Uterine fibroids are benign tumors in the reproductive tract that contain large amounts of collagen which cause pelvic discomfort and pain, decreased fertility, pregnancy complications, increased rate of miscarriage, uterine bleeding, prolonged menstrual bleeding and frequent urination. Uterine fibroids are the leading cause of hysterectomies in the U.S., accounting for approximately 250,000 hysterectomies and 30,000 myomectomies each year. There are up to $9.4 billion in annual direct costs (surgery, hospital admissions, outpatient visits and medications).

BioSpecifics supported preclinical research with highly purified collagenase as an early step in the potential treatment for uterine fibroids. Studies at Duke Medicine indicated 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 showed that treatment of fibroids with determined doses of purified collagenase causes a statistically significant decrease in the stiffness or the tissue and suggests that CCH couldĀ potentially be a minimally invasive treatment for this condition.

BioSpecifics is conducting a Phase 1 open-label dose escalation study in fifteen subjects treated prior to hysterectomy at the Department of Gynecology & Obstetrics at Johns Hopkins University, all patients have been fully enrolled. The primary endpoint assesses the safety and tolerability of CCH following a one-time injection directly into uterine fibroids at three doses under transvaginal ultrasound guidance. The secondary endpoints assess symptoms of pain and bleeding, quality of life throughout the study, size, collagen content and rate of apoptosis in CCH treated fibroids. Additional exploratory endpoints measure the tissue elasticity and assess relative stiffness using SWEI (Shear Wave Elasticity Imaging).

BioSpecifics expects to present top-line data from this trial in the fourth quarter of 2018.

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 CCHcan 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 CCH 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 CCH 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.

In March 2018, positive interim data from the ongoing Phase 1 study of CCH for the treatment of uterine fibroids were presented in a poster on March 9, 2018 at the Society for Reproductive Investigation's 65th Annual Scientific Meeting in San Diego, CA. The data show the safety and effectiveness of the injection method in five patients. Three patients were injected with CCH and underwent a hysterectomy 24-96 hours after the injection. Two patients subsequently were injected with a higher dose of collagenase, and underwent a hysterectomy 63 days after the injection. The collagenase-treated tissue samples showed not only the reduction of collagen content, but also the disruption of the tissue pattern, while in control tissues the collagen remained abundant and compact. The digestion of collagen did not extend beyond the capsule of any fibroid. No adverse event occurred in these patients.