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 24, 2017
Allergan reported data from a multicenter, randomized, double-blind, placebo-controlled, phase II study of BOTOX for major depressive disorder (MDD). The study evaluated two different doses of BOTOX (30 units or 50 units) relative to placebo in adult females with MDD over duration of up to 24 weeks. The BOTOX 30 U dose demonstrated numerically superior efficacy in MADRS total score compared to placebo. The treatment (LS mean) difference for 30 U was -4.2 at three weeks (p=0.005); -3.7 at week six (p=0.053) and -3.6 at week nine (p=0.049). The primary end point was at week six. The 50 U did not demonstrate superior efficacy over placebo (LS mean difference was 1.3). Both secondary efficacy variables (CGI-S and HAMD-17) showed numerically superior efficacy over placebo and trended in the same direction as the primary efficacy variable for 30 U, but not for 50 U. Both 30 U and 50 U were well-tolerated. The company plans to move forward and develop a phase III program.
Genentech reported interim results from the phase III HAVEN 2 study evaluating emicizumab prophylaxis in children less than 12 years of age with hemophilia A and inhibitors to factor VIII. HAVEN 2 is a single-arm, multicenter, open-label study. The interim analysis, after a median of 12 weeks of treatment, included 19 children. The study will enroll a total of 60 children for its final analysis, planned after 52 weeks of treatment with emicizumab. At this interim analysis, after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time. These findings are consistent with results from the phase III HAVEN 1 study in adults and adolescents (12 years of age or older) with hemophilia A and inhibitors to factor VIII, in which emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time compared to no prophylaxis, as well as compared to prior prophylaxis with bypassing agents. The most common adverse events with emicizumab in the HAVEN 2 study were injection site reactions and nasopharyngitis. Two additional phase III studies of emicizumab are ongoing.
Novocure announced data from a post hoc analysis of the EF-14 pivotal phase III clinical trial of Optune in combination with temozolomide for the treatment of newly diagnosed glioblastoma. The objective of the pre-specified post hoc analysis was to evaluate the efficacy and safety of Optune when added to physician’s best choice second-line treatment after first disease recurrence among patients enrolled in the EF-14 trial. The analysis shows that the median overall survival of patients treated with Optune increased by 28% compared to patients treated with physician’s best choice second line chemotherapy alone from 9.2 months to 11.8 months (HR=0.70, p=0.049). Bevacizumab, alone or in combination with chemotherapy, was the most frequently used second-line treatment. The analysis also shows that the median overall survival of patients treated with Optune in combination with bevacizumab increased by 31% compared to patients treated with bevacizumab alone from nine months to 11.8 months (HR=0.61, p=0.043).
OncoMed Pharmaceuticals issued results of a 145-patient, phase II trial of tarextumab (anti-Notch2/3, OMP-59R5) in combination with etoposide plus either cisplatin or carboplatin chemotherapy in previously untreated patients with extensive-stage small cell lung cancer. The double-blind, multicenter trial randomized two study arms that received either 15mg/kg of tarextumab every three weeks in combination with six cycles of etoposide and either cisplatin or carboplatin chemotherapy followed by tarextumab maintenance to progression or six cycles of chemotherapy and a placebo. The median progression-free survival (mPFS) for tarextumab plus chemotherapy was 5.6 months versus 5.5 months for chemotherapy plus placebo (HR=0.969). The median overall survival (mOS) analysis did not show a benefit for tarextumab in combination with chemotherapy (mOS=9.3 months) compared to the chemotherapy plus placebo arm (10.3 months; HR=1.01). Five individual Notch biomarkers (Hes1, Hes6, Hey1, Hey2 and Notch3) failed to identify a definitive subset of patients with a treatment effect on either mPFS or mOS. Overall response rates were 68.5% and 70.8% in the tarextumab and placebo arms respectively. The combination of tarextumab plus chemotherapy was well-tolerated.