Foresee Pharmaceuticals announced the complete enrollment of its pivotal Phase 3 Casppian trial for central precocious puberty (CPP), marking a significant milestone in addressing an unmet pediatric medical need. The Taiwan and US-based biopharmaceutical company is evaluating leuprolide mesylate (FP-001) 42 mg controlled-release formulation in children with this rare endocrine disorder.
Trial Design and Objectives
The Casppian Phase 3 study is an open-label, multicenter clinical trial evaluating the efficacy, safety, and pharmacokinetics of leuprolide (FP-001) 42 mg controlled-release in patients with central (Gonadotropin-Dependent) precocious puberty over 48 weeks. The study's primary endpoint measures the percentage of patients with serum LH concentration < 4 mIU/mL 30 minutes following a GnRH agonist stimulation test at Week 24.
Leuprolide (FP-001) 42 mg will be considered effective for treating children with CPP if ≥80% of patients exhibit LH suppression < 4 mIU/mL at Week 24. This threshold reflects the clinical standard for hormonal suppression in CPP treatment.
Clinical Significance and Disease Impact
Central precocious puberty is a condition causing early sexual development in children when their "hypothalamus - pituitary gland - gonadal axis" activates prematurely, leading to puberty onset between 2 and 9 years of age. According to the National Organization for Rare Disorders (NORD), CPP occurs in 1 out of 5,000 to 10,000 children, with approximately 80-90% of cases being idiopathic, particularly in females, who are affected at a female-to-male ratio of around 20:1.
Children with CPP face significant long-term consequences, including risk of short adult stature and psychological complications such as lower self-esteem, stress, anxiety, and depression, which may negatively impact their quality of life. Gonadotropin-releasing hormone (GnRH) agonists, including leuprolide, represent the most widely used treatment approach for CPP.
Development Timeline and Regulatory Strategy
Topline results from this pivotal trial are expected by the end of 2025. Based on successful study completion by mid-2026, Foresee plans to submit a comprehensive New Drug Application (NDA) package to regulatory authorities by Q3 2026.
"Reaching full enrollment in this pivotal Phase 3 trial is a crucial step toward addressing a significant unmet need in central precocious puberty. We are deeply committed to rigorously evaluating Leuprolide mesylate therapy's potential to make a meaningful difference for affected children with CPP and their families," stated Yisheng Lee, M.D., Chief Medical Officer.
Existing Product Portfolio
Foresee's leuprolide injectable emulsion, 42 mg (marketed under the brand name CAMCEVI®), is currently approved for treating adult patients with advanced prostate cancer and has demonstrated positive efficacy and safety results with statistically significant treatment effects. CAMCEVI 42 mg is approved in the U.S., Canada, EU, Taiwan, Israel, and the UK, with U.S. launch occurring in April 2022.
The company has also submitted a U.S. NDA for the 3-month version of CAMCEVI, which has been accepted for review with a PDUFA date of August 29, 2025. EU regulatory submission for the 3-month version remains under preparation.
"The efficient completion of enrollment in the Casppian Phase 3 study reflects the dedication of our clinical development team, investigators, and participating patients and their families. We are unwavering in our commitment to executing the trial with the highest safety and quality standards to generate robust data for regulatory submission," stated Bassem Elmankabadi, M.D., Senior Vice President of Clinical Development.
Company Pipeline
Beyond CPP, Foresee is developing CAMCEVI 6-month LAI formulation in a Phase 3 clinical trial for premenopausal breast cancer in China through a partnership. The company's broader pipeline includes Aderamastat (FP-025), a highly selective oral MMP-12 inhibitor that showed positive outcomes in a Phase 2 proof-of-concept study in allergic asthmatic patients, and Mirivadelgat (FP-045), an oral ALDH2 activator currently in Phase 2 WINDWARD study for pulmonary hypertension-interstitial lung disease patients.
