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 FD&C 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.
Public Health Relevance Statement: PROJECT NARRATIVE Over 10 million women in the US and 100 million women globally suffer from Heavy Menstrual Bleeding (HMB). Current therapeutic options are complex, expensive, and require an operating room environment, or lack effectiveness and have severe side effects. To address this issue, Gynion is developing Menorrx, a drug/device combination that will be an easy-to-use and cost-effective system to safely and consistently treat HMB in the office setting.
Project Terms: Adoption; Affect; After Care; After-Treatment; post treatment; Aftercare; Anesthesia; Anesthesia procedures; Local anesthesia; Conduction-Blocking Anesthetics; Local Anesthetics; Animals; Blood Transfusion; Liquid Chromatography; Clinical Research; Clinical Study; cost effectiveness; Pharmaceutical Preparations; Drugs; Medication; Pharmaceutic Preparations; drug/agent; Endometrium; Uterine lining; Environment; Extravasation; Leakage; Spillage; Mammalian Oviducts; Fallopian Tubes; Salpinx; Uterine Tubes; oviduct; Goals; hemodynamics; Hemorrhage; Bleeding; blood loss; Hospitalization; Hospital Admission; Human; Modern Man; Hysterectomy; implantable device; biomedical implant; implant device; indwelling device; Intrauterine Devices; Intrauterine Contraceptive Devices; intra-uterine contraception; intra-uterine contraceptive device; intra-uterine device; intra-uterine system; intrauterine contraception; intrauterine system; Marketing; Medical Device; Menstrual cycle; Methods; Methodology; Operating Rooms; Pain; Painful; Legal patent; Patents; Patients; Pilot Projects; pilot study; Plasma; Blood Plasma; Plasma Serum; Reticuloendothelial System, Serum, Plasma; Postoperative Complications; post-operative complications; pressure; Production; Progestins; Corlutina; Corluvite; Cyclogest; Gestagenic Agents; Gestagens; Gestiron; Gestone; Lipo-Lutin; Luteohormone; Lutocyclin; Lutocylin M; Lutogyl; Lutromone; Progestagenic Agents; Progestasert; Progestational Agents; Progestational Compounds; Progestational Hormones; Progesterone Agents; Progestogel; Progestogens; Progestol; Progeston; Prolidon; Proluton; Syngesterone; Therapeutic Progestin; Utrogestan; Quality of life; QOL; Risk; Safety; Sales; Family suidae; Pigs; Suidae; Swine; porcine; suid; Time; Tissues; Body Tissues; Toxicology; Trichloroacetic Acid; trichloro-acetic acid; Uterus; womb; Viscosity; Woman; Measures; Catheters; health care; Healthcare; Caring; Iron deficiency anemia; Organ; Blood Sample; Blood specimen; Procedures; Cervical; Surface; Clinical; Phase; Chemicals; Endometrial; Endometrial Cavity; Uterine cavity; Gynecologist; Therapeutic; Hour; Complex; Oral; System; Hormonal; Drug Formulations; Operative Surgical Procedures; Operative Procedures; Surgical; Surgical Interventions; Surgical Procedure; surgery; interest; Ablation; Services; Animal Model; Animal Models and Related Studies; model of animal; Toxic effect; Toxicities; Histopathology; Modality; Devices; social; Excision; Abscission; Extirpation; Removal; Surgical Removal; resection; Positioning Attribute; Position; Emotional; Modeling; Pharmaceutical Agent; Pharmaceuticals; Pharmacological Substance; pharmaceutical; Pharmacologic Substance; Effectiveness; Address; Dose; Breast Cancer Risk Factor; breast cancer risk; Data; International; Qualifying; in vivo; Perioperative; Small Business Innovation Research Grant; SBIR; Small Business Innovation Research; Process; Development; developmental; cost; mass spectrometer; cost effective; innovate; innovative; innovation; new drug treatments; new drugs; new pharmacological therapeutic; new therapeutics; new therapy; next generation therapeutics; novel drug treatments; novel drugs; novel pharmaco-therapeutic; novel pharmacological therapeutic; novel therapy; novel therapeutics; commercialization; LMIC; low and middle-income countries; Formulation; systemic toxicity; experiment; experimental research; experiments; experimental study; Injections; side effect; optimal drug dosage; optimal drug dose; appropriate dose; clinical trial enrollment; cost effective therapy; cost effective treatment; pig model; piglet model; swine model; porcine model; Diameter
