Periodic Reporting for period 1 - Chelator (IRON CHELATOR NBMI FOR THE TREATMENT OF IRON OVERLOAD IN BETA-THALASSEMIA MAJOR)
Reporting period: 2019-06-01 to 2020-04-30
Iron is an essential metal, crucial for the body, always bound to proteins. If free, not bound to proteins, it is toxic, causing major oxidative stress and damage to organs. This happens in blood transfusion dependent Iron overload diseases such as Beta-Thalassemia Major with over ten thousand patients in the EU, of which most in Italy, Greece, Cyprus. Each unit of red cell concentrate contains approximately 250 mg of iron. As there is no physiological mechanism for the excretion of iron, after about 50 transfusions, the storage capacity of about 10–15 g is saturated, and tissue damage may occur. To remove this excess iron, iron chelators are used. These drugs are decades old, inefficient, complicated and have substantial side effects which also affect patient's compliance - and in spite of this have huge sales. The annual treatment cost of a patient is tens of thousands of Euros and for the EU hundreds of millions of Euros, plus indirect costs and poor life quality. There is a medical need for a better iron chelator. Emeramide (NBMI) is a novel metal chelator that has shown promise. During this feasibility study, a business plan has been developed that will guide clinical validation and commercialization efforts.
Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far
A team of regulatory consultants have been engaged to generate a plan of how to take the drug to market, which is included in the report along with the associated costs for the program. The report also includes a market assessment and a discussion on pricing.
Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)
The project has lead to a plan of how to take the drug to market, which is crucial should we decide to develop the drug. Thus, there have been no socio-economic impacts or wider societal implications as the research studies.