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MolMed: corporate governance strengthening

Co-optation of two new members of the Board of Directors, resignation of three Directors

Milan, October 22, 2015 – Today the Board of Directors of MolMed S.p.A. (Milan: MLM) co-opted Dr. Riccardo Palmisano and Prof. Didier Trono as new members of the Board of Directors following the resignation from office, during the same meeting, of Mrs. Marina Del Bue, Mr. Germano Carganico and Mr. Lorenzo Salieri, respectively executive Director in charge of internal control and risk management, executive Director and nonexecutive Director. The resigning Directors did not serve on any of the Company’s Internal Committees. In addition, the Board of Directors postponed to the next meeting, scheduled on the 9th of November, any resolution on the co-optation of the third Director, who must comply with applicable legislation upon “gender quotas”, and the verification of the independence requirements of the new Directors and their compliance with the maximum number of offices held in other companies, as defined by the Company.

Marina Del Bue and Germano Carganico decided to resign from the Board to allow the corporate governance strengthening and, maintaining their positions respectively as General Manager Corporate Governance & Administration and Director of Business Development and Strategic Affairs of MolMed, will focus on advancing the numerous and significant activities of the Company.

Lorenzo Salieri’s decision aims to foster a change in the Board of Directors’ composition, allowing members with specific international professional skills in the field to join to the Board; added to the decrease in the number of shares Science Park Raf S.p.A. – winding up – holds in MolMed S.p.A. and the termination of the shareholders agreement concerning the Company, expired on March 2015. The Chairman Prof. Claudio Bordignon, highlighting the outstanding support rendered by the resigning Directors, together with all the members of the Board of Directors and Board of Statutory Auditors, expressed their heartfelt thanks to the outgoing Directors for their profuse and sincere commitment in the performance of their duties.

Dr. Palmisano is non-executive Chairman of the Board of Directors of Genzyme s.r.l., a cutting-edge biotech company, leader in research and development of orphan drugs for rare diseases. During his career, Dr. Palmisano successfully launched a large number of important products in many therapeutic areas, in both retail and hospital markets, participated and supported several international projects, and successfully negotiated pricing and reimbursement for several innovative drugs with the Italian Authorities. In 2008, Dr. Palmisano joined Assobiotec, the trade association, part of Federchimica, brings together Italian biotech companies, becoming Vice President, responsible for biotech pharma and health, in 2010. In recent years, he became involved in several initiatives dedicated to research and innovation: supports a number of start-up companies, in accomplishing the various steps needed to enter into the market; he is a member of the evaluation committee of the “Area Salute” projects at UniCredit Startlab; a member of the Steering Committee of Rare Partners, a non-profit company, dedicated to the development of new therapeutics and diagnostics in the field of rare diseases; as well as a member of the projects evaluation group at AriSLA. Medical Doctor by education (Parma University), his career in the Italian pharmaceutical industry started in 1986 at Farmitalia Carlo Erba (1986), he then moved on to Gruppo Menarini (1988) where he contributed to the development and internationalisation of the first Italian pharmaceutical Group, becoming Director of the Pharmaceutical Division Italy in 1993 and General Manager at Lusofarmaco in 1995. His career in multinational companies started in 2000, year in which he set up all the operations of the Italian branch of Shire Pharmaceutical, in 2003 he was appointed Vice President Commercial Retail Market in GlaxoSmithKline Verona and, lastly, Vice President & General Manager Italy at Genzyme (2005). After the acquisition of Genzyme by Sanofi he was appointed Director of Business Strategy and Development at Sanofi Italia (2012- 2013).

Prof. Trono, already a member of MolMed’s Scientific Advisory Board, from which he resigned following today’s appointment, is a regarded scientist and opinion leader internationally renowned in the field of lentiviral vectors. Prof. Trono has a long-standing interest for interactions between viruses and their hosts. This led him to study the biology of pathogens such as human immunodeficiency virus and hepatitis B virus, and to develop virusbased delivery systems for human gene therapy. In the last decade, his research shifted towards the field of epigenetics, to explore the impact of retro-elements and their controlling mechanisms on the development and physiology of higher organisms, including humans.

