channel Activator for Lumbar Spinal Stenosis

BETHESDA, Md., Dec. 23, 2013 (GLOBE NEWSWIRE) -- Sucampo Pharmaceuticals, Inc. SCMP (Sucampo) today announced the results of its double-blind, placebo-controlled Phase 2a proof of concept study evaluating safety and efficacy of an intravenously (IV) administered compound of the company's proprietary ion channel activator program in patients with lumbar spinal stenosis (LSS). A responder analysis of data from the trial revealed that patients receiving the ion channel activator experienced a statistically significant improvement in pain, as determined by improvements in the visual analog scale (VAS) score, versus placebo (94.4% versus 62.5%; p=0.035).

Improvements were also observed in the primary and other efficacy measures, including pain-associated quality of life measures for patients treated with the ion channel activator, although the extent of improvement was not statistically significant.

The ion channel activator was well-tolerated and was not associated with serious adverse events. Additionally, no worsening of any of the efficacy measures in the active group was recorded.

"These findings suggest that our 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," said Ryuji Ueno, M.D., Ph.D., Ph.D., Chairman, Chief Executive Officer, and Chief Scientific Officer of Sucampo. "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. 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."

This multicenter, randomized, double-blind, placebo-controlled, dose-escalation study enrolled 51 hospitalized patients between the ages of 20 and 75 with a confirmed diagnosis of lumbar spinal stenosis.  Patients were administered 30 or 60 mcg of the ion channel activator or placebo twice daily for two weeks. Common adverse events included headache, diarrhea, injection site inflammation and feeling cold. No serious adverse events were observed. In addition, data from a previous Phase 1 study demonstrated a favorable safety profile for the IV formulation of this ion channel activator.

Lumbar spinal stenosis is a common degenerative disease that affects an estimated 6 million Americans, with approximately 1.4 million diagnosed annually.¹ The disease is caused by the narrowing of and diminished blood flow to the spinal cord, leading to pain, numbness and muscle weakness in the lower extremities. Current treatment options are limited to pain-killers and invasive surgery. In the United States, lumbar spinal stenosis is the most common reason for back surgery in people over the age of 65.²

About the Intravenous (IV) Ion Channel Activator

The (IV) compound of Sucampo's proprietary ion channel activator program is an investigational prostone compound in development as a potential treatment for severe lumbar spinal stenosis. Sucampo holds worldwide, exclusive rights to 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. A Phase 1b study of the oral compound will begin in early 2014.

About Sucampo Pharmaceuticals, Inc.

Sucampo Pharmaceuticals, Inc. is focused on the discovery, development and commercialization of drugs based on ion channel activators knows as prostones. Discovered by the company's scientific founder, prostones are naturally occurring fatty acid metabolites with unique physiological activities. Sucampo has two marketed products – AMITIZA^® and RESCULA^® – and a pipeline of prostone-based product candidates in clinical development. A global company, Sucampo is headquartered in Bethesda, Maryland, and has operations in the United Kingdom, Switzerland and Japan. For more information, please visit www.sucampo.com.

The Sucampo logo and the tagline, The Science of Innovation, are registered trademarks of Sucampo AG. AMITIZA is a registered trademark of Sucampo AG. RESCULA is a registered trademark of R-Tech Ueno, Ltd, and has been licensed to Sucampo AG.

Sucampo Forward-Looking Statement

This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements are based on management's current expectations and involve risks and uncertainties, which may cause results to differ materially from those set forth in the statements. The forward-looking statements may include statements regarding product development, product potential, future financial and operating results, and other statements that are not historical facts. The following factors, among others, could cause actual results to differ from those set forth in the forward-looking statements: the impact of pharmaceutical industry regulation and health care legislation; Sucampo's ability to accurately predict future market conditions; dependence on the effectiveness of Sucampo's patents and other protections for innovative products; the risk of new and changing regulation and health policies in the U.S. and internationally and the exposure to litigation and/or regulatory actions. No forward-looking statement can be guaranteed and actual results may differ materially from those projected. Sucampo undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Forward-looking statements in this presentation should be evaluated together with the many uncertainties that affect Sucampo's business, particularly those mentioned in the risk factors and cautionary statements in Sucampo's most recent Form 8-K and 10-K, which Sucampo incorporates by reference.

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Sources:

1.     Prevalence of Symptoms of Cervical and Lumbar Stenosis Among Participants in the Osteoporotic Fractures in Men Study (Vogt, et al.) Spine. 2006;31(13):1445-1451.

2.     Ciol MA, Deyo RA, Howell E, Kreif S. An assessment of surgery for spinal stenosis: time trends, geographic variations, complications, and reoperations. J Am Geriatr Soc 1996;44:285-90.