Antisense Pharma initiates Phase III brain tumor trial

The Sapphire study will be carried out in Europe, America and Asia. Approximately 70 hospital centers will participate. The Phase III study is designed as a randomized, active-controlled, open-label clinical trial with two treatment arms: trabedersen in a dose of 10 micromolar concentration will be compared to current standard therapy with either temozolomide or BCNU.

Clinical centers conducting the Sapphire study aim to enroll a total of approximately 130 adult patients with recurrent or refractory anaplastic astrocytoma. Trabedersen will be administered intratumorally via one single catheter using convection-enhanced delivery on an outpatient treatment basis. The treatment period lasts up to six months consisting of seven-day cycles every other week.

The primary efficacy endpoint is the survival rate at 24 months. Further efficacy endpoints include overall survival and time to death. The 14-month progression rate is the surrogate endpoint for an interim analysis.

The results of the previous randomized, active-controlled Phase IIb study show that the novel, targeted therapy holds much promise, said Antisense Pharma.

Karl-Hermann Schlingensiepen, CEO of Antisense Pharma, said: “We have taken trabedersen all the way from drug discovery to the pivotal Phase III clinical trial. The enrollment of the first patients in the Sapphire study is a key milestone in our drug development program for trabedersen.

“It is also in Phase I/II clinical development for advanced pancreatic carcinoma, malignant melanoma and colorectal carcinoma. Trabedersen with its unique mode of action can lead to a paradigm shift towards tackling malignant tumors at their roots while providing a better quality of life for patients.”