Like many aspects of healthcare business space, drug development is in a state of constant change for the purpose of better health outcomes. Here is a summarised report of new drug therapies currently in clinical trials worldwide as from this week.
|Sponsor||Study Phase||Drug Name/Code||Clinical Indication||Status||Comment|
|Akcea Therapeutics||Phase II||AKCEA-APO(a)-LRx||Cardiovascular Disease (CVD) and Hyperlipoproteinemia (A)||This randomized, double-blind, placebo-controlled, dose-ranging Phase II study included 286 patients with established CVD and high lipoprotein/Lp (a) (baseline mean of approximately 100 mg/dL [250 nmol/L] – more than three times the upper limit of normal). All subjects were treated for at least six months, with some treated up to one year. The primary study objective was to characterize the safety and tolerability of AKCEA-APO(a)-LRx and to inform dose and dose frequency selection for the planned Phase III cardiovascular outcomes study. A key finding was that it achieved statistically significant dose-dependent reductions of Lp(a) compared to placebo at all dose levels, including low monthly doses of AKCEA-APO(a)-LRx.||The most common adverse event was injection site reactions (ISRs). ISRs were mostly mild and occurred in a minority of patients. There were no reports of any patient in the study experiencing a confirmed platelet level below 100,000/mm3. The incidence of platelet levels below normal (140,000/mm3) was comparable between the active (10.5%) and placebo (14.9%) groups. Roughly 90% of patients completed treatment and the rate of treatment discontinuation was comparable between the active and placebo groups.|
|Poxel SA||Phase III||Imeglimin||Type 2 Diabetes||The TIMES 2 trial of the Phase III program for Imeglimin, an investigational therapeutic agent for type 2 diabetes, for patient registration in Japan has been completed. Otherwise referred to as TIMES (Trials of IMeglimin for Efficacy and Safety), the Imeglimin Phase III registration program in Japan includes three pivotal trials to evaluate the efficacy and safety of Imeglimin in over 1,100 patients. The TIMES 2 trial is a 52-week, open-label, parallel-group study to assess the long-term safety and efficacy of Imeglimin in Japanese patients with type 2 diabetes. In this particular study, Imeglimin is administrated as a monotherapy or combination therapy to over 700 patients with existing hypoglycemic agents, including a DPP4 inhibitor, SGLT2 inhibitor, biguanide, sulphonylurea and GLP1 receptor agonist.||Imeglimin is the first clinical candidate in a new chemical class of oral agents called Glimins by the WHO. Imeglimin has a unique mechanism of action targetting mitochondrial bioenergetics. Imeglimin acts on all three key organs - the liver, muscles and pancreas, all of which play an important role in the treatment of type 2 diabetes and it has shown glucose lowering efficacy by increasing insulin secretion in response to glucose, improving insulin sensitivity and suppressing gluconeogenesis.|
|Genentech||Phase III||Baloxavir Marboxil||Influenza Types A/H3N2 & B||This Phase III trial called CAPSTONE-2 was a multicentre, randomized, double-blind study that evaluated the efficacy and safety of a single dose of baloxavir marboxil compared with placebo and oseltamivir in people 12 years or older who are at a high risk of complications from influenza. The CAPSTONE-2 study revealed that Baloxavir Marboxil has efficacy (reduced time to improvement of influenza symptoms) in influenza type A/H3N2 (median time of 75.4 hours and 100.4 hours; p<0.05) and type B (median time of 74.6 hours and 100.6 hours; p<0.05) versus placebo. Furthermore, results for the overall patient population of the study showed numerically shorter time to improvement of influenza symptoms of Baloxavir Marboxil versus oseltamivir with a median time to improvement of symptoms of 73.2 hours for Baloxavir Marboxil compared with 81.0 hours for oseltamivir (p=0.8347).||Baloxavir Marboxil showed efficacy in comparison to placebo and oseltamivir for key secondary endpoints, including reducing the time that the virus continued to be released from the body (viral shedding; median time of 48.0 hours for Baloxavir Marboxil versus 96.0 hours for both placebo and oseltamivir; p<0.0001). Baloxavir Marboxil also lowered the use of antibiotics and incidence of influenza-related complications (3.4 percent and 2.8 percent respectively) compared to placebo (7.5 percent and 10.4 percent; p=0.01 and p<0.05). Baloxavir Marboxil had a numerically lower overall incidence of reported adverse events (25.1 percent) compared with placebo (29.7 percent) or oseltamivir (28.0 percent).|
|Zealand Pharma A/S||Phase III||Glepaglutide||Short Bowel Syndrome (SBS)||This Phase III global study which enrolled it first patient on the 4th October, 2018, seeks to demonstrate efficacy and safety of once- and twice-weekly subcutaneous injections of 10 mg Glepaglutide in SBS patients on parenteral support. 129 subjects will be enrolled at roughly 40 highly dedicated investigational sites across the United States, Canada and Europe. The study will be placebo-controlled, randomized, parallel-group, double-blind, and with fixed dose injection in design. The primary objective is to confirm the efficacy of glepaglutide in reducing parenteral support volume in SBS patients.||The secondary objectives of the study will be to evaluate additional efficacy endpoints, as well as safety and tolerability. With recent FDA orphan drug designation being granted for Glepaglutide for SBS treatment, this is a long-acting GLP-2 analog therapy with an effective half-life of approximately 50 hours. The preceding Phase II Glepaglutide study in SBS patients showed increases in intestinal absorption following only 3 weeks of treatment, thereby providing the basis for initiating this global Phase III study.|
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