A Phase 1 clinical trial of a potential treatment for endometriosis, known as FOR-6219, is soon to begin in the U.K., after that country’s Medicines and Healthcare products Regulatory Agency (MHRA) granted permission for the study, Forendo Pharma announced.
The trial will be a randomized, double-blind, placebo-controlled study in healthy postmenopausal women 45 to 65 years of age. It will evaluate the safety, tolerability, and pharmacokinetics (movement of a drug within the body) of single and multiple oral doses of FOR-6219.
Results are expected before the end of this year.
Estrogens are essential hormones for female sexual and reproductive development. There are several types of estrogens, such as estrone and estradiol.
High estrogen levels are found in the endometriotic tissue of endometriosis patients. The HSD17B1 enzyme is part of the mechanism that regulates estrogen action, by enabling the conversion of non-active estrone into active estradiol within endometrial cells. When this pathway is blocked, the buildup of estradiol is prevented, limiting the formation of endometriotic lesions.
FOR-6219 is designed to specifically inhibit the HSD17B1 enzyme in endometriotic tissue, so as not to impact hormone levels elsewhere. Studies in a primate model of endometriosis supported the potential effectiveness of this new treatment approach.
“Commencing the first clinical study of FOR-6219 marks an important milestone for Forendo Pharma,” Risto Lammintausta, the company’s CEO, said in a press release. “FOR-6219 has the potential to address significant unmet needs in women’s health, and potentially offers multiple benefits over the current endometriosis therapies including long term use, as well as the opportunity for earlier intervention to this chronic disease.”
“Forendo Pharma has world class experience in steroid hormone biology and the emerging field of intracrinology. The team has developed a novel, first in class therapy for a new target that holds the potential to be a game changer in the treatment of endometriosis,” said Stephan Christgau, chairman of Forendo Pharma and a senior partner at Novo Seeds. “FOR-6219 is a unique molecule that may provide an efficacious and safe treatment for women suffering from the severe debilitating disorder of endometriosis.”