Foresee Pharmaceuticals has received a crucial safety endorsement for its ongoing Phase 3 Casppian trial investigating CAMCEVI (leuprolide FP-001) in pediatric patients with central precocious puberty (CPP). The independent Data and Safety Monitoring Board (DSMB) has recommended continuing the trial without modifications, marking its third positive safety review.
The multicenter, multinational open-label study, which has reached approximately 75% enrollment, is evaluating a 42mg controlled-release formulation of leuprolide administered every six months. This development represents a potential advancement in CPP treatment, where consistent hormone suppression is crucial for managing premature sexual development.
Clinical Development Progress and Safety Profile
"Early efficacy signals and continued safety validation strengthen our confidence in this therapy's potential as a groundbreaking advancement for pediatric CPP patients," stated Bassem Elmankabadi, M.D., Senior Vice President of Clinical Development at Foresee. The study utilizes the company's proprietary Stabilized Injectable Formulation (SIF) technology, which has already demonstrated success in adult applications.
CAMCEVI's current approval for advanced prostate cancer treatment has shown statistically significant treatment effects, providing a foundation for its potential expansion into pediatric indications. The six-month dosing schedule could offer a significant advantage over more frequently administered alternatives.
Understanding CPP and Treatment Implications
CPP affects approximately 1 in 5,000 to 10,000 children, with a notably higher prevalence in girls, showing a female-to-male ratio of around 20:1. The condition triggers premature activation of the hypothalamus-pituitary-gonadal axis, leading to early puberty onset between ages 2 and 9.
The implications of CPP extend beyond physical development. Affected children face risks of shortened adult height and significant psychosocial challenges, including decreased self-esteem, anxiety, and depression. Approximately 80-90% of cases are idiopathic, particularly in females, highlighting the importance of effective treatment options.
Future Outlook and Development Timeline
Dr. Ben Chien, CEO and Chairman of Foresee, emphasized the significance of this milestone: "This positive DSMB recommendation brings us closer to providing safe and effective treatments for children in need." The company anticipates releasing top-line results from the Casppian trial in 2025.
The development program builds on Foresee's expanding portfolio, which includes various formulations of CAMCEVI. The company has submitted a U.S. NDA for a 3-month version with a PDUFA date of August 29, 2025, while simultaneously preparing for European regulatory submission.
