20th Jun 2005 12:05
For Immediate ReleaseStatement on release of positive TKT Hunter Syndrome dataBasingstoke, UK and Philadelphia, US - June 20, 2005 - Shire PharmaceuticalsGroup plc (LSE: SHP, NASDAQ: SHPGY, TSX: SHQ) makes the following statement onthe announcement by Transkaryotic Therapies, Inc (NASDAQ:TKTX) of results fromthe company's pivotal phase 3 clinical trial (AIM study) evaluating itsinvestigational human enzyme replacement therapy (ERT), iduronate-2-sulfatase(I2S), for the treatment of patients with Hunter syndrome:Shire Chief Executive, Matthew Emmens said:"We are very pleased that the announcement of the AIM study results from TKTare positive and that they plan to file the product license applications in theUS and Europe as targeted, in the fourth quarter of 2005. ERT productdevelopment is lower risk than other new chemical entities. This lower riskprofile is one of the reasons why TKT is such an attractive fit for Shire."Patients with Hunter syndrome are now one step nearer to being able to obtaintreatment for this debilitating and life threatening condition with the releaseof this new trial data from TKT."Shire announced on April 21, 2005 that it had signed a definitive agreement toacquire TKT. The transaction is subject to approval by the shareholders of bothcompanies, as well as regulatory approvals and satisfaction of other customaryclosing conditions. The transaction is expected to close in the summer of 2005.TKT will host a conference call/webcast, today, June 20, 2005, at 9:00 a.m ET/2pm BST to discuss the AIM study. To participate by telephone, dial (913)981-4905. A live audio webcast can be accessed on the TKT website at http://www.tktx.com within the Investor Information section. A replay of the call willbe available for two weeks by dialling (719) 457-0820; (888) 203-1112 and usingthe access code: 3246105. A replay of the webcast will be archived on the TKTwebsite under Events in the Investor Information section.The full text of the TKT press announcement is pasted overleaf/below: TKT Reports Positive Top-Line Results of Hunter Syndrome Pivotal Trial Primary Endpoint Achieves Statistical SignificanceCambridge, MA, June 20, 2005 - Transkaryotic Therapies, Inc. (Nasdaq: TKTX)today announced positive top-line results from the company's pivotal Phase IIIclinical trial evaluating its investigational human enzyme replacement therapy,iduronate-2-sulfatase (I2S), for the treatment of patients with Huntersyndrome. Hunter syndrome, also known as MPS II, is a rare, life-threateninggenetic disorder with no available treatment. In the trial, patients whoreceived 0.5 mg/kg of I2S on a weekly basis showed a statistically significantimprovement in the primary efficacy endpoint (p=0.0049) compared to patientsreceiving placebo. Based on these results, TKT expects to file for regulatoryapproval of I2S in both the United States and Europe in the fourth quarter of2005.The primary efficacy endpoint of the trial, also referred to as the AIM study("Assessment of I2S in MPS II") was a composite endpoint of two clinicalmeasures previously used to assess clinical benefit in MPS disorders - forcedvital capacity and six-minute walk test. The mean improvement from baseline toweek 53 in percent predicted forced vital capacity was 3.4% in patientsreceiving I2S compared to 0.8% in patients receiving placebo. The mean increasefrom baseline to week 53 in the distance walked by patients receiving I2S was44 meters as compared to 7 meters in the placebo group.Joseph Muenzer, M.D., Ph.D., of the University of North Carolina at ChapelHill, an internationally recognized leader in the diagnosis and treatment ofMPS disorders and the lead investigator of the AIM study said, "These findingsare very encouraging for the medical and patient communities as we believeenzyme replacement therapy can bring new hope for patients and familiesaddressing many of the symptoms associated with Hunter syndrome."Treatment with I2S was generally well-tolerated by patients in the trial. Themost common adverse events observed were associated with the clinicalmanifestations of Hunter syndrome. Of the adverse events considered possiblyrelated to I2S, infusion related reactions were the most common and weregenerally mild. No patient withdrew from the trial due to an adverse eventconsidered related to I2S."We are extremely excited about the outcome of the study. In addition, we arevery thankful to all the patients and their families who participated in thisone year trial. Their commitment to this program was instrumental in generatingthe data which we believe will support regulatory approval of I2S," said KipMartha, M.D., Senior Vice President and Chief Medical Officer of TKT.TKT expects full data will be presented at a medical meeting in the autumn of2005.Trial DesignThe AIM study was a Phase III double-blind, placebo-controlled clinical trialconducted at nine sites around the world, including the United States, theUnited