Six AI-driven Drug Discovery Companies Enabling Precision Oncology

Artificial Intelligence (AI) is steadily making its presence felt across various sectors, including the pharmaceutical industry. Among the multiple applications of AI in this field, immunotherapy - a treatment method that utilizes the body's immune system to combat diseases, notably cancer - is seeing a significant influence. The integration of AI can potentially enhance treatments and patient care by making it more precise and personalized.

Making sense of complex biological systems

The human immune system is a densely interconnected network involving numerous immune cells, signaling molecules, and genes. The interactions within this network are intricate, dynamic, and largely unpredictable. The vast amount of biological data often surpasses the comprehension ability of humans and the scope of traditional statistical methods.

This is where AI, with its capacity to understand and predict from data, comes into play. Deep learning algorithms can handle enormous amounts of genetic, proteomic, and clinical data, discern patterns and correlations, and contribute to the identification of new immunotherapeutic targets.

What is immunotherapy?

Immunotherapy refers to a set of medical treatments that use the body's natural defenses – the immune system – to identify, fight, and eliminate diseases, particularly cancer. Instead of directly attacking the disease, immunotherapies equip the immune system to recognize disease cells and stimulate an immune response against them. There are several types of immunotherapies, each with a different mechanism of action - checkpoint inhibitors, Chimeric Antigen Receptor T-cell (CAR-T cel)l therapy, cancer vaccines, monoclonal antibodies, Immune system modulators.

Enhancing personalized immunotherapies

Personalized immunotherapies, such as CAR-T cell therapy, have shown promise in cancer treatment. In this therapy, T cells are removed from a patient's body, genetically modified to produce receptors (CARs) that target specific proteins on cancer cells, and then infused back into the patient.

However, predicting which patients will respond favorably to these therapies has been a persistent challenge. AI algorithms can process data from various sources, including genomics, clinical trials, and real-world evidence. This helps to discover biomarkers and predict patients' responses to treatment. Thus, AI can assist in selecting patients who are likely to benefit most from the therapy, leading to improved treatment outcomes and cost-effectiveness.

Refining Combination Therapies

Immunotherapies often yield better results when combined with other treatments. Yet, determining the optimal combination can be a time-consuming process. AI can aid in identifying effective drug combinations, potentially speeding up the process and the development of more efficient treatment strategies.

Improving Clinical Trials 

Clinical trials play a pivotal role in the development of new immunotherapies. However, they can be expensive and lengthy. AI can potentially expedite the clinical trial process, right from patient selection to data collection and analysis. Predictive models powered by AI can identify suitable patients more accurately, reduce drop-out rates, and enable real-time data analysis.

Below is an alphabetically ordered list of 