After obtaining an M.D. from the University of Geneva and completing his clinical training in pathology, internal medicine and infectious diseases in Geneva and at the Massachusetts General Hospital in Boston, Didier Trono embarked in his scientific career at the Whitehead Institute for Biomedical Research of MIT. In 1990, he joined the faculty of the Salk Institute for Biological Studies (La Jolla – CA) to launch a centre for AIDS research. In 1997, he moved back to Europe, where he took over as Chairman of the Department of Genetics and Microbiology at the University of Geneva (2000-2004), and became Dean of the Basic Sciences Section of the Faculty of Medicine (2001-2004). From 2004 to 2012, he was Deputy Director of “Frontiers in genetics” at the Swiss National Science Foundation, and Dean of the School of Life Sciences at the Swiss Institute of Technology in Lausanne, where he is still a Professor. He is a member of EMBO, the French NationalAgency forAIDS Research, the Swiss National Science Foundation and the Swiss National Research Council. He is the author or co-author of more than 200 publications and holds several patents.

“The appointment of Dr. Palmisano and Prof. Trono”, said Prof. Claudio Bordignon, Chairman and CEO of MolMed S.p.A.,” reflects the Board’s desire to strengthen the Company’s corporate governance with a higher number of directors of great international standing and having the technical expertise in the Company’s most important areas of activity. This also in light of the recent addition to the pipeline. On the other hand the resigning Directors Marina Del Bue and Germano Carganico will continue to support the Board’s activity as executives of the Company”. According to disclosures made to the public and to the Company, to date, Marina Del Bue, Germano Carganico, Lorenzo Salieri, Riccardo Palmisano and Didier Trono do not hold any ordinary shares of MolMed S.p.A.. Prof. Claudio Bordignon, Chairman and CEO of the Company, has been appointed Director in charge of internal control and risk management. The curricula vitae provided by Dr. Palmisano and Prof. Trono, are available on Company’s website (www.molmed.com).

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.

—— About MolMed MolMed S.p.A. is a medical biotechnology company focused on research, development and clinical validation of novel anticancer therapies. MolMed’s pipeline includes antitumour therapeutics in clinical and preclinical development: Zalmoxis® (TK) is a cell-based therapy enabling bone marrow transplants from partially compatible donors, in absence of post-transplant immune-suppression, currently in Phase III in high-risk acute leukaemia and under evaluation by EMA for a Conditional Marketing Authorization; NGR-hTNF is a novel therapeutic agent for solid tumours which displays antitumor activity through its specific binding to blood vessels feeding the tumour mass, currently investigated in a broad clinical programme; CAR-CD44v6, an immuno-gene therapy project potentially effective for many haematological malignancies and several epithelial tumours, currently in preclinical development. MolMed also conducts cell and gene therapy projects in collaboration with third parties, offering resources and expertise covering preclinical to Phase III trials activities. These projects include the development and the validation of the manufacturing process and control strategy, and the cGMP production of clinical-grade viral vectors and manufacturing of patient-specific genetically engineered cells. MolMed has its headquartered at the San Raffaele Biotechnology Department (DIBIT) in Milan, Italy, and secondary office at OpenZone, in Bresso (Milan). MolMed is listed on the main market (MTA) of the Milan stock exchange managed by Borsa Italiana (ticker Reuters: MLMD.MI).

Source: MolMed.com

#MolMed #MolMed Claudio Bordignon #MolMed Board of Directors

MolMed New Data At ASCO On NGR-hTNF Show Significantly Extended Survival

A statistically significant 40% improvement of both overall survival and progression-free survival in a large population of patients, identified by a pre-specified analysis on the prior treatment-free interval, and who presented a very dismal prognosis was reported by MolMed S.p.A. (MLM.MI) this weekend, at the 50th ASCO annual meeting.

These efficacy results were observed in an international randomized Phase III study evaluating the investigational drug NGR-hTNF in combination with best investigator choice in 400 patients with malignant pleural mesothelioma (MPM) who had previously failed a first-line chemotherapy.

