Milan (Italy), 10 December 2012 – MolMed S.p.A. (MLM.MI) announces the presentation of the long term clinical benefit and safety data of its investigational cell-based therapy TK at the 54th Annual Meeting of the American Society of Hematology (ASH) in Atlanta (GA, U.S.).
The overall analysis comprises 128 patients with high-risk haematological malignancies who were treated in different countries in 10 Phase I-II clinical trials with TK cells, i.e. genetically engineered donor T cells administered after haematopoietic stem cell transplants from healthy donors to improve the graft-versus-leukaemia effect and to hasten patient immune reconstitution. Notably, no adverse events correlated to the use of TK cells were ever reported. Graft versus Host Disease (GvHD) was reported in 28 patients which, in all cases was rapidly and completely controlled thanks to the TK technology without the need of high-dose immunosupression following the transplant. These results confirm the feasibility, safety and effectiveness of this approach on a large patient population.
Most importantly, 34 patients – predominantly enrolled in MolMed’s Phase I/II study TK007 – who did not find a fully-compatible donor and received TK cells from a partially-compatible (haplo-identical) donor. Out of those, 25 patients quickly achieved immune reconstitution and nine patients are alive and completely disease-free after a median follow-up of 7 years. Also in these patients GvHD, when occurring, was always controlled thanks to the TK technology without the need of further pharmacological treatment.
These long-term clinical data are relevant in light of the severe disease status and the advanced age of the patient population treated. The results further confirm the overall high benefit/risk ratio of the TK cell therapy and, most relevantly, outline its potential for curing high-risk patients.
MolMed is conducting an international pivotal randomised Phase III trial for high-risk leukaemia patients undergoing bone marrow transplants from haplo-identical donors, ongoing in Europe and the US. Moreover, based on this accumulated data and the rarity of the indication (TK has obtained Orphan Drug designation), the Company expects to file a request for Conditional Marketing Authorisation to the European Authority in mid-2013.
About Phase III trial TK008
TK008 is a pivotal randomised Phase III trial in adult patients affected by high-risk leukaemia undergoing transplant of haematopoietic stem cells collected from partially compatible family donors (haplo-transplant). The trial design has disease-free survival as the primary end-point – which includes both transplant-related mortality and disease relapse – evaluated on a patient population of 170 patients. The trial will compare the outcome of haplo-transplants with or without TK add-backs, with a 3:1 randomisation ratio in favour of the TK arm. Secondary end-points include overall survival, reduction of transplant-related mortality, safety and patients’ quality of life. (Trial identifier on ClinicalTrials.gov: NCT00914628).
TK is a cell therapy product, based on the use of genetically engineered donor T cells given after heamatopoietic transplants from healthy donors, and specifically from partially compatible family donors (haplo-transplants), for the treatment of high-risk leukaemia. Add-backs of TK have the potential to allow the retention of immune-protection and anti-leukaemia effects of donor T cells, while promptly controlling and abrogating the possible onset of Graft-versus-Host Disease (GvHD). TK has been granted Orphan Drug designation in both the European Union and the United States.This press release is written in compliance with public disclosure obligations established by CONSOB (Italian securities & exchange commission) resolution no. 11971 of 14.5.1999, as subsequently amended.
MolMed S.p.A. is a biotechnology company focused on research, development and clinical validation of novel anticancer therapies. MolMed’s pipeline includes two novel therapeutics in clinical development: TK, a cell-based therapy enabling bone marrow transplants from partially compatible donors, in Phase III in high-risk acute leukaemia; NGR-hTNF, a novel vascular targeting agent, in Phase III in malignant pleural mesothelioma and in Phase II in six more indications: colorectal, lung (small-cell and non-small-cell), liver and ovarian cancer, and soft tissue sarcomas. MolMed also offers top-level expertise in cell and gene therapy to third parties to develop, conduct and validate projects from preclinical to Phase III trials, including scale-up and cGMP production of clinical-grade viral vectors, and manufacturing of patient-specific genetically engineered cells. MolMed is headquartered at the San Raffaele Biomedical Science Park in Milan, Italy. The Company’s shares are listed on the main market (MTA) of the Milan Stock Exchange. (Ticker Reuters: MLMD.MI)
FONTE : MOLMED