Frost & Sullivan released a report titled “Nuoyuan Medical: Class I New Drug Pemefolacianine Completes Phase I Clinical Trial, Advancing Intraoperative Precision Diagnosis.” The report highlights how Nanjing Nuoyuan Medical Devices Co., Ltd. (Nuoyuan Medical) independently developed Class I new drug, Pemefolacianine, recently completed Phase I clinical trials in China with promising results. As an innovative fluorescent imaging agent targeting folate receptor alpha (FRα), Pemefolacianine demonstrated excellent safety and favorable pharmacokinetics.
Adopting a dual-track development model of “drug-device integration”, Nuoyuan Medical focuses on technology convergence and clinical translation, driving the joint development of Pemefolacianine and a series of molecular imaging contrast agents along with supporting imaging systems. Pemefolacianine is expected to systematically address the limitations of current contrast agents in intraoperative precision diagnosis of tumors. When combined with a fluorescence imaging system, it offers a superior, real-time diagnostic tool for surgery. Nuoyuan Medical is currently conducting a Phase II clinical trial of Pemefolacianine in China, while clinical trial preparations are underway in the United States. The company has completed patent filings in many countries, demonstrating its strong global competitiveness and forward-looking strategy in the field of molecular imaging.
I. The Contemporary Needs of the Precision Tumor Diagnosis and the Advancement of Molecular Imaging Contrast Agents
Limitations of Traditional Fluorescent Contrast Agents
Indocyanine Green (ICG) is a widely used near-infrared fluorescent agent that has been applied in clinical diagnostics and intraoperative image-guided procedures since its approval in 1956. As a representative passively-targeted agent, ICG has limited specificity for tumor tissues, often leading to a high rate of false-positive findings. Additionally, its relatively low fluorescence quantum yield restricts imaging performance. These limitations have become major bottlenecks to further advances in precision diagnosis during oncologic surgery.
The Emergence and Significance of Targeted Fluorescent Contrast Agents
To address urgent clinical needs, targeted fluorescent contrast agents have been developed. These contrast agents typically consist of three key components: a targeting ligand, a linker, and a fluorochrome. The fluorochrome emits fluorescence at a defined wavelength, while the targeting ligand binds specifically to biomolecules or cell receptors that are overexpressed on the surface of tumor cells. These novel contrast agents enable precise localization of tumors, improve the accuracy of intraoperative tumor margin identification, allow detection of small lesions, and reduce the risk of residual tumor tissue, ultimately helping to lower postoperative recurrence rates.
II. Clinical Value of Pemefolacianine
– Folate Receptor Alpha (FRα) is the Key Tumor Biomarker
Folate receptors (FRs) are glycoproteins located on the cell membrane and include several subtypes. Among them, alpha subtype (FRα) and beta subtype (FRβ) have drawn particular attention due to their distinct biological features in tumor development and progression. Studies have shown that FRα is overexpressed in various malignancies such as lung cancer and ovarian cancer, while its expression in normal tissues is minimal. This stark contrast makes FRα an ideal molecular marker for tumor diagnosis and therapy. In comparison, FRβ is upregulated in acute myeloid leukemia (AML) and tumor-associated macrophages (TAMs), and also exhibits a certain degree of expression in normal tissues. These marked differences in expression patterns between FR subtypes provide a solid theoretical basis for developing contrast agents that specifically target FRα.
– Design Concept and Mechanism of Action of Pemefolacianine
Pemefolacianine is a next-generation near-infrared fluorescent (NIRF) agent developed by Nuoyuan Medical using its Artificial Intelligence for Drug Discovery (AIDD) platform. Its core design aims to achieve highly specific targeting of FRα. Preclinical studies have shown that Pemefolacianine has strong selectivity for FRα, with an affinity approximately eight times higher than for FRβ. It can precisely identify and bind to FRα, which is overexpressed on tumor cell surfaces, enabling accurate localization and real-time imaging of tumor tissue. This specific molecular recognition mechanism significantly improves the spatial resolution and specificity of tumor imaging, while effectively reducing the false-positive rate. Pemefolacianine holds a first-mover advantage in the field of fluorescent contrast agents and is currently the only FRα-targeted contrast agent undergoing clinical development worldwide. It is well positioned to provide a new technological foundation for precision tumor diagnosis and treatment.
– The Analysis of OTL-38 (Cytalux) Clinical Application
OTL-38 is the first near-infrared fluorescent (NIRF) contrast agent targeting folate receptors (FR) to be approved by the U.S. Food and Drug Administration (FDA). In clinical use, it enables precise detection of small lesions, including those potentially missed by conventional methods, thereby significantly enhancing lesion detection efficiency. OTL-38 was approved by the FDA in 2021 and 2022 for intraoperative identification of malignant lesions in ovarian cancer and lung cancer, respectively. Despite its favorable contrast imaging effect, OTL-38 has certain limitations. Firstly, it lacks selectivity between FRα and FRβ, which has led to a relatively high false-positive rate in clinical trials. Secondly, in terms of pharmaceutical stability, OTL-38 must be stored and transported at temperatures below -20°C.
– Advantages of Pemefolacianine
With a strong foundation in AIDD, Nuoyuan Medical has developed Pemefolacianine, a structurally novel contrast agent designed to offer a more effective real-time diagnostic for precision tumor surgery.
– Enhanced Targeting: Pemefolacianine selectively binds to FRα, which may help reduce the clinical false-positive rate.
– Improved Stability: Pemefolacianine remains stable at 2-8°C or 25°C, greatly easing storage and transportation requirements.
– Better Compatibility: Pemefolacianine is compatible with 0.9% sodium chloride injection or 5% glucose injection, offering better suitability for diabetic patients.
