Cellectar Biosciences Inc (NASDAQ:CLRB) stock jumped Tuesday after it said the US Food and Drug Administration has granted an exemption to the “import alert” faced by the Centre for Probe Development and Commercialization (CPDC), linked to the use of Cellectar’s lead cancer candidate CLR 131.
The biopharma said the exemption will allow it to begin enrolling patients in its Phase 1 pediatric study for the treatment of relapsed or refractory solid tumors including neuroblastoma, lymphomas and malignant brain tumors.
READ: Cellectar Biosciences shares soar after its cancer drug receives Rare Pediatric Disease Designation
Shares of the Madison, Wisconsin-based company soared more than 12% to $3.07.
Separately, Cellectar is already enrolling patients in its Phase 1 and Phase 2 multiple myeloma and select B-cell lymphoma studies of CLR 131 for its hematology investigational new drug application.
“We are grateful that the FDA has granted this additional exemption for CLR 131, which allows us to immediately initiate our pediatric clinical study in children battling life-threatening cancers,” said Cellectar Biosciences President and CEO, James Caruso. “These patients have a very poor prognosis and low rates of survival as a result of limited effective treatment options. Based on our preclinical and ongoing clinical studies, we are optimistic that CLR 131 has the potential to provide a meaningful treatment option for children suffering from cancers with high unmet medical needs.”
Phase 1 pediatric study of CLR 131
The Phase 1 open-label, dose-escalation study will evaluate the safety and tolerability of a single intravenous administration of CLR 131 in 30 children with cancers, including neuroblastoma, sarcomas, malignant brain tumors and lymphomas, including Hodgkin’s lymphoma.
The secondary objective is to identify the recommended Phase 2 dose of CLR 131 and to determine preliminary antitumor activity or treatment response to CLR 131 in children and young adults.
The company plans to initiate the Phase 1 study in seven pediatric cancer centers within and outside the United States.
In December 2014, the FDA granted orphan drug designation to CLR 131 for the treatment of multiple myeloma.
In September 2018, the biopharma’s cancer CLR 131 treatment also received Rare Pediatric Disease Designation from the FDA as a treatment for osteosarcoma, a rare pediatric cancer that begins in the cells that form bones.
The Rare Pediatric Disease Designation is granted for diseases that affect children from birth to 18 years old and affect fewer than 200,000 persons in the US.
The designation has been granted to CLR 131 in four pediatric cancers, including neuroblastoma, rhabdomyosarcoma and Ewing’s Sarcoma.
An RPDD can lead to a priority review voucher, which cuts down the FDA’s review time in half to six months.
Contact Uttara Choudhury at [email protected]