Evaxion's AI-Immunology Platform Gains Validation in Clinical Trials

Evaxion Biotech has achieved a notable breakthrough with its AI-Immunology platform, demonstrating its capability to select effective cancer vaccine targets in clinical trials. This AI-powered platform, integrating proprietary models like PIONEER, EDEN, RAVEN, and ObsERV, identifies neoantigens—tumor-specific targets that can stimulate immune responses. In three separate trials, a strong correlation was observed between high AI-predicted scores and robust immunological responses in patients. Furthermore, in one trial, a statistically significant link was found between AI predictions and Progression Free Survival (PFS), highlighting the platform's clinical relevance.

The company's pipeline includes three personalized cancer vaccines: EVX-01, EVX-02, and EVX-03. EVX-01, targeting metastatic melanoma and used in combination with Merck’s Keytruda (clinical trial), showed promising results with a 67% patient benefit rate, compared to the 33-40% expected from PD-1 inhibitors alone. Currently, EVX-01 is undergoing Phase 2 trials in the U.S., Europe, and Australia. EVX-02, a DNA-based vaccine, and EVX-03, aimed at advanced solid cancers, are also advancing in development.

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Here is a brief overview of Evaxion's AI platform:

Oncology: PIONEER and ObsERV

At the heart of Evaxion’s immunotherapy work is PIONEER, a model positioned to rapidly identify neoantigens—mutated proteins that emerge in cancer cells. The company suggests that PIONEER can accurately select the most promising neoantigens, thereby guiding the development of highly personalized vaccines. By doing so, Evaxion aims to offer a precision-based alternative to the conventional "one-size-fits-all" approach, potentially minimizing unwanted immune responses.

Key highlights of PIONEER:

• Accurate neoantigen prediction: Said to drive T-cell responses with higher accuracy compared to other state-of-the-art tools.

• Multiple neoantigen targeting: Aims to address tumor heterogeneity and immune escape.

• Personalized therapy: PIONEER tailors treatments based on patient-specific tumor profiles and HLA types.

In clinical trials, PIONEER reportedly triggered neoantigen-specific T-cell responses in 100% of patients treated with EVX-01, with a 67% overall response rate.

ObsERV: Harnessing Endogenous Retroviruses

ObsERV, another key component of Evaxion's platform, focuses on endogenous retroviruses (ERVs)—ancient viral remnants embedded in human DNA. While typically dormant in healthy cells, these ERVs can be reactivated in cancer cells, offering a potential source of antigens for personalized cancer immunotherapies.

Key highlights of ObsERV:

• Personalized ERV antigen treatment: ObsERV identifies patient-specific ERV antigens based on sequencing data and designs tailored immunotherapies.

• Proof of concept: Preclinical models have shown that ObsERV-based therapies can trigger durable tumor protection and stimulate robust T-cell responses (both CD8+ and CD4+).

• Expanding immunotherapy: Adding ObsERV to PIONEER could help target cancers with low tumor mutational burden (TMB), where neoantigens are scarce.

Evaxion suggests that ERVs could be particularly valuable for patients with cancers that exhibit few mutations, offering new treatment possibilities. Large-scale analyses have indicated that cancers with low TMB may still express significant numbers of ERVs, opening the door for immunotherapy in a broader patient population. ObsERV’s integration into personalized treatments is already under discussion with regulatory authorities.

Bacterial Infectious Diseases: EDEN

EDEN brings innovation to bacterial vaccine development, particularly for antibiotic-resistant strains. Evaxion promotes EDEN as a rapid solution for discovering bacterial antigens, asserting that it can identify protective antigens in a matter of days. The platform narrows down thousands of bacterial proteins to a select few that show the most potential for vaccine development.

Key highlights of EDEN:

• Rapid antigen identification: Supposedly identifies vaccine targets in weeks rather than years.

• Broad protection: Designed to target a wide range of bacterial strains, including antibiotic-resistant pathogens like MRSA.

Viral Infectious Diseases: RAVEN

RAVEN extends the AI-Immunology platform into viral diseases, including rapidly mutating viruses. RAVEN is presented as a model capable of identifying both B-cell antigens and T-cell epitopes, combining them to produce broader and longer-lasting immune responses.

Key highlights of RAVEN:

• Dual response: Designed to trigger both B-cell and T-cell immune responses.

• Resilience to viral mutations: The platform aims to ensure durability and broader protection against viral variants.

Image credit: Wirestock, iStock

Topics: Clinical Trials