AI-powered Cancer Vaccine In The Works

The company said the U.S. FDA has cleared the IND application for its self-developed EVM14, a tumor-associated antigen vaccine

Key Takeaways:

• The FDA has cleared Everest Medicine's EVM14 cancer vaccine application, and its EVM16 vaccine has been administered on the first patient under an investigator-initiated trial
• Everest Medicines has localized its own clinically validated advanced mRNA technology platform, which is now advancing a pipeline of innovative therapeutics

AI-powered healthcare has ignited a frenzy in capital markets and also reshaped the biopharma landscape. A key early innovator in that race is Everest Medicines Ltd. (1952.HK), China's leading biopharma company to integrate AI into mRNA cancer vaccine research. The company is advancing its AI-powered mRNA oncology pipeline into clinical trials, driving breakthroughs in target screening, sequence design, and delivery optimization.

Recent advances in mRNA research are a primary force behind the company's strategic shift to a "dual-engine" model, integrating in-house R&D capabilities with AI drug discovery technologies to propel the development of next-generation cancer immunotherapies.

Everest's latest breakthrough comes from its highly touted mRNA technology platform that is emerging as one of the company's crown jewels. The company announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for EVM14, a tumor-associated antigen (TAA) vaccine injection. Developed using Everest's proprietary mRNA technology platform, EVM14 has successfully received FDA IND authorization, becoming the company's first mRNA therapeutic cancer vaccine to enter international clinical development. This milestone represents a breakthrough for Everest in translating mRNA technology from fundamental research to global clinical application.

Everest also said in early March that that the first patient has been dosed with its internally developed personalized mRNA cancer vaccine EVM16 as part of an investigator-initiated clinical trial (IIT). Such moves demonstrate Everest's growing prowess in mRNA technology and are drawing in investors, with the stock price up about 150% over the last year, well ahead of other biotech companies.

While achieving a "commercial-level profit", the company's revenue is growing rapidly as more of its drugs reach the market, moving it closer to generating sustainable profits. Its latest report shows its revenue of 302 million yuan ($42 million) in the first half of last year was up sharply from 9 million yuan a year earlier.

The potential and strategy of mRNA vaccines

mRNA cancer vaccines are at the forefront of next-generation cancer immunotherapy, showcasing significant potential. The most advanced candidate, mRNA-4157, has completed patient enrollment for a phase three clinical trial, with developer Moderna suggesting it could be launched as early as 2027. This could mark a pivotal moment in the field, given the rapid development and flexibility of mRNA technology in targeting various cancers. The success of mRNA technology in Covid-19 vaccines has also propelled its application in oncology, building on the momentum from the pandemic.

Everest is among the first biopharmaceutical companies in China to integrate AI into mRNA-based oncology drug development, positioning it to reap big revenue from a global mRNA therapeutics market that Precedence Research expects will reach $20.83 billion by 2025 and $42.64 billion by 2034.

Everest Medicines began laying the groundwork for development of its mRNA technology as early as 2021, successfully localizing its clinically validated proprietary mRNA platform through roughly four years of R&D. It has established a comprehensive end-to-end industrial chain platform — one of the few globally — spanning antigen design, mRNA sequence optimization, LNP delivery technology development, and industrial-scale production.

The company holds global rights to its mRNA platform and pipeline, which is structured around four key areas: personalized cancer vaccines (PCV), tumor-associated antigens (TAA), immune-modulatory cancer vaccines, and in vivo CAR-T therapies. The company has its own commercial scale manufacturing facility, ensuring its core capabilities across the entire process from R&D to commercialization for mRNA oncology and autoimmune therapeutics.    

EVM16 is a novel personalized therapeutic mRNA cancer vaccine internally developed by Everest. It contains neoantigens with high immunogenicity potential, predicted based on the unique tumor mutations of each patient using Everest's proprietary AI-powered neoantigen prediction algorithm, EVER-NEO-1. In August 2024, Everest launched an investigator-initiated trial (IIT) to evaluate EVM16's safety, tolerability, immunogenicity and preliminary efficacy as both a standalone therapy and in combination with PD-1 antibodies for treatment of advanced or recurrent solid tumors.

