Innovent Gains NMPA Breakthrough Therapy Status for IBI363 in Melanoma

SAN FRANCISCO and SUZHOU, China, March 30, 2025 -- Innovent Biologics, Inc., a leading biopharmaceutical enterprise, has announced a significant achievement in the development of its novel drug IBI363. The Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) has awarded Breakthrough Therapy Designation (BTD) to IBI363, a pioneering PD-1/IL-2α-bias bispecific antibody fusion protein. This designation is for use as a monotherapy in treating unresectable, locally advanced or metastatic mucosal or acral melanoma patients who have not undergone prior systemic therapy.

Innovent has commenced its initial pivotal study for IBI363, with the first patient already dosed. This study aims to assess the efficacy and safety of IBI363 monotherapy compared to pembrolizumab (Keytruda®) in patients with advanced melanoma who have not received systemic therapy before. Additionally, IBI363 has been granted two fast track designations (FTD) by the U.S. Food and Drug Administration (FDA) for treating squamous non-small cell lung cancer and melanoma.

In earlier clinical trials, IBI363 exhibited remarkable efficacy in immunotherapy-naïve melanoma patients. These trials, namely Phase 1a/1b (NCT05460767) and Phase 2 (NCT06081920), enrolled 26 patients suffering from advanced acral or mucosal melanoma. IBI363 achieved an impressive objective response rate (ORR) of 61.5% and a disease control rate (DCR) of 84.6%, surpassing current domestic standards for immunotherapy.

Extended follow-up periods have shown that IBI363 induces sustained tumor responses and long-term benefits, indicating its potential superiority over existing therapies in treating melanoma. Innovent's Senior Vice President, Dr. Hui Zhou, emphasized IBI363's unique mechanism as a first-in-class next-generation immuno-oncology therapy. It works by selectively inhibiting the PD-1/PD-L1 pathway while activating the IL-2 pathway. This dual-action approach provides regulatory recognition of its clinical value, addressing unmet medical needs, particularly in non-cutaneous melanoma subtypes like mucosal melanoma, which are prevalent in China and show resistance to immunotherapy.

The NMPA's Breakthrough Therapy Designation aims to accelerate the development and review of investigational drugs for serious conditions, based on preliminary clinical evidence suggesting significant improvement over existing treatments. This designation allows a drug candidate to receive expedited review and facilitates communication between the sponsor and regulatory authorities, thus accelerating approval processes to meet patients' clinical needs swiftly.

IBI363 is Innovent's independently developed first-in-class drug candidate, designed to enhance efficacy while reducing toxicity. The drug features a PD-1/IL-2 bispecific antibody fusion protein that optimizes therapeutic effects and minimizes side effects. Its IL-2 component is engineered for selective IL-2 delivery and PD-1 blockade, facilitating precise targeting and activation of tumor-specific T cells. Preclinical studies have demonstrated IBI363's potent anti-tumor activity across various pharmacological models, including those resistant to PD-1 inhibitors and metastatic models, while keeping a favorable safety profile.

Innovent is currently conducting clinical trials for IBI363 in China, the United States, and Australia to evaluate its safety, tolerability, and preliminary efficacy in patients with advanced malignancies. The pivotal study focuses on treating immunotherapy-naïve mucosal or acral melanoma. The U.S. FDA has granted IBI363 two fast track designations for melanoma and squamous NSCLC, further recognizing its potential.

Melanoma, though comprising only 3% of skin cancer types, has the highest mortality rate and a high likelihood of metastasis. The incidence and mortality rates of melanoma are rising in China. Melanoma is divided into three subtypes: cutaneous, acral, and mucosal. The characteristics of melanoma among Chinese patients differ significantly from those in European and American Caucasian populations, particularly in pathogenesis and treatment response. Advanced melanoma treatments vary depending on the presence of the BRAF V600 mutation and may include targeted therapies or chemotherapy combined with anti-angiogenic drugs. However, new treatment options are urgently needed for advanced mucosal melanoma, which remains resistant to current immunotherapy approaches.