MolMed S.p.A. (Milan:MLM) announces that the Company has filed to the European Medicines Agency an application for Conditional Marketing Authorisation for TK, its novel investigational cell-gene therapy. TK is an adjunctive treatment in hematopoietic stem cell transplantation (HSCT) for patients affected by high risk leukaemia.
It is estimated that in EU approximately 5,000 leukaemia patients/year lack a fully compatible donor: TK offers to these patients the option of transplantation from a mismatched donor without post-transplant immunosuppression. If approved, TK would be the first cell-gene therapy product on the market for this life threatening condition.
The Conditional Marketing Authorisation application is supported by cumulative efficacy and safety results obtained from a completed Phase I-II, multicentre trial (TK007). These data were included in presentations given at the 49th annual congress of the American Society of Clinical Oncology (ASCO) and at the 55th Annual Meeting of the American Society of Hematology (ASH) in 2013. Results show that TK can reduce transplant-related mortality and increase overall survival in high risk leukaemia patients transplanted from a mismatched HSCT donor.
The application also includes initial data from the currently ongoing pivotal randomised Phase III study (TK008), which have been submitted to the next ASCO congress taking place in May in Chicago. The TK therapy was granted Orphan Drug Designation by the European Commission.
Claudio Bordignon, Chairman of the Board and CEO of MolMed, commented: ”With the submission of the Conditional Marketing Authorisation, we have achieved an important milestone which represents a turning point in the clinical development of our TK technology. We believe that by providing fast immune reconstitution and eliminating the need for post-transplant immune-suppression, TK may represent a new and powerful tool able to provide an efficacious treatment option for those high risk leukaemia patients who lack a fully matched donor. With more than 120 patients treated worldwide and an ongoing randomised Phase III trial, our efforts in the implementation of the TK technology witnesses our dedication to the treatment of leukaemia patients lacking a valid treatment option, and MolMed competitiveness in the challenging field of immune-gene therapy of cancer”.
Conditional Marketing Authorisation
The Conditional Marketing Authorisation represents an expedite path for early market authorisation ahead of completion of the pivotal registration studies. Such anticipated authorisation is mainly based on efficacy and safety evidences accumulated in early studies.
A Conditional Marketing Authorisation may be granted only if all the following requirements are met:
- the risk-benefit balance of the medicinal product is positive;
- it is likely that the applicant will be in a position to provide the comprehensive clinical data;
- unmet medical needs will be fulfilled;
- the benefit to public health of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required.
A Conditional Marketing Authorisation is valid for one year, on a renewable basis. The holder is required to complete ongoing studies or to conduct new studies with a view to confirming that the benefit-risk balance is positive.
TK is a cell therapy product, based on the use of genetically engineered donor T cells carrying a “suicide gene”. These cells are administered to patients during the haematopoietic stem cell transplantation for the treatment of high risk leukaemia. TK therapy allows to eliminate the post-transplant immunosuppression treatment thus accelerating the immune reconstitution and controlling the immunological consequences arising from the genetic differences with the donor, known as Graft versus Host Disease (GvHD).
In virtue of this approach, HSCT from partially compatible donors is a safer and more effective option, thus potentially increasing the number of candidates for transplantation.
TK008 is a pivotal randomised Phase III trial (TK008) in adult patients affected by high-risk leukaemia undergoing transplant of haematopoietic stem cells collected from partially compatible (haploidentical) family donors.
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.
With the aim to provide additional clinical benefit to patients and to significantly increase the potential participation of centres in the trial, the Company implemented in 2012 two important changes in the protocol design of Phase III trial TK008. The first consists in broadening the enrolment criteria to include patients in leukaemic relapse, in addition to those in disease remission; the second change provides for the introduction of a further treatment option in the control arm, based on the use of an unmanipulated transplant followed by cyclophosphamide administration during the post-transplantation period.
This press release is written in compliance with public disclosure obligations established by CONSOB (Italian securities & exchange commission) resolution no. 11971 of 14 May 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 antitumour therapeutics in clinical development: TK, a cell-based therapy enabling bone marrow transplants from partially compatible donors, in absence of post-transplant immune-suppression, 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)