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 (Phase I study) until conclusion of Phase III studies necessary for registration and placing the drug on the market. 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 positive concluding a Phase II and a Phase III study in children with Duchenne’s Muscular Dystrophy, studies necessary for the registration request of givinostat for the treatment of Duchenne Muscular Dystrophy, the Clinical R&D Department in collaboration with the Regulatory team submitted the registration dossier to EMA and FDA. In this indication, a long-term study aimed at demonstrating the long-term safety of the drug is currently under way and a Phase III study will begin shortly in Duchenne boys who have lost their walking ability. Still in area of dystrophinopathies, the Clinical R&D Department has completed a Phase II study in Becker’s Muscular Dystrophy.

In the onco-hematological 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 Department 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 and in 2022, a Phase I study (First Time in Human) in oncology has been started.

The Clinical R&D Department currently includes a senior group of scientists (e.g. physicians, personnel graduated in other bio-medical areas, project managers, statisticians) who are responsible for the design and implementation of the clinical development plans of the molecules’ portfolio of the Clinical R&D Department. The clinical trials conduction 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.