Clinical Proof of Concept for TGF-beta 2-Inhibitor AP 12009 in Phase Iib EMEA provides Guidance on Phase III Design and Approval Re...(or 10 μM AP 12009 inrecurrent or refrac...),Health

An international, randomized, active-controlled, Phase III study to evaluate the efficacy and safety of AP 12009 as monotherapy in adult patients with recurrent or refractory anaplastic astrocytoma will start in Q3 2008. The study drug will be compared to standard chemotherapy with either Temozolomide or BCNU. Antisense Pharma plans to conduct an interim analysis using the progression rate at 14 months as the surrogate endpoint. Positive results will allow submission for conditional approval by the EMEA. The SAWP at the EMEA indicated that, due to the rarity of the disease, the surrogate endpoint would be accepted, provided that the response data are supported by survival data. The fixed timepoint analysis at 24 months will be acceptable as the primary endpoint for full approval. Furthermore, a time-to-event analysis will be performed. "We are very pleased with this explicit statement by the Scientific Advice Working Party to proceed as we have planned. We sincerely hope that this will accelerate access to treatment for patients in need," says Dr. Hubert Heinrichs, Chief Medical Officer of Antisense Pharma. The Phase III study aims to enroll recurrent or refractory anaplastic astrocytoma patients in about 50 centers in 12 countries.

In the international randomized, active-controlled Phase IIb study, reported at ASCO, recurrent or refractory glioblastoma patients also showed long-lasting tumor responses in the AP 12009 groups (2). As of April 2008 the odds ratio for 10 μM AP 12009 versus control is 0.54. The risk to die is thus reduced by approximately 50% for glioblastoma patients receiving the study drug, as compared to patients under standard chemotherapy treatment. "Based on these data, a pivotal clinical trial in glioblastoma patients with good prognosis, for e
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