Replies to post #7535 on GTC Biotherapeutics (fka GTCB)

[DewDiligence]

“Seventeen additional patients have been treated, of which 16 are considered evaluable. One patient is considered unevaluable due to the course of treatment deviating from the clinical protocol. None of the additional patients had clinically relevant DVTs or other thromboembolisms during the evaluation period. On this basis, the study has already met the statistical requirements for non-inferiority. Although the primary endpoint has already been met, enrollment is being extended through February to include an additional patient to ensure that the minimum number of evaluable patient procedures agreed upon with the FDA is satisfied.”

Nice job, GTC.

[aslan2772]

This is good news, as expected. Congrats longs! The next hurdle for this trial is immunogenicity. I don't expect any significant problems there, but it is a bigger hurdle than efficacy, IMHO (only bad luck or inadequate trial design could have interfered with reaching the primary endpoint). GTCB has moved a step closer to profitability, which is fundamentally important since cash has long been their biggest concern.

Cheers.

[DewDiligence]

Last (17th) Patient Enrolled In ATryn Pivotal Study

[This is good news insofar as it keeps the ATryn BLA on the previously announced timeline. The study has already met its primary endpoint irrespective of what happens with the 17th patient.]

http://biz.yahoo.com/bw/080225/20080225005488.html?.v=1

>>
Monday February 25, 8:30 am ET

FRAMINGHAM, Mass.--(BUSINESS WIRE)--GTC Biotherapeutics, Inc. ("GTC", Nasdaq: GTCB) announced today that ATryn® has completed enrollment in the pivotal study for the treatment of patients with hereditary antithrombin deficiency, or HD, undergoing high-risk surgical or childbirth procedures. As previously announced, the pivotal study has met the primary endpoint of demonstration of non-inferiority to plasma-derived antithrombin in preventing clinically relevant deep vein thromboses, or DVTs, or other thromboembolisms.

A minimum of 31 evaluable HD patients were required for the ATryn® treatment trial. Results for 14 of these patients were already obtained from the previous study that supported ATryn®’s approval in the European Union for HD patients undergoing surgical procedures. Seventeen evaluable patients have been treated to complete the pivotal study. There were no clinically relevant DVTs or other thromboembolisms during the evaluation period in any of the patients treated in the pivotal study.

The previously completed comparator arm of the study is based on historical data gathered under a prospective clinical protocol from patients who had been previously treated with plasma-derived antithrombin products while undergoing similar high-risk procedures to those performed in the active arm. There were no clinically relevant DVTs or other thromboembolisms in these patients.

“Completing enrollment in this pivotal study is another important stepping stone in validating the application of our transgenic production technology to the development of recombinant protein therapeutics. We look forward to completing our rolling Biologics License Application as planned around mid-2008, when we submit the full clinical data package,” stated Geoffrey F. Cox, PhD, GTC’s Chairman and CEO. “ATryn® is unique in being the only recombinant form of human antithrombin to be developed for therapeutic use, as recognized in its orphan drug status for the HD indication.”

The full clinical data package will include 90-day follow-up tests for antibody generation to ATryn®, which is a typical regulatory requirement and was also part of the approval process successfully completed for the European Union.
<<