MyoKardia, Inc. announced positive results from the Phase II PIONEER-HCM clinical study of the investigational agent mavacamten in symptomatic, obstructive hypertrophic cardiomyopathy (oHCM) patients, including results from a low-dose patient cohort (“Cohort B”), which studied once-daily 2mg and 5mg oral doses of mavacamten. PIONEER-HCM is a Phase II open-label study to assess the efficacy, safety, pharmacokinetics, pharmacodynamics, and tolerability of mavacamten in patients with symptomatic oHCM. PIONEER-HCM consists of two dosing cohorts: in the first cohort, subjects received a once-daily 10mg, 15mg or 20mg dose of mavacamten and were required to discontinue background therapy including beta blockers prior to study entry; and Cohort B, in which subjects received a once-daily 2mg or 5mg oral dose of mavacamten and nine out ten patients remained on beta blocker therapy. Baseline patient characteristics were similar across both patient cohorts.
Poxel SA announced today the initiation of the TIMES 2 and TIMES 3 trials for the Phase III program for Imeglimin, an investigational therapeutic agent for type 2 diabetes, in Japan. Referred to as TIMES (Trials of IMeglimin for Efficacy and Safety), the Imeglimin Phase III program in Japan includes three pivotal trials to evaluate Imeglimin’s efficacy and safety in approximately 1,100 patients. TIMES 2: A Phase III, 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 study, Imeglimin will be administrated orally as a monotherapy or combination therapy with existing hypoglycemic agents, including a DPP4 inhibitor, SGLT2 inhibitor, biguanide, sulphonylurea and GLP1 receptor agonist. TIMES 3: A Phase III, 16-week, double-blind, placebo-controlled, randomized study with a 36-week open-label extension period to evaluate the efficacy and safety of Imeglimin in combination with insulin in Japanese patients with type 2 diabetes and inadequate glycemic control on insulin therapy.
AbbVie and Neurocrine Biosciences, Inc. announced that its Phase III ELARIS UF-II trial of elagolix for uterine fibroids met its primary endpoint. Results from the second of two pivotal Phase III studies demonstrated at month six that elagolix (300 mg twice daily), in combination with low-dose hormone (add-back) therapy (estradiol 1.0 mg / norethindrone acetate 0.5 mg), reduced heavy menstrual bleeding with 76.2 percent (p<0.001) of women with uterine fibroids achieving clinical response compared to placebo (10.1 percent), as measured by the alkaline hematin method. Hypoestrogenic effects, such as hot flush and reduction in bone mineral density, from elagolix treatment were observed in the study. The overall safety profile for elagolix was consistent with what was observed in Phase II studies and the first Phase III study (ELARIS UF-I) in uterine fibroids.1,5-6 Data from the ELARIS UF-II Phase III study will support regulatory submissions for elagolix. Safety data, including most common adverse events, continue to be collected in this ongoing study.
Abbott announced new late-breaking clinical trial data from the MOMENTUM 3 clinical study. The MOMENTUM 3 Investigation Device Exemption (IDE) study is a prospective, multi-center, randomized, unblinded study evaluating the safety and effectiveness of the HeartMate 3 LVAD. HeartMate 3 LVAD is a small, implantable mechanical circulatory support (MCS) device for advanced heart failure patients who are awaiting transplantation or are not candidates for heart transplantation. Patients receiving HeartMate 3 LVAD had significant improvements compared to the HeartMate II LVAD in functional capacity and quality of life scores at two years compared to baseline. Patients with the HeartMate 3 LVAD had a survival rate of 82.8 percent at two years compared to 76.2 percent for those with the HeartMate II LVAD. Stroke rate was significantly lower (10 percent) for the HeartMate 3 LVAD compared to the HeartMate II LVAD (19 percent). Rates of all other adverse events were similar between the HeartMate 3 LVAD and historical rates seen in the HeartMate II LVAD.