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 January 23, 2017
AbbVie issued results of a phase Ib/II
PCYC-1102 trial and PCYC-1103 extension study of single-agent Imbruvica (ibrutinib) for chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Patients received either 420mg or 840mg once daily until disease progression or unacceptable toxicity. In this analysis, 89% of treatment-naïve (TN) and relapsed/refractory (R/R) patients with CLL/SLL, including those with high-risk disease, showed a complete or partial response. Among R/R patients, 34% had del17p, 35% had del11q, 47% had del13q, and 78% had unmutated IGVH. Almost one-third of patients (29%) who received ibrutinib as their first treatment for the disease achieved a complete response (CR), and patients lived without disease progression longer when treatment was started earlier in the course of the disease. In the five years of follow-up, the overall response rate (ORR) in patients treated with Imbruvica was 89%, with 14% of patients achieving CR [87% ORR with 29% CR in TN patients (n=31) and 89% ORR with 10% CR in R/R patients (n=101)]. Median time on study was 62 months for TN patients and 49 months for R/R patients. At five years, progression-free survival (PFS) was 92% in TN patients and 43% in R/R patients, and overall survival (OS) was 92% for TN patients and 57% for R/R patients. Median PFS was not reached in the TN group and was 52 months for previously treated R/R patients. Median OS was not reached for TN or R/R patients.
Keryx Biopharmaceuticals reported results of a phase III study of ferric citrate for iron deficiency anemia (IDA) in non-dialysis-dependent chronic kidney disease (NDD-CKD). The pivotal study randomized 234 patients (233 patients received at least one starting dose of ferric citrate) at 32 clinical sites in the U.S. NDD-CKD patients with hemoglobin levels between 9g/dL and
11.5g/dL and who were intolerant to or had inadequate response to oral iron supplements were randomized 1:1 (ferric citrate versus placebo), n=117 and n=116, respectively. Patients enrolled in the study were not allowed to receive any IV or oral iron, or ESAs during this study. The study had a 16-week, randomized, double-blind, placebo-controlled efficacy period followed by an eight-week open-label safety extension period in which all patients remaining in the study, including the placebo group, received ferric citrate. During the 16-week efficacy period, ferric citrate was administered at a starting dose of three tablets per day with food and could be titrated every four weeks by an additional three tablets for up to a maximum of 12 tablets per day; the mean dose received in ferric citrate treated patients was five tablets per day. The primary endpoint was the proportion of patients achieving a =1g/dL increase in hemoglobin at any point during the 16-week efficacy period. Baseline laboratory values were similar between the treatment arms. Use of ferric citrate in patients with NDD-CKD and IDA, as highlighted above, is investigational and has not been determined to be safe or efficacious. Ferric citrate delivered a clinically meaningful 1g/dL increase in hemoglobin levels for the majority (52.1% (61/117)) of patients treated at any point during the 16-week efficacy period, increases were seen as early as one to two weeks after start of treatment and were sustained for the majority of patients who achieved the primary endpoint. Ferric citrate was generally well-tolerated and adverse events were consistent with its known safety profile, with diarrhea reported as the most common adverse event.
Sunovion Pharmaceuticals announced results of a phase II/III study of dasotraline for attention deficit hyperactivity disorder (ADHD). SEP360-202 was a six-week, randomized, double-blind, multicenter, placebo-controlled, parallel-group efficacy and safety trial that compared dasotraline with placebo in children ages 6 to 12 years with a primary diagnosis of ADHD (DSM-5 criteria), ADHD RS-IV HV score of =28 and CGI-S score of =4 at study baseline. Of those enrolled, 342 patients were randomized 1:1:1 to receive dasotraline 2mg/day (n=111) or 4mg/day (n=115), or placebo (n=116) once daily. Patients in the 4mg/day arm started at the 2mg/day dose for the first week of the trial and were increased to 4mg/day at week two. Children taking dasotraline 4mg/day experienced a statistically significant improvement in ADHD symptoms compared to placebo, as measured by the ADHD Rating Scale IV: Home Version (ADHD RS-IV HV) total score (least squares [LS] mean change from baseline at week six: -17.53 [95% CI:-20.12, -14.95] vs -11.36 [-13.89, -8.83], respectively; effect size (ES)=0.48, p<0.001). This statistically significant and clinically relevant improvement over placebo was maintained each week through week six. Improvements in Clinical Global Impression-Severity of Illness Scale (CGI-S) scores were also statistically significant in the 4mg/day dose arm compared to placebo. The 2mg/day dose arm did not demonstrate a statistically significant difference from placebo in the ADHD RS-IV total score and did not statistically separate from placebo in the CGI-S scores. Both dasotraline 4mg and 2mg arms were generally well-tolerated with an adverse event (AE) profile consistent with completed adult dasotraline studies. The most common treatment-emergent adverse events (TEAE) (reported in 5% or more of patients and greater than placebo) included insomnia, decreased appetite and decreased weight. Pending successful completion of ongoing studies and discussions with the FDA, Sunovion intends to submit a New Drug Application (NDA) to the FDA in 2017 for ADHD in children and adults.