Unmet Need

MAFLD / NAFLD / NASH


Nonalcoholic fatty liver disease (NAFLD), now better referred to as MAFLD (Metabolic Associated Fatty Liver Disorder) is a condition characterized by an increasing retention of fat in the liver (steatosis), and is currently thought to be the most common type of liver disease in both adults and adolescents, with rates of up to 25-30% of the U.S. population.

Nonalcoholic steatohepatitis (NASH) is a progressive form of MAFLD where inflammation, in addition to steatosis, leads to further liver damage and scarring (fibrosis). Worsening of fibrosis over time can cause extensive scarring of the liver up to development of cirrhosis. Patients with advanced stages of NASH and fibrosis are at higher risk for serious complications including liver failure, leading to transplant, and cancer (hepatocellular carcinoma). Patients with MAFLD and NASH show also a higher risk for cardiovascular problems, heart disease being the number one cause of death for patients with MAFLD.

Early diagnosis of the disease presents several challenges and MAFLD can go undetected, due to lack of apparent symptoms and evidences in standard liver testing Imaging tests (ultrasound, CAT scan or MRI) can provide a useful diagnostic tool for steatosis, but liver biopsy, despites all its limitations, is currently still the only clinically accepted method used to determine the extent of the liver damage and measuring the degree of fibrosis (scar tissue). Despite being early and clinical research in the area of metabolic diseases a very lively, dynamic and popular field, there are currently still no approved medications specific for the treatment of NAFLD/NASH, making it a high unmet need.

Obesity


Obesity, a metabolic condition in which too much fat is accumulated by the body, has tripled worldwide since 1975 and, in 2016, over 650 million people have been estimated being obese. It affects adults (13% of people aged 18 years and older) and children (38 million of children under the age of 5). According to WHO, a body mass index (BMI) over 25 is considered overweight, and over 30 is obese. Due to obesity or overweight, over 4 million people are dying each year. Usually, obesity is due to a multitude of factors, some of which are inherited and some other related to the environment and personal diet and life-style choices. Obesity is not just a matter of aesthetic, but it is a severe medical problem which heavily increases the risk of other serious medical conditions such as cardiovascular disorders, diabetes, high blood pressure and cancer. Currently, very few medications are specifically approved for the treatment of obesity and, for this reason, active research is ongoing worldwide.

Metastatic Breast Cancer


Breast cancer is the most diagnosed cancer among women worldwide, but is also affecting men. Metastatic breast cancer, or stage 4 breast cancer, refers to cancer that is spread beyond the breast tissue and local lymph nodes into other areas and tissues of the body (liver, brain, bones…). There’s currently no cure for stage 4 breast cancer. Available treatments are limited and depend on the cancer Type (Hormone-receptor positive or negative, HER2 positive or negative, triple negative) and may include hormone therapy (for Hormone-receptor positive ER+, PR+), chemotherapy (for Hormone-receptor negative cancers), immunotherapy (which uses drugs that stimulate your immune system to destroy cancer cells), and their combination. Triple negative stage 4 breast cancer is not responding to any hormone treatment, is usually treated with chemotherapy and is responsible for the vast majority of breast cancer associated deaths. A new approach to the treatment of metastatic breast cancer is highly needed and micro-RNA based drugs might represent the answer.