CycloSam® Broader Indications

Initial research has identified multiple initial potential indications for CycloSam®. Read about them below.

Metastatic Bone Cancers

Metastatic bone cancers originate from other parts of the body but have metastasized to the bone. They are difficult and oftentimes impossible to treat. The most common metastatic bone cancers originate from cancers of the prostate, breast, and lung. Seven out of every 10 breast and prostate cancer patients will have bone metastases.

CycloSam® may or is proposed to be an effective tool for the treatment of metastatic bone cancers.

  • Single-agent for pain palliation (similar to Xofigo® population)

  • In combination with immunotherapy for potential systemic effect​

  • Sm-153 increases tumor antigenicity in animal models, suggesting potential synergy with immunotherapy

Osteosarcoma/Ewing's Sarcoma:
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

Bone Marrow Ablation

Certain cancers, immune system diseases, blood diseases, and sickle cell disease require bone marrow ablation/transplantation (BMA/T). The standard protocol for BMA/T consists of high dose chemotherapy and/or Total Body Irradiation (TBI). This standard treatment is physically devastating to patients, especially younger and older patients.

Radiopharmaceuticals are potentially the best option because there may be fewer patient side effects. Many radiopharmaceuticals have excellent safety records. However, current products have long half-lives and high levels of impurities, meaning patients could be immunocompromised for unacceptably long periods of time.

CycloSam® may be able to become the new standard of care. The short half-life (46 hours) and low impurity profile means a patient may be able to be treated and transplanted a few days later.

Bone Marrow Ablation

High Dose Chemotherapy Agents

    • Multiple doses over multiple days
    • Disrupts the short-term cell production cycle resulting in mucositis
    • Damage to soft tissue
    • Can take days to be eliminated while side effects can last months
    • Cytotoxic
    • Multiple contraindications

CycloSam® Sm-153 DOTMP*

✓ One day dosage

✓ Not shown to disrupt short-term cell production

✓ Not shown to damage soft tissue

✓ CycloSam® radiation is directed to the bone and away from soft tissue

✓ Most of the non-bone-bound dose can be eliminated from the body right away

✓Not cytotoxic

✓May be used when chemotherapy is contraindicated

*based on animal and in vitro data to date