In February 2016, we initiated an open-label Phase II single arm trial for PNH patients with eculizumab resistance due to C5 polymorphisms, pursuant to a clinical trial protocol approved by the European Union national regulatory authority
The primary objective of the eculizumab-resistance program is to provide patients who have clinically demonstrated resistance to eculizumab with early access to nomacopan as a potentially lifesaving alternative. Results from this patient have demonstrated complete complement inhibition (Elisa CH50 < 8 Eq/ml, lower limit of quantification) and marked LDH reduction to around 1.5 times the upper limit of normal, or ULN.
This patient has been transferred to the long-term safety and efficacy CONSERVE study, and in aggregate has been treated for more than 28 months of therapy as of June 30, 2018 and has been self-administering nomacopan.
In May 2018, we enrolled a second PNH patient with eculizumab resistance due to C5 polymorphisms in an open-label six-month Phase II single arm trial. The trial is being conducted at a study site in New York to determine the safety and efficacy of nomacopan.
At the end of the trial, the patient will have the option of remaining on nomacopan and being entered into our long-term safety and efficacy CONSERVE study.