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"There is a significant unmet need in diagnosing and treating millions ofpatients with fibromyalgia. Potential new treatments that address the varioussymptoms can have a significant impact on patients' quality of life," said I.Jon Russell, M.D., Ph.D., the study's lead investigator and AssociateProfessor of Medicine, Division of Clinical Immunology and Rheumatology, andDirector, University Clinical Research Center, University of Texas HealthScience Center at San Antonio.
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The 14-week study included 548 adult patients with fibromyalgia randomizedto one of three treatment arms: sodium oxybate 4.5 g/night, sodium oxybate 6g/night or placebo. The primary outcome measure, viewed by both U.S. and EUregulatory authorities as a clinically meaningful endpoint, was the proportionof patients who achieved at least 30 percent reduction in pain from baselineto endpoint based on the Pain Visual Analog Scale (VAS). In the EU it is alsoconsidered that Fibromyalgia Impact Questionnaire (FIQ) data is equallyrelevant. FIQ data are considered supportive data by U.S. regulators.
In the top-line results, a significant number of patients treated withsodium oxybate achieved 30 percent or greater improvement in their paincompared to patients treated with placebo. Of those patients receiving sodiumoxybate treatment, 46.2 percent of patients on 4.5 g/night and 39.3 percent ofpatients on 6 g/night reported this level of pain relief, compared with 27.3percent of patients on placebo. These results were highly statisticallysignificant.
Patients' physical functioning and ability to perform daily tasks, asmeasured by the FIQ, were significantly different from placebo for the 4.5g/night dose and approached significance for the 6 g/night dose.
Patients receiving sodium oxybate also reported significant improvement infatigue, another common symptom of fibromyalgia, at both active dosage levels.
Adverse events were similar to those seen in previous experience withsodium oxybate. The most common adverse events, with incidence greater than orequal to 5 percent and at least twice the rate of placebo, were headache,nausea, dizziness, vomiting, diarrhea, anxiety, and sinusitis. Sodium oxybatewas generally well tolerated, with the majority of adverse events reportedbeing mild to moderate in nature.
"Achieving positive sodium oxybate (JZP-6) data in fibromyalgia from thisPhase III clinical trial is an exciting milestone for Jazz Pharmaceuticals,and supports our commitment to developing products that can improve outcomesfor patients with serious medical conditions," said Samuel Saks, M.D., ChiefExecutive Officer of Jazz Pharmaceuticals.
"As UCB continues to focus on serious diseases of the central nervoussystem and immunology, we are delighted to partner with Jazz Pharmaceuticalsin bringing new hope for patients with this under-treated condition," saidRoch Doliveux, Chief Executive Officer of UCB.
Only primary efficacy and safety data have been reviewed at this time.Further analyses will be undertaken to examine the full results, includingsecondary endpoints, in greater detail. The sodium oxybate Phase III clinicaltrial program also includes a second randomized, double blind,placebo-controlled study, which is continuing at sites in the U.S. and Europe.More than 90% of the subjects have been enrolled in this second Phase IIItrial. Jazz Pharmaceuticals anticipates submitting a New Drug Application forsodium oxybate to the U.S. Food and Drug Administration by the end of 2009.UCB anticipates filing in the EU shortly after. UCB has the exclusivemarketing and distribution rights to sodium oxybate for fibromyalgia in Europeand some other countries outside North America and will manage registrationsaccordingly.
Jazz Pharmaceuticals will host an investor conference call and live audiowebcast to discuss the preliminary top-line results from this clinical trialon November 20, 2008 commencing at 5:00 p.m. Eastern Time/2:00 p.m. PacificTime. The live webcast may be accessed on Jazz Pharmaceuticals' website athttp://www.JazzPharmaceuticals.com. Please connect to the website prior to thestart of the conference call to ensure adequate time for any softwaredownloads that may be necessary. An archived version of the webcast will beavailable through December 4, 2008. Investors may participate in theconference call by dialing 866-730-5765 in the U.S., or 857-350-1589 outsidethe U.S., and entering passcode 52336179. A replay of this call will beavailable until December 4, 2008 at 888-286-8010 or 617-801-6888(international), using the passcode 46982308.
