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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 April 25, 2016

Marathon Pharmaceuticals released results of a phase III study of deflazacort for Duchenne Muscular Dystrophy (DMD). The pivotal, randomized, double-blind, placebo controlled and active comparator study enrolled 196 DMD patients. Patients were randomized to either deflazacort 0.9mg/kg/day, deflazacort 1.2mg/kg/day, prednisone 0.75mg/kg/day or placebo for 12 weeks. The primary endpoint was change in average muscle strength from baseline to week 12 with deflazacort and prednisone compared to placebo measured by the Medical Research Council Index (MRC). Deflazacort met the primary endpoint at week 12 v. placebo (p=0.0173 and, p=0.0003 for 0.9mg/kg/d and 1.2mg/kg/d doses, respectively v. -0.10 for placebo). After 12 weeks, placebo patients were re-randomized to either of the three active treatments from weeks 12 to 52 and those not randomized to placebo were maintained on their blinded active treatment through 52 weeks. Muscle strength and timed functional tests were included in the analysis. The analyses of non-ambulatory patients were post-hoc and not pre-specified. Muscle strength in non-ambulatory DMD patients, deflazacort at 0.9mg/kg/d and 1.2mg/kg/d, were assessed, with the deflazacort 1.2mg/kg/d arm reaching statistical significance at week 12 v. placebo (p=0.04). The FDA has granted Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation for deflazacort for DMD. Marathon expects to submit an NDA for deflazacort in May 2016 and, if approved by the FDA, deflazacort could be made commercially available in the U.S. in early 2017. 

Morphotek reported results of a phase III trial of farletuzumab (MORAb-003) in combination with a platinum standard chemotherapy regimen (carboplatin plus taxane) in platinum-sensitive ovarian cancer patients in first relapse. All subjects received carboplatin plus paclitaxel or docetaxel (for six cycles combined with randomized test product 1:1:1 farletuzumab 1.25mg/kg, farletuzumab 2.5mg/kg, or placebo), and single-agent test product was continued weekly until disease progression. Neither farletuzumab dose met the study’s primary progression-free survival (PFS) endpoint when compared to placebo, with PFS of 9.0, 9.5 (hazard ratio [HR]=0.99) and 9.7 months (HR=0.86) for the placebo, farletuzumab 1.25mg/kg, and farletuzumab 2.5mg/kg arms, respectively. There was no difference in overall survival. Prespecified subgroup analyses demonstrated that subjects with a low CA125 level (less than three times the upper limit of normal) correlated with longer PFS (HR=0.49) and OS (HR=0.44) for farletuzumab 2.5mg/kg versus placebo. Subjects with higher farletuzumab exposure also showed superior PFS and OS compared to placebo. The most common adverse events were those known to be associated with chemotherapy, including alopecia, nausea, neutropenia, fatigue, thrombocytopenia and neuropathy. Morphotek has initiated a phase II trial to investigate the potential clinical benefit observed in patients with a low CA125 level.

Zafgen issued results of a phase III study of beloranib for Prader-Willi syndrome (PWS). The pivotal, double-blind, placebo-controlled trial enrolled 107 patients randomized to receive twice-weekly subcutaneous injections of either 2.4mg or 1.8mg of beloranib or placebo. The co-primary efficacy endpoints for this trial were an improvement in hyperphagia-related behaviors and reduction in body weight. Patients in the ZAF-311 trial were markedly obese at baseline. Patients randomized to receive placebo displayed substantial (4.15%) increase in body weight over the course of the six months of randomized treatment. Body weight gain in this patient population was anticipated, and typically occurs throughout life generally due to a lack of effective treatments for managing obesity. Patients treated with beloranib, in contrast to placebo, experienced a reduction in weight, with the 2.4mg dose arm displaying a 5.3% reduction from baseline, with a placebo-adjusted weight loss of 9.45%. The HQ-CT is a PWS-specific study instrument that provides an assessment by caregivers of the food-seeking behaviors exhibited by patients. The scale provides a composite value from nine questions, each rated on a scale of zero to four units (total range of score of zero to 36). Patients in the ZAF-311 trial were enrolled only if their baseline HQ-CT total score was greater than 12 units, representing moderate-to-severe hyperphagia related behaviors at baseline. While hyperphagia-related behaviors were stable over six months of treatment in the placebo arm, both the 2.4mg and 1.8mg beloranib arms showed highly statistically significant reductions in HQ-CT total score, indicative of reduced hunger-associated behaviors.

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