Research and Development Platform
Employing the antisense principle, the platform technology .R.A.D.A.R.
enables the rational design of synthetic oligonucleotides to act as mRNA antagonists. This unique technology allows the explicit selection of molecules for disease-specific targets. This systematic selection of a few so-called oligonucleotide sequences replaces time-consuming and expensive conventional screening approaches. The .R.A.D.A.R. technology thereby enables straightforward and efficient development of novel antisense drugs.
Antisense drugs belong to a new generation of so-called informational drugs. In contrast to conventional medicines, their specificity is based on their sequence rather than on their molecular structure. This is why various antisense drugs – although designed for different clinical applications – have comparable properties with regard to their pharmaceutical manufacturing, pharmacology and safety profile and thus facilitate cost-effective development and production
Our Product Pipeline
Antisense Pharma develops antisense technology-based drugs for the targeted treatment of cancer and other as yet incurable diseases. Our main therapeutic approach is to foster the body’s own immune response against tumor development and invasion by targeting oncogenes such as transforming growth factor–beta (TGF-beta).
Our lead product, trabedersen (AP 12009), is currently being investigated in an Phase III clinical trial, known as the ‘SAPPHIRE’ study, for the treatment of adult patients with recurrent or refractory anaplastic astrocytoma, a highly malignant brain tumor (WHO grade III).
Trabedersen is also being tested in a clinical Phase I/II study for the treatment of advanced pancreatic carcinoma and malignant melanoma.
A clinical Phase I/II trial for the treatment of colorectal cancer has been closed already.
Trabedersen selectively inhibits the production of the tumor factor TGF-beta2 (TGF-β2), essential the onset and progression of a variety of highly aggressive tumors. Trabedersen has been shown in clinical trials to decrease tumor size or tumor progression and to increase survival time of patients with with different cancers.
The tumor-promoting factor TGF-beta1 (TGF-β1) plays the most prominent role in the proliferation and metastazation of lung and prostate cancer. For the treatment of these cancers, Antisense Pharma is developing the drug AP 11014, which is currently in an advanced stage of pre-clinical testing.
The antisense drug AP 15012 for the treatment of other cancers further expands our product pipeline. Additional molecules and other drug technologies for cancer and non-cancer indications are in the discovery stage.
Mode of action of trabedersen (AP12009) in Cancer Treatment
Malignant brain tumors (high-grade glioma), pancreatic cancer and malignant melanoma are among the most aggressive tumors known. Antisense Pharma is vigorously testing its product trabedersen (AP 12009) in different clinical studies with the aim to meet the unmet medical need for effective and innovative approaches to treat these cancers. Trabedersen downregulates the generation of one of the most important tumor-promoting factors - TGF-beta2 (TGF-β2) - at the molecular level. With its proangiogenic and promigratory effects, TGF-β2 plays a pivotal role in explosive tumor progression and the aggressive spread of tumor cells throughout the body (metastazation). In addition, tumor-derived TGF-β2 overexpression builds up a protective shield around the tumor. This shield results in an inactivation of immune cells and allows the tumor to escape the body’s immune surveillance. The tumor cells can spread unhindered throughout the body. The antisense drug trabedersen blocks the translation of the TGF-β2 mRNA and thus downregulates its synthesis, destroying the protective shield around the tumor. Further tumor growth is consequently inhibited and the body’s own immune system is reactivated, enabling the immune cells to combat the cancer cells.
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