Cellectar Biosciences Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration for Iopofosine I 131 in Relapsed or Refractory Pediatric High-Grade Glioma

Core Insights - Cellectar Biosciences announced that the FDA granted Rare Pediatric Drug Designation for iopofosine I 131, a treatment for inoperable relapsed or refractory pediatric high-grade glioma, highlighting its potential to address a critical need in pediatric oncology [1][3][15] - Interim results from the CLOVER-2 Phase 1b trial showed significant improvements in progression-free survival (PFS) and overall survival (OS) for patients treated with iopofosine I 131, indicating its promise as a novel therapeutic option [5][6][12] Company Overview - Cellectar Biosciences is a late-stage clinical biopharmaceutical company focused on developing drugs for cancer treatment, leveraging its proprietary Phospholipid Drug Conjugate™ delivery platform [13][14] - The company aims to provide targeted cancer therapies that improve efficacy and safety, with iopofosine I 131 being a key asset in its pipeline [14] Clinical Study Insights - The CLOVER-2 trial involves children, adolescents, and young adults with relapsed or refractory pediatric high-grade gliomas, assessing the safety and tolerability of iopofosine I 131 [12] - Interim data revealed that patients receiving a minimum of 55 mCi total administered dose experienced an average PFS of 5.4 months and OS of 8.6 months, with some patients achieving even longer survival rates [6][9] Safety Profile - Iopofosine I 131 demonstrated a favorable safety profile, with no significant cardiovascular, renal, or liver toxicities reported, and manageable hematologic adverse events [9][10] - The treatment's selective targeting resulted in clinically negligible off-target effects, reinforcing its potential as a safe therapeutic option for pediatric patients [9] Case Studies - Two case studies presented in the trial showed promising results: a 25-year-old male with a target lesion reduction of over 50% and a PFS of 10.9 months, and a 15-year-old female with a target lesion reduction and a PFS of 11.2 months [7][8]