BioSpecifics Technologies announced positive topline data from its Phase 1 trial of Collagenase Clostridium Histolyticum (CCH) for the treatment of uterine fibroids. The study met the primary endpoint of safety and tolerability with no observed clinically significant adverse reactions. Pharmacodynamic changes were noted in all secondary endpoints with the exception of apoptosis. Statistically significant reductions in collagen content were observed as compared to control fibroids with a median reduction of 39 percent (p<0.05), as well as statistically significant reductions in collagen distribution as compared to control fibroids with an average reduction in density of collagen bundles of 21 percent.
"These topline data highlight the potential promise of this non-surgical treatment option, to safely and effectively reduce the collagen content of uterine fibroids, which are extremely prevalent tumors of the female reproductive tract," said Thomas L. Wegman, President of BioSpecifics. "We are excited to see the pharmacodynamic changes observed in situ in the treated uterine fibroids and we look forward to presenting the full data and subsequently progressing this program into Phase 2 studies. Uterine fibroids have very few non-surgical treatment options and it is our hope to bring CCH to these patients in order to not only treat their fibroids, but also improve their quality of life."
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"Non-invasive surgical options for uterine fibroid management are extremely limited, but necessary for the thousands of patients who deal with the symptoms of this common condition," said Phyllis Leppert, M.D., Ph.D., Professor Emeritus of Obstetrics and Gynecology, Duke University, senior author of preclinical studies of CCH for treatment of uterine fibroids. "The preliminary safety and efficacy of CCH suggests that increased stiffness in the extracellular matrix of fibroids is a crucial aspect of the disease process. I look forward to seeing the Phase 1 data mature as CCH for the treatment of uterine fibroids demonstrates a promising, non-invasive treatment option for patients with uterine fibroids."
Twelve subjects with uterine fibroids were injected transvaginally under ultrasound guidance with CCH in stepped increased dosages and three subjects with uterine fibroids were injected with saline and methylene blue. Only one fibroid per patient was treated with CCH injection. Treated and control tissue samples were harvested at surgery from 24 hours to 60 days following injection. The effects of the CCH injection on collagen content and distribution of collagen bundles and apoptosis were compared between the control arm and the injected fibroids.
Statistically significant reductions in collagen content as compared to control fibroids were observed with a median reduction of 39 percent (p <0.05). Treated fibroid samples had an average 21 percent reduction in density of collagen bundles compared to control tissues. No increases in apoptosis in treated tissues compared to control tissues were observed. CCH injections were safe and well tolerated in all patients with no clinically significant adverse events related to study drug reported. In patients who reported pain at baseline, the pain decreased within 24 to 48 hours and remained low until the last visit at 60 to 90 days.