Fort Lauderdale, Florida – May 4, 2011 – Sucampo Pharmaceuticals, Inc. (“Sucampo”), (NASDAQ: SCMP) today reported data from a proof of concept phase 2 clinical trial conducted by R-Tech Ueno, Ltd. (“RTU”, JASDAQ: 4573), of UF-021 (unoprostone isopropyl) in 112 patients with retinitis pigmentosa (RP). The purpose of the phase 2 study was to test the effects of unoprostone isopropyl in protecting and improving the central vision in mid-stage to late-stage RP patients. These data were reported today at the annual conference of The Association for Research in Vision and Ophthalmology (ARVO), in Fort Lauderdale, Florida.
The primary endpoint of the study was a change from baseline in retinal sensitivity in the central 2 degrees as measured by Microperimeter-1 (MP-1). The results showed that the high-dose group met the primary endpoint (p=0.018). The low-dose group did not meet the primary endpoint although there was a trend towards improvement. Safety and tolerability were good with mainly mild irritation in some patients; there were no serious adverse reactions.
The secondary endpoints included change from baseline in retinal sensitivity and in the mean deviation (MD) as measured by Humphrey perimetry 10-2, as well as change from baseline in visual acuity (logMAR) and vision-related quality of life (VFQ-25). Additional post-hoc analysis was performed on the change from baseline within the central 2 degrees by Humphrey perimetry.
Ryuji Ueno, M.D., Ph.D., Ph.D., Chairman and Chief Executive Officer of Sucampo Pharmaceuticals, said, “Sucampo is exploring the most efficient means to develop unoprostone isopropyl for this potential indication, which would support our mission of creating effective drugs for the patients who need them. We believe that these data, which demonstrate a dose-dependent improvement of visual function in retinitis pigmentosa patients, support further clinical development of unoprostone isopropyl.”
Sucampo Pharmaceuticals, Inc., through its subsidiaries (Sucampo Pharma Americas, Inc. and Sucampo Manufacturing & Research AG), holds research, development and commercialization rights to unoprostone isopropyl for all territories worldwide, except Japan, Korea, Taiwan and the People’s Republic of China, through agreements signed in April 2009 and March 2011.
Data describing the primary endpoint results, contained in poster #4992/A416 titled Microperimetry Shows Protection of Central Vision in Retinitis Pigmentosa Patients Treated with UF-021: A Phase 2 Study, included these highlights
- In a comparison of changes from baseline within groups, there was a statistically significant (p=0.018) increase in central retinal sensitivity threshold for the high-dose group.
- In a comparison among the groups in changes from baseline in mean central retinal sensitivity, the placebo group showed almost no change. The low-dose group showed some trend for improvement, and the high-dose group showed consistent improvement as early as 4 weeks, which continued over time.
- In a comparison of change from baseline in mean retinal sensitivity among the three groups, there was a trend toward a dose-dependent response (p=0.097). When adjusted for baseline difference in Humphrey perimetry 10-2 MD values, a dose-dependent improvement (p=0.038) in mean central sensitivity was demonstrated.
- There were statistically significant differences between the placebo and high-dose groups in the change from baseline in central retinal sensitivity (p=0.036) and in the proportion of patients with worsening of the retinal sensitivity by =4 decibels (dB) (placebo 21.2%, high dose 2.6%).
- When comparing the outcomes for patients with mean central retinal sensitivity of less than 12 decibels at baseline, there was a positive dose-responsiveness (p=0.037) for changes in retinal sensitivity of the central 2 degrees.
Among the data reported today were these results regarding the secondary endpoint results in poster #4994/A418, titled Humphrey Perimetry 10-2 Assessment of Retinitis Pigmentosa Patients in Phase 2 Study of UF-021:
- In a comparison of change from baseline in mean retinal sensitivity among the three groups, a statistically significant (p=0.018) dose-dependent improvement was demonstrated.
- A significantly (p=0.033) greater number of patients in the high-dose group experienced improvement of central retinal sensitivity by more than 3 dB than in the placebo group.
- Patients whose retinal sensitivity increased by three or more dB also showed significant improvement in logMAR visual acuity (p<0.05) and contrast sensitivity (p<0.01).
- In a comparison within groups of changes from baseline in patients’ VFQ-25 total points values, the changes within the high-dose group were statistically significant (p=0.048).
- A comparison among groups of the change from baseline in VFQ-25 total points values showed a trend to dose-responsiveness (p=0.12).
- In a comparison among groups, there was a statistically significant (p=0.001) dose-dependent response in the change from baseline in the VFQ-25 scores for “social life functions due to vision.”
- In a comparison among the groups in changes from baseline in mean retinal sensitivity, the placebo group showed almost no change. The low-dose group showed variable change, and the high dose group showed improvement as early as 4 weeks, which continued over time.
About the Phase 2 Clinical Study Design of UF-021 for Retinitis Pigmentosa
This phase 2 clinical study was a multi-center, randomized, double-masked, three parallel group, placebo-controlled study, conducted in patients with mid- to late-stage retinitis pigmentosa (RP) who ranged in age from 20 to 65 years, and with a visual acuity of 0.5 or greater. They received placebo, or one or two drops of UF-021 twice daily for 24 weeks. A total of 112 patients were enrolled and 109 patients were evaluated; three patients discontinued treatment due to irritation at time of instillation, worsened diabetes and enterocolitis. This phase 2 study was conducted at several sites in Japan: Chiba University Graduate School of Medicine (Chiba); Hirosaki University (Hirosaki); Iwate Medical University (Iwate); Juntendo University (Tokyo); Miyazaki University (Miyazaki); and, Wada Yuko Eye Clinic (Sendai).
About Retinitis Pigmentosa (RP)
Retinitis Pigmentosa is a genetic disease characterized by progressive, irreversible vision loss and decreasing visual acuity. As RP progresses, daily life becomes increasingly more difficult. Blindness from all causes is among the most significant injuries to a patient’s quality of life and is a major driver of patient-based cost of care and life-style maintenance. There are no drugs or therapeutic procedures currently approved for the treatment of RP today.
About Sucampo Pharmaceuticals, Inc.
Sucampo Pharmaceuticals, Inc., founded in the U.S. in 1996, is an international pharmaceutical company based in Bethesda, Maryland, focused on the development and commercialization of medicines based on prostones. The therapeutic potential of prostones, which occur naturally in the human body as a result of enzymatic (15-PGDH) transformation of certain fatty acids, was first identified by Ryuji Ueno, M.D., Ph.D., Ph.D., Sucampo Pharmaceuticals’ Chairman and Chief Executive Officer. Dr. Ueno founded Sucampo Pharmaceuticals in 1996 with Sachiko Kuno, Ph.D., founding Chief Executive Officer and currently Executive Advisor, International Business Development and a member of the Board of Directors. For more information about Sucampo Pharmaceuticals, please visit www.sucampo.com.
About R-Tech Ueno
R-Tech Ueno was founded in Japan in 1989 by Ryuji Ueno, M.D. Ph.D., Ph.D., and has been a pharmaceutical venture corporation focusing on research, development, manufacturing and sales promotion of prescription drugs mainly in the area of ophthalmic diseases. R-Tech’s current President and Chief Executive Officer is Yukihiko Mashima, M.D., Ph.D., a certified ophthalmology specialist in Japan. Products manufactured by R-Tech Ueno serve ophthalmology and gastrointestinal patient populations. Utilizing the highest level of expertise in the field of ophthalmology, R-Tech Ueno’s “physicians oriented new drug innovation” is making advances into the development of new drugs that target ophthalmic diseases with no currently effective medications. R-Tech Ueno also offers comprehensive support services to venture companies which seek new drug development.
To learn more about R-Tech Ueno Ltd. and its products, visit http://www.rtechueno.com/en/index.php
Sucampo Forward-Looking Statement Any statements in this press release about future expectations, plans and prospects for Sucampo Pharmaceuticals are forward-looking statements made under the provisions of The Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the words “project,” “believe,” “anticipate,” “plan,” “expect,” “estimate,” “intend,” “should,” “would,” “could,” “will,” ”may” or other similar expressions. Forward-looking statements include statements about the potential utility of UF-021 to treat particular indications and expected data availability dates. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including those described in Sucampo Pharmaceuticals’ filings with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K for the year ended December 31, 2010, and other periodic reports filed with the SEC. Any forward-looking statements in this press release represent Sucampo Pharmaceuticals’ views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. Sucampo Pharmaceuticals anticipates that subsequent events and developments will cause its views to change. However, while Sucampo Pharmaceuticals may elect to update these forward-looking statements publicly at some point in the future, Sucampo Pharmaceuticals specifically disclaims any obligation to do so, whether as a result of new information, future events or otherwise.
