Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of December 11, 2017

Daiichi Sankyo announced that updated safety and efficacy data from two subgroups of patients with metastatic breast cancer from an ongoing phase I study of DS-8201. Updated preliminary results in a subgroup analysis of 57 efficacy evaluable patients with HER2-positive metastatic breast cancer pre-treated with ado-trastuzumab emtansine (T-DM1) showed that DS-8201 demonstrated a confirmed overall response rate of 61.4% (35 of 57 patients) and a disease control rate of 94.7% (54 of 57 patients). Among 50 of these patients who also were pre-treated with pertuzumab, DS-8201 demonstrated a confirmed overall response rate of 62% (31 of 50 patients) and a disease control rate of 94% (47 of 50 patients). In 39 efficacy evaluable HER2-positive patients with hormone-receptor positive disease, DS-8201 demonstrated an overall response rate of 56.4% (22 of 39 patients) and disease control rate of 92.3% (36 of 39 patients). The majority of patients with HER2-positive metastatic breast cancer were continuing to receive treatment at the time of data cut-off. Preliminary estimates of median progression-free survival have reached 10.4 months.

Merck & Co. released pivotal phase III study results of PREVYMIS (letermovir) for prophylaxis (prevention) of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT). In the study, significantly fewer patients in the PREVYMIS arm (37.5%, n=122/325) compared to the placebo arm (60.6%, n=103/170) developed clinically significant CMV infection, discontinued treatment or had missing data through Week 24 post-transplant (p<0.001), the primary efficacy endpoint. The treatment effect for PREVYMIS in preventing clinically significant CMV infection was consistent across pre-specified high- and low-risk strata for CMV reactivation both at week 14 (end of treatment) and at week 24 post-transplant. All-cause mortality in patients receiving PREVYMIS was lower compared to placebo at Week 24 post-transplant and at week 48 post-transplant. This double-blind study randomized adult CMV-seropositive allogeneic hematopoietic-cell transplant recipients to receive PREVYMIS or placebo orally or intravenously through week 14 post-transplant. PREVYMIS was dosed at 480mg/day (or 240mg/day when co-administered with cyclosporine).  

xBiotech reported results of a phase II study evaluating MABp1 for Hidradenitis Suppurativa (HS). The 20 patient, double-blind, placebo-controlled study randomized patients 1:1 to receive either MABp1 or placebo every two weeks for 12 weeks. Patients in the study underwent primary assessment of efficacy using Hidradenitis Suppurativa Clinical Response (HiSCR) scores at 12 weeks, continued by a follow up phase to assess time to relapse after an additional 12 weeks without therapy. Efficacy measures include assessment of HiSCR scores, a validated method for evaluating efficacy in HS patients, as well as quality of life assessment and ultrasonographic evaluation. The publication highlights the efficacy of MABp1, in which the study’s primary endpoint was met in 60% of MABp1 treated patients compared to 10% of placebo patients (odds ratio 13.50, 95% confidence intervals 1.19-152.51; p=0.035). The clinical efficacy of MABp1 was maintained until week 24 (12 weeks after discontinuation of treatment) at which time point, no patients treated with placebo had a positive HiSCR score (0%) compared to four out of 10 patients (40%) treated with MABp1. Treatment with MABp1 was also accompanied by better patient-reported outcomes. Decrease of the visual analogue scale (VAS) was found in 30% of placebo patients compared with 70% of patients treated with MABp1.

Zogenix announced data from its phase III trial (Study 1) of ZX008 (low-dose fenfluramine hydrochloride) for Dravet syndrome. The randomized, double blind, placebo-controlled study enrolled 119 patients across sites in the U.S., Canada, Europe and Australia. The median age of patients was 8 years (range, 2-18 years). Following a six-week baseline observation period, patients were randomized to one of three treatment groups: ZX008 0.8mg/kg/day (30mg maximum daily dose; n=40), ZX008 0.2mg/kg/day (n=39) and placebo (n=40) in which ZX008 or placebo was added to current regimens of antiepileptic drugs. Patients were titrated to their target dose over two weeks and then remained at that fixed dose for 12 weeks. The mean baseline convulsive seizure frequency across the study groups was approximately 40 seizures per month.