Novel cellular therapy, CMD-003, granted orphan drug designation for treatment of EBV-positive non-Hodgkin lymphomas • First patient treated with CMD-003 in CITADEL Phase II clinical trial
London, UK, 18 March 2015 – Cell Medica, a leading cellular immunotherapy company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the Company’s cancer immunotherapy treatment, referred to as CMD-003, under development for Epstein-Barr Virus (EBV) positive non-Hodgkin lymphomas. Additionally, the company announced the treatment of the first patient in the CITADEL Phase II clinical trial investigating the safety and efficacy of CMD-003 for the treatment of aggressive extranodal NK/T cell lymphoma (ENKTCL) in patients who have failed previous therapies. The patient was treated at the Dana-Farber Cancer Institute in Boston, MA under the care of Dr Eric Jacobsen.
Dr Jacobsen, clinical director of the adult lymphoma program at Dana-Farber Cancer Institute and an assistant professor of medicine at Harvard Medical School, said: “Extranodal NK/T cell lymphoma can be a difficult disease to treat. Until recently there has been limited research devoted to this relatively rare disease. We are very excited to have treated the first patient in the CITADEL trial. Cytotoxic T cells are an exciting therapeutic area in oncology and I am hopeful that this approach will offer an effective new treatment option for these patients.”
CMD-003 is a novel way to target a range of cancers associated with the oncogenic Epstein-Barr Virus (EBV). The treatment has been developed by Cell Medica in collaboration with the Center for Cell and Gene Therapy (CAGT) at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital. CMD-003 is produced from patient immune cells taken from a blood sample and sent to the Company’s manufacturing site for activation and expansion through a proprietary procedure developed for commercial-scale use. CAGT (Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital) has treated more than 250 patients with research prototypes of CMD-003 and has reported promising clinical results across a range of malignancies.
The CITADEL Trial will include NK/T cell lymphoma patients who have failed conventional treatment of advanced extranodal NK/T cell lymphoma. The trial will include patients recruited at 24 centers in five countries as part of a clinical development programme aimed at regulatory approval in the USA, Europe and South Korea.
Chief investigator Dr Helen Heslop, Interim Director of CAGT at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital, said: “It is very gratifying to see an innovative cell therapy approach, developed here by Dr Cliona Rooney and other CAGT investigators, progressing towards regulatory approval and clinical adoption.”
Gregg Sando, CEO of Cell Medica, commented: “The CITADEL trial will investigate a new approach for treating patients with EBV-associated NK/T cell lymphoma by harnessing the human immune system to target and kill the cancer. With the Orphan Drug Designation, we can look forward to FDA regulatory support and incentives for the development of this novel therapy. These are important steps which show we are making real progress with cellular immunotherapy as a powerful new technology with the potential to transform the treatment of cancer patients.”
The development of CMD-003 and the CITADEL Trial are funded in part by an investment from the Cancer Prevention and Research Institute of Texas (CPRIT) alongside Cell Medica’s other strategic investors including Imperial Innovations, Invesco, Woodford Investment Management and The Wellcome Trust.
Orphan Drug designation is granted by the FDA’s Office of Orphan Products Development for drugs that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits across all stages of drug development, which include accessibility to grants to support clinical development, seven years of market exclusivity following marketing approval and tax credits on U.S. clinical trials.
Cell Medica is a cellular therapeutics company engaged in the development, manufacturing and marketing of cellular immunotherapy products for infectious disease and cancer. The Company’s Cytovir™ product family for immune reconstitution is for the treatment of infections caused by latent viruses in immunosuppressed patients following allogeneic haematopoietic stem cell (bone marrow) transplant. In the field of cancer immunotherapy, Cell Medica is targeting a range of cancers associated with the oncogenic Epstein Barr Virus (EBV) including lymphoma and nasopharyngeal carcinoma.
The Center for Cell and Gene Therapy, based in Houston, Texas, was established by Baylor College of Medicine, Houston Methodist Hospital and Texas Children’s Hospital to provide a research infrastructure to rapidly translate novel cell and gene therapy protocols from the laboratory to the clinic.
CPRIT was established to expedite cancer research innovation and commercialization and to enhance access to evidence-based prevention programs and services throughout the State. Company commercialization awards are a critical part of the Institute’s initiative to support the advancement of the most promising opportunities towards regulatory approval and market launch.
EBV is a ubiquitous virus infecting 90% of the human population and resides in a latent state in B cells and epithelial cells of the nasopharynx. The association between EBV infection and certain cancers has been well established in subtypes of lymphoma, nasopharyngeal carcinoma and gastric cancer. The expression of EBV antigens by tumor cells provides the opportunity to use the patient’s own immune cells to target and kill the cancer.
Extranodal NK/T-cell lymphoma (ENKTCL) is a rare type of non-Hodgkin lymphoma (NHL) recognized as a separate entity by the World Health Organization. ENKTCL is almost always positive for Epstein-Barrvirus (EBV). The incidence in the US is approximately 650 cases per annum with an estimated prevalence of 4,500 patients but higher in Asia where it represents 6% – 9% of NHL. It is an aggressive lymphoma with median survivals of 13-42 months and 5-year overall survivals (OS) of 20% to 65%.
T cell immunotherapy involves harnessing the power and precision of the human immune system to treat disease. Extensive academic research in the field of clinical immunology has provided early evidence that T cells can be used as a cellular therapeutic for the treatment of viral infections. Certain cancers are also associated with infection by viruses such as the Epstein Barr Virus (EBV). Ongoing clinical research at UCL, Baylor College of Medicine and other institutions is exploring how T cell immunotherapy may be used to eliminate malignant cells which express viral antigens.
18th March 2015