REGENSBURG, Germany, Oct. 19 /CNW/ -- The biopharmaceutical company Antisense Pharma GmbH has announced today that it has received the approval by Health Canada for its pivotal Phase III clinical trial SAPPHIRE in patients with recurrent or refractory anaplastic astrocytoma.

The SAPPHIRE study is a randomized, active-controlled, clinical trial designed to confirm the efficacy and safety of the investigational drug trabedersen (AP 12009), observed in previous clinical studies. Trabedersen is being investigated as monotherapy compared to current standard therapy with temozolomide (alternatively BCNU (carmustine)). The results of a previous randomized, active-controlled Phase IIb study show that the novel, targeted therapy holds significant promise. Currently recruiting study centers will be published on www.anticancer.de.

International Phase III study design

The SAPPHIRE study is being carried out in Europe, North, Middle and South America along with Asia. Up to 100 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 umol will be compared to current standard therapy with temozolomide (alternatively BCNU (carmustine)). 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 (CED) on an outpatient treatment basis. The treatment period lasts up to 6 months consisting of 7-day cycles every other week.

Study objectives

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. Safety parameters will include adverse events, serious adverse events, ECG parameters, neurological examination and vital signs. Also the patient's quality of life is an important parameter of the study.

Great need for new approaches in cancer therapy

"The diagnosis of recurrent or refractory anaplastic astrocytoma is devastating since the therapeutic possibilities available to treat such patients are inadequate. The life expectancy of such patients is unfortunately very limited. Hospitals worldwide are committed to improve this situation in the framework of the SAPPHIRE study. The results of the previous studies show that this new, targeted therapy holds significant promise", remarked Dr. Rolando Del Maestro, Director of Brain Tumor Research at the Montreal Neurological Institute and Hospital, McGill University. Dr. Del Maestro is the World Coordinating Investigator of the SAPPHIRE study.

Targeted therapies drive market growth

Trabedersen is a first-in-class targeted therapy. This novel compound acts multimodally via inhibition of transforming growth factor-beta 2 (TGF-beta-2). Unlike non-specific therapies, e.g. chemotherapy or radiotherapy, targeted therapies act much more specifically at the molecular basis of the disease.