Kingdom, Germany and Brazil. The primary goal of the study was toevaluate the safety and efficacy of 0.5 mg/kg of I2S administered weeklycompared to placebo. Additionally, the trial evaluated 0.5 mg/kg of I2S everyother week compared to placebo. Ninety-six patients with Hunter syndrome wererandomized to one of three groups with each patient receiving a total of 52infusions of either I2S, I2S alternating weekly with placebo, or placebo. Ofthe 96 who enrolled, 94 completed the study and they all elected to participatein the open-label extension study of I2S at a dose of 0.5 mg/kg weekly.About I2S and Hunter SyndromeI2S is a human iduronate-2-sulfatase produced by genetic engineering technologyintended for long-term treatment of Hunter syndrome. TKT's I2S replaces anenzyme that is deficient in patients with Hunter syndrome, and therefore couldpotentially either stop or ameliorate the clinical manifestations of thedisease. TKT's I2S product has been designated an orphan drug in both theUnited States and the European Union. There is currently no effective therapyfor Hunter syndrome.Hunter syndrome is a hereditary disorder characterized by the body's inabilityto produce the enzyme iduronate-2-sulfatase, which is essential in thecontinuous process of replacing and breaking down glycosaminoglycans (GAG). Asa result, GAG remains stored in cells in the body causing progressive damage.The symptoms of Hunter syndrome are usually not visible at birth, but usuallystart to become noticeable after the first year of life. Often the firstsymptoms may include hernias, frequent ear infections, runny noses, andabnormal facial appearance.As the disease progresses, a variety of symptoms appear including, enlargedliver and spleen, heart failure, decreased endurance, obstructive andrestrictive airway disease, sleep apnea, joint stiffness, and, in some cases,central nervous system involvement. If central nervous system involvementexists, the life expectancy for patients with Hunter syndrome is typically10-15 years of age, however, some patients can survive into the fifth or sixthdecade of life. TKT believes there are approximately 2,000 patients worldwideafflicted with Hunter syndrome in countries where reimbursement may bepossible.Additional information about Hunter syndrome is available online at http://www.hunterpatients.com.For further information please contact:Investor Relations Clĩa Rosenfeld (Rest of the World) +44 1256 894 160 Brian Piper (North America) +1 484 595 8252 Media Jessica Mann (Rest of the World) +44 1256 894 280 Matthew Cabrey (North America) +1 484 595 8248 Notes to editorsShire Pharmaceuticals Group plcShire Pharmaceuticals Group plc (Shire) is a global specialty pharmaceuticalcompany with a strategic focus on meeting the needs of the specialist physicianand currently focuses on developing projects and marketing products in theareas of central nervous system (CNS), gastrointestinal (GI), and renaldiseases. Shire has operations in the world's key pharmaceutical markets (US,Canada, UK, France, Italy, Spain and Germany) as well as a specialist drugdelivery unit in the US.For further information on Shire, please visit the Company's website: www.shire.com."SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF1995Statements included herein that are not historical facts are forward-lookingstatements. Such forward-looking statements involve a number of risks anduncertainties and are subject to change at any time. In the event such risks oruncertainties materialize, Shire's results could be materially affected. Therisks and uncertainties include, but are not limited to, risks associated withthe inherent uncertainty of pharmaceutical research, product development,manufacturing and commercialization, the impact of competitive products,including, but not limited to, the impact of those on Shire's Attention Deficit& Hyperactivity Disorder (ADHD) franchise, patents, including but not limitedto, legal challenges relating to Shire's ADHD franchise, government regulationand approval, including but not limited to Health Canada's suspension ofADDERALL XR sales in Canada and the expected product approval dates ofMETHYPATCH⨠(MTS) (ADHD), SPD503 (ADHD), SPD465 (ADHD), SPD476 (ulcerativecolitis), SPD 480 (ulcerative colitis) and NRP104 (ADHD), including itsscheduling classification by the Drug Enforcement Agency in the United States,Shire's ability to secure new products for development and other risks anduncertainties detailed from time to time in Shire's filings with the Securitiesand Exchange Commission, including its Annual Report on Form 10-K for the yearended December 31, 2004Page 4 of 4Hampshire International Business Park Chineham Basingstoke Hampshire RG24 8EP United Kingdom Tel +44 (0)1256 894000 Fax +44 (0)1256 894708 www.shire.com Press Release Registered in England 2883758 Registered Office as aboveENDShire Pharmaceuticals Group PLCRelated Shares:
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