IRON CHELATOR NBMI FOR THE TREATMENT OF IRON OVERLOAD IN BETA-THALASSEMIA MAJOR

Información del proyecto

Chelator

Identificador del acuerdo de subvención: 868024

DOI

10.3030/868024

Proyecto cerrado

Fecha de la firma de la CE 24 Mayo 2019

Fecha de inicio 1 Junio 2019

Fecha de finalización 30 Abril 2020

Financiado con arreglo a

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Coste total

€ 71 429,00

Aportación de la UE

€ 50 000,00

Coordinado por

NBMI SCIENCE LIMITED
Ireland

Periodic Reporting for period 1 - Chelator (IRON CHELATOR NBMI FOR THE TREATMENT OF IRON OVERLOAD IN BETA-THALASSEMIA MAJOR)

Período documentado: 2019-06-01 hasta 2020-04-30

Resumen del contexto y de los objetivos generales del proyecto

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.

Trabajo realizado desde el comienzo del proyecto hasta el final del período abarcado por el informe y los principales resultados hasta la fecha

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.

Avances que van más allá del estado de la técnica e impacto potencial esperado (incluida la repercusión socioeconómica y las implicaciones sociales más amplias del proyecto hasta la fecha)

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.

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