Bioverativ announced that the first patient has been dosed in the Phase III clinical program of its investigational therapy BIVV009 for cold agglutinin disease (CAgD). The Phase III program includes two parallel Phase III trials, Cardinal and Cadenza, which are evaluating the efficacy and safety of BIVV009 in adult patients with primary CAgD, a disease with no approved therapies. The study assessed the long-term efficacy, safety and PK/PD profile of BIVV009 in six severely anemic primary CAgD patients. Primary and secondary outcome measures were achieved in the CAgD patients in the study. Hemoglobin levels increased in all six patients (median >4g/dl) which eliminated the need for transfusions while on treatment. There were no serious AEs assessed as related to BIVV009 by the investigator.
Dermira announced that the investigational treatment olumacostat glasaretil (formerly DRM01) did not meet the co-primary endpoints in its two Phase III pivotal trials (CLAREOS-1 and CLAREOS-2) in patients nine years of age and older with moderate-to-severe acne vulgaris. The Phase III clinical program included two randomized, multi-center, double-blind, parallel-group, vehicle-controlled trials, CLAREOS-1 and CLAREOS-2. The program assessed the efficacy and safety of olumacostat glasaretil compared to a potential New Drug Application (NDA) submission to the U.S. Food & Drug Administration (FDA). The program enrolled a total of 1,503 patients (CLAREOS-1, n=759 and CLAREOS-2, n=744) at 94 sites in the United States, Canada and Australia. In each trial, patients were randomized in a 2:1 fashion to receive either olumacostat glasaretil at a concentration of five percent or vehicle twice daily for 12 weeks. No treatment-related serious adverse events were reported, and no new or unexpected events were observed. None of these co-primary endpoint results were statistically significant.
Aimmune Therapeutics announced that the results of its pivotal Phase III PALISADE trial of AR101 for the treatment of peanut allergy. PALISADE studied the efficacy and safety of AR101 in peanut-allergic patients by assessing reductions in clinical reactivity to peanuts. PALISADE enrolled a total of 554 patients ages 4-49 with the primary analysis of patients ages 4–17. The trial met its primary and secondary endpoints, and AR101 demonstrated an encouraging tolerability and safety profile over the course of approximately one year of treatment. After treatment, patients completed an exit DBPCFC. The median tolerated dose of peanut protein in the entry DBPCFC was 10 mg, the equivalent of approximately 1/30 of a peanut, and, at baseline, 43 percent of patients had a peanut-specific IgE level >100 kU/L. There were no deaths or suspected, unexpected serious adverse reactions (SUSARs) in the trial, and the incidence of serious adverse events (SAEs) was low, as 1.1 percent of AR101 patients experienced SAEs that were possibly related to treatment. Most AR101 patients (85.5 percent} did not experience any investigator-reported systemic hypersensitivity reactions (SHRs) during the trial.
Esperion announced positive top-line results from the first pivotal, Phase III study (Study 4 or 1002-048) of bempedoic acid. The 12-week study met its primary endpoint with LDL-C lowering totaling 28 percent (p<0.001). The LDL-C lowering for the bempedoic acid group was 23 percent from baseline, as compared to an LDL-C increase of five percent for the placebo group. Patients treated with bempedoic acid also achieved a significantly greater reduction of 33 percent in high-sensitivity C-reactive protein (hsCRP), compared to the placebo group which had an increase of two percent (p<0.001). The 12-week, global, pivotal, Phase III randomized, double-blind, placebo-controlled, multicenter study evaluated the efficacy and safety of bempedoic acid 180 mg/day versus placebo as add-on therapy in patients with atherosclerotic cardiovascular disease (ASCVD), or at a high risk for ASCVD. The primary objective was to assess the 12-week LDL-C-lowering efficacy of bempedoic acid versus placebo when added to ezetimibe and up to the lowest starting dose of a statin.