The magnitude of treatment effect increased with NGR-hTNF duration and was particularly marked in patients receiving at least three months of therapy, with a median survival time nearly doubled in patients treated with NGR-hTNF compared to control patients: 16.5 vs 9.8 months, respectively.

Claudio Bordignon, Chairman and CEO of MolMed, commented: “The results presented at ASCO on the efficacy of NGR-hTNF in the treatment of the patient affected by the more aggressive form of malignant pleural mesothelioma confirm, in a large Phase III trial, the therapeutic potential of this molecule already observed in Phase II in chemo-resistant squamous NSCL carcinoma, soft-tissue sarcoma, and ovarian carcinoma. Having been able to extend the median survival time to more than 16 months in patients treated for at least 12 weeks is a success for the study and, more important, offers the perspective of a relevant clinical benefit to patients affected by the more aggressive form of mesothelioma. These results represent for MolMed the basis to aggressively pursue the next crucial steps for the future of this molecule: the definition of the best and fastest registration path, and the identification of a suitable partner for offering to NGR-hTNF the possibility to express its full potential as an anti-cancer agent of broad application.”

The data reported at ASCO, mainly obtained in combination with either gemcitabine or vinorelbine in a very aggressive and chemo-resistant disease, assume particular relevance as they are confirmatory of the efficacy previously shown by NGR-hTNF plus gemcitabine in the first-line Phase II study in squamous lung cancer patients.

Furthermore, NGR-hTNF confirmed in this large patient population its very favourable tolerability profile in combination with the three different chemotherapeutic agents administered in this study (gemcitabine, vinorelbine and doxorubicin).

An additional two randomized Phase II studies reported at ASCO meeting clearly established the effect of NGR-hTNF on survival.

In the four-arm randomized Phase II study in sarcoma patients, the low-dose weekly NGR-hTNF plus doxorubicin regimen induced a statistically significant doubled survival time, as compared with the other schedules given at high dose in combination with doxorubicin or as monotherapy at low or high dose. The 3-year survival rate with this schedule exceeded 40% and, notably, similar results were reported for both chemo-naïve and pretreated patients, thus confirming the elevated NGR-hTNF efficacy in more aggressive, chemo-resistant disease.

In the randomized Phase II study in resistant / refractory ovarian cancer patients, NGR-hTNF in combination with an anthracycline improved overall survival in patients with normal or high baseline lymphocyte counts, as compared to patients receiving an anthracycline alone.

Taken together, these clinical evidences are also consistent with the drug mechanism of action, based on both an increased intratumoral chemotherapy uptake and interaction with host immune system.

About NGR-hTNF NGR-hTNF is a novel therapeutic agent for solid tumours which displays antitumor activity through its specific binding to blood vessels feeding the tumour mass. NGR-hTNF is being investigated in a large clinical program, including a Phase III trial in malignant pleural mesothelioma (second line), a Phase II trial in malignant pleural mesothelioma (first-line maintenance therapy) and five Phase II trials in colorectal, lung (small-cell and non-small-cell), liver and ovarian cancer, and soft tissue sarcomas.

NGR-hTNF has been granted Orphan Drug designation for the treatment of mesothelioma and liver cancer in both the EU and the US.

About MolMed 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)

FONTE: MolMed.com

#MolMed #MolMed NGRhTNF #MolMed NGRhTNF ASCO

MolMed TK ASCO new data from the pivotal Phase III randomized trial TK008

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”.

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.

TK008 is coordinated by Dr. Fabio Ciceri, M.D. from the San Raffaele Scientific Institute, principal investigator of the trial who illustrated the results at ASCO and commented: “Initial results of the Phase III study indicate that in haploidentical transplant recipients TK-cell infusions abate non-relapse mortality and leukemia relapse by providing a fast immune recovery in a dose-dependent manner. HSV-TK suicide gene machinery effectively and timely controlled the graft-vs-host disease (GvHD) in 100% of affected patients with no requirement for long-term immune-suppressive treatment. Results obtained so far appear largely superior to the outcomes reported by large EBMT surveys for the two treatment options of TK008 control arm: T-cell depleted and T-cell replete followed by high-dose cyclophosphamide. These results are of particular relevance in light of the more accessible standard option represented by haploidentical transplants.” About TK 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. Based on the efficacy and safety data and on the Orphan Drug designation, the Company filed a request for Conditional Marketing Authorisation of TK with the European Medicine Authority in March 2014.

