QSAM Biosciences Receives Rare Pediatric Disease Designation From FDA for CycloSam in the Treatment of Osteosarcoma

QSAM Biosciences Inc. recently announced the US FDA has granted Rare Pediatric Disease Designation to QSAM’s clinical-stage drug candidate, CycloSam, for the treatment osteosarcoma, a devastating form of bone cancer that afflicts mostly children and young adults.

Douglas Baum, QSAM’s CEO, stated “Combined with the orphan designation for osteosarcoma that we received last year from the FDA, the Rare Pediatric Disease Designation may allow QSAM to potentially bring CycloSam to market more rapidly through additional incentives and eligibilities that ultimately help these young patients for whom there is currently little hope. Patients with this disease are eligible to participate in our current Phase 1 clinical trial, however, we anticipate that we will initiate a separate clinical trial in the coming year specifically focused on primary bone cancers such as osteosarcoma and Ewing’s Sarcoma. We are dedicated as a company to making a difference in the lives of children and their families battling these forms of bone cancer.”

Rare Pediatric Disease Designation (RPDD), defined as diseases primarily affecting fewer than 200,000 Americans under the age of 18 each year, may provide substantial financial incentives by making companies eligible for a Priority Review Voucher (PRV) upon drug approval by the FDA. A PRV grants accelerated FDA review of a drug candidate for any indication, reducing the review period to 6 months and potentially gaining early market access. PRVs may be used by the recipient company for any drug development program, or alternatively, sold or transferred to larger pharmaceutical companies.

Osteosarcoma is the most common form of bone cancer in children and young adults with primary high-grade bone malignancy. There have been few advancements over the last 40 years for this debilitating and often deadly disease, with treatment often resulting in limb amputation. Accordingly, there is a large unmet market need for a better treatment that is more efficacious against pediatric osteosarcoma and better tolerated by patients.

In August 2021, the company received orphan designation for CycloSam for the indication of osteosarcoma. Orphan Drug Designation (ODD) affords QSAM certain additional benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees, and seven years of market exclusivity, if approved.

QSAM Biosciences, Inc. is developing next-generation nuclear medicines for the treatment of cancer and related diseases. QSAM’s initial technology, CycloSam (Samarium-153 DOTMP), is a clinical-stage bone targeting radiopharmaceutical​ developed by IsoTherapeutics Group LLC, pioneers in the nuclear medicine space who also developed the FDA-approved Quadramet (Samarium-153 EDTMP) radiopharmaceutical product, which is indicated for pain palliation. QSAM is led by an experienced executive team and Board of Directors that have completed dozens of FDA approvals and multiple successful biotech exits.

CycloSam has demonstrated preliminary safety and efficacy in animal studies and a single patient FDA-cleared human trial performed in 2020 at the Cleveland Clinic. This nuclear technology uses low specific activity Samarium-153 (resulting in far less europium impurity) and DOTMP, a chelator which is believed to eliminate off-target migration and targets sites of high bone turn over making it, in management’s opinion, an ideal agent to treat primary and secondary bone cancers. Since CycloSam delivers targeted radiation selectively to the skeletal system, it is also believed to be an effective agent to perform bone marrow ablation as pre-conditioning for bone marrow transplantation, and in procedures to reduce external beam radiation to bone tumors. This multi-patented drug candidate utilizes an FDA approved radioisotope combined with a novel chelant that has demonstrated preliminary increased efficacy and decreased side effects in animal models and veterinary treatment of bone cancer in dogs. Further, CycloSam utilizes a streamlined, just-in-time manufacturing process that is already in place. Given these factors, management believes there is a strong pathway to commercialization for CycloSam.