The Clinical Research and Development (R&D) Department, in close cooperation with other company functions, is responsible for the research and development of new molecules from the first administration in humans to their registration and marketing. In particular, the Clinical R&D Department concluded clinical studies aimed at recording a new liquid formulation of riluzole (Teglutik) in the United States and a biosimilar in Europe.
In addition, after concluding a study in children with Duchenne’s Muscular Dystrophy demonstrating that givinostat - a Histone Deacetylase inhibitor developed by Italfarmaco - prevents the replacement of muscle fibers with scar tissue, and after having met the European and US Regulatory Authorities, the Clinical R&D Department has started the studies necessary for the registration of givinostat for Duchenne’s Muscular Dystrophy. In this claim, a Phase III study and a long-term study aimed at demonstrating the long-term safety of the drug, are currently under way. Also in the area of dystrophy, the Clinical R&D Department has started a Phase II study in Becker’s Muscular Dystrophy.
In the onco-haematological field, the Clinical R&D Department is currently conducting studies to assess the potential of givinostat in chronic myeloproliferative neoplasms. In particular, after having completed three Phase Ib/II studies, and after having met the European and US Regulatory Authorities, the Clinical R&D Directorate started the studies necessary for the application for registration of givinostat for Polycythemia Vera. In this claim, a Phase III study and a long-term study are currently under way to demonstrate safety and to observe the maintenance of the drug’s long-term efficacy.
The Clinical R&D Department has initiated the development of other Histone Deacetylase inhibitors that are currently at different stages of development. One of these new molecules should begin phase I in 2022.
The Clinical R&D Department currently includes a senior group of qualified bio-medical scientists who are responsible for the design and implementation of the clinical development plans of the molecules’ portfolio of the Clinical R&D Department. The conduct of clinical trials is also delegated to external CRO with which the Clinical R&D Department works in project teams.
The company’s projects are developed by the Clinical R&D Department according to the modern criteria of developing new drugs, therefore using as much as possible translational medicine approaches, which provides for early patient testing and which allows for the collection of data on the same, and then return to better formulate the candidate drug for development more targeted on the pathology.