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Will MSTX Data Be Epic?

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Sickle cell disease is an inherited genetic disorder caused by a mutation in the gene that encodes for hemoglobin, resulting in distorted red blood cells. The red blood cells, which become crescent shaped or like a sickle, cling to each other and to the walls of blood vessels, severely impairing the blood flow to the organs. An estimated 100,000 Americans and millions worldwide are affected by sickle cell disease.

The hallmark of sickle cell disease is recurring episodes of severe, debilitating pain commonly known as sickle cell crisis or vaso-occlusive crisis. The only drug that is FDA labeled for the treatment of sickle-cell disease is hydroxyurea, and it was approved in 1998.

Exploring a treatment for this inherited, lifelong disorder is Mast Therapeutics Inc. (MSTX), and the drug candidate in focus is Vepoloxamer. This compound works by binding to damaged surfaces of the cell membranes, restoring the cells' natural, hydrated, and non adhesive surface.

A phase III study evaluating Vepoloxamer, dubbed EPIC, in patients with sickle cell disease experiencing vaso-occlusive crisis, is underway. A total of 388 patients, ages four to 46 are enrolled in the study. Patient enrollment in this pivotal study was completed in February of 2016, and the company is slated to release top-line results this month.

MSTX closed Monday's trading at $0.58, down 5.06%.

Will the trial live up to its name?

Stay tuned for updates in the coming days...

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