– Higher Safety Profile: In the Phase I clinical trial of Pemefolacianine, no drug-related adverse events were observed.
III. The Results of Pemefolacianine Phase I Clinical Trial in China
The Phase I clinical trial of Pemefolacianine in China has been successfully completed with positive results. The study was initiated following approval from the ethics committee (YW2025-010(F1)) and was registered on the Chinese Clinical Trial Register (CTR20250567). A total of 32 healthy participants were enrolled and randomized at a 6:2 ratio to receive either Pemefolacianine for injection or a placebo.
No drug-related adverse events were reported during the trial, indicating that Pemefolacianine has a favorable safety and tolerability profile.
- Pharmacokinetic Characteristics
The area under the AUC (area under the curve) and the maximum plasma concentration (Cmax) of Pemefolacianine increased linearly with dose. The elimination half-life ranged from 1.59 to 7.86 hours. Additionally, both fluorescence intensity and central point spectral values exhibited a dose-dependent correlation.
Table: Key Results of Pemefolacianine Phase I Clinical Trial in China
IV. Global Clinical Development and Market Strategy
Nuoyuan Medical recognizes the strategic importance of globalizing innovative drug development and has implemented a dual-track clinical development and market strategy for Pemefolacianine across both Chinese and international markets. To support this goal, the company has adopted a “China U.S. dual filing and approval” approach. In addition, Pemefolacianine has secured patent authorizations in China, the U.S., Europe, and Japan, establishing a strong foundation for global commercialization and intellectual property protection.
– Domestic Development Plan: Initiation of Phase II Clinical Trial in China
Following the successful completion of the Phase I trial and confirmation of its safety profile, Nuoyuan Medical has launched a Phase II clinical trial of Pemefolacianine in China. This trial aims to systematically evaluate the efficacy of Pemefolacianine in intraoperative tumor imaging, along with further assessment of its safety.
– International Strategy: FDA IND Approval and Global Expansion
After the U.S. FDA approved Cytalux (OTL-38), the first folate receptor-targeted contrast agent, Pemefolacianine became the first FRα-targeted fluorescent contrast agent from China to receive Investigational New Drug (IND) approval from the FDA. Nuoyuan Medical is actively preparing for clinical trials in the United States to accelerate its global registration, with the goal of positioning Pemefolacianine as a leading targeted fluorescent contrast agent worldwide.
V. Robust Product Pipeline and Forward-Looking Strategy
In addition to Pemefolacianine, Nuoyuan Medical has established a diverse molecular imaging product pipeline, reflecting its strong capabilities in innovative drug development and forward-looking strategic planning. With a comprehensive portfolio spanning both fluorescent and radionuclide imaging, and encompassing both broad-spectrum and targeted contrast agents, the company is well-positioned for resilience and long-term growth in the molecular imaging market. Currently, the next-generation broad-spectrum fluorescent contrast agent Glydocyanine Green and the next-generation SPECT-CT radionuclide contrast agent NY-99mTc-FAPI-068 have both entered the IND submission stage in China and the United States.
Indocyanine Green (ICG) for Injection: As a broad-spectrum fluorescent imaging agent, ICG has received marketing approval from China’s National Medical Products Administration (NMPA) and passed dual consistency evaluations, meeting international quality standards.
Glydocyanine Green: A next-generation broad-spectrum fluorescent imaging agent, Glydocyanine Green addresses the limitations of conventional ICG in targeting and imaging performance. It offers enhanced tumor-targeting ability, higher fluorescence efficiency, and favorable in vivo pharmacokinetics. Its imaging window exceeds 72 hours, making it suitable for visualizing solid tumors and the upper urinary tract.
NY-99mTc-FAPI-068: A next-generation fibroblast activation protein (FAP) targeted radionuclide imaging agent. Data from investigator-initiated trials (IITs) show that it delivers high tumor uptake, low uptake in normal organs such as the liver, an extended imaging window, and a strong safety profile. It is intended for use in the diagnosis, staging, and follow-up of multiple tumor types, including pancreatic cancer, lung cancer, and neuroendocrine tumors.
VI. Integration of Drug and Device Innovation to Elevate Precision Oncology to New Heights
Nanjing Nuoyuan Medical Devices Co., Ltd. is a technologically advanced enterprise based in Jiangsu Province, China, dedicated to the R&D, manufacturing, and commercialization of high-end medical devices and novel targeted contrast agents. The company has adopted an innovation-driven model that promotes coordinated development of drugs and devices, with a drug-led approach, aiming to deliver comprehensive solutions for precision oncology. Its core technology platforms include molecular fluorescence, Raman spectroscopy, CMOS imaging, medical artificial intelligence, and AIDD. Nuoyuan Medical has established a robust product pipeline, consisting of multiple series of oncology diagnostic and therapeutic systems and molecular imaging contrast agents. The company holds fully independent intellectual property rights, with over 160 granted patents in China and abroad, reflecting its strong global competitiveness.
As a pioneer in China’s molecular imaging field, Nuoyuan Medical has developed a portfolio of internationally advanced molecular imaging systems, including surgical fluorescence imaging systems, 4K endoscopic fluorescence imaging systems, 4K 3D endoscopic fluorescence systems, and AI-assisted Raman spectroscopy-based multimodal fluorescence navigation systems. The company has also independently developed innovative fluorescent probes and radionuclide imaging contrast agents, such as Pemefolacianine, Glydocyanine Green, and NY-99mTc-FAPI-068, achieving true synergy between imaging technologies and diagnostic contrast agents. With continued implementation of its dual-engine “drug-device integration” strategy, Nuoyuan Medical is establishing a full-chain solution for clinical diagnosis and treatment integration, and is enabling the transition to a new era of submillimeter-precision oncologic surgery.
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