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news.Genzyme, a biotechnology company, has reported that the Phase II clinical trial of its investigational oral therapy Genz-112638 for Gaucher disease type 1 met its primary endpoint.
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The study had a composite primary efficacy endpoint: a clinically meaningful response in at least two of three endpoints in individual patients after the 52-week study period. The ongoing open-label study concluded in October 2008. Approximately 22 of 26 study participants completed at least 52 weeks of treatment. After the study concluded, all 20 of the patients who were eligible chose to remain on treatment.
More than half of the participants have completed more than two years of treatment. Medical centers in North America, South America, Europe, Asia and the Middle East are participating in this study. The data for the 52-week analysis indicate that Genz-112638 demonstrated robust efficacy results in that 91% of those who completed 52 weeks achieved the primary endpoint, the company said.
The results approach those observed in patients after one year of Cerezyme treatment. Patients in the trial continue to be treated and longer-term efficacy data are currently being collected, the company added. The safety analysis demonstrated that Genz-112638 was well tolerated and has a promising safety profile.
The company said that it is developing Genz-112638, a capsule taken orally, to provide a convenient treatment alternative for adult patients with Gaucher disease type 1, and to provide a broader range of treatment options for patients and physicians to achieve individual therapeutic goals. Currently, Genzyme’s Cerezyme, the standard of care for patients with Gaucher disease type 1, is administered through intravenous infusions.
Genzyme is currently finalizing protocols for two Phase III trials that it expects to initiate in mid-2009. One trial will include untreated patients with Gaucher disease type 1, and the other a switch trial in which patients who have achieved their therapeutic goals with Cerezyme will be maintained.
Geoff McDonough, senior vice president of Genzyme, said: As we progress through the clinical development plan, we believe that Genz-112638 will become a meaningful treatment option for the management of Gaucher disease type 1, offering patients and physicians more flexibility to individualize therapy for optimal management of the disease.