Selexys Pharmaceuticals Corp. is enrolling patients in a Phase 2 trial of a drug designed to treat sickle cell disease.
The Oklahoma City-based company obtained $23 million in venture capital financing last fall to help pay for clinical study.
Selexys' study will include about 175 patients nationwide. Patients randomly will receive a high dose of Selexys' SelG1 or a placebo, with or without standard treatment.
The study will examine the effectiveness of SelG1 in reducing the rate of sickle cell-related pain crises.
“With the limited therapies available to patients with sickle cell disease, this trial with a novel P-selectin inhibitor is particularly exciting,” said lead investigator Dr. Kenneth Ataga, a professor at the University of North Carolina at Chapel Hill. “Not only does it have the potential to improve the clinical outcomes in patients, its effects may be additive or synergistic with those of hydroxyurea, the only drug currently approved by the FDA for treatment of complications due to sickle cell disease.”
Sickle cell disease is the most common genetic disorder in the United States. It affects an estimated 100,000 Americans, including one in 500 blacks.
Sickle cell anemia results in abnormally shaped red blood cells, which get stuck inside the blood vessels. That makes it difficult for those cells to deliver oxygen throughout the body, causing severe pain and harming organs, muscles and bones.
“Data from preclinical sickle cell disease models suggest that blockade of P-selectin effectively prevents the painful stoppage of circulation in small blood vessels called vasoocclusion and maintains patent blood flow,” said Selexys Executive Vice President Dr. Russell Rother. “Results from our recently completed Phase I study indicate that SelG1 is well tolerated in healthy human subjects.
“We now look forward to investigating its safety and efficacy in the sickle cell disease patient population.”
With the limited therapies available to patients with sickle cell disease, this trial with a novel P-selectin inhibitor is particularly exciting.”
Dr. Kenneth Ataga,
Lead investigator and a professor at the University of North Carolina at Chapel Hill