Oncos Therapeutics granted orphan drug designation for CGTG-102 in soft tissue sarcoma (STS) by FDA and EMA
Oncos Therapeutics Ltd, a private, clinical-stage biotechnology company focused on the development and commercialization of targeted oncolytic immunotherapies for solid tumors, announced today that the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted orphan drug designation for CGTG-102, a granulocyte-macrophage colony stimulating factor-coding oncolytic adenovirus (Ad5/3-D24-GMCSF) for the treatment of soft tissue sarcoma.
“We are pleased to receive orphan drug designations for CGTG-102 from the two leading regulatory agencies in the world. These designations confirm the need for developing novel therapies for rare cancers and are an important step in the development of CGTG-102″, said Frans Wuite, M.D., President and Chief Executive Officer of Oncos.
Orphan Drug Designation applies to drugs that seek to treat rare diseases or conditions for which there may be few adequate therapies. In USA, the designation will provide Oncos with the opportunity of seven years of marketing exclusivity, grant funding to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential waiver of the FDA’s application user fee. Similarly, incentives are provided for Europe by EMA, which include fee reductions on future activities and the potential for 10 years market exclusivity.
About Soft Tissue Sarcoma
Soft tissue sarcoma is estimated to affect 3-4 in 10,000 people in the EU and US.
About 50% of soft tissue sarcomas are found in the early stage and advanced stage patients have an average overall survival of 13 months with today’s standard of care. Several efforts to develop additional chemotherapeutics for STS have failed, including two products in Phase III development this year. Novel therapies with a different mechanism of action are therefore needed.
Oncos’ lead product candidate, CGTG-102, is an oncolytic immunotherapy based on a purposefully modified, GM-CSF encoding adenovirus that selectively replicates in tumor cells and eventually kills them. Upon tumor cell death, newly synthesized viruses are released and infect neighboring tumor cells. Simultaneously, tumor antigens are exposed activating a tumor specific, systemic response by the patient’s immune system. This response is further enhanced by the CGTG-102 induced production of GM-CSF in tumor cells.
A Phase I study with CGTG-102, will be completed this year and Phase II studies are scheduled to start in 2014. Prior to the Phase I study, 115 patients with chemotherapy refractory solid tumors had been treated with CGTG-102 in an individualized treatment program regulated by the Finnish Medicines Agency as determined by the EU Regulation on Advanced Therapy Medicinal Products, EC/1394/2007.