On September 17, 2018 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR 131 for the treatment of osteosarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, SEP 17, 2018, View Source [SID1234530175]). CLR 131 is Cellectar’s lead Phospholipid Drug Conjugate (PDC) product candidate.
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"CLR 131 has demonstrated promise as an anticancer agent in preclinical and clinical settings, and we are working now to establish its impact on various rare and deadly pediatric cancers," said John Friend, M.D., chief medical officer of Cellectar. "Cellectar is pleased to have the opportunity to work closely with the FDA on our planned Phase 1 trial for these indications and we remain committed to advancing the pediatric programs as rapidly as possible."
Since May 2018 the company has received RPDD for CLR 131 in four pediatric cancers: neuroblastoma, rhabdomyosarcoma, Ewing’s Sarcoma and, most recently, osteosarcoma. Should any of these indications reach approval, the RPDD may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future NDA or BLA submission, which would reduce the FDA review time from 12 months to six months. Currently, these vouchers can also be transferred or sold to another entity. Over the last 16 months, five priority review vouchers were sold for between $110 million and $150 million each.
The FDA grants Rare Pediatric Disease Designation for diseases that primarily affect children from birth to 18 years old, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
About Osteosarcoma
Osteosarcoma derives from bone forming mesenchymal, or connective tissue, cells and is the most commonly diagnosed primary bone malignancy among children and adolescents. The incidence is about 4.4 cases per 1 million per year in children younger than 24 years [Mirabello 2009]. While there is a 70% cure rate among patients with localized disease, 5-year overall survival rates are approximately 20% for among patients who develop metastatic disease [Saraf 2018]. Additionally, among patients who experience disease progression or recurrence survival for is less than 30% [Chou 2005].
About CLR 131
CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131 is in a Phase 2 clinical study in R/R MM and a range of B-cell malignancies and a Phase 1b clinical study in patients with R/R MM exploring fractionated dosing. The objective of the multicenter, open-label, Phase 1b dose-escalation study is the characterization of safety and tolerability of CLR 131 in patients with R/R MM. Patients in Cohorts 1-4 received single doses of CLR 131 ranging from 12.5 mCi/m2 to 31.25 mCi/m2. All study doses have been deemed safe and well tolerated by an independent Data Monitoring Committee. The company is currently initiating a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma and is planning a second Phase 1 study in combination with external beam radiation for head and neck cancer.