MolMed TK ASCO New Data from the Pivotal Phase III Randomized Trial TK008

Presented at ASCO data on the first set of patients from the pivotal Phase III TK008 study: the survival benefit exceeds trial target in acute leukaemia patients

MolMed S.p.A. (MLM.MI) provided at the 50th ASCO annual meeting new data from the ongoing randomised pivotal Phase III study on its cell therapy product TK for high-risk acute leukaemia patients transplanted from partially matched (haplo-identical) donors.

The intent-to-treat analysis of the first 24 patients treated with TK indicates a 74% 1-year disease free survival (DFS) as the primary study endpoint: this result largely exceeds the target of 52% DSF for the TK arm vs 30% for the control arm.

Notably, 86% of patients treated with TK were alive at one year (the key secondary study endpoint) and the corresponding figures for patients who achieved immune reconstitution rose to 85% for disease free survival and 100% for overall survival.

The direct impact of TK cells on transplant outcome was confirmed by a very low incidence of relapse (16% - with no relapse in patients receiving higher TK cell doses) and non-relapse mortality (10% - with no deaths observed in patients achieving immune reconstitution).

Claudio Bordignon, Chairman and CEO of MolMed, commented: "We are particularly proud of the results presented today at the ASCO meeting on the pivotal Phase III trial of the TK cell therapy for acute high risk leukemia patients transplanted from partially matched family donors. When we started this study, our goal was to provide all patients affected by the most aggressive forms of leukemia with the opportunity to undergo bone marrow transplantation, by far their best chance for a permanent cure. For this purpose we developed the TK therapy that allows patients lacking a fully matched donor to access a safer transplantation from partially matched family donors, an approach available to nearly all patients. TK is based on the genetic engineering of the donor immune system that can offer a graft without post-transplant immune-suppression and provide for a rapid and complete reconstitution of the immune system. This therapeutic strategy is characterized by low infection risk, low leukemia relapse rate and, consequently, by a higher probability of cure. Today we report data on the first patients enrolled in this study with an outcome that exceed the target of the trial design. These results strongly support our commitment to complete this large pivotal Phase III study. Moreover, the relevance of the data presented at ASCO is an important validation of our strategic decision to pursue Conditional Marketing Authorization for TK in Europe".

TK MolMed Asco Maggio 2014
MolMed has been investing on the TK cell therapy that represents today the largest clinical experience of immuno-gene therapy of tumours worldwide. This extensive program was carried out through the implementation of a centralised manufacturing in a single facility for global distribution. This experience, coupled with the new facilities built by MolMed, represent today an ideal platform for all new technologies of tumour immune-gene therapy based on the different strategies of genetic engineering of the immune system.

The Company would like to thank all patients and their families who placed confidence in this new technology and all the investigators from all transplantation centres who participate the Phase III trial and who contributed to the previous studies.