Osteosarcoma

Osteosarcoma is the initial proposed indication for CycloSam®. QSAM believes CycloSam® in this population will potentially qualify for:

One or more Expedited Programs (Breakthrough Therapy, Fast Track, Accelerated Approval, or Priority Review)
- “Between 2012-2017, the FDA approved 58 new cancer drugs, 55 (95%) of which were expedited under at least one program”
FDA Priority Review Voucher (PRV) for Rare Pediatric Cancers
- In August 2019, AstraZeneca purchased a PRV for $95 MM (non-dilutive)

Clinical Landscape of Initial Indication

The most common primary bone cancer (US Incidence 800 to 900 cases)
Affects adolescents and adults (typically people aged 10-30 years)
Current treatments include surgery, amputation, radiation therapy, chemotherapy
Localized disease: 70% cure rate with chemotherapy
Metastatic disease has no cure with short life expectancies: long-term survival rates of <25%
For localized disease, prognosis is dependent on response to neoadjuvant therapy (chemotherapy +/- radiation therapy)
Surgery in the form of limb-sparing resection is preferred to amputation to optimize function
Neoadjuvant therapy improves the chances of avoiding amputation
Quadramet® is currently in the guidelines for second-line relapsed/refractory disease

US IND cleared by FDA and clinical study approved to start enrolling patients at Albert Einstein College of Medicine
Up to 7 additional centers are being recruited (e.g. John Hopkins, Cleveland Clinic, MD Anderson Cancer Center)
Most centers estimate they will be approved to enroll patients in early 2021
Study calls for 5 cohorts of 3 patients at an increasing dose levels
Study is “open label” allowing for early efficacy and safety information
Potential exists to allow for current study to rollover into pivotal study once optimal dose is determined