Mast Therapeutics To Start Phase 3 Sub-Study For Sickle Cell Drug

Mast Therapeutics announced that has started patient enrollment in the Phase III EPIC sub-study investigating MST-188 in sickle cell disease (SCD).

In previous studies, MST-188 has demonstrated the ability to reduce viscosity and adhesive frictional forces in the blood, improving microvascular blood flow. Enhancing flow of oxygen-carrying blood to organs and tissues can lead to shorter duration of vaso-occlusive crisis and better tissue oxygenation. This in turn limits organ damage and end-organ dysfunction and failure in sickle cell disease.

The EPIC sub-study will assess the efficacy of MSS-188 on tissue oxygenation in subjects with sickle cell disease who are undergoing vaso-occlusive crisis as a primary trial endpoint.  Tissue oxygenation will be determined using a non-invasive, FDA-approved device.

Brian M. Culley, CEO of Mast Therapeutics, said, “Organ failure, which is the leading cause of premature death in adults with sickle cell disease, is thought to be the consequence of a lifetime of repeated vaso-occlusive events and the resulting ischemia.  We believe that measuring effects on microvascular blood flow and tissue oxygenation during vaso-occlusive crisis will be useful to understanding MST-188's potential to improve long-term outcomes for sickle cell patients where multi-decade, survival trials are impractical.  MST-188 has already demonstrated an ability to improve microvascular blood flow.  Now, this sub-study will demonstrate whether MST-188 also improves tissue oxygenation.” Culley said that the results will help shed light on MST-188’s potential to address end-organ failure and related premature death in patients with sickle cell disease.

According to the National Heart, Lung, and Blood Institute, sickle cell disease is a serious disorder in which the body produces sickle-shaped red blood cells instead of the normal disc-shaped red blood cells. This flaw causes sickle cells to block blood flow in vessels, which can lead to pain and organ damage. Sickle cell disease also increases risk of infection for affected patients.

Last April, the company presented data for MST-188 showing that the investigational treatment reduced the mean erythrocyte sedimentation rate (ESR) by 50 percent associated with control in blood collected from patients with SCD. ESR describes the rate of red blood cell travel through anti-coagulated blood. The study data were presented at the 8th Annual Sickle Cell Disease Research & Educational Symposium held in Miami.