Trial Results

Momenta Reports Positive Top-Line Phase I Data for M281 in Healthy Volunteers

January 8, 2018

Momenta Pharmaceuticals reported positive top-line data showing safety, tolerability and proof of mechanism for M281 in a Phase I single ascending dose (SAD) and multiple ascending dose (MAD) study of normal human volunteers. Over the 98-day MAD study, M281 exhibited no serious adverse events, was well-tolerated and decreased circulating IgG levels up to 89% with a mean reduction of 84%. M281 is a fully human anti-neonatal Fc receptor (FcRn) aglycosyl­ated immunoglobulin G (IgG1) monoclonal antibody, engineered to reduce circulating pathogenic IgG antibodies, in excess of that achieved by any current treatments, by com­pletely blocking endogenous IgG recycling via FcRn. Momenta Pharmaceuticals will finalize development strategy and initiate a proof of concept clinical trial in the second half of 2018, pending regulatory feedback. The Phase 1 randomized, double-blind, placebo-controlled study evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics of M281.

Akebia Announces Results of Vadadustat in Anemia Associated with Dialysis- Dependent Chronic Kidney Disease

Akebia Therapeutics announced positive top-line results from its Phase II study of vadadustat in patients with anemia associ­ated with dialysis-dependent chronic kidney disease (DD-CKD) in Japan. The results are consistent with findings from previous stud­ies of vadadustat. Akebia’s partner, Mitsubi­shi Tanabe Pharma Corporation (MTPC), is conducting a Phase III study of non-dialysis dependent (NDD-CKD) patients in Japan and, based upon the data, is expected to begin Phase III studies in DD-CKD patients in Japan in 2018. The double-blind, placebo-controlled, dose-finding Phase II study was designed to evaluate the efficacy, safety and tolerability of orally-administered vada­dustat in Japanese patients with anemia associated with DD-CKD. This 16-week study evaluated 60 patients during a six-week placebo-controlled, fixed-dose period and a 10-week active treatment, dose adjust­ment and maintenance period. The primary efficacy endpoint was mean hemoglobin change from baseline to week six comparing vadadustat to placebo. Statistically significant improvements in the primary endpoint were observed in the vadadustat groups, 150mg (p=0.0004), 300mg (p<0.0001) and 600mg (p<0.0001), compared to placebo. The data indicate a dose-response for vadadustat.

NxThera Announces Three- Year Outcomes Data of Rezum System for Enlarged Prostate

NxThera announced the three-year outcomes data from the Rezūm II pivotal clinical trial of its minimally invasive Rezūm System, dem­onstrating significant, effective and durable lower urinary tract symptom (LUTS) relief, improved quality of life and preserved sexual function for men treated for benign prostatic hyperplasia (BPH). The Rezūm II randomized, controlled trial enrolled 197 men from 15 sites in the U.S., and one-, two- and now three-year data from this trial has demonstrated durable symptom relief with preserved sexual function in patients who were treated with the Rezūm System. The seven individual IPSS domains, including urgency and nocturia, indicated significant relief of symptoms at one month, remained significant throughout three years (p<0.0001). Sexual function was preserved in patients treated with the Rezūm System, as measured via the International Index of Erectile Function (IIEF-15) and Male Sexual Health Questionnaire (MSHQ) through three years of follow-up. The ejaculatory bother score (MSHQ-EjD) improved over baseline from 12 to 36 months (p<0.004). No latent related adverse events occurred and no de novo erectile dysfunction was reported. Surgical retreatment rate was 4.4 percent (six out of 135 subjects) over three years of follow-up. Four of these six secondary interventions were related to an untreated median lobe at baseline. Thirty of the patients received treat­ment to the median lobe or elevated central zone, in addition to treating the lateral lobes; these 30 patients not only demonstrated sig­nificant improvements in symptom (IPSS) and urinary flow rates (Qmax), but also demon­strated decreased post-voiding residual (PVR) urine from 24 months to 36 months (p<0.04). Surgical retreatment rate was 4.4 percent (six out of 135 subjects) over three years of follow-up. Four of these six secondary interventions were related to an untreated median lobe at baseline.