VIVUS announces 'positive' results from Phase 1 clinical trial of VI-0106
VIVUS announced positive preliminary results from a Phase 1 clinical study evaluating the pharmacokinetic profile of its VI-0106 in healthy volunteers. VIVUS is developing VI-0106, a proprietary soft capsule formulation of tacrolimus for the treatment of pulmonary arterial hypertension, a degenerative disease that makes it difficult for the heart to pump blood through the lungs to be oxygenated and may ultimately lead to heart failure. The U.S. Food and Drug Administration approved tacrolimus in 1994 for use in lowering the risk of organ rejection in patients undergoing kidney transplant, and the drug is currently indicated for use in additional organ transplant settings and to treat atopic dermatitis. Tacrolimus has been shown to increase signaling through the bone morphogenetic protein receptor 2 pathway, which is down-regulated in PAH patients. The single-center, UK-based Phase 1 PK study was conducted in two parts. Sixteen healthy volunteers were enrolled in Part 1 and each patient sequentially received single doses of three different tacrolimus prototype regimens administered at least 10 days apart. An interim analysis was conducted following the third dosing period to select a formulation prototype with the most desirable PK profile. Three subsequent periods assessed the selected formulation at different doses, or under different fed/fasted conditions. The second part of the study evaluated steady-state PK parameters in 12 healthy volunteers receiving daily doses of the selected prototype formulation. VIVUS is developing VI-0106 under the FDA's 505(B)(2) regulatory pathway, which will allow the Company to leverage the FDA's finding of safety and effectiveness for tacrolimus as well as a significant body of previously published tacrolimus safety and efficacy data. This is expected to reduce the time and cost to develop VI-0106 as a potential breakthrough therapy for the treatment of PAH. In September 2017, results of a university-run Phase 2a clinical study of tacrolimus in patients with PAH were published in the European Respiratory Journal. Study results demonstrate the safety of tacrolimus in patients with PAH. The currently approved formulation of VI-0106 tacrolimus was also provided for compassionate use in three class 3 or 4 PAH patients. The compassionate use demonstrated dramatically reduced rates of hospitalizations and functional class improvements were observed. VIVUS believes that VI-0106 could become an important new treatment option for PAH patients and intends to request both Fast Track and Breakthrough Therapy Designation from the FDA for VI-0106 in this indication.