Adaptimmune Receives Orphan Drug Designation in the European Union for its NY-ESO SPEAR® T-cell Therapy for Treatment of Soft Tissue Sarcoma

Adaptimmune previously received orphan drug destination from the U.S. Food and Drug Administration for its NY-ESO SPEAR T-cell therapy in this indication.

"We are delighted to have received the European Commission's orphan medicinal product designation," said Dr. Rafael Amado, Adaptimmune's Chief Medical Officer. "We look forward to progressing our NY-ESO SPEAR T-cell therapy through clinical trials with the goal of eventually making this product available in Europe to patients with advanced soft tissue sarcoma."

Orphan drug designation by the European Commission provides certain regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. The designation provides incentives for companies seeking protocol assistance and scientific advice from the European Medicines Agency (EMA) during the product development phase and a 10-year period of marketing exclusivity in the EU following product approval.

Data from recent published epidemiological studies estimate the prevalence of soft tissue sarcoma in the EU to be 2.86 per 10,000 which corresponds to approximately 146,918 people based on the total population of 513.7 million people in the EU, Norway, Iceland, and Liechtenstein as of January 1, 2015 [EUROSTAT 2015].

The European Commission's decision follows the positive opinion adopted by the EMA's Committee for Orphan Medicinal Products recommending Adaptimmune's SPEAR T-cell therapy targeting NY-ESO for designation as an orphan medicinal product for the treatment of soft tissue sarcoma announced by Adaptimmune on June 20, 2016.

About Soft Tissue Sarcoma
Soft tissue sarcomas can develop from soft tissues including fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues. There are approximately 50 types of soft tissue sarcomas, including synovial sarcoma, a cancer of the connective tissue around the joints. Soft tissue sarcomas can develop at almost any anatomic site, such as the extremities, trunk or thorax, abdomen and retroperitoneum, pelvis and the head and neck region. The more common soft tissue sarcomas originate from muscle, nerve tissue, fat, or deep skin tissue. For a number of sarcomas, such as synovial sarcoma, the tissue origin is not well characterized. Surgical resection is the standard therapy for localized disease and radiation therapy (preoperative or postoperative) and/or chemotherapy is added in selected cases. 

About Adaptimmune
Adaptimmune is a clinical stage biopharmaceutical company focused on novel cancer immunotherapy products based on its SPEAR ^®  (Specific Peptide Enhanced Affinity Receptor) T-cell platform. Established in 2008, the company aims to utilize the body's own machinery - the T-cell - to target and destroy cancer cells by using engineered, increased affinity TCRs as a means of strengthening natural patient T-cell responses. Adaptimmune's lead program is a SPEAR T-cell therapy targeting the NY-ESO cancer antigen. Its NY-ESO SPEAR T-cell therapy has demonstrated signs of efficacy and tolerability in Phase 1/2 trials in solid tumors and in hematologic cancer types, including synovial sarcoma and multiple myeloma. Adaptimmune has a strategic collaboration and licensing agreement with GlaxoSmithKline for the development and commercialization of the NY-ESO TCR program. In addition, Adaptimmune has a number of proprietary programs. These include SPEAR T-cell therapies targeting the MAGE-A10 and AFP cancer antigens, which both have open INDs, and a further SPEAR T-cell therapy targeting the MAGE-A4 cancer antigen that is in pre-clinical phase with IND acceptance targeted for 2017. The company has identified over 30 intracellular target peptides preferentially expressed in cancer cells and is currently progressing 12 through unpartnered research programs. Adaptimmune has over 250 employees and is located in Oxfordshire, U.K. and Philadelphia, USA. For more information: http://www.adaptimmune.com

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