MX-1520: Sickle Cell Anemia Therapeutic

Orally-active agent to prevent vaso-occlusive episodes

The Medical Need

In the US, between 80,000-100,000 people suffer from sickle cell anemia, an inherited blood disease that can cause episodes of severe pain, damage to vital organs, and death. Expenditures for sickle cell complications each year are a major burden to the US healthcare system and are estimated at $1.5+ billion annually.

Sickle cell anemia results from a single mutation in the gene that encodes hemoglobin (Hb), the protein that transports oxygen in the circulatory system. The mutant or sickled hemoglobin (HbS) does not transport oxygen to the tissues effectively and forms rod-like polymers when it gives up oxygen, causing red blood cells to become rigid and irregularly-shaped. These irregular and inflexible sickled cells create blockages in the blood vessels and prevent normal blood and oxygen flow to tissues. These vessel blockages, or vaso-occlusions, characterize sickle cell anemia and are responsible for most of the severe complications of the disease.

Medinox's Approach

Medinox is addressing the need for a safe and effective preventative therapeutic for the vaso-occlusive episodes of sickle cell anemia with its orally active new chemical entity, MX-1520. Medinox's scientists have shown this molecule to improve the oxygen-carrying properties of HbS.

Medinox believes that MX-1520 may be an alternative to hydroxyurea, an approved preventative therapeutic for sickle cell patients. Since it has an excellent safety profile, MX-1520 has the potential to be a safer alternative to hydroxyurea, which is a toxic cancer chemotherapeutic agent that is not recommended for patients under the age of 18.

Laboratory studies of MX-1520 are very encouraging and show that the compound has high potential as a new orally-active preventative therapeutic for vaso-occlusive episodes. In a key transgenic animal model of sickle cell disease, MX-1520 showed proof of concept by reducing the number of sickled cells and improving the oxygen carrying properties of red blood cells.

For additional information on sickle cell anemia, please visit the Sickle Cell Society.