Sangart Initiates Phase 1b Study of MP4CO in Patients With Sickle Cell Disease

Sangart, Inc., a global biopharmaceutical company dedicated to developing life-saving medicines specifically designed to enhance the perfusion and oxygenation of ischemic (oxygen deprived) tissues through targeted oxygen and other gas delivery, today announced the initiation of a Phase 1b study designed to evaluate the safety of MP4CO treatment in patients with sickle cell disease. MP4CO is designed to deliver therapeutic levels of carbon monoxide (CO) to patients suffering from a sickle cell crisis. This initial study will involve patients who are not currently undergoing a painful vaso-occlusive crisis. Preclinical studies show that carbon monoxide stabilizes the hemoglobin of patients with sickle cell disease and prevents the sickling of red blood cells.

We are encouraged by the preclinical data on MP4CO and look forward to evaluating the safety and potential efficacy of this therapy in helping alleviate pain associated with a sickle cell crisis, while also potentially reducing the severity and duration of a crisis,” said Dr. Jo Howard, at Guy’s and St Thomas’ Hospital, London, UK. “There is currently a dearth of treatments for painful vaso-occlusive crises associated with sickle cell disease, and MP4CO represents a potential treatment option for these patients.”

Brian O'Callaghan - President and Chief Executive Officer: Sangart

The clinical trial is a multi-center, randomized, double-blind, controlled study that will enroll approximately 32 clinically stable adult patients with sickle cell disease who are not undergoing a painful vaso-occlusive crisis. The trial will be conducted at four sites across three countries, including the United Kingdom, France and Jamaica. The primary objective of the study is to establish the safety of treatment with MP4CO. The study will comprise ascending dose levels in five groups of single dose infusions followed by three groups of fractionated doses.

This trial initiation is a key step towards providing a treatment to patients suffering from a sickle cell crisis. The strong preclinical data suggest that a clinical evaluation of MP4CO in patients could yield meaningful results,” said Brian O’Callaghan, President and CEO of Sangart. “Along with our other investigational therapy, MP4OX, we remain committed to developing medicines designed to enhance the delivery of oxygen and other gases to tissues and organs.

Sickle cell disease is an inherited hemoglobin disorder affecting red blood cell circulation in millions of people around the world. People with sickle cell disease have red blood cells that contain mostly hemoglobin S, an abnormal type of hemoglobin. Due to the presence of hemoglobin S, these red blood cells become crescent or sickle-shaped and have difficulty passing through small blood vessels. When sickle-shaped cells block small blood vessels, less blood circulates through the body, causing severe pain and damage to tissues that do not receive a normal blood flow. This vaso-occlusion is known as a “sickle cell crisis“. There are currently no approved medications to treat sickle cell crises, and only symptomatic relief is available.

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