Prolong Pharmaceuticals’ investigational drug for treatment of sickle cell disease (SCD)-associated complications, Sanguinate, showed promising results in patients experiencing vaso-occlusive crisis (VOC).
According to the research study, “SANGUINATE Returns RBCs To More Normal Morphology In Patients With VOC,” presented at the 2017 Annual Symposium of the Foundation for Sickle Cell Disease Research (FSCDR) in Fort Lauderdale, Florida, this new treatment was able to restore the shape of red blood cells, improving the VOC symptoms in SCD patients.
“Vaso-occlusive crisis is a debilitating, painful condition caused by sickling of the red blood cells,” Gershwin Blyden, principal investigator at the FSCDR, said in a press release. “These findings add to the mounting clinical evidence supporting the effects of Sanguinate to ameliorate a painful crisis in the acute phase.”
Preclinical studies showed that Sanguinate can facilitate the transfer of oxygen to oxygen-deprived cells and tissues. It was also shown to down-regulate the inflammatory process that is commonly activated in SCD patients. By overcoming these two biological process, the investigational drug has the potential to reduce the symptoms and many complications of sickle cell disease.
Phase 2 trials for VOC treatment in SCD patients (NCT02411708 and NCT02672540) are ongoing and still recruiting participants. Sanguinate has received orphan drug status from the U.S. Food and Drug Administration.
Interim results from one of the Phase 2 trials demonstrated that 10 sickle cell patients who received intravenous Sanguinate showed a reduced number of abnormally shaped red blood cells within few hours after treatment. This effect was sustained for at least 72 hours, indicating a prolonged effect. In comparison, placebo-treated patients did not show any alteration in the number of abnormal blood cells.
“Sanguinate is a dual-gas transfer agent that has been shown to revert sickled red blood cells to a more normal shape. These latest findings are consistent with prior in vitro study results showing that Sanguinate may offer a prolonged therapeutic effect on cell morphology,” said Ronald Jubin, vice president of research and development at Prolong Pharmaceuticals.
“We look forward to advancing our clinical program to further evaluate Sanguinate’s potential benefits for people suffering from sickle cell disease,” he said.