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Italfarmaco S.p.A joins EU-funded EndoStem consortium to support clinical study with Givinostat in Duchenne Muscular Dystrophy

The EndoStem consortium (, a large scale scientific collaboration coordinated by Dr David Sassoon (UPMC/Inserm, Paris, France), and co-financed by the European Commission via the 7th Framework Programme, has welcomed Italfarmaco (Milano, Italy) into the consortium and project as a full partner, to augment the number of clinical trials being performed to treat muscle disorders.

As part of their participation and collaboration, Italfarmaco is initiating clinical trials using the HDAC inhibitor Givinostat, to treat Duchenne Muscular dystrophy. Patients with muscular dystrophy suffer debilitating muscle loss that gets worse as they age. Dr. Pier Lorenzo Puri, associate professor at Sanford-Burnham and Italy’s Dulbecco Telethon Institute, has performed a preclinical trial using HDAC inhibitors showing the promoting effect in regeneration in dystrophic animal models1,2. His team demonstrated that exposure to HDACi forces muscle resident progenitors to adopt the myogenic lineage. The data also supported the efficacy of the HDAC inhibitor Givinostat (from Italfarmaco) in promoting regeneration of dystrophic muscles while reducing the formation of fibrotic scars and fat deposition. This treatment was largely more effective than the treatment with the control HDAC inhibitor. Based on clinical data, Givinostat was previously granted orphan drug designation by EMA for the treatment of Polycythemia Vera, a blood disorder in which the bone marrow makes too many red blood cells, and for Systemic-onset Juvenile Idiopathic Arthritis demonstrating its safety in humans including children. Given the suitability of Givinostat for treatment of paediatric patients, the collaboration is moving forward with the planning of a clinical trial in DMD patients. An important aim of this clinical trial is to correlate the beneficial effects of molecules used and their effect on endogenous stem cells. “The joint effort of Endostem and Italfarmaco is the fuel for accelerating the translation of pre-clinical data generated by my group at the Dulbecco Telethon Institute/Fondazione Santa Lucia into a clinical trial with boys affected by Duchenne Muscular Dystrophy".” says Dr. Puri. “Italfarmaco is fortunate and proud to join Endostem, the ideal partner for effectively moving to the development of Givinostat in such debilitating diseases as muscle disorders.” says Dr Mascagni Pre-Clinical R&D Senior Director from Italfarmaco. “Italfarmaco is a perfect fit for the work encompassed in Endostem as they complement extremely well the work going on in and among the existing partners in the consortium," says Dr Sassoon. About Endostem EndoStem ( is a partnership of 15 research and clinical teams from globally recognised academic centres, small biotech and large pharmaceutical companies working together to develop new strategies aimed at stimulating stem cells that are resident in damaged tissue to repair it in situ. This approach is recognised as one of the most promising approaches to targeting stem cells for regenerative medicine due to the alignment with existing therapeutic development approaches used by large industry and recent advances in understanding the key barriers for tissue regeneration. Coordinated by Professor David Sassoon, co-financed by the European Commission via the 7th Framework Programme the aims of the project over are: - Implementation of clinical trials, with muscular dystrophies as the primary clinical target using innovative biopharmaceuticals - Development of novel best in class biopharmaceuticals with highly specific and well defined modes of action - Fast track clinical translation based on a constant feedback loop between emerging patient responsiveness to new drugs and the development of the next generation of therapeutics - Better understanding of the key issues preventing effective tissue repair matched with approaches to circumvent them About Italfarmaco S.p.A Italfarmaco S.p.A. one of the leading Italian pharmaceutical groups, operates in both the pharmaceutical and the fine chemical industries. The company was founded in Milan in 1938.The Italfarmaco Group now markets ethical products in Italy, Switzerland, Spain, Portugal, Greece, Russia, Turkey, Chile, Peru, Morocco and also for the subsidiary Chemi SPA in USA and Brazil. Total employees is over 2100 and total turnover exceeds 500 million euros. Its products, all of which have a high therapeutic content, are mainly used in the cardiovascular, immuno-oncological, gynaecological, dermatological, orthopaedic and neurological areas. The Italfarmaco Group is particularly active in the field of research, mainly oriented towards the cardiovascular area, oncohematology and diseases associated with alterations of the immunological system. References 1- Minetti GC, Colussi C, Adami R, Serra C, Mozzetta C, Parente V, Illi B, Fortuni S, Straino S, Gallinari P, Steinkhuler C, Capogrossi M, Sartorelli V, Bottinelli R, Gaetano C, Puri PL. Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors. Nature Medicine 2006; 12 (10): 1147-50. 2- Consalvi S, Saccone V, Giordani L, Minetti G, Mozzetta C, Puri PL. Histone deacetylase inhibitors in the treatment of muscular dystrophies: epigenetic drugs for genetic diseases. Mol Med. 2011 Feb 7.


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