About Sodium Oxybate
Sodium oxybate is the sodium salt form of gamma-hydroxybutyrate, anendogenous neurotransmitter and metabolite of GABA. While the precisemechanism of action is unknown, the effects may be mediated in part throughinteraction with GABA(B) and GHB receptors. Sodium oxybate is the activeingredient in XYREM(R), approved by the FDA for the treatment of excessivedaytime sleepiness (EDS) and cataplexy (the sudden loss of muscle tone) inadult patients with narcolepsy. The American Academy of Sleep Medicinerecommends sodium oxybate as a standard of care for the U.S. Food and DrugAdministration-approved indications. It is also approved by the EuropeanMedical Evaluation Agency (EMEA) for the treatment of narcolepsy withcataplexy in adult patients. Most commonly reported adverse drug reactions innarcolepsy patients are dizziness, nausea and headaches. Sodium oxybate hasthe potential to induce respiratory depression and neuropsychiatric events.Sodium oxybate has not been evaluated by regulators for the treatment offibromyalgia and is not approved for this use.
About Fibromyalgia
Fibromyalgia, a chronic condition characterized by widespread pain,affects 0.5% - 5% of adults worldwide. Fibromyalgia is believed to be acentral nervous system condition, resulting from neurological changes in howthe brain perceives and responds to pain. In addition to pain, the mainsymptoms are fatigue, disturbed sleep and morning stiffness.
The exact causes of fibromyalgia are unknown. It may be triggered byphysical trauma, emotional stress, chronic pain or infection. Genetics,neurochemicals that affect pain modulation, neurohormones and sleep physiologyabnormalities are thought to play a role. Research also has suggested arelationship between sleep and pain. Fibromyalgia patients experience a highprevalence of sleep problems, including a reduction in non-restorative or deepsleep.
About Jazz Pharmaceuticals, Inc.
Jazz Pharmaceuticals is a specialty pharmaceutical company that identifies,develops and commercializes innovative treatments for important, underservedmarkets in neurology and psychiatry. The Company has an unwavering commitmentto improving care for patients with serious psychiatric and neurologicalconditions through innovative treatments and distinctive and valuable programsfor patients and physicians. For further information seehttp://www.JazzPharmaceuticals.com.
About UCB
UCB (Brussels, Belgium, http://www.ucb-group.com) is a biopharmaceuticalcompany dedicated to the research, development and commercialization ofinnovative medicines with a focus on central nervous system and immunologydisorders. Employing around 12,000 people in over 40 countries, UCB achievedrevenue of EUR 3.6 billion in 2007. UCB is listed on Euronext Brussels(symbol: UCB).
Jazz Pharmaceuticals "Safe Harbor" Statement under the Private SecuritiesLitigation Reform Act of 1995
This press release contains forward-looking statements related to thedevelopment of Jazz Pharmaceuticals' sodium oxybate (JZP-6) product candidatefor the treatment of fibromyalgia, including the submission of a New DrugApplication to the FDA. These forward-looking statements are based on thecompany's current expectations and inherently involve significant risks anduncertainties. Jazz Pharmaceuticals' actual results and the timing of eventscould differ materially from those anticipated in such forward-lookingstatements as a result of these risks and uncertainties, which include,without limitation, the risk that clinical trial results may require JazzPharmaceuticals to discontinue development of the sodium oxybate (JZP-6)product candidate, risks related to Jazz Pharmaceuticals' ability to obtainadditional funds sufficient to support its operations, risks related to JazzPharmaceuticals' reliance on third parties to conduct the clinical trials forits product candidates, including the second Phase III clinical trial of thesodium oxybate (JZP-6) product candidate, and risks that regulatory filingsmay not be made, or may be delayed, and that the sodium oxybate (JZP-6)product candidate for the treatment of fibromyalgia may not be approved formarketing by regulatory authorities. These and other risk factors arediscussed under "Risk Factors" in the Quarterly Report on Form 10-Q for thequarter ended September 30, 2008 filed by Jazz Pharmaceuticals with theSecurities and Exchange Commission on November 14, 2008. Jazz Pharmaceuticalsundertakes no duty or obligation to update any forward-looking statementscontained in this release as a result of new information, future events orchanges in its expectations.
UCB Forward-looking Statement
This press release contains forward-looking statements based on currentplans, estimates and beliefs of management. Such statements are subject torisks and uncertainties that may cause actual results to be materiallydifferent from those that may be implied by such forward-looking statementscontained in this press release. Important factors that could result in suchdifferences include: changes in general economic, business and competitiveconditions, effects of future judicial decisions, changes in regulation,exchange rate fluctuations and hiring and retention of employees.PRELIMINARY TOP LINE RESULTS OF FIRST PHASE III TRIAL SHOW SIGNIFICANT DECREASES IN PAIN AND FATIGUE, AND IMPROVEMENT IN DAILY FUNCTION, IN FIBROMYALGIA PATIENTS
SOURCE Jazz Pharmaceuticals, Inc.
