Nanjing Nuoyuan Medical Devices Co., Ltd. has achieved a significant milestone in precision oncology with the successful completion of Phase I clinical trials for Pemefolacianine, a next-generation fluorescent imaging agent designed to improve intraoperative tumor detection. The novel contrast agent, which targets folate receptor alpha (FR-α), demonstrated excellent safety and favorable pharmacokinetics in the completed study.
Addressing Critical Limitations in Current Imaging Technology
Traditional fluorescent contrast agents like Indocyanine Green (ICG), approved in 1956, face significant limitations in oncologic surgery. As a passively-targeted agent, ICG exhibits limited specificity for tumor tissues, often resulting in high false-positive rates. Its relatively low fluorescence quantum yield further restricts imaging performance, creating bottlenecks in precision diagnosis during cancer surgery.
Pemefolacianine represents a paradigm shift toward targeted fluorescent contrast agents, which consist of three key components: a targeting ligand, a linker, and a fluorochrome. These agents enable precise tumor localization, improve accuracy of intraoperative tumor margin identification, allow detection of small lesions, and reduce the risk of residual tumor tissue.
Targeting Folate Receptor Alpha for Enhanced Specificity
Folate receptor alpha emerges as an ideal molecular marker due to its overexpression in various malignancies including lung cancer and ovarian cancer, while maintaining minimal expression in normal tissues. This stark contrast differentiates FR-α from the beta subtype (FR-β), which is upregulated in acute myeloid leukemia and tumor-associated macrophages and exhibits expression in normal tissues.
Developed using Nuoyuan Medical's Artificial Intelligence for Drug Discovery (AIDD) platform, Pemefolacianine demonstrates strong selectivity for FR-α with approximately eight times higher affinity compared to FR-β. Preclinical studies show the agent can precisely identify and bind to FR-α overexpressed on tumor cell surfaces, enabling accurate localization and real-time imaging of tumor tissue while significantly improving spatial resolution and specificity.
Phase I Trial Results Demonstrate Safety and Efficacy
The Phase I clinical trial, approved by ethics committee (YW2025-010(F1)) and registered on the Chinese Clinical Trial Register (CTR20250567), enrolled 32 healthy participants randomized at a 6:2 ratio to receive either Pemefolacianine injection or placebo.
The study revealed no drug-related adverse events, indicating favorable safety and tolerability profiles. Pharmacokinetic analysis showed that both area under the curve (AUC) and maximum plasma concentration (Cmax) increased linearly with dose, with elimination half-life ranging from 1.59 to 7.86 hours. Both fluorescence intensity and central point spectral values exhibited dose-dependent correlations.
Four Key Advantages Over Existing Agents
Pemefolacianine offers distinct advantages through its novel structure developed via AIDD technology. Enhanced targeting through selective FR-α binding may help reduce clinical false-positive rates. The agent maintains stability at both 2-8°C and 25°C, significantly easing storage and transportation requirements.
Compatibility with 0.9% sodium chloride injection or 5% glucose injection provides better suitability for diabetic patients. Most notably, the Phase I clinical trial observed no drug-related adverse events, establishing a higher safety profile compared to existing alternatives.
Global Development Strategy and Market Positioning
Following successful Phase I completion, Nuoyuan Medical has launched Phase II clinical trials in China to systematically evaluate Pemefolacianine's efficacy in intraoperative tumor imaging alongside further safety assessment. The company has implemented a dual-track clinical development strategy across Chinese and international markets through a "China U.S. dual filing and approval" approach.
Pemefolacianine became the first FR-α-targeted fluorescent contrast agent from China to receive FDA Investigational New Drug (IND) approval, following the FDA's approval of Cytalux (OTL-38), the first folate receptor-targeted contrast agent. The company is actively preparing for U.S. clinical trials to accelerate global registration, positioning Pemefolacianine as a leading targeted fluorescent contrast agent worldwide.
Patent authorizations have been secured in China, the U.S., Europe, and Japan, establishing a strong foundation for global commercialization and intellectual property protection.
Comprehensive Product Pipeline and Drug-Device Integration
Beyond Pemefolacianine, Nuoyuan Medical has established a diverse molecular imaging product pipeline spanning both fluorescent and radionuclide imaging. The next-generation broad-spectrum fluorescent contrast agent Glydocyanine Green and the SPECT-CT radionuclide contrast agent NY-99mTc-FAPI-068 have both entered IND submission stages in China and the United States.
The company's drug-device integration strategy encompasses multiple molecular imaging systems, including surgical fluorescence imaging systems, 4K endoscopic fluorescence imaging systems, and AI-assisted Raman spectroscopy-based multimodal fluorescence navigation systems. With over 160 granted patents in China and abroad, Nuoyuan Medical demonstrates strong global competitiveness in establishing comprehensive solutions for precision oncology.
