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

NIH RePORTER · NIH · R44 · $995,099 · view on reporter.nih.gov ↗

Abstract

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.

Key facts

NIH application ID
10931704
Project number
5R44CA278570-02
Recipient
PHARMAIN CORPORATION
Principal Investigator
Joshua Alfaro
Activity code
R44
Funding institute
NIH
Fiscal year
2024
Award amount
$995,099
Award type
5
Project period
2023-09-19 → 2026-08-31