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Cell Medica Announces Orphan Drug Designation in the European Union for Cytovir ADV

BIOGY

Cell Medica Limited today announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products has issued a positive opinion on an application for orphan designation of a novel T cell immunotherapy under development by the company. The cell therapy, Cytovir ADV, targets the treatment of adenovirus infections in patients following allogeneic hematopoietic stem cell (bone marrow) transplant and is comprised of adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo. Adenovirus infections are a frequent cause of complications and death in patients, particularly children, who have received bone marrow transplants.

Cytovir ADV is based upon a new paradigm for the treatment of infections in patients following bone marrow transplantation. Derived from T-cells which provide immunity to the virus in the bone marrow donor, Cytovir ADV is being investigated as a novel method for transferring adenovirus immunity from the donor to the patient following the bone marrow transplant procedure. These patients are often profoundly immuno-compromised for a period of 6-9 months after the procedure and hence vulnerable to life-threatening infections. No antiviral drugs are currently approved for adenovirus infections post transplant. Cell Medica is developing a related product, Cytovir CMV, for cytomegalovirus infections in the same patient group.

Cytovir ADV is currently being investigated in the ASPIRE Phase I/II clinical trial under way at Great Ormond Street Hospital (GOSH) in London, one of the world’s leading centres for bone marrow transplantation in children.

“Cytovir ADV represents a potential breakthrough for the treatment of life-threatening adenovirus infections in children following a bone marrow transplant and we are very excited about the continued progress for this cell therapy,” said Gregg Sando, Chief Executive Officer of Cell Medica. “Receiving orphan designation for Cytovir ADV in the European Union is a significant milestone for Cell Medica. The benefits include 10 years of market exclusivity from product launch in the EU as well as fee reductions.”

The development of Cytovir ADV and the ASPIRE Phase I/II Clinical Trial have been supported by collaborative research grants from the UK’s innovation agency, the Technology Strategy Board.

About Cytovir ADV

Cytovir ADV is comprised of naturally occurring T lymphocyte cells (T cells) which demonstrate immune response functions when exposed to adenovirus antigens. The ASPIRE trial will explore the safety and activity of adenovirus-specific T cells infused in paediatric patients. The development program will establish whether Cytovir ADV reconstitutes immediate and long-lasting immunity against the virus, thereby potentially avoiding the medical costs, hospitalization and mortality associated with adenovirus infections in this patient group.

About Cell Medica

Cell Medica is a cellular therapeutics company engaged in the development, manufacturing and marketing of T cell immunotherapy approaches for the treatment of infections and cancers related to oncogenic viruses. The Company’s lead cell product, CytovirTM CMV, for the treatment and prevention of cytomegalovirus (CMV) infections in patients following allogeneic bone marrow transplant, is available in the UK and is under development in other territories. Cytovir ADV is being developed for the treatment of adenovirus infections in pediatric patients following bone marrow transplantation. Cell Medica is also working with the Center for Cell and Gene Therapy, Baylor College of Medicine, to develop an autologous T-cell product, CMD-003, for the treatment of malignancies associated with the oncogenic Epstein Barr Virus, including lymphomas and nasopharyngeal carcinoma.

About Great Ormond Street Hospital for Children

Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH) is the country’s leading centre for treating sick children, with the widest range of specialists under one roof. With the UCL Institute of Child Health, GOSH represents the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future.

About Technology Strategy Board

The Technology Strategy Board is the UK’s innovation agency. Its goal is to accelerate economic growth by stimulating and supporting business-led innovation. Sponsored by the Department for Business, Innovation and Skills (BIS), the Technology Strategy Board brings together business, research and the public sector, supporting and accelerating the development of innovative products and services to meet market needs, tackle major societal challenges and help build the future economy. For more information please visit www.innovateuk.org.

About Orphan Designation in the EU

The EU’s Orphan Medicinal Product Designation is designed to promote the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. Following approval for the treatment of adenovirus infections in patients post bone marrow transplant, Cytovir ADV will enjoy 10 years exclusivity in the market. The designation also provides eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.

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