Development of an immunomodulating peptide as a therapy for osteosarcoma in canine and human

Osteosarcoma (OSA) is the most common primary malignancy of bone in children and
adolescents, accounting for approximately 5% of all childhood tumors (www.cancer.gov).
Osteosarcoma occurs primarily in the long bones of the limbs, including most commonly the
femur, tibia, and humerus1. The current standard of care therapy for human patients diagnosed
with OSA includes multi-agent neoadjuvant chemotherapy (doxorubicin + cisplatin +/-
methotrexate) followed by surgical resection of the primary lesion and adjuvant chemotherapy,
curing approximately 60 to 70% of patients 1–3. However, up to 40% of newly diagnosed patients
presenting with localized disease eventually develop metastases to the lung, indicating that
many patients already have micrometastases present in the lung at the time of diagnosis or
develop them during the perioperative period 4. The 5-year survival rate for children with
metastatic OSA is extremely low, hovering around 20%-30%, a rate that has not improved in
over 30 years 2. Clearly, novel therapies are in critical need, specifically molecules capable of
inhibiting the process of OSA metastasis. PharmaIN is developing immunomodulating peptide
with unique anti-tumor and anti-fibrotic properties for the treatment of osteosarcoma and other
cancer types. The peptide’s anti-tumor activity has been demonstrated as a monotherapy and
combination with other immuno/chemotherapies in multiple cancer models, including OSA
metastatic model. Pilot tox data has been obtained and PharmaIN proposes to conduct a clinical
trial in canine OSA patients as a translation tool for human and concurrent product development
for veterinary applications. The additional proposed work is IND enabling for a Phase 1 human
clinical trial and consists of manufacturing drug substance and drug product needed for GLP-tox
and clinical studies, completion of GLP-tox studies and submission of the IND for the OSA
indication.

Grant Number: 5R44CA278570-02
NIH Institute/Center: NIH
Principal Investigator: Joshua Alfaro