International clinical trial for patients with brain tumours, co-ordinated at The Neuro

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 μM 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 (The Neuro), McGill University. Professor 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-b2). Unlike non-specific therapies, e.g. chemotherapy or radiotherapy, targeted therapies act much more specifically at the molecular basis of the disease. Commanding up to 80% of the growing oncology market, the targeted therapies like trabedersen are substantially driving the growth of the pharmaceutical market.1 A successful marketing authorization would make trabedersen the first TGF-beta targeting drug for the treatment of cancer.

Combating cancer at its roots

“We have taken trabedersen all the way from drug discovery to the pivotal Phase III clinical trial. The approval of the SAPPHIRE study by Health Canada is an important step in our efforts to make trabedersen available to those who need it as quickly as possible. Trabedersen is also in Phase I/II clinical development for advanced pancreatic carcinoma, malignant melanoma and colorectal carcinoma. With its unique mode of action, we believe that trabedersen has the potential to lead to a paradigm shift towards tackling malignant tumors at their roots while providing a longer life and a better quality of life for patients,” commented Dr Karl-Hermann Schlingensiepen, Chief Executive Officer of Antisense Pharma.

About the Montreal Neurological Institute and Hospital

Celebrating 75 years

The Montreal Neurological Institute and Hospital (The Neuro) is a unique academic medical centre dedicated to neuroscience. The Neuro is a research and teaching institute of McGill University and forms the basis for the Neuroscience Mission of the McGill University Health Centre. Founded in 1934 by the renowned Dr. Wilder Penfield, The Neuro is recognized internationally for integrating research, compassionate patient care and advanced training, all key to advances in science and medicine. Neuro researchers are world leaders in cellular and molecular neuroscience, brain imaging, cognitive neuroscience and the study and treatment of epilepsy, multiple sclerosis and neuromuscular disorders. For more information, please visit www.mni.mcgill.ca.

For more information on the SAPPHIRE trial please visit the website www.anticancer.de