Therapeutic areas
As one of the world’s most critical medical challenges, cancer is one of our primary focus areas
Cancer remains the second-leading cause of death in the industrialized world, and incidence rates are growing. The cause of cancer is manyfold, and genetics, environment and lifestyle factors play a role in the evolution of cancer in different parts of the world. Even though there have been important breakthroughs in recent decades, there is still a high unmet need in the treatment of cancer.
Cancer immunotherapy has quickly become one of the key treatment opportunities against several cancer types. The therapies available today, checkpoint inhibitors being at the forefront, benefit only 20-30% of cancer patients with durable responses, and some cancer types do not respond at all. The need for additional and novel approaches addressing the untapped potential of activating the immune system, such as Nykode Therapeutics’ cancer vaccines, is still valid. Combining insights into genetic alterations and environmental exposures and activation of the immune system will continue to be an important part of cancer therapy evolution for years to come. Individualized cancer therapy, with treatment approaches tailored to each patient, is expected to be increasingly important in the fight against cancer. Combining individualized approaches with activation of the immune system is an attractive and increasingly emerging approach. Therapeutic cancer vaccines, with their ability to specifically activate the immune system, in particular CD8 killer T cells, and target specific cancer antigens, is one such approach.
HPV-driven cancers (abi-suva)
Nykode’s wholly owned lead product candidate, abi-suva (formerly VB10.16), is a DNA-based therapeutic vaccine targeting cancers caused by Human Papillomavirus 16 (HPV16). HPV is the cause of 630,000 cancer cases annually. There are several types of high-risk HPV-causing cancers, with HPV16 being the most common. HPV-induced oropharyngeal cancer, a type of head and neck (H&N) cancer, is rapidly growing among both women and men in the Western world, particularly in Northern Europe and North America. Immune checkpoint inhibitors are an important part of the clinical development landscape in HPV-driven tumors, but despite the advances seen in the treatment of HPV-driven cancers, there is still an unmet medical need.
Abi-suva’s clinical program spans four trials. VB-C-01, a first-in-human study in 34 women with high-grade cervical intraepithelial neoplasia, showed abi-suva monotherapy was well tolerated with early signs of efficacy. VB-C-02, a Phase 2 trial in 52 women with advanced or recurrent cervical cancer, showed abi-suva combined with atezolizumab was well tolerated with higher response rates and longer survival than historical standard-of-care controls, most pronounced in PD-L1-positive patients on their first line of therapy. VB-C-03, a Phase 1/2a trial combining abi-suva with pembrolizumab in HPV16-positive, PD-L1-positive recurrent/metastatic HNSCC, showed encouraging activity with a 38.5% objective response rate, supporting continued development in the first-line HNSCC setting.
Building on these results, Nykode has initiated Abili-T, a randomized Phase 2 trial evaluating abi-suva plus pembrolizumab (KEYTRUDA®) versus pembrolizumab alone as first-line treatment for HPV16-positive, PD-L1-positive recurrent/metastatic HNSCC. The trial is currently enrolling, with a first interim efficacy analysis expected in 2027.
Individualized cancer therapy (VB10.NEO)
Every patient’s tumor is unique, and to effectively address this challenge, the principle of individualized treatments is emerging quickly as an important part of future cancer therapy options. By focusing on individual characteristics and mutational alterations in each patient’s tumor, therapies can be uniquely tailored and customized.
VB10.NEO is a wholly owned, DNA-based individualized neoantigen therapy (INT) built on Nykode’s proprietary antigen-presenting cell (APC)-targeting technology and NeoSELECT™, an AI-driven neoantigen selection engine.
Nykode has established a clinically validated, end-to-end manufacturing process for VB10.NEO, achieving a 100% manufacturing success rate across two clinical trials. Recent improvements have meaningfully reduced manufacturing time, with a current process of under six weeks from biopsy to release, positioning Nykode as one of the fastest and most robust INT developers on the market. A future in-house manufacturing process is expected to create a scalable, time- and cost-competitive supply chain, where Nykode’s simpler plasmid DNA process offers an inherent cost-of-goods and needle-to-needle time advantage over alternative technologies such as mRNA-LNP.
NeoSELECT™ has demonstrated a significant correlation between highly ranked neoantigens and their actual immunogenicity in patients, and the platform was recently strengthened by a new U.S. patent extending protection through 2039.
Across two clinical trials in advanced solid tumors, VB10.NEO was safe and well tolerated, with no serious vaccine-related adverse events. Vaccine-specific immune responses were observed in 88–100% of patients, including de novo T cell responses in 85% of patients, with responses remaining durable for more than one year after the final dose.
Nykode is actively exploring partnerships to advance VB10.NEO across a broad range of tumor types.
PIONEERING THE BATTLE AGAINST AUTOIMMUNE DISEASES THROUGH INNOVATIVE THERAPEUTIC STRATEGIES
The complex world of autoimmune diseases
Autoimmune diseases, where the immune system mistakenly attacks the body’s own cells, present a significant and growing health challenge. These conditions, ranging from rheumatoid arthritis to multiple sclerosis, affect millions worldwide, manifesting in various forms and intensities. The complexity of these diseases lies in their diverse etiologies, involving genetic, environmental, and lifestyle factors. Despite advancements in understanding the immune system, there remains a substantial unmet need for effective, targeted treatments for many autoimmune conditions. Current therapies rely primarily on broadly immunosuppressive agents that unavoidably enhance the risk of adverse effects comprising infection and cancer. The goal of future autoimmune disease therapy is to restore tolerance to the self-antigen(s) that causes the pathology by targeting autoreactive T cells while preserving immune competence to prevent infections and malignancies.
Nykode’s approach to autoimmune diseases
Our approach to treating autoimmune diseases is grounded in a thorough comprehension of the immune system, augmented by the capabilities of our technology platform to precisely target antigens to antigen-presenting cells. Specifically, in the context of autoimmune disorders, we focus on directing these antigens to specialized antigen-presenting cells that are adept at eliciting antigen-specific immune suppressive T cell responses. This strategy is opposite of the pro-inflammatory responses triggered by conventional vaccines. As a result, vaccines developed for autoimmune conditions are often referred to as “inverse” vaccines. Additionally, our versatile DNA platform allows for the incorporation of DNA-encoded immune suppressive cytokines. This integration further conditions the antigen-presenting cells, thereby enhancing the immune suppressive response and offering a more targeted therapeutic approach.
