Development strategy and status

This study was completed and preliminary data was released in June 2011. Data from this study was presented at the European Society for Organ Transplantation in September 2011 in Glasgow and at the American Society of Nephrology Renal Week in November 2011 in Philadelphia.

This Phase lll study successfully demonstrated non-inferiority in predefined endpoints compared to Prograf. The Phase lll open label conversion (switch) study in 326 stable kidney transplant recipients, with Prograf as the comparator, met all its primary efficacy and safety endpoints. The study also showed a trend towards superior rejection rates based on central laboratory pathology assessment with rates of 0.6% for LCP-Tacro and 3.1% for Prograf (p=0.214).

This clinical Phase III study in de novo kidney transplant patients was initiated in October 2010. Patient enrolment was completed in the first quarter of 2012, with 543 patients enrolled. Data from the completed study is expected to be available mid-2013.

Study 3002 is a randomised, double-blind, multicentre study that compares once-daily LCP-Tacro against twice-daily Prograf in de novo adult kidney transplant patients. The primary endpoint of the study, a composite endpoint (BPAR, graft failure, loss to follow up or death), will be evaluated after a 12-month treatment period to demonstrate the non-inferiority of LCP-Tacro compared to Prograf. Secondary endpoints will include safety, tolerability and renal function assessments. The study is being conducted at approximately 90 transplant centres, primarily in the United States and Europe. Patients will participate in a 12-month extension period on treatment for follow-up safety assessments. This study is being conducted under an SPA, such that the FDA has assessed the protocol for the study and has agreed that it will not later alter its perspective on the issues of the agreed design, execution, or analyses proposed in the protocol(s) unless public health concerns unrecognised at the time of protocol assessment under this process are evident.

Based on the favourable results of study 3001 and the totality of an extensive Phase I, II and III clinical safety, efficacy and pharmacokinetics programme, the Company intends to file an MAA in the European Union in 2013 and is currently evaluating whether to wait for the results of the second Phase III study (in de novo patients) before making this submission. Filing in the United States is targeted for the second half of 2013, following completion of the ongoing study 3002.

Veloxis has initiated one Phase IIIb/IV study and plans to initiate several additional studies to further examine the potential clinical differences between LCP-Tacro and existing therapies, including Prograf.

This is a phase IIIb study of LCP-Tacro in kidney transplant patients experiencing tremors on standard tacrolimus formulations. It is designed to explore whether converting patients who have symptomatic tremor from treatment with standard twice-daily tacrolimus capsules (such as Prograf) to sustained release once-daily LCP-Tacro tablet, leads to a measurable improvement in tremor. This study was initiated in December 2011 and a preliminary analysis of the results from the first 16 patients demonstrated a trend toward a reduction in tremor following the switch from twice-daily tacrolimus to once-daily LCP-Tacro. The study continues to enroll and final data is expected in first half of 2013.