Recently, HC016 Lipid Complex Injection (hereinafter referred to as “HC016”), an innovative tumor immunotherapy product independently developed by Zhejiang Haichang Biotech Co., Ltd., has achieved two significant milestones: firstly,The product was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) on December 31, 2025, for the treatment of osteosarcoma.Second,The Phase I clinical study of this product successfully enrolled the first subject on January 5, 2026, and completed the first dosing the following day.

Introduction to HC016

HC016 is a Toll-like receptor 9 (TLR9) agonist independently developed by Haichang Biotech. The product innovatively utilizes QTsome.^TMLipid Nanoparticle (LNP) technology encapsulates novel C-type cytidine-phosphate-guanosine oligodeoxynucleotide (CpG-ODN) products and directly delivers them to the tumor site via intratumoral injection. This delivery technology not only activates TLR9 and downstream signaling pathways, generating specific immune responses against tumors, but also significantly reduces systemic drug exposure, enhancing treatment safety, with the potential for treating a broad spectrum of malignant tumors.

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HC016 Osteosarcoma Indication Granted FDA Orphan Drug Designation in the U.S.

Osteosarcoma (OS) is the most common primary malignant bone tumor in children and adolescents, characterized by malignant cells producing osteoid or immature bone tissue.^[1]The United States sees approximately 1,000 new confirmed cases of OS each year.^[2], officially recognized as a rare disease by the National Organization for Rare Disorders (NORD)^[3]For patients with metastasis, the efficacy of existing therapies is limited, the prognosis is poor, and the five-year survival rate is only 20%-30%.^[4], there is an urgent unmet clinical need.

On December 31, 2025, HC016 successfully obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the indication of osteosarcoma. Due to the comprehensive submission materials and robust data of HC016, the FDA granted approval without issuing any Information Requests (IR) during the review period, demonstrating Haichang Biotech's rigorous scientific approach, exceptional R&D efficiency, and high-standard global registration capabilities in the field of rare disease drug development. Additionally, obtaining orphan drug designation marks a key milestone in the development process of HC016, providing significant policy support for the subsequent clinical development of the drug and accelerating its progress to benefit patients worldwide.

First Subject Enrolled in Phase I Clinical Study of HC016

On January 5, 2026, the Phase I clinical study (CTR20254445) of HC016 completed the enrollment of the first subject, and the first dosing was successfully completed on January 6. The Phase I clinical study is divided into a dose escalation phase and a dose expansion phase, aiming to evaluate the safety, tolerability, pharmacokinetic characteristics, pharmacodynamic characteristics, and preliminary efficacy of HC016 in patients with advanced or recurrent solid tumors (including melanoma, squamous cell carcinoma of the head and neck, and sarcoma).

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