The European Medicines Agency (EMA) has granted Orphan Drug Designation to THERAVECTYS'  therapeutic vaccine candidate for the treatment of Adult T-cell Leukaemia/Lymphoma (ATL/L).

ATL/L is a rare malignancy of a certain type of white cells, T-Lymphocytes, caused by the Human T-Cell Lymphotropic Virus type 1 (HTLV-1) with a prevalence up to 1 per cent of the overall population in Japan. Approximately 5 per cent of all patients infected with HTLV-1 will develop ATL/L, in their lifetime.

Four ATL/L subtypes have been described, with a poor prognosis (less than a year for the two most aggressive forms). Patients are confronted with a lack of well-tolerated and/or performant treatment options which, to-date, include biological treatments with serious adverse reactions as well as aggressive chemo and antiviral therapies or, when eligible, long and uncertain hematopoietic stem cell transplantation.

The biological compound is an investigational therapeutic vaccine aiming at inducing an immune response against HTLV antigens born by ATL/L with the aim of enabling the patients’ immune system to fight leukaemic cells.

“On World Cancer Day 2015, we are proud to be one of the most innovative players in the immuno-oncology space, developing a new kind of treatment for more sustainable remission. In our continued effort to improve patient care, we are moving to the next stage in our fight against cancer with the launch of our clinical trial for virally-induced Adult Leukaemia/Lymphoma,” Says Renaud Vaillant, chief executive officer.

“Preclinical immunogenicity results obtained to-date are very promising and we are really excited by the perspective bringing a safe and better-tolerated alternative to patients who are desperately in need of a treatment,” says Déborah REVAUD, senior scientist in charge of the development project.

In Europe, the Orphan designation is granted to drugs in development intended for the treatment, the prevention or the diagnosis of life-threatening or chronically-debilitating diseases of a prevalence lesser than 5 in 10,000 people. The designation allows sponsors to benefit from an accelerated development process as well as incentives and a 7 years market exclusivity once the drug is placed on the market.

“We are extremely pleased that the European Medicines Agency has granted an Orphan Drug status to our vaccine candidate against ATL/L,” says Emmanuelle Sabbah-Petrover, Head of Regulatory Affairs. “This is a significant milestone for THERAVECTYS and we intend to take full advantage of all incentives associated with this designation to pursue and further accelerate the development of the compound. We expect to recruit our first patients towards the end of Q3 2015 in Europe and advance further developments in the US and in Japan in 2016.”

Should the vaccine candidate demonstrate a convincing safety and efficacy profile during its development against ATL/L, the company is already considering the perspective of further developing the same vaccine candidate for HTLV-related infections as a therapy and possibly as a prophylactic approach.

THV02 is an experimental treatment composed of two lentiviral vectors to be used in a prime/boost regimen in ATL/L patients infected by the HTLV-1 virus. Both investigational drugs encode the same antigens, derived from four proteins of the HTLV-1 virus.

During preclinical evaluation, THV02 has demonstrated to be safe and has presented an unprecedented immunogenicity profile in several models. THERAVECTYS is privately-owned, fully-integrated discovery and clinical development biotech company originating from the Pasteur Institute.

The company capitalises over 15 years of fundamental research in the field of lentiviral vectors and has secured worldwide exclusive rights to Pasteur Institute intellectual property. Based on its lentiviral vector technology platform, THERAVECTYS develops therapeutic vaccines and immunotherapies to fight cancers and infectious diseases, including a proprietary and differentiated CAR T-cell technology platform.

The company is strongly supported by renowned investors and former global biopharmaceutical executives and is rapidly progressing in its R&D activities and in-house GMP production capabilities. Alone and in collaboration with partners, THERAVECTYS is accelerating its clinical development programmes and is planning to initiate two additional phase I/II clinical trials in oncology in 2015.

THERAVECTYS is privately-owned, fully-integrated discovery and clinical development biotech company originating from the Pasteur Institute. The company capitalizes over 15 years of fundamental research in the field of lentiviral vectors and has secured worldwide exclusive rights to Pasteur Institute intellectual property.