Uterine fibroids are benign tumors that form in the reproductive tract and contain large amounts of collagen which cause pelvic discomfort and pain, decreased fertility, pregnancy complications, miscarriage, heavy bleeding and frequent urination. Uterine fibroids are the primary indication for hysterectomy in the U.S., accounting for approximately 200,000 hysterectomies and 30,000 myomectomies each year. There are up to a $9.4 billion in annual direct costs (surgery, hospital admissions, outpatient visits, medications).

BioSpecifics supported preclinical research with highly purified collagenase as an early step in the potential treatment for uterine fibroids. Studies at Duke Medicine indicate that the use of highly purified collagenase can reduce the stiffness of human uterine fibroid tissue in laboratory experiments. Increased tissue rigidity has been implicated as a cause of the morbidity associated with uterine fibroids. Laboratory research shows that treatment of fibroids with determined doses of purified collagenase causes a statistically significant decrease in the stiffness or the tissue and suggests that XIAFLEX couldĀ potentially be a minimally invasive treatment for this condition.

In October 2014, promising preclinical data from this collaborative study with Duke Medicine were presented at the Mechanotransduction in the Reproductive Tract conference. The data demonstrated that XIAFLEX can reduce the rigidity of human uterine fibroid tissue, potentially shrink uterine fibroid tumors and lend further support to continued research on the potential treatment of XIAFLEX in uterine fibroids.

In May 2016, BioSpecifics announced the publication of data highlighting the efficacy of collagenase clostridium histolyticum (CCH) for the treatment of uterine fibroids in the May 2016 issue of the American Journal of Obstetrics & Gynecology in an article titled, "Loss of Stiffness in Collagen-Rich Uterine Fibroids after Digestion with Purified Collagenase Clostridium Histolyticum." The data show that highly purified CCH can reduce the stiffness of human uterine fibroids ex-vivo and potentially decrease their size.

In March 2017, the potential use of XIAFLEX for the treatment of uterine fibroids was highlighted at the Uterine Fibroids 2017 Conference: A Case for Women's Health Conference, in Durham, NC. The investigator, Dr. James Segars, Director of Reproductive Science and Women's Health Research at Johns Hopkins School of Medicine, presented data in a presentation titled, "An Injectable Drug to Treat Fibroids: A Phase 1 clinical trial," demonstrating accuracy of the injection technique in three patients.

BioSpecifics has developed a clinical protocol with investigators from John Hopkins for a pilot safety and tolerability study in this indication and a Phase 1 clinical trial was initiated in the second quarter of 2017. The Phase 1 open-label dose escalation study in fifteen female subjects treated prior to hysterectomy is taking place at the Department of Gynecology & Obstetrics at Johns Hopkins University. Three subjects have been injected with saline to evaluate the accuracy of the injection method prior to the first patient being dosed with XIAFLEX, 12 subjects will receive XIAFLEX. The primary endpoint will assess the safety and tolerability of CCH following a one-time injection directly into uterine leiomyomas at three doses under transvaginal ultrasound guidance. The secondary endpoints will assess symptoms of pain and bleeding, quality of life throughout the study, shrinkage of CCH treated fibroids in size, increased rates of apoptosis in treated fibroids and the collagen content of treated fibroids.