Sunday, April 12, 2015

Drugs in Clinical Pipeline: Sanguinate

Sanguinate is an investigational biopharmaceutical product that through its anti-vaso-constrictive properties facilitates the transfer of oxygen to oxygen-deprived cells and tissues; and is the only biological product currently in clinical development for the multiple comorbidities of Sickle Cell Disease (SCD).  Many of the comorbidities of SCD are caused by a spiraling cycle of sickling, hemolysis and blood vessel inflammation.  These comorbidities include vaso-occlusive crisis, acute chest syndrome, leg ulcers and pediatric and adult stroke.  By correcting oxygen levels and down-regulating inflammation, Sanguinate has the promise of effectively treating many of the debilitating, acute comorbidities associated with Sickle Cell Disease [1].

In April 2015, the US Food and Drug Administration (FDA) granted Orphan Drug Designation for Prolong Pharmaceuticals' flagship product Sanguinate for the treatment of sickle cell disease (SCD).

In a Phase I trial, three cohorts of eight healthy volunteers received single ascending doses of 80, 120, or 160 mg/kg of Sanguinate. Two volunteers within each cohort served as a saline control. There were no serious adverse events. Serum haptoglobin decreased, but did not appear to be dose related. The T1/2 was dose dependent and ranged from 7.9 to 13.8?h. In addition to the Phase I trial, Sanguinate was used under an expanded access emergency Investigational New Drug. Sanguinate was found to be safe and well tolerated in a Phase I clinical trial, and therefore it will advance into further clinical trials in patients [2].

Sanguinate is now in a Phase II study for the reduction or prevention of delayed cerebral ischemia following subarachnoid hemorrhage.  Phase II trials are also planned for vaso-occlusive crisis and leg ulcers secondary to SCD as well as for preventing delayed graft function following kidney transplantation. The product is being evaluated for the treatment of beta-thalassemia in international trials.

2. Misra, H.; et. al. PEGylated carboxyhemoglobin bovine (SANGUINATE): results of a phase I clinical trial. Artif Organs 2014, 38(8), 702-707.