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ARTICLE | Clinical News

Ra reports mixed data from Phase II trial of PNH candidate

December 8, 2017 7:03 PM UTC

Ra Pharmaceuticals Inc. (NASDAQ:RARX) reported data from a Phase II trial to treat paroxysmal nocturnal hemoglobinuria (PNH) showing that subcutaneous RA101495 met the primary endpoint of reducing lactate dehydrogenase (LDH) levels from baseline to the mean of weeks 6-12 in a cohort of 10 Soliris eculizumab-naïve patients (p=0.002). However, in a cohort of patients who had switched from Soliris to RA101495, 7 of 11 transfusion-dependent patients experienced breakthrough hemolysis and reverted back to Soliris. Ra said all 5 transfusion-independent patients in the switch cohort successfully switched to RA101495 as indicated by stable LDH levels and no episodes of breakthrough hemolysis.

The open label, international trial enrolled 29 patients across 3 cohorts, including 10 Soliris-naïve patients, 16 patients who switched from Soliris to RA101495 and 3 patients who had an inadequate response to Soliris. Patients received 0.3 mg/kg RA101495 at day 1, followed by 0.1 mg/kg RA101495 daily...

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