Imagine a world where cancer treatment is personalized, powered by AI, and shows remarkable results. That's exactly what Evaxion's groundbreaking phase 2 trial of EVX-01, an AI-designed personalized cancer vaccine, has achieved. But here's where it gets controversial: can a small trial with just 16 patients truly revolutionize cancer treatment? And this is the part most people miss: the potential implications of a 75% objective response rate in advanced melanoma patients, a condition notoriously difficult to treat.
Evaxion (NASDAQ: EVAX) unveiled the two-year results of its phase 2 trial on October 17, 2025, showcasing an impressive 75% objective response rate (ORR) in 12 out of 16 advanced melanoma patients. This included 4 complete responses, with 92% of patients still responding at the 24-month mark. The vaccine, EVX-01, targets multiple neoantigens—unique proteins arising from cancer mutations—and demonstrated an 81% success rate in inducing specific T-cell responses. Tumor reduction was observed in 15 out of 16 patients, and 54% experienced deepened responses during treatment. The treatment was well-tolerated, with no significant safety concerns reported.
But let's dive deeper into the details. The trial, though small, presents a compelling case for the potential of AI-driven personalized medicine. EVX-01, developed using Evaxion's AI-Immunology™ platform, is tailored to each patient's unique tumor profile and immune characteristics. This precision approach has yielded results that are not only statistically significant but also clinically meaningful. For instance, the 75% ORR is a substantial improvement over traditional treatments, and the durability of responses—with 92% of patients still responding after two years—is particularly noteworthy.
However, the trial's limitations cannot be overlooked. The small cohort size of 16 patients raises questions about the generalizability of the results. Additionally, the primary endpoint was not formally analyzed due to reduced enrollment, and key secondary endpoints like progression-free survival (PFS) and overall survival (OS) were not yet reportable at the data cut-off. These factors introduce uncertainty and highlight the need for larger, confirmatory studies.
Here's where the debate heats up: While the results are undeniably promising, some critics argue that the small sample size and lack of formal analysis of the primary endpoint may overstate the vaccine's efficacy. Others counter that the biological signals and clinical activity observed in this trial are too compelling to ignore, especially given the dire prognosis typically associated with advanced melanoma. The question remains: is EVX-01 a game-changer, or is it too early to tell?
Evaxion's leadership is optimistic. Birgitte Rønø, CSO and interim CEO, stated, 'A 75% ORR is unprecedented in this hard-to-treat patient population and underscores the significant potential of EVX-01 as a new treatment option.' Professor Muhammad Adnan Khattak, a key opinion leader and trial investigator, added, 'This study sets a new benchmark in personalized immunotherapy... Evaxion’s AI-Immunology™ approach is not just innovative; it’s clinically meaningful and ready to shape the future of oncology.'
The data were presented at the European Society for Medical Oncology (ESMO) Congress 2025 in Berlin and will be further discussed in a webinar on October 22, 2025. These platforms provide opportunities for stakeholders to engage with the findings and for Evaxion to address lingering questions. The company is also planning to publish full datasets, including PFS and OS data, and is exploring partnerships for larger, controlled studies to confirm these initial findings.
So, what's next for EVX-01 and personalized cancer vaccines? The road ahead is both exciting and challenging. If further studies validate these results, EVX-01 could become a cornerstone of cancer treatment, offering hope to patients with limited options. But the journey from promising trial data to widespread clinical use is fraught with regulatory, financial, and logistical hurdles. Evaxion's ability to navigate these challenges will determine whether EVX-01 fulfills its potential.
Thought-provoking question for our readers: Given the promising but preliminary nature of these results, should regulatory bodies fast-track approval for personalized cancer vaccines like EVX-01, or should they demand larger, more definitive trials? Share your thoughts in the comments below—we'd love to hear your perspective!
