Development of an endometrial ablation drug-device combination to treat heavy menstrual bleeding

PROJECT SUMMARY Over 10 million women in the US and 100 million women globally suffer from Heavy Menstrual Bleeding (HMB). Oral medications lack effectiveness and, like hormonal IUDs (intrauterine devices), have significant side effects. Currently available endometrial ablation options, which is another therapeutic alternative, are complex and require advanced anesthesia, which significantly limits their in-office use. Because of the high cost, complexity, and/or clinical limitations of the available options, millions of women receive no treatment or are compelled to undergo a hysterectomy. An estimated 2-3 million hysterectomies are done for HMB worldwide, including ~400,000 just in the US. Gynion will address this unmet need with Menorrx, a drug/device combination that will be a non-invasive, easy-to-use, and cost-effective therapy for HMB. It is intended for use by obstetrician- gynecologists (OB-GYN) practitioners in an office setting with the requirement of only local anesthesia. Menorrx is a proprietary drug formulation that uses Trichloroacetic Acid (TCA) as an active ingredient. The formulation is instilled into the uterine cavity which then chemically ablates an endometrial layer using an innovative delivery system that assures procedural safety and consistent results. The long-term goal of this SBIR project is to market Menorrx globally, making HMB treatment more available and affordable due to its simplicity, safety, high efficacy, and cost-effectiveness. To achieve this goal, Gynion needs to obtain regulatory approvals in the US and internationally. Such approvals require the company to conduct animal and human clinical studies to demonstrate product safety, including toxicological safety, and efficacy. The focus of Aim 1 is to demonstrate that the Yorkshire pig is a viable in vivo animal model for intrauterine instillation of Menorrx. A species-appropriate dose of Menorrx will be developed, and three pigs will be treated in the 96-hour non-GLP study. After the treatment, a histopathology assessment will confirm dose safety. Aim 2 is to assess the low initial systemic toxicity of Menorrx that is administered by intrauterine instillation and determine when the peak of toxicity occurs. In Phase II, Gynion will conduct a 72-hour and 30-day GLP-compliant survival study using this pig model to assess systemic toxicity levels and confirm no long-term systemic toxicity effects of TCA. This will allow Gynion to establish preliminary toxicology safety and initiate a human toxicity study as part of the regulatory approval process. After Gynion obtains New Drug Application (NDA) approval in the US under section 505(b)(2) of the FDandC Act, and a CE Mark, it will commercialize Menorrx in the global market that is projected to reach $1.6B with a small direct US sales force and with international distributors. Once commercial viability and rapid adoption are demonstrated, the company will be well-positioned for acquisition by a major medical device or pharmaceutical company.