Iltamiocel cell therapy in women with recurrent or persistent stress urinary incontinence: Evidence from two double-blind, randomized, placebo-controlled trials

Abstract

Rationale:

Recurrent or persistent female stress urinary incontinence (SUI) following prior surgery represents an unmet medical need and a significant clinical challenge for physicians.

Objectives:

To report the integrated clinical experience from placebo-controlled studies of iltamiocel, an investigational autologous muscle cell therapy, in women with a history of prior SUI surgery.

Findings:

Results from two multicenter, randomized, placebo-controlled studies of iltamiocel were pooled. Efficacy assessments were based on change from baseline to 12 months in number of SUI episodes, quality of life, and symptom severity. A total of 92 female participants, 61 iltamiocel and 31 placebo, were enrolled. At 12 months after injection, a significantly greater proportion of iltamiocel-treated participants achieved $\ge$75% reduction in SUI episodes compared with placebo (44.3% vs 16.1%, p=0.007). This treatment response was maintained out to 24 months in 84.2% of iltamiocel participants. In addition, significant differences were observed at 12 months between iltamiocel and placebo responders for the Incontinence Impact Questionnaire-7 (-31.7 vs -15.2; p=0.038) and the Severity Index for Urinary Incontinence in Women (-2.8 vs -0.3; p=0.011). No serious adverse reactions related to iltamiocel were reported. Adverse events related to the treatment procedures were reported in 6 (9.8%) iltamiocel participants and 4 (16.1%) placebo participants.

Conclusions:

Iltamiocel demonstrates significant and sustained efficacy in reducing SUI episodes and improving quality of life in women with recurrent or persistent SUI following prior surgical interventions. It offers a safe, effective and minimally invasive treatment option, addressing the need for robust management strategies in this challenging patient population.