QSAM Biosciences Receives Rare Pediatric Disease Designation from FDA for CycloSam in the Treatment of Osteosarcoma
QSAM Biosciences has received Rare Pediatric Disease Designation from the FDA for its clinical-stage drug, CycloSam, aimed at treating osteosarcoma, a severe bone cancer primarily affecting children and young adults. This designation may expedite market access, providing potential incentives, including a Priority Review Voucher upon FDA approval. QSAM also holds Orphan Drug Designation for CycloSam, which offers additional benefits, such as tax credits and seven years of market exclusivity if approved. The company plans a clinical trial focused on primary bone cancers in the upcoming year.
- FDA granted Rare Pediatric Disease Designation for CycloSam.
- Potential to expedite market access with Priority Review Voucher.
- Received Orphan Drug Designation for osteosarcoma, offering tax credits and market exclusivity.
- Osteosarcoma has seen few advancements in treatment over the last 40 years, indicating a challenging market.
Austin, TX, Feb. 02, 2022 (GLOBE NEWSWIRE) -- QSAM Biosciences Inc. (OTCQB: QSAM), a company developing next-generation therapeutic radiopharmaceuticals, including Samarium-153-DOTMP (CycloSam®), for the treatment of cancer and related diseases, today announces that the United States Food and Drug Administration (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.
About QSAM Biosciences
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®.
Legal Notice Regarding Forward-Looking Statements: This news release contains "Forward-looking Statements". These statements relate to future events or our future financial performance. These statements are only predictions and may differ materially from actual future results or events. We disclaim any intention or obligation to revise any forward-looking statements whether as a result of new information, future developments or otherwise. There are important risk factors that could cause actual results to differ from those contained in forward-looking statements, including, but not limited to our ability to fully commercialize our technology, risks associated with changes in general economic and business conditions, regulatory risks, early stage versus late stage product safety and efficacy, actions of our competitors, the extent to which we are able to develop new products and markets, the time and expense involved in such development activities, the ability to secure additional financing, the ability to consummate acquisitions and ultimately integrate them, the level of demand and market acceptance of our products, and changes in our business strategies. This is not an offering of securities and securities may not be offered or sold absent registration or an applicable exemption from the registration requirements.
Contact
Investors:
Jason Nelson
CORE IR
ir@qsambio.com
516-222-2560
Media:
Jules Abraham
CORE IR
917-885-7378
julesa@coreir.com
FAQ
What is the significance of the Rare Pediatric Disease Designation for QSAM?
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