A new type of treatment for endometriosis, a chronic and often debilitating condition affecting millions, has been cleared by US regulators to begin human testing. The FDA's approval of the Investigational New Drug application for ENDO-205 allows developer EndoCyclic Therapeutics to initiate a first-in-human clinical study, marking a significant step for a field where new therapeutic approaches are urgently needed.
The drug candidate, ENDO-205, represents a first-in-class, non-hormonal targeted peptide therapeutic. It is designed to directly eliminate the lesions caused by endometriosis—where tissue similar to the uterine lining grows outside the uterus—without impacting healthy tissue. This targeted mechanism aims to avoid the hormonal manipulation, surgical intervention, and systemic toxicity associated with current management strategies. The condition itself is a leading cause of female infertility and affects an estimated 10% of women and girls of reproductive age globally.
"This milestone marks an important step forward for the more than 190 million girls and women worldwide affected by endometriosis, which still remains one of the most overlooked diseases in medicine," said Tanya Petrossian, Founder and CEO of EndoCyclic Therapeutics. The company plans to initiate a Phase 1 clinical study evaluating the treatment in healthy pre-menopausal women. The programme is built on over a decade of research into the root cause of the disease and has been recognized as an NIH SBIR Success Story, supported by multiple awards from the National Institutes of Health.
The upcoming Phase 1 trial will focus on assessing the safety and tolerability of ENDO-205 in healthy volunteers. If successful, it will pave the way for further studies in patients living with endometriosis, offering a hopeful outlook for a novel therapeutic that addresses the underlying biology of the condition without disrupting the body's hormonal balance.