Shanghai, China, September 30, 2025 - Shanghai Henlius Biotech, Inc. (2696.HK) announced the investigational new drug (IND) application for its HLX13, a proposed ipilimumab biosimilar independently developed by the company, was approved by the U.S. Food and Drug Administration (FDA) as a first-line treatment for patients with unresectable hepatocellular carcinoma.

Immune checkpoint inhibitors are playing a crucial part in immunotherapy, which has emerged in recent years as a novel approach to combating tumour cells with distinct advantages and enormous promise ^[1]. In addition to PD-1/L1 and LAG-3, CTLA-4 is also an inhibitory immune checkpoint expressed on regulatory T cells and activated T cells and has a higher affinity to B7 molecules compared with CD28, thus can competitively bind to B7 molecules to inhibit the proliferation and activation of T cells ^[2]. Anti-CTLA-4 fully human monoclonal antibody (mAb) can block the inhibitory effects of CTLA-4 on the co-stimulation signal necessary to T cell activation and can increase the number of reactive T-effector cells which mobilize to mount a direct T-cell immune attack against tumour cells. Also, anti-CTLA-4 mAb may selectively deplete T-regulatory cells at the tumour site, leading to an increase in the intratumoral T-effector/T-regulatory cell ratio which drives tumour cell death. Meanwhile, results showed that the combination therapy of anti-CTLA-4 mAb and anti-PD-1 mAb is expected to have a synergistic effect and may bring additional clinical benefit to patients ^[3-4]. The reference product of HLX13, YERVOY^®, the world’s first CTLA-4 inhibitor, has been approved in various countries and regions in combination with nivolumab for the treatment of melanoma and hepatocellular carcinoma, among other indications. 

HLX13 is a biosimilar of ipilimumab independently developed by Henlius in accordance with international biosimilar guidelines, including those of National Medical Products Administration (NMPA), EMA and FDA. Henlius has conducted preliminary comparative analytical assessment and a series of head-to-head preclinical studies to evaluate the similarity of HLX13 and the reference product ipilimumab. The results demonstrated a high similarity or no significant difference between HLX13 and the reference product ipilimumab. In April 2025, Henlius entered into a license agreement with Sandoz, granting Sandoz exclusive commercialization rights for HLX13 in the United States, 42 European countries and regions, Japan, Canada, and Australia.

Henlius has established an integrated global platform for R&D, regulatory registration, and clinical operations, backed by a manufacturing and quality management system that meets global regulatory standards, successfully driving the approvals of six products in markets beyond China. Henlius is committed to advancing the global development of HLX13 and working with partners to provide more high-quality, affordable treatment options for patients worldwide.

*YERVOY^® is a registered trademark of Bristol-Myers Squibb (US)

【Reference】

[1] Robert C. A decade of immune-checkpoint inhibitors in cancer therapy[J].Nature Communications,2020,11(1). 

[2] Wolchok J D, Saenger Y.The Mechanism of Anti‐CTLA‐4 Activity and the Negative Regulation of T‐Cell Activation[J].The Oncologist,2008,13 Suppl 4(Supplement 4):2-9.

[3] Victor T S, Rech A J, Maity A,et al.Radiation and Dual Checkpoint Blockade Activates Non-Redundant Immune Mechanisms in Cancer[J].Nature, 2015, 520(7547).

[4] Baas P, Scherpereel A, Nowak AK, et al. First-line nivolumab plus ipilimumab in unresectable malignant pleural mesothelioma (CheckMate 743): a multicentre, randomised, open-label, phase 3 trial. Lancet. 2021;397(10272):375-386. doi:10.1016/S0140-6736(20)32714-8