25th March 2009, 11:24 PM
|
 |
Administrator
|
|
Join Date: Mar 2009
Posts: 103
Thanks: 0
Thanked 2 Times in 2 Posts
|
|
Puricase
Press Release:
Savient Reports Additional Positive Trial Data for Secondary Endpoints from Puricase(R) (pegloticase) Phase 3 Studies
Quote:
EAST BRUNSWICK, N.J., Feb 04, 2008; Savient Pharmaceuticals, Inc. (NASDAQ:SVNT), today announced additional positive results for secondary efficacy endpoints in the two replicate Phase 3 studies for Puricase(R) (pegloticase) for treatment-failure gout. Results were favorable for the treatment effect on clinical outcomes assessed by the reduction in the number of tender and swollen joints, and in improvement in Patient Reported Outcomes (PRO) as measured by Short Form-36 (SF-36) and the Health Assessment Questionnaire - Disability Index (HAQ-DI). These endpoints were assessed in a pre-specified pooled analysis from the two replicate studies and are reported for the Intent-to-Treat population. The improvement reported in these outcomes was clinically meaningful as determined by the pre-specified definitions.
The company previously reported that pegloticase had met the pre-specified primary efficacy endpoint, i.e. normalization of plasma uric acid for every two week and every four week dose administration, independently, in each of the two Phase 3 studies. In addition, the 8 mg every two week dose arm attained statistical significance versus placebo in the pre-specified pooled analysis, a secondary endpoint, in the proportion of patients who had a "complete response" for the elimination of gout tophi. The treatment effect of pegloticase on gout flares, another secondary endpoint, was reported as a favorable numerical trend in both treatment arms versus placebo, but neither treatment arm attained significance. The assessment of safety across the two Phase 3 studies was reported to be favorable and to have shown that the only adverse safety signal was the occurrence of infusion reactions. Seven patients (2/85 in the Q2 week arms, 5/84 in the Q4 week arms) had a total of 11 severe infusion reactions (muscle cramps, back or chest pain). Approximately 25 percent of all patients in the Q2 and Q4 treatment arms experienced a mild or moderate infusion reaction.
Following on this previously reported data, additional analysis showed statistical significance for both the every two week and every four week treatment arms versus placebo was achieved for:
-- a reduction in the number of tender and swollen joints
-- a reduction in the number of tender joints
-- an improvement in the clinician's global assessment (a 10 cm Visual Analog Scale)
For both treatment groups, the degree of improvement was greater for patients defined as plasma uric acid responders than for non-responders, but uric acid non-responders appeared to show improvement versus placebo as well. Further analysis of this finding is ongoing.
Two Patient Reported Outcomes instruments were employed in the pegloticase program, Short Form-36 (SF-36) and the Health Assessment Questionnaire - Disability Index (HAQ-DI), which have been used extensively in Rheumatology studies to assess clinically meaningful change in various rheumatic diseases. The results of the Phase 3 trials show that in the pre-specified pooled analysis, both treatment arms attained statistical significance for improvement versus placebo for the SF-36 physical component scores. The Arthritis-Specific Health Index form SF-36 also demonstrated statistically significant improvement versus placebo for both treatment arms and again showed that even the plasma uric acid non-responders had improvement versus placebo. The SF-36 bodily pain scale also demonstrated statistical significance for both treatment arms versus placebo and also showed that the plasma uric acid non-responders appeared to improve but only about half as well as the responders.
The results of the HAQ-DI assessment on arthritis pain showed statistically significant improvement versus placebo for the Q2 week dose group. The patient's own assessment of their overall functioning, a component of the HAQ-DI, showed that both treatment arms attained statistically significant improvement versus placebo.
"These results provide valuable evidence of clinical benefit in our upcoming regulatory submissions and are expected to be presented in full detail in future scientific forums and in peer-reviewed publications," stated Zeb Horowitz, M.D., Sr. VP and Chief Medical Officer. "The strength of the evidence from the two GOUT trials has the potential to change the way physicians and patients view treatment-failure gout, a view currently centered almost exclusively on the unsuccessful control of uric acid and the acute symptomatic relief of painful gout flares, gradually leading to increasingly advanced disease. In light of the pegloticase data, gout treatment-failure patients and their physicians have reason to hope for a better treatment option."
The company is scheduled to have a pre-BLA meeting with the reviewing division of the U.S. Food and Drug Administration (FDA) on April 17, 2008 and plans to file the BLA with the FDA as soon as practicable following the pre-BLA meeting based on the positive results from its Phase 3 trials.
|
|
Linear Mode
