RenJing Biotechnology Announces FDA IND Approval for First-in-Class mRNA Therapeutic Vaccine RG002 Targeting HPV-Related Cervical Intraepithelial Neoplasia

Today, RinuaGene Biotechnology Co., Ltd. (hereinafter referred to as “RinuaGene”) announced that its independently developed therapeutic mRNA vaccine for HPV-related tumors, RG002, received Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) on October 27, 2023. The vaccine is indicated for the treatment of cervical intraepithelial neoplasia grade 2 or 3 (CIN2/3) associated with human papillomavirus (HPV) types 16 and/or 18, making it the first mRNA-LNP therapeutic vaccine globally approved to initiate clinical trials for this indication. The company is simultaneously advancing its clinical trial application with the National Medical Products Administration (NMPA).

HPV infection (particularly with HPV types 16 and 18) is the primary cause of cervical precancerous lesions and cervical cancer. The persistent progression of these precancerous lesions ultimately leads to the development of cervical cancer, which is the fourth most common malignant tumor threatening women's health worldwide.^[1] . Surgery is the primary treatment for cervical precancerous lesions, but it carries numerous risks, including a significantly increased rate of preterm birth and miscarriage, as well as a high rate of persistent or recurrent disease.^[2]. Furthermore, HPV infection is directly associated with oropharyngeal cancer, anal cancer, and genital system tumors such as vaginal cancer, penile cancer, and vulvar cancer.^{[3] [4]}. Although prophylactic HPV vaccines are already on the market, HPV-related cancers continue to pose a broad threat, with more than 850,000 new cases diagnosed annually and a high case fatality rate, resulting in nearly 450,000 deaths each year.^[1], andCurrently, no therapeutic vaccine targeting HPV-related precancerous lesions and malignant tumors has been approved for market launch worldwide.

RG002 utilizes RinuaGene’s proprietary mRNA sequences and lipid nanoparticle (LNP) delivery system, and has undergone comprehensive optimization based on multiple patented technologies, including antigen design, sequence optimization, and regulatory elements.The delivery system employed by the company demonstrates high delivery efficiency and excellent in vivo safety. Preclinical studies indicate that RG002 monotherapy can induce robust specific cellular immunity and tumor cell cytotoxicity, effectively inhibiting the growth of HPV-associated tumors; furthermore, it exhibits favorable synergistic and potentiating effects when combined with anti-tumor agents such as immune checkpoint inhibitors (e.g., PD-L1 antibodies).

The robust preclinical data provide a solid foundation for the use of RG002 in treating HPV-related precancerous lesions and malignant tumors. RinuaGene will continue to efficiently advance the clinical development of RG002, extensively arranging clinical trials for relevant indications globally, and actively exploring RG002 as monotherapy and combination therapy for cervical precancerous lesions, cervical cancer, head and neck squamous cell carcinoma, and rare tumors.Addressing the substantial clinical need for treating HPV-related diseases to benefit patients worldwide.

Professor Wu Lingying, Director of the Department of Gynecologic Oncology at the Cancer Hospital, Chinese Academy of Medical Sciences, and Chairperson of the CSCO Gynecologic Oncology Expert Committee“It is indicated that the incidence of cervical cancer in China is trending toward younger age groups. With the widespread adoption of early screening for cervical cancer, an increasing number of precancerous lesions and early-stage cervical cancers are being detected at earlier stages. For patients with precancerous lesions who desire fertility preservation or are unsuitable for surgical treatment, there is a need for non-surgical therapeutic options. For advanced cervical cancer, the long-standing lack of effective treatments has resulted in poor patient prognosis. In recent years, immunotherapy has achieved breakthrough progress in cervical cancer; however, drugs such as PD-1 monoclonal antibodies have limited response rates. Therefore, there is an urgent need for new therapeutic strategies to further improve overall efficacy and survival benefits. RG002, a novel mRNA-LNP drug independently developed by RinuaGene, offers a completely new mechanism of action by exerting clinical efficacy through immune activation against HPV-specific antigens. Its combination with existing standard chemotherapy and PD-1 monoclonal antibodies holds broad prospects for clinical development, warranting anticipation from both clinicians and patients.”

RinuaGene settled in Suzhou BioBAY in March 2021, dedicated to developing innovative mRNA-based therapeutics and vaccines to address significant unmet clinical needs globally. The company has established a pipeline of more than 10 candidates, advanced four of them to the Investigational New Drug (IND) application stage, and initiated three IND submissions, one of which has already received FDA IND clearance. We extend our sincere gratitude to all partners, investors, and BioBAY for their continued trust and support. RinuaGene remains committed to efficiently advancing the global development of its pipeline and iteratively upgrading its technology platform.

The Company holds comprehensive independent intellectual property rights and a global patent portfolio, having filed over 30 invention patents and obtained two software copyright registrations. It operates four core technology platforms: linear mRNA, circular RNA, novel LNP-targeted delivery, and CMC. Located in BioBAY, the Company maintains an mRNA innovation and R&D center along with a pilot-scale cleanroom facility, enabling end-to-end independent R&D and clinical-grade manufacturing capabilities for mRNA therapeutics and vaccines.

At this stage, the company focuses on the research and development of therapeutic cancer vaccines, infectious disease vaccines, cancer immunotherapy drugs, protein replacement products, and novel delivery systems. Upholding its corporate culture of innovation, openness, collaboration, and mutual benefit, the company welcomes multifaceted cooperation in areas such as product development, combination therapies, technology platforms, and novel delivery systems, to jointly promote the development and application of RNA technology across multiple fields.

References:

1.Sung H, Ferlay J, Siegel RL, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries[J]. CA:A Cancer Journal for Clinicians, 2021, 71(3): 209 – 249.

2. Bi Hui, Li Mingzhu, Zhao Chao, et al. Chinese Expert Consensus on the Management of Cervical Low-Grade Squamous Intraepithelial Lesions[J]. Chinese Journal of Clinical Obstetrics and Gynecology, 2022, 23(4):3.

3. Jia Xinhua, Su Zheng, Li Yufei, et al. Research progress on the epidemiology of human papillomavirus-related head and neck cancer [J]. Chinese Journal of Public Health, 2022, 38(11):5.

4. National Medical Products Administration. Technical Guidelines for Clinical Trials of Human Papillomavirus Vaccines (Trial), 2023.