TK008 study 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 compares the outcome of haplo-transplants with or without TK add-backs, with a 3:1 randomisation ratio in favour of the TK arm.

The primary study end-point is disease-free survival (DFS) – which includes both transplant-related mortality and disease relapse – evaluated on 170 patients. The study is powered to detect an increase in 1-year DFS from 30% in the control arm to 52% in the experimental 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.

FONTE: MolMed.com

#MolMed #MolMed TK leukaemia #MolMed TK ASCO

MolMed reports top line results of NGR-hTNF in the phase III trial in malignant pleural mesothelioma

MolMed S.p.A. (MLM.MI) today announced the results of the double-blinded, placebo-controlled Phase III trial of NGR-hTNF versus best investigator choice in 400 patients with malignant pleural mesothelioma who had previously failed a first-line chemotherapy.

Despite not meeting its primary endpoint of improving overall survival (OS) in the entire population, the study showed a statistically significant (unstratified p=0.02; stratified p=0.01) 40% improvement of OS in patients with poorer prognosis who had progressed during or shortly after first-line chemotherapy. These patients represent 50% of the entire patient population and were identified by a pre-specified analysis based on prior treatment-free interval. This clinical parameter will allow to easily identifying patients who derive most clinical benefit from NGR-hTNF in combination with the chemotherapy of choice. Consistent with the overall survival improvement in this patient population, a 40% percent longer progression-free survival (PFS) was also reported for the NGR-hTNF treated arm in those patients who presented a more aggressive, chemo-resistant disease. In addition to the efficacy data, NGR-hTNF confirmed in this large patient population its very favourable tolerability profile also in combination with the three chemotherapeutic agents administered in this study (gemcitabine, vinorelbine and doxorubicin).

NGR-hTNF has already been granted Orphan Drug designation for the treatment of mesothelioma in both the EU and the US. Claudio Bordignon, Chairman and CEO of MolMed, commented: “The results obtained in a pre-specified population of high-risk patients represent an important reward for the Company’s mission and effort in improving survival and quality of life of cancer patients, even if the primary endpoint was not reached in the entire population. For the first time in malignant pleural mesothelioma a highly significant clinical benefit was achieved in a large subpopulation with the worst prognosis, represented by patients refractory or rapidly progressing after first line treatment. In addition, these data provide confirmation of NGR-hTNF effect on survival already observed in Phase II studies in other indications. Pending a more in depth evaluation, these top line results already provide potential to pursue conditional marketing authorisation and further clinical development in mesothelioma and other indications.”

For the same pathology the Company is running a randomised Phase II study on NGR-hTNF as maintenance therapy after completion of first line chemotherapy (NGR019). The study aims at extending the treatment-free interval of those patients who did not progress after first line, providing them with a long term therapy with a high tolerability profile.

About NGR-hTNF

NGR-hTNF is a novel therapeutic agent for solid tumours which displays antitumor activity through its specific binding to blood vessels feeding the tumour mass. NGR-hTNF is being investigated in a large clinical program, including a Phase III trial in malignant pleural mesothelioma (second line), a Phase II trial in malignant pleural mesothelioma (first-line maintenance therapy) and five Phase II trials in colorectal, lung (small-cell and non-small-cell), liver and ovarian cancer, and soft tissue sarcomas. In particular, completed randomised clinical trials showed statistically significant increase in overall survival in squamous cell NSCLC and soft tissue sarcomas. Recent results from the Phase III study in malignant pleural mesothelioma confirmed the ability of NGR-hTNF in treating particularly challenging cancer indications. Updated results from MolMed clinical development program will be presented at most relevant forthcoming meetings, including the ASCO 2014 in June. NGR-hTNF has been granted Orphan Drug designation for the treatment of mesothelioma and liver cancer in both the EU and the US.

