FDA Grants Orphan Drug Designation to HCB101 for Gastric Cancer

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to HCB101, an investigational immunotherapy, for the treatment of gastric cancer, according to a news release from HanchorBio, Inc. The designation applies broadly to gastric cancer, including advanced gastric adenocarcinoma in both HER2-positive and HER2-negative disease, and highlights the ongoing need for new options for patients facing this diagnosis.

HCB101 is a next-generation therapy designed to help the immune system recognize and destroy cancer cells. Early clinical findings suggest the drug may produce antitumor activity with a safety profile intended to reduce some of the blood-related side effects that limited earlier therapies targeting the same pathway.

"Receiving our first FDA orphan drug designation is a major milestone for HanchorBio and important validation of our scientific, regulatory, and development strategy," said Scott Liu, founder, chairman, and CEO of HanchorBio. "Gastric cancer remains an area of profound unmet medical need, and this designation reinforces our commitment to developing differentiated immunotherapies that can meaningfully improve outcomes for patients. This designation strengthens HCB101's profile as a globally relevant asset and represents a strategically important step as we advance the program toward U.S. and international development. It further supports our ongoing engagement with multinational partners as we explore collaboration and licensing opportunities for HCB101 and our broader immunotherapy pipeline."

Orphan drug designation is granted to treatments for rare diseases and provides development incentives, including potential tax credits, waiver of certain FDA fees and, if approved, seven years of market exclusivity in the United States.

What is HCB101 and how does it work?

HCB101 targets the CD47–SIRPα pathway, an “innate immune checkpoint” that cancer cells use to avoid being eaten by immune cells called macrophages. By blocking this signal, the therapy is designed to restore the immune system’s ability to attack tumors and improve antigen presentation, which may help activate broader immune responses.

Unlike earlier anti-CD47 approaches, HCB101 was engineered to reduce binding to red blood cells. This design aims to limit hematologic toxicity and allow the drug to be combined more easily with standard treatments such as chemotherapy and targeted therapy.

Researchers are currently studying HCB101 in combination with ramucirumab and paclitaxel for patients with advanced gastric cancer in the second-line setting. Early data have shown tumor shrinkage in some patients, although the therapy remains investigational.

Why new treatments are needed for gastric cancer

Gastric cancer remains a serious disease with significant unmet need, particularly for patients whose cancer has progressed after first-line therapy. Although targeted therapies and immune checkpoint inhibitors have improved outcomes for some patients, responses are often not durable and treatment-related toxicities can limit use.

In the United States, gastric cancer is considered a rare disease, which qualifies it for orphan drug designation. Despite its rarity, outcomes in advanced disease remain poor, especially in the second-line setting, where available regimens offer limited long-term benefit.

Researchers continue to explore new strategies that activate both the innate and adaptive immune systems, with the goal of improving response rates and durability without substantially increasing side effects.

How HCB101 is being studied

HCB101 is being evaluated in an ongoing phase 1b/2a clinical trial that is testing the drug in combination with ramucirumab and paclitaxel for patients with previously treated advanced gastric cancer. The study is assessing safety, dosing, tumor response and other clinical outcomes.

The therapy was developed using AI-assisted structural modeling to selectively bind CD47 on cancer cells while minimizing interaction with red blood cells. Investigators are also examining how well the drug engages its target and whether it can be integrated into standard treatment schedules.

At this stage, the available data are early and primarily focused on safety and signs of antitumor activity. Larger studies will be needed to determine whether the therapy improves survival or other long-term outcomes.

Because HCB101 is still in clinical testing, it is not yet available outside of a trial. The current study is enrolling patients with advanced gastric cancer who have received prior therapy, including those with HER2-positive and HER2-negative disease. Eligibility for participation depends on multiple factors, including prior treatments, overall health and specific study criteria. Patients interested in clinical trials should speak with their oncology team to determine whether a study may be appropriate.

Additional findings and next steps

Orphan drug designation does not mean the therapy is approved, but it can help accelerate development by providing regulatory and financial support. The designation also reflects the FDA’s recognition of the need for new treatment approaches in gastric cancer.

Investigators plan to continue global clinical development of HCB101 and explore its use as a potential backbone immunotherapy across multiple solid tumors. Researchers are particularly interested in combination strategies that may enhance immune responses without increasing toxicity.

For patients, the development of therapies targeting the innate immune system represents a different approach from currently approved PD-1/PD-L1 inhibitors. Although more research is needed, early findings suggest this strategy may offer another pathway to improving outcomes in difficult-to-treat gastric cancer.

As clinical trials continue, patients and clinicians will be watching closely to see whether HCB101 can translate early antitumor activity into meaningful long-term benefit.

Reference

1. “HanchorBio Receives FDA Orphan Drug Designation for HCB101 in Gastric Cancer.” News Release. HanchorBio.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI and reviewed by a human editor.

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