Cell Therapy for Critical Limb Ischemia Shows Positive Interim Data
An interim analysis has met prespecified safety and efficacy checkpoints.
Positive interim data from the phase 3 SALAMANDER trial (NCT03174522) of REX-001 has demonstrated sufficient safety and efficacy in patients with critical limb-threatening ischemia (CTLI) for the trial to continue, Ixaka announced.
While the unblinded data has not yet been released, the trial’s independent Data Monitoring Committee (DMC) has assessed the interim data from the first 30% of enrolled patients 12 months after treatment and has stated that the trial has met prespecified safety and efficacy checkpoints.
“We are delighted with the positive outcome in this interim analysis of the Phase 3 trial for REX-001 in CLTI. With such poor treatment options available and the high number of patients affected, this is a significant milestone in this devastating disease. The positive interim analysis confirms the potential of REX-001 as an effective treatment of the disease”, Joe Dupere, chief executive officer, Ixaka, said in a statement.
REX-001 is an investigative, autologous, multi-cell therapy designed to treat CLTI in patients with diabetes. It is designed to address the underlying processes such as plaque deposition, inflammation, ischemia, vessel degeneration, and ulcer formation that lead to clinical progression in CLTI. Ixaka, formerly Rexgenero, believes that the therapy could meet a high unmet need in this therapeutic space as REX-001 is currently the furthest advanced product in this indication.
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“This first Phase 3 interim analysis is a critical milestone for our lead cell therapy candidate REX-001. The positive benefit-risk evaluation by the DMC provides reassurance that REX-001 is an effective and safe treatment for CLTI, and that it is on the right path towards marketing authorization,” Gilbert Wagener, MD, senior vice president and chief medical officer, Ixaka, added to the statement.
The DMC stated that the interim data from the randomized, double-blind, placebo-controlled, adaptive SALAMANDER trial shows efficacy in line with previously clinical trials. The preceding phase 2 clinical trial (NCT03111238) demonstrated that REX-001 treatment resulted in complete ulcer healing in 75% of patients compared to 14% of patients in the control group. They observed no significant safety concerns.
“Based on these results, I am confident that we can provide our innovative and much-needed product to patients suffering from CLTI in the near future,” Wagener added.
SALAMANDER is enrolling patients with diabetes and Rutherford category 5 CLTI. The trial’s primary endpoint is complete ulcer healing or change in Rutherford classification from Category 5 to Category 4 or lower 12 months after treatment. It is recruiting in sites across Europe, including the UK, Spain, Austria, Portugal, Poland, Hungary, the Netherlands, the Czech Republic, Latvia, Lithuania and Georgia.
The interim analysis is 1 of 2 planned, the second of which will occur when 50% of patients enrolled have reached the 12-month follow-up visit. Over 50% of patients have been treated so far. No unblinded data will be released until the trial’s completion, which is set for the end of April 2022.
