Shire presents positive data from TKT-025 EXT Phase I/II study for VPRIV in type 1 Gaucher disease patients

Shire plcJul 2 2010

Shire plc (LSE: SHP, Nasdaq: SHPGY), the global specialty biopharmaceutical company, today announced positive data from its long-term extension to its open label Phase I/II study (TKT-025 EXT) for VPRIV® (velaglucerase alfa), the company's enzyme replacement therapy for patients with type 1 Gaucher disease.  The data were presented at the 9th Annual European Working Group on Gaucher Disease (EWGGD) in Cologne, Germany, and add to the growing body of clinical evidence which supports the safety and sustained efficacy of VPRIV.

A post-hoc analysis was undertaken of TKT-025 EXT to evaluate the achievement of long-term therapeutic goals, including improvement in bone mineral density (BMD), among all 8 type 1 treatment naive Gaucher patients who participated in TKT-025 EXT and were treated for a minimum of 48 months.  The initial dose of 60 U/kg administered intravenously every other week (EOW) was lowered in a step-wise fashion to 45 U/kg EOW and then to 30 U/kg EOW after patients achieved at least 2 of 4 predefined therapeutic goals following 1 year of treatment.  Seven of the 8 patients were maintained at the lower dose.  All of the patients achieved the therapeutic goals for hemoglobin concentrations, platelet counts, liver and spleen volumes, and improvement in BMD within 4 years of initiation of treatment with VPRIV.

A separate post-hoc analysis of the change in BMD among patients in TKT-025 EXT through 69 months was also reported.  

"The data presented provide additional and compelling support that VPRIV is able to provide patients with continuous improvement of key therapeutic goals, including bone, in a clinically meaningful way," said Professor Ari Zimran from the Shaare Zedek Medical Center, Jerusalem, Israel.

TKT-025 EXT BMD Results Highlights Include:

• Among those treated with VPRIV alone, 3 patients with osteopenia (2 LS and 1 FN) achieved normal BMD and 1 patient with osteoporosis (FN) became osteopenic by 69 months.
• Among the ITT population, lumbar and femoral BMD improved significantly by months 24 and 33, respectively (LS: 0.39; 95%CI 0.06, 0.72 and FN: 0.39; 95%CI 0.16, 0.62)).
• At baseline, Z-scores among ITT patients were significantly lower than those seen in age and gender matched controls and showed significant improvement over time during VPRIV treatment (LS and FN p< 0.001 for all measures).
• Among VPRIV-only treated patients, significant improvement was seen over time [the Z-score LS y-intercept was -1.29>

Also presented at the EWGGD were data from a Phase III clinical trial (TKT-034) that demonstrated patients were safely transitioned to VPRIV (15-60U/kg EOW) from the same number of units of imiglucerase. Clinical measures of disease remained stable over 12 months.

TKT-034 Results Highlights Include:

• No patients developed IgG antibodies to VPRIV, including 3 patients who tested positive for anti-imiglucerase antibodies at screening.
• Hemoglobin concentrations, platelet counts, and liver and spleen volumes remained stable over the course of the one year study.

"We are very pleased with the opportunity to present these findings at the EWGGD, a key scientific meeting for the Gaucher community," said Ferd Massari, VP and Global Head of Clinical and Medical Affairs, Shire Human Genetic Therapies. "The data presented underscore the long term safety and efficacy of VPRIV for treatment naïve Gaucher patients, as well as for patients who switched from imiglucerase to VPRIV. Moreover, they demonstrate that VPRIV may offer benefits regarding the achievement of therapeutic goals, including improvements in bone parameters."