Sangart, Inc. an international biopharmaceutical company, has initiated a phase 1b established to evaluate the safety of the treatment MP4CO patients with sickle cell anaemia.
MP4CO is designed to provide therapeutic levels of carbon monoxide (CO) to patients with sickle cell disease crises. The original study will include patients who do not suffer from painful vaso-occlusive crises. Preclinical studies indicate that carbon monoxide stabilizes haemoglobin in patients with sickle cell disease and prevents sickling of red blood cells.
"The preclinical data are encouraging MP4CO and we are pleased to be able soon to evaluate the safety and efficacy of this treatment to alleviate the pain associated with sickle cell disease crises, while reducing the severity and duration of a crisis, "said Dr. Jo Howard , of Guy's and St Thomas' Hospital, London, UK. "There are very few treatments for painful vaso-occlusive crises associated with sickle cell anaemia, and MP4CO is a potential treatment option for these patients. "
The clinical trial is multi-centre, randomized, double-blind, involving about 32 clinically stable adult patients with sickle cell anaemia who are not painful vaso-occlusive crisis. The trial will be conducted in four sites across three countries, including the United Kingdom, France and Jamaica. The main objective of the study is to establish the safety of treatment with MP4CO. The study will include ascending dose levels administered in five groups of single-dose infusions, followed by three groups of fractional doses.
"This initiative represents a first step forward to provide treatment to patients with sickle cell disease crises. The strong preclinical data suggest that clinical evaluation of patients MP4CO could generate significant results, "said Brian O'Callaghan , President and CEO of Sangart. "Simultaneously with our other experimental treatment, MP4OX, we remain committed to finding drugs designed to improve the delivery of oxygen and other gases in the tissues and organs. "
Sickle cell anaemia is an inherited disorder of haemoglobin on the movement of red blood cells of millions of people around the world. People with sickle cell disease have red blood cells that contain mostly haemoglobin S, an abnormal type of haemoglobin. Due to the presence of haemoglobin S, the red blood cells adopt a crescent or sickle-shaped and have difficulty passing in the small vessels. When sickle-shaped cells block the vessels, less blood flows through the body, causing severe pain and tissue damage that are no longer regularly irrigated by blood. This vaso-occlusion is known as a "crisis of sickle cell. "There is currently no approved therapy to treat sickle cell disease crises, and only the symptoms can be treated.
Platform products Sangart is based on the MP4 molecule, an experimental biopharmaceutical product designed to improve tissue perfusion of oxygen-deprived (ischemic) and provide targeted delivery of oxygen in the capillaries. Using a new approach to PEGylation, Sangart produces the MP4 molecule developed according to the optimal oxygen affinity, diffusion potential and molecular size to perfuse capillaries and target the delivery of oxygen to tissues that are particularly at risk undergoing ischemia. MP4 can bind carbon monoxide to deliver hemoglobin in red blood cells and heme proteins in tissue to produce anti-inflammatory and anti-apoptotic (cell death anti-programmed). Once the carbon monoxide was unloaded, the MP4 is oxygen when flow in the lung and the book in the ischemic tissues.
Sangart is an international biopharmaceutical company engaged in the development of drugs designed to save the lives of patients and specifically designed to improve perfusion and tissue oxygenation odes ischemic (oxygen deprived) through a delivery targeted oxygen.