Pharmaceutical Business review

FDA grants orphan drug status for ARCA’s rNAPc2 as potential treatment for Ebola

Recombinant Nematode Anticoagulation Protein c2 (rNAPc2) has previously showed post-exposure efficacy in non-human primate models of Ebola hemorrhagic fever virus (HFV) and it was originally developed as a cardiovascular therapy for thrombosis and other indications.

As a result, the drug candidate has an extensive human clinical record, and has been safely evaluated in more than 700 human patients in nine Phase I and II clinical trials for cardiovascular disease.

rNAPc2 is a potent and selective inhibitor of tissue factor (TF), the protein responsible for starting the extrinsic coagulation pathway, the primary coagulation mechanism in humans.

The company said that the rationale for rNAPc2 as a HFV therapy arises from the role of TF in HFV mediated disseminated intravascular coagulation (DIC), an often fatal complication of the HFV disease syndrome that leads to spontaneous hemorrhage.

The pilot studies of rNAPc2 in non-human primates have showed potential efficacy against two of the most deadly known HFVs, Ebola and Marburg.

Results from these studies has showed that, when administered as a post-exposure therapy, rNAPc2 showed evidence of efficacy in improving survival and inhibiting the DIC process.

Additionally, in these studies, rNAPc2 showed anti-inflammatory and anti-viral properties.

Currently, the company is exploring options to develop rNAPc2 including seeking development partners, out-licensing the compound and applying for grant or government funding.