About Phase III trial NGR015

NGR015 is a pivotal randomised, double-blind, placebo-controlled, international, multicentre Phase III trial performed on 400 malignant pleural mesothelioma patients (enrolled in 48 centres in the EU, US, Canada and Egypt) whose disease progressed after a pemetrexed-based chemotherapy. The trial is powered to detect a survival benefit for NGR-hTNF, when combined with best investigator’s choice (BIC), where BIC includes either best supportive care alone or in combination with chemotherapy (either doxorubicin, or gemcitabine, or vinorelbine). NGR-hTNF is administered according to the dose and schedule that were confirmed as the most efficacious in Phase II trials: 0.8 µg/m2 weekly, until disease progression.

About Phase III trial NGR019

NGR019 is a randomised, double-blind, placebo-controlled, international, multicentre Phase II trial on 100 adult pleural mesothelioma patients with non-progressive disease after a pemetrexed-based chemotherapy. The trial is powered to detect a progression-free survival benefit for NGR-hTNF, when combined with best supportive care (BSC). NGR-hTNF is administered according to the dose and schedule that were confirmed as the most efficacious in Phase II trials: 0.8 µg/m2 weekly, until disease progression. Secondary endpoints include overall survival, tumour response, safety and quality of life.

Conditional Marketing Authorisation

The Conditional Marketing Authorisation represents an expedite path for early market authorisation ahead of completion Phase III clinical development. 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:

1. the risk-benefit balance of the medicinal product is positive; 2. it is likely that the applicant will be in a position to provide the comprehensive clinical data; 3. unmet medical needs will be fulfilled; 4. 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 allows the Company to commercialise the product in advance and 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.

About pleural mesothelioma

Pleural mesothelioma is a form of cancer strongly related with repeated exposure to asbestos fibres (for more information please refer to Park et al, Global Magnitude of Reported and Unreported Mesothelioma, Environmental Health Perspectives, 2011). Pleural mesothelioma is a tumor that develops in the tissue that lines the chest cavity. With an incidence of approximately 1/100,000, pleural mesothelioma is still a relatively rare type of cancer, but has been progressing quickly in the past 20 years as incidence rates have continuously increased. Pleural mesothelioma has a long latency period and symptoms are non-specific, so that in most cases diagnosis is difficult before the advanced stage of the disease is reached. Research indicates that pleural mesothelioma may affect each year more than 10,000 people in the EU and the US. Currently, only one therapy has been approved for first-line treatment, whilst the elevated need for first-line maintenance and second line treatment remains unmet. Clinical development of NGR-hTNF is aimed at filling this therapeutic gap.

FONTE: MolMed.com

#MolMed #NGR-hTNF #malignant pleural mesothelioma

TK submission for Conditional Approval validated by EMA

MolMed S.p.A. (Milan:MLM) announces that the European Medicines Agency (EMA) has validated the submission of the Conditional Marketing Authorisation for TK, a novel proprietary investigational cell-gene therapy. The data review of the submitted dossier starts today.

TK is an adjunctive treatment in hematopoietic stem cell transplantation for patients affected by high risk leukaemia. TK has been granted orphan drug status by the European Commission. MolMed is also glad to announce that the Company has been invited by the European Society for Blood and Marrow Transplantation (EMBT) to illustrate the scientific and regulatory path of TK which – with more than 120 patients treated so far – is the largest cell-gene therapy application ever performed. Claudio Bordignon, CEO and president of the Board, will give a presentation on the topic next Tuesday 1st of April during the plenary session of the 40th annual meeting of the EMBT, which will be held in Milan from March 30th to April 2nd.

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:

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.

About TK 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 study 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.

SOURCE: MolMed.com

#MolMed #TK #Conditional Marketing Authorisation #Molmed TK

MolMed TK therapy submitted for Conditional Marketing Authorisation in EU

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”.

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.

TK008 study 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.

SOURCE: MolMed.com

#MolMed #TK #Conditional Marketing Authorisation