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 July 25, 2016

AbbVie reported results of a phase III study of HUMIRA (adalimumab) for moderate to severe fingernail psoriasis and moderate to severe chronic plaque psoriasis. During the first 26 weeks (Period A), enrolled patients (n=217) were randomized to receive HUMIRA 40mg every other week (n=109) following an initial 80mg dose or matching placebo (n=108). After week 16 in Period A, if psoriasis-affected body surface area increased by 25% from baseline, patients could transition to receive open label HUMIRA for 26 weeks (Period B). HUMIRA-treated patients met primary endpoints at week 26: 46.6% achieved at least a 75% improvement in modified Nail Psoriasis Severity Index (mNAPSI) versus 3.4% for placebo (p<0.001) and 48.9% achieved Physician’s Global Assessment-fingernail-psoriasis of 0 (clear) or 1 (minimal) with at least a two-point improvement from baseline versus 6.9% for placebo (p<0.001). Adverse events (AEs) were reported by 56.9% of HUMIRA-treated patients and 55.6% of placebo-treated patients. Serious AEs were reported by 7.3% and 4.6%, respectively, including serious infections in 3.7% of those treated with HUMIRA compared to 1.9% on placebo.

Merck & Co. and Pfizer issued results for two phase III studies (VERTIS Mono and VERTIS Factorial) of ertugliflozin for type 2 diabetes. The study results showed statistically significant reductions in A1C (a measure of average blood glucose) for both ertugliflozin doses tested (5mg and 15mg daily). VERTIS Mono, a 26-week, randomized, double-blind, placebo-controlled investigational study enrolling 461 patients, met its primary endpoint, showing that patients randomized to ertugliflozin 5mg and 15mg had significantly greater A1C reductions of 0.99% and 1.16%, respectively, compared with placebo (p<0.001, for both comparisons). In addition, significantly more patients taking ertugliflozin 5mg and 15mg achieved the A1C treatment goal of less than 7% (28.2% and 35.8%, respectively) compared with placebo (13.1%) (p<0.001, for both comparisons), which was a secondary endpoint of the study. The rates of discontinuations due to AEs were low across all groups (2.6% for ertugliflozin 5mg; 2% for ertugliflozin 15mg; 3.3% for placebo). A higher incidence of genital mycotic infections in females was observed in patients taking ertugliflozin 15mg (22.6%) and ertugliflozin 5mg (16.4%) compared with placebo (5.6%). VERTIS Factorial, another 26-week, randomized, double-blind investigational study enrolling 1,233 patients, evaluated the co-administration of ertugliflozin and Merck’s DPP-4 inhibitor JANUVIA (sitagliptin). This study also met its primary endpoint, with greater reductions in A1C observed in patients taking ertugliflozin in combination with sitagliptin compared to ertugliflozin or sitagliptin alone. An A1C reduction of 1.5% was observed in both combinations studied (ertugliflozin 5mg or 15mg with sitagliptin 100mg), as compared with A1C reductions of 1% with ertugliflozin 5mg alone, 1.1% with ertugliflozin 15mg alone, and 1.1% with sitagliptin 100mg alone (p<0.001 for both combinations v. individual treatments). Merck and Pfizer plan to submit New Drug Applications to the FDA for ertugliflozin and the two fixed-dose combination tablets (ertugliflozin plus JANUVIA, and ertugliflozin plus metformin) by the end of 2016. VERTIS Mono and VERTIS Factorial are a part of the VERTIS clinical development program comprised of a total of nine phase III trials in approximately 12,600 adults with type 2 diabetes. 

Ultragenyx Pharmaceutical reported results of a phase III study of recombinant human beta-glucuronidase (rhGUS, UX003) for the treatment of Mucopolysaccharidosis 7 (MPS 7, Sly syndrome). The randomized, placebo-controlled, blind-start clinical study, conducted at four sites in the U.S., was designed to assess the efficacy and safety of rhGUS in 12 patients between 5 and 35 years of age. Patients were randomized to one of four groups. Patients were dosed with 4mg/kg of rhGUS every other week for up to a total of 48 weeks, and all groups received a minimum of 24 weeks of treatment with rhGUS. The study met its primary endpoint of reducing urinary GAG (dermatan sulfate) excretion after 24 weeks of treatment, demonstrating a reduction from baseline of 64.8 percent (p<0.0001). The study provides evidence of clinical improvement with rhGUS treatment. The Multi-domain Responder Index (MDRI) score at 24 weeks of treatment, a secondary endpoint, demonstrated an overall mean improvement (±SD) of +0.5 domains (±0.80) (p=0.0527). Six of the 12 patients had an improvement in their MDRI score of +1 or more. Five patients demonstrated no worsening of this progressive disease, or an MDRI score of 0. One patient had an MDRI score of -1. The MDRI is a summation of scores from each of the following domains: the six-minute walk test (6MWT), forced vital capacity (FVC), shoulder flexion, visual acuity and the Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) fine motor and gross motor function. For the 6MWT, the improvement (±SE) was 20.8 (±16.75) meters at 24 weeks of treatment based on the estimates from nine patients who had any change from baseline data. Three of these patients demonstrated an improvement of a magnitude equal or greater than the MID with increases of 65 meters, 80 meters and 83 meters at 24 weeks compared to baseline. For the fatigue scores, four patients improved at or above the MID level after 24 weeks of treatment and nine of 12 showed improvement at some point during the study. Based on these data, Ultragenyx plans to meet with the FDA and EMA this year to discuss plans to submit regulatory filings in the first half of 2017.