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New Medical Therapies™

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 February 23, 2015

Baxter International reported results of a phase III study of BAX 855 for hemophilia A. The prospective, global, multicenter, openlabel, two-arm phase III study evaluated BAX 855 among 137 previously treated hemophilia A patients (PTP) who were 12 years or older. Patients were assigned either to twice weekly prophylaxis (40-50IU/kg, n=120) or on-demand treatment (10-50IU/kg, n=17). In addition to a reduced annualized bleed rate (ABR), BAX 855 also was effective in treating bleeding episodes, 96% of which were controlled with one or two infusions at a median dose of 29IU/ kg per infusion. Treatment was rated excellent or good for nearly all episodes (96.2%). In the prophylactic group (n=101), 40% of patients experienced no bleeds. No patients developed inhibitors to BAX 855 and no treatment-related serious adverse events, including hypersensitivity, were reported. Seven adverse reactions in six patients, including headache, diarrhea, nausea and flushing were reported. The study findings supported Baxter’s December 2014 submission for approval of BAX 855 to the FDA. Baxter’s continuation study for patients who completed the pivotal trial and the phase III study among previously treated patients under the age of 12 with severe hemophilia A remain ongoing. Upon completion of the pediatric study, Baxter expects to file for marketing authorization with the EMA in 2016.

Chiasma issued results of a phase III study of octreotide in the treatment of adults with acromegaly. The open-label, dose-titration, single-arm, multi-center, baseline-controlled phase III trial enrolled 155 people diagnosed with acromegaly and managed on somatostatin analogs. During the dose-titration phase of the study, patients initially received 40mg (20mg BID) daily of octreotide capsules, which could be increased, as required, to 60mg/day or 80mg/day (two 40mg doses) to maintain clinical and biochemical response. The dose-titration phase was followed by a fixed-dose phase for up to seven months; at that point, patients could elect to continue into a voluntary six-month extension phase up to 13 months. The study found 89% of patients were either completely or partially responding to parenteral treatment at baseline. 65% of patients who received octreotide capsules twice a day achieved disease control, as measured by circulating concentrations of insulin-like growth factor-1 (IGF-1) and growth hormone (GH) concentrations, at seven months (core treatment period), meeting the primary endpoint, and 62% achieved disease control at 13 months (end of treatment). 85% of responders, at the end of the dose-titration phase, had sustained response up to 13 months. 80% of patients who entered the fixed-dose phase improved or maintained their acromegaly symptoms (54% improved, 26% maintained), at the end of treatment. 89% percent of patients experienced an adverse event (AE); 92% of the events were mild to moderate. The most common AEs were reported in the gastrointestinal system, nervous system and musculoskeletal system, consistent with the known safety profile of octreotide and the disease burden of acromegaly, with no AEs related to the oral route of administration. The majority of the gastrointestinal AEs reported in the study were mild to moderate, occurring within the first two months of treatment and resolved on treatment (median duration 13 days). Based on these results, Chiasma intends

Genzyme released results of a phase III study of Cerdelga (eliglustat) in treatment-naïve patients with Gaucher disease type 1. The randomized, double-blinded, placebo-controlled, multinational registration trial enrolled 40 treatment-naïve patients with Gaucher disease type 1 who had splenomegaly in addition to thrombocytopenia and/or anemia at study entry. Patients were stratified by baseline spleen volume and randomized 1:1 to receive Cerdelga (50mg or 100mg twice daily) or placebo for nine months. The primary efficacy endpoint of the study demonstrated a statistically significant reduction from baseline in spleen size by a mean of 28% compared in Cerdelga patients with a mean increase of 2% in placebo patients, for an absolute difference of 30% (P<0.0001). Hemoglobin levels increased from baseline by an absolute difference of 1.2g/dL compared with placebo (P=0.0006). Liver volume decreased from baseline by an absolute difference of 6.6% compared with placebo (P=0.0072). Platelet levels increased from baseline by an absolute difference of 41% compared with placebo (P<0.0001). There were no serious adverse events associated with either treatment group.

Mesoblast reported results of a phase III study of MSC-100-IV for severe steroid-resistant acute graft versus host disease (aGVHD). The pediatric trial enrolled 160 children. Overall response (defined as partial + complete response) at day +28 was 64%, and this response correlated with statistically significant improved survival compared to non-responders at day +100 after an MSC-100-IV infusion (81% v. 39%, p=0.0001). The day 28 overall responses by grade were 74% for grade B, 66% for grade C and 59% for grade D. Overall responses by organ involvement at day +28 were 62% for GI, 77% for skin and 53% for liver. Approximately 80% of grade B/C patients and over 50% of grade D patients survived to day 100. A single-arm, open-label, phase III registration trial to support product approval by the FDA, which will include approximately 60 pediatric patients with aGVHD, is in progress.

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