SHANGHAI, July 4, 2025 /PRNewswire/ -- Everest Medicines (HKEX: 1952.HK), a biopharmaceutical company focused on the discovery, clinical development, manufacturing and commercialization of innovative medicines, recently hosted the "2025 Everest Medicines mRNA Platform R&D Day" in Shanghai. The event unveiled significant advancements in the company's proprietary AI+mRNA platform and highlighted key cancer and autoimmune pipeline programs developed through the platform, substantially progressing the company's "dual-engine" strategy.

The R&D Day showcased Everest's proprietary mRNA platform driven by AI and its three leading pipeline assets in cancer and autoimmune disease: the personalized cancer vaccine (PCV) EVM16, the off-the-shelf tumor associated antigen (TAA) vaccine EVM14 and the in vivo CAR-T program.

Everest has built an industry-leading, fully integrated, and localized AI+mRNA platform that accelerates mRNA product development. EVM16, a personalized cancer vaccine, is built upon a proprietary AI-based neoantigen prediction algorithm, EVER-NEO-1, and the third generation mRNA sequence optimization model. mRNA sequences encoding each patient's tumor-specific neoantigens are encapsulated into lipid nanoparticles (LNP) and administered to the patient to elicit an antigen specific T cell immune response. Preclinical studies of EVM16 in mouse melanoma models demonstrated efficacy and synergistic effects when combined with PD-1 antibody. The Investigator-Initiated Clinical Trial (IIT) dosed the first patient in March 2025. Early results showed strong immunogenicity and specific T-cell responses even at a low starting dose in advanced cancer patients.

EVM14, an off-the-shelf therapeutic mRNA cancer vaccine, targets five tumor-associated antigens and is applicable across multiple types of squamous cell carcinomas. Preclinical studies have demonstrated its potential to induce immune memory and reduce tumor recurrence. EVM14 has received a U.S. Food and Drug Administration (FDA) Investigational New Drug (IND) clearance and is progressing toward IND submission in China.

Everest's in vivo CAR-T program is founded upon its proprietary targeted LNP (tLNP) delivery system and has shown promising results in both humanized mouse models and non-human primates. It offers key advantages including off-the-shelf availability, lymphodepletion-free administration, and dose controllability.

These advancements, together with progress across the proprietary AI+mRNA platform and pipeline, best exemplify Everest's strategic transition from a 'license-in' model to one focused on driving internal innovation.

"Most existing anti-cancer therapies fail to generate lasting immune memory and offer limited protection against recurrence. Even after surgery, chemotherapy, and/or immunotherapy, patients with resectable advanced tumors face a high risk of recurrence or metastasis within 2-3 years," said Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines. "mRNA vaccines, with the potential to induce immune memory, hold promise to effectively prevent tumor recurrence and metastasis, and have shown breakthrough results in this field." "Recent publications in Nature^[1] and Science^[2] validate the potential of personalized mRNA cancer vaccines and in vivo CAR-T therapies, respectively. The clinical data confirm that personalized mRNA cancer vaccines in combination with PD-(L)1 inhibitors and chemotherapy can significantly reduce recurrence risk in resectable stage I–III pancreatic cancer. Meanwhile, in vivo CAR-T therapy demonstrated rapid and deep tumor clearance in humanized mouse models, paving the way for new options for cancer and refractory autoimmune diseases."

The advancement of CAR-T and mRNA therapies marks a turning point in cell and gene therapies, with mRNA driving the next wave of medical innovation. As a genetic information-based modality, mRNA turns the body into a "programmable medicine factory," with broad applications across oncology, autoimmune disorders, infectious diseases, and rare disease.

As one of the few global biopharmaceutical companies with a fully integrated and proprietary mRNA platform, Everest has built end-to-end capabilities across the whole value chain: covering antigen design, sequence optimization, LNP delivery and scalable manufacturing. Backed by third-generation AI algorithms, our AI+mRNA platform significantly boosts expression and immunogenicity, laying a solid foundation for mRNA-based cancer vaccine development.

The platform has previously been clinically validated. In a global Phase II clinical trial enrolling 374 subjects, Everest's mRNA COVID-19 vaccine candidate PTX-COVID19-B was directly compared with Comirnaty^®, Pfizer/BioNTech's U.S. FDA-approved mRNA vaccine. PTX-COVID19-B demonstrated non-inferiority compared to Comirnaty^®. Additionally, it was generally well-tolerated, with a safety and tolerability profile similar to Comirnaty^®. 

We are engaging with the top 20 global pharmaceutical companies, focusing our BD strategy on leveraging international resources to accelerate global development of innovative assets. Meanwhile, we believe our AI+mRNA platform can drive sustained breakthroughs in cancer and autoimmune therapeutics, bringing real change to patients by not only extending life but improving quality of life."

"We are pleased to present the latest advancement of Everest's proprietary AI+mRNA platform. As a key delivery tool for mRNA vaccine development, we have established and optimized our proprietary LNP delivery system, with patent coverage particularly in ionizable lipids and stealth lipids. Our internal lipid library (>500 lipids) supports multiple programs, including vaccines and in vivo CAR-T therapies. Extensive in vivo studies were carried out to evaluate efficacy, safety, and tolerability of lipids.  We have established both 'passive' and 'active' targeting strategies for extrahepatic and tissue/cell-specific delivery. The platform is scalable with a strong intellectual property portfolio," said Jennifer Yang, Ph.D, Chief Scientific Officer of Everest Medicines. "EVM16CX01 is the first-in-human trial for EVM16, aiming to assess the safety, tolerability, immunogenicity, and preliminary efficacy of EVM16 as monotherapy and in combination with a PD-1 antibody in patients with advanced or recurrent solid tumors. Preliminary data showed that EVM16 elicits strong immunogenicity and specific T-cell responses in advanced patients even at low starting dose, validating the reliability of our EVER-NEO-1 algorithm.

The global, multi-center Phase I clinical trial of EVM14 adopts a staggered design to efficiently determine the recommended Phase II dose (RP2D). The study will enroll patients with three major tumor types: squamous non-small cell lung cancer (sq-NSCLC), head and neck squamous cell carcinoma (HNSCC), and esophageal squamous cell carcinoma (ESCC). The primary endpoints are safety and tolerability. Additionally, preliminary evaluation of anti-tumor efficacy in advanced cancers patients will also be explored. Both MD Anderson Cancer Center in the United States and Shanghai Chest Hospital in China will participate in the trial.

The EVM14 C101 Phase 1 clinical trial is expected to enroll its first patient in Q3 2025. The first clinical batch of EVM14 was successfully released from Everest's Jiashan manufacturing site on June 9, and is anticipated to be delivered to U.S. clinical centers by mid-August.

Everest's in vivo CAR-T program, developed based on the proprietary targeted LNP (tLNP) system, has made significant progress. In humanized mouse models and non-human primate (NHP) models, it demonstrated high T-cell transfection rates, excellent CAR expression, and effective B-cell clearance.

About Everest Medicines

Everest Medicines is a biopharmaceutical company focused on discovering, developing, manufacturing and commercializing transformative pharmaceutical products and vaccines that address critical unmet medical needs for patients in Asian markets. The management team of Everest Medicines has deep expertise and an extensive track record from both leading global pharmaceutical companies and local Chinese pharmaceutical companies in high-quality discovery, clinical development, regulatory affairs, CMC, business development and operations. Everest Medicines has built a portfolio of potentially global first-in-class or best-in-class molecules in the company's core therapeutic areas of renal diseases, infectious diseases and autoimmune disorders. For more information, please visit its website at www.everestmedicines.com.

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