MINNEAPOLIS–(BUSINESS WIRE)–March 24, 2004–Orphan Medical, Inc. (Nasdaq:ORPH) announced today that patient enrollment is complete for its Phase III(b) trial assessing Xyrem(R) (sodium oxybate) oral solution to treat excessive daytime sleepiness (EDS) associated with narcolepsy. Xyrem is currently the first and only approved treatment for cataplexy associated with narcolepsy. EDS is a universal symptom of narcolepsy frequently accompanied by unavoidable episodes of sleep.
This Phase III(b) trial, referred to as SXB-15, is a double-blind, placebo-controlled, randomized study designed to evaluate improvement in EDS when Xyrem is used in conjunction with stimulants. Improvement is measured by the Epworth Sleepiness Score and Clinical Global Impressions of Change measure, and includes the objective measure Maintenance of Wakefulness Test (MWT) in all patients on four occasions during the study. The trial includes 200 patients in four parallel groups (three different Xyrem doses and a placebo group.) The Company expects preliminary data to be available before or at the Associated Professional Sleep Societies (APSS) meeting held in June.
Orphan Medical also has a second Phase III(b) trial underway to evaluate the relative efficacy of Xyrem with or without modafinil in treating EDS. Patient enrollment in this trial, known as EXCEEDS, is nearing completion as well. The primary endpoint of the trial is the reduction of excessive daytime sleepiness using the objective measure Maintenance of Wakefulness Test as well as investigator assessments of narcolepsy symptoms using the Clinical Global Impressions of Change measure.
The results of the two Phase III(b) trials will be analyzed and, if positive, will be submitted to the U.S. Food and Drug Administration (FDA) as a supplemental New Drug Application (sNDA) in the second half of this year.
“The completion of patient enrollment for SXB-15 is an important milestone for Orphan Medical,” said John Bullion, Orphan Medical Chief Executive Officer. “These are difficult and demanding trials. They have been conducted in North America and six countries in Europe and have required substantial commitment on the part of patients and investigators. The subsequent sNDA filing should allow the positioning of Xyrem as a medication that effectively addresses the full range of narcolepsy symptoms.”
Narcolepsy is a chronic, debilitating neurological disease. Cataplexy associated with narcolepsy, a sudden loss of muscle tone, is usually triggered by strong emotions such as laughter, anger, or surprise. As such, patients often selectively isolate themselves from interaction with others resulting in a dramatic effect on a patient’s quality of life. Xyrem is the first and only approved medication for the treatment of cataplexy and was commercially launched in October of 2002. Sixty to ninety percent of the 100,000 to 140,000 Americans with narcolepsy suffer from cataplexy, with about 50,000 receiving some form of treatment for their disease.
Orphan Medical acquires, develops, and markets pharmaceuticals of high medical value for inadequately treated and uncommon CNS diseases treated by specialist physicians. The lead product for the company is Xyrem, which is the first and only approved treatment for cataplexy associated with narcolepsy. Xyrem is also being assessed as a treatment for the full range of narcolepsy symptoms including excessive daytime sleepiness. Orphan Medical’s pipeline includes development stage products for fibromyalgia and pain. Orphan Medical’s Internet Web site address is www.orphan.com.
The information in this press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. A number of factors could cause actual results to differ materially from the Company’s assumptions and expectations. These are set forth in the cautionary statements included in Orphan Medical’s most recent Form 10-Q or Form 10-K filed with the Securities and Exchange Commission. (These documents can be accessed through the Orphan Medical Web site at http://www.orphan.com). All forward-looking statements are qualified by, and should be considered in conjunction with, such cautionary statements.