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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 August 24, 2015

Biogen has issued results of a phase III study of Alprolix for severe hemophilia B. B-YOND enrolled 116 males, including 93 participants (81%) who completed B-LONG, and 23 (100%) of those who completed Kids B-LONG. The open-label extension study was 27.6 months for adults and adolescents and 47.7 weeks for children under age 12. The median overall median annualized bleeding rates (ABR) was zero for children under age 6 who received weekly prophylaxis (n=9). For children between 6 and 12 years old, median overall ABRs were 2.65 (n=10), 2.37 (n=5) and 3.13 (n=1) in weekly, individualized and modified prophylaxis regimens, respectively. In each age group, the median average weekly dose for participants previously on prophylaxis was similar for individuals in the weekly and individualized treatment arms. Safety results were typical of the hemophilia B populations studied. The most common adverse events (incidence of greater than or equal to 5 percent) for adults and adolescents included headache, common cold and vomiting. For children under age 12, the most common adverse events were falls, common cold and seasonal allergy.

Boehringer Ingelheim has reported results of a phase IIIb study of Spiolto Respimat (tiotropium/olodaterol) for chronic obstructive pulmonary disease (COPD). OTEMTO 1&2 build on the pivotal phase III TONADO trials that demonstrated Spiolto Respimat provides significant improvements in lung function, quality of life, breathlessness and reduction in rescue medication use over Spiriva Respimat right from the initial disease stages when patients first need maintenance therapy. OTEMTO 1&2 are part of the >15,000 patient TOviTO phase III clinical trial program. OTEMTO trials show Spiolto Respimat provides clinically meaningful improvements in breathlessness compared to placebo (measured by a 1.62 point improvement in Transition dyspnoea index focal score); consistent improvements in lung function, breathlessness and quality of life compared to Spiriva (tiotropium); and a safety profile similar to Spiriva or placebo. Incidence of adverse events (AEs) was broadly similar across treatment groups, with a higher incidence of AEs leading to discontinuation in the placebo groups compared to the treatment groups. The FDA recently accepted for review an sNDA to include the OTEMTO quality-of-life data in the Stiolto Respimat label.

CytoDyn has issued results of a phase IIb trial of PRO 140 for HIV. PRO 140 reduces viral loads by as much as 1.8log with one dose per week. In the monotherapy study, some HIV patients are experiencing suppressed viral load for 11 months. The first self-injectable antibody, PRO 140, has documented a 98% success rate. The company has a phase III trial planned for 300 patients, expected to begin by the end of Q3 2015. The company may apply for a Breakthrough designation with the FDA and plans to submit its NDA for final approval of PRO 140 in November 2016. PRO 140’s previous Fast Track designation carries a possibility of accelerated approval.

Merz Neurosciences has issued results of a phase III study of incobotulinumtoxinA for post-stroke upper limb spasticity. The trial was a prospective, multicenter, randomized, doubleblind, placebo-controlled main period (12-week duration) with a single incobotulinumtoxinA 400 U treatment or placebo, followed by a 36-week open-label extension in which subjects (n=259) could receive up to three additional treatment cycles. The main period randomization rate of incobotulinumtoxinA to placebo was 2:1. In the primary analysis, the co-primary variables were improvements in muscle tone defined as “change in Ashworth Scale score from baseline to week four” and improvements in functional outcomes, defined as “investigator’s Global Impression of Change at the week four visit.” Both showed statistical significance, with p<0.001 and p=0.003 respectively. The trial also met a key secondary outcome measure, in which subjects with an improvement =1 on the Ashworth Scale at week four were classified as responders and showed significant improvements in disability associated with spasticity (p<0.001). Treatment-related adverse events were reported for 3.8% and 1.9% of subjects treated with incobotulinumtoxinA and placebo, respectively. Merz has submitted an sBLA to the FDA to seek approval for the use of incobotulinumtoxinA in the treatment of post-stroke upper limb spasticity and anticipates FDA action prior to the end of this calendar year.

Omeros has released results of a phase II trial of OMS721 for the treatment of thrombotic microangiopathies (TMAs). The trial is designed to enroll primarily hemolytic uremic syndrome (aHUS) patients but also patients with thrombotic thrombocytopenic purpura (TTP) and hematopoietic stem cell transplant (HSCT)-related TMA. There was no placebo arm. In each three-patient cohort, OMS721 is dosed for four weeks. In this trial, platelet counts in all three aHUS patients in the mid- and high-dose cohorts (two in the mid-dose and one in the high-dose cohort) were normal after the treatment period, with a statistically significant mean increase from baseline of about 68,000 platelets/mL (p=0.0055). In the mid-dose cohort, the two patients with plasma therapy-resistant aHUS demonstrated 47% increase in mean platelet count, resulting in both patients having counts in the normal range; 86% decrease in mean schistocyte count, with schistocytes disappearing in one patient; 71% increase in mean haptoglobin, with both patients reaching the normal range during treatment and one slipping slightly below normal at one week following the last dose; and 5% decrease in the mean levels of LDH, with levels in both patients remaining slightly elevated above normal range. The company is discussing a phase III trial design with the FDA.

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