EVM14 is an off-the-shelf mRNA mRNA cancer vaccine targeting multiple tumor-associated antigens, which is designed to treat various cancers, including non-small cell lung cancer and head and neck cancer.

EVM14 induced a dose-dependent, antigen-specific immune response in mice and significantly inhibited tumor growth in multiple syngeneic tumor models. As a therapeutic vaccine, EVM14 demonstrated the ability to induce immune memory and prevent tumor recurrence. The vaccine has the potential to allow patients to achieve "long-term cancer-free survival", a valuable feature in the field of tumor treatment.

Neither immunotherapy nor targeted therapy can offer long term benefits to cancer patients. EVM14's unique mechanism offers the potential to complement current treatments by enhancing the efficacy via combination therapies and delaying disease recurrence, aligning with current trends in oncology drug development. With a relatively standardized production process, the vaccine boasts lower manufacturing costs and broader applicability across various tumor types.

Another notable product in development is Everest's in vivo mRNA CAR-T therapy, which has demonstrated promising preclinical efficacy, with advantages over conventional CAR-T, including lower production costs, eliminating lymphodepletion, and outpatient administration needs. In parallel, the CAR-T cell therapy sector is evolving, with in vivo CAR-T development gaining prominence. This was highlighted by AstraZeneca's acquisition of EsoBiotech for $1 billion, underscoring the intense competition among pharmaceutical companies.

"Everest has successfully transitioned from a license-in model to a dual-engine strategy combining in-house R&D with strategic licensing, establishing a proprietary mRNA platform," said Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines. "EVM14, along with EVM16, which is a personalized cancer vaccine in clinical development and in-vivo CAR-T programs, will serve as foundational elements of our innovation strategy in oncology and autoimmune diseases."

Supercharging cancer and autoimmune diseases development with AI

DeepSeek's recent success with large language model AI is drawing renewed attention to the technology's potential, including in the pharmaceutical industry. In drug development, AI can enhance efficiency in drug discovery, compound synthesis, clinical trial cohort selection and data analysis. A report in TechEmergence says AI can boost the new drug success rates by 16.7% while slashing time costs by 40% to 60%. Governments around the world are accelerating AI adoption in pharmaceuticals, with the U.S. launching a $500 billion Stargate Project in January this year to redesign mRNA-based cancer therapies using AI.

Everest's "AI+mRNA" technological integration is attracting its own investor attention. The strategy sees the company embed AI across critical mRNA development stages, including in neoantigen prediction, mRNA sequence design and LNP research to boost R&D efficiency.

In mRNA neoantigen vaccine development, accurate prediction of immunogenic mutations is crucial for designing vaccines that activate immune attacks on tumor cells. EVER-NEO-1, the AI-powered neoantigen prediction algorithm developed in-house by Everest, can identify the majority of reported tumor neoantigens, as well as several previously unreported neoantigens.  Furthermore, it has outperformed the tumor neoantigen identification system developed by Memorial Sloan Kettering Cancer Center in certain aspects.

Everest's self-developed mRNA sequence design algorithm has currently moved to its third generation after four years of upgrades. For LNP delivery systems — the workhorses of mRNA vaccines — Everest's proprietary LNPs enhance protein expression and T-cell immunity, with AI deeply integrated into its in-house lipid library screening to accelerate discovery.

Following its recent gains, Everest's stock now trades at a high price-to-sales (P/S) ratio of about 30 times. Within the AI-powered drug development sector, such companies, which possess both commercial products and AI platform capabilities, look like strong bets in the crowded field of publicly traded Chinese biotech companies. As AI continues to redefine drug discovery and development, Everest's expertise in AI-powered mRNA technology positions it as an attractive partner for global pharmaceutical companies seeking cutting-edge innovations and accelerated development timelines.