Akari Therapeutics' (NASDAQ:AKTX) coversin Phase II trial in paroxysmal nocturnal hemoglobinuria (PNH) has cautious expert optimism after full complement ablation was achieved at the same dose in an earlier study.
The close correlation between Phase I's complement ablation endpoint and Phase II's serum lactate dehydrogenase (LDH) measurement, as well as Phase II's dose monitoring protocol, underscores success confidence, they said. Some noted, however, a lack of previous patient data, alongside Phase II's small study size may hinder its success prospects.
Similarly to Alexion Pharmaceuticals' (NASDAQ:ALXN) Soliris (eculizumab) - the only approved PNH treatment -- enhanced infection risk and a subsequent black-box warning are expected for coversin, some experts added. Coversin's leukotriene B4 (LTB4) inhibition effects require safety assessments, one said.
Coversin could read peak sales of up to USD 507m, according to an analyst report. A separate report notes Phase II outcome uncertainty due to coversin's previously small trial size yet its shared mechanism-of-action (MOA) with Soliris' validated clinical pathway gives some hope for success. Several analyst reports expect results before YE16.
Coversin is a small, recombinant protein that inhibits the formation of the membrane attack complex (MAC), according to Akari's website. PNH involves uncontrolled complement activation by the MAC, resulting in haemolysis. Symptoms include kidney disease, thrombosis, liver dysfunction and pulmonary hypertension.