The phase 2a open-label dose ascending study to assess the safety, tolerability, and efficacy of VMB-100 in female patients with moderate SUI is expected to begin in the first half of 2024.
The FDA has accepted an investigational new drug (IND) application for VMB-100, a potency enhanced mRNA encoding human insulin-like growth factor-1 (IGF-1), for the treatment of patients with stress urinary incontinence (SUI), announced Versameb, the developer of the therapy, in a news release.1
The clearance will initiate the launch of a phase 2a open-label dose ascending study to assess the safety, tolerability, and efficacy of VMB-100 in female patients with moderate SUI. The trial is expected to begin in the first half of 2024.
"The IND clearance by the FDA represents a significant milestone for the VMB-100 development program and for Versameb," said Klaas Zuideveld, Chief Executive Officer of Versameb AG, in the news release.1 "This brings us a step closer to providing an effective therapeutic option to patients suffering from SUI. Our proprietary VERSagile platform is uniquely designed to improve potency of mRNA therapeutics, and we believe VMB-100 can potentially become a game changer for SUI treatment. We look forward to initiating this trial in the first half of 2024 to evaluate the potential clinical benefits of VMB-100 in SUI."
VMB-100 encodes IGF-1 and is intramuscularly locally delivered to provide sustained muscle regeneration following short-term treatment. The treatment utilizes enzymatic doggybone DNA (dbDNA) technology developed by Touchlight, which, according to the company, “can manufacture genes of interest of more than 20kb and accommodate sequences typically unstable as plasmid DNA in E. coli, such as those found in viral vector and mRNA production.”2
The dbDNA platform is being used as a critical starting material for the manufacturing of VMB-100, making it the 3rd product utilizing the platform to enter clinical development.
“Touchlight’s dbDNA vector technology has proven instrumental in supporting the development of our novel RNA-based therapeutics, including VMB-100. We are pleased with the ongoing progress achieved through our manufacturing agreement with Touchlight, and we believe the Company’s dbDNA technology will continue to enhance efficiency in manufacturing and supplying VMB-100, as well other pipeline products. We are excited to launch our Phase 2a study to further evaluate the safety, tolerability, and efficacy of VMB-100 and move closer to bringing the first FDA-approved SUI therapeutic to market,” Zuideveld added in a news release from Touchlight.2
In preclinical studies, VMB-100 was shown to induce the expression of IGF-1 in human muscle cells. The treatment also led to accelerated regeneration of the urinary sphincter muscle and restored urinary sphincter function following 1 dose of the treatment in animal models. The phase 2a study of VMB-100 would be a first-in-human trial of the treatment.
"Stress urinary incontinence significantly impacts the quality of life of millions of women globally and is a major unmet medical need with no approved therapeutics available currently. IGF-1 plays a key role in promoting muscle regeneration and re-establishment of muscle function. We believe VMB-100 has the potential to fill the current therapeutic gap as a first-in-class mRNA therapy that can restore muscle function and thereby effectively treat incontinence,” concluded Roger R. Dmochowski, MD, MMHC, FACS, in the news release.1 Dmochowski is the Chief Medical Officer of Versameb AG as well as a professor of urology, surgery and gynecology and vice chair for faculty affairs and professionalism, and associate surgeon in chief at Vanderbilt University Medical Center in Nashville, Tennessee.
1. Versameb AG announces FDA clearance of IND application for VMB-100 in the treatment of stress urinary incontinence. News release. Versameb. November 16, 2023. Accessed November 22, 2023. https://www.prnewswire.com/news-releases/versameb-ag-announces-fda-clearance-of-ind-application-for-vmb-100-in-the-treatment-of-stress-urinary-incontinence-301990373.html
2. Touchlight’s enzymatic doggybone DNA used in the manufacture of Versameb’s VMB-100 for first-in-human clinical study. News release. Touchlight. November 21, 2023. Accessed November 22, 2023. https://www.businesswire.com/news/home/20231121090119/en/Touchlight%E2%80%99s-enzymatic-doggybone-DNA-used-in-the-manufacture-of-Versameb%E2%80%99s-VMB-100-for-first-in-human-clinical-study