Indication for CycloSam®


Osteosarcoma: Initial Proposed Indication for CycloSam®

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

Immediate Path to Human Trials

  • 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