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 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.
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
“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
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