Oxford BioMedica, a leading gene therapy company, said its partner MolMed has received regulatory approval to start a phase III trial of TK therapy for high risk acute leukaemia. The start of the trial triggers an undisclosed milestone payment to Oxford BioMedica.

Under a license agreement with Oxford BioMedica, MolMed's TK therapy product employs Oxford BioMedica's retroviral ex vivo gene delivery technology.

AIFA, the Italian Health authority, authorised the start of a Phase III trial (TK008), in Italy, of MolMed's TK cell therapy in patients affected by high risk acute leukaemia and receiving haematopoietic stem cell transplantation from a partially compatible family donor (haplo-HSCT). AIFA clearance, the very first one in Italy for a phase III of a cell/gene therapy, requires the completion of analytical characterisation of TK components within the treatment of 20 per cent of patients involved in the study, and follows approval of the clinical protocol, obtained in December 2007 by the Ethical Committee of the first clinical centre involved.

EMEA supported MolMed in preparing phase III, by providing protocol assistance and scientific advice as granted by the Orphan Drug designation of TK. The multicentric, randomised phase III trial will assess the very positive outcome of phase I/II trial TK007 that resulted in an exceptional improvement in survival of patients by promoting rapid and sustained immune reconstitution, and proved safety and efficacy of haplo-HSCT, thus enabling feasibility of transplantation from partially incompatible family donors.

Claudio Bordignon, president and chief executive officer, MolMed, said, "The remarkable results obtained in the phase I/II trial show the importance of TK therapy in making stem cell transplantation available for all candidate patients lacking a fully compatible donor, who are approximately 60 per cent of those who could benefit from this potentially curative treatment".

TK therapy is based on the use of genetically engineered (TK+) donor T lymphocytes, used in association with haplo-HSCT. TK+ donor lymphocytes can control the main complications associated with haplo-HSCT, while maintaining the anti-leukaemia effects of the transplant, thereby increasing both patients' survival and the number of available donors. Orphan drug designation for TK has been granted by the EMEA in 2003 and by the FDA in 2005. MolMed's strategic partner Takara Bio Inc. (Japan) is developing TK for the Asian markets.

Oxford BioMedica is a biopharmaceutical company specialising in the development and commercialisation of novel therapeutic vaccines and gene-based therapies with a focus on oncology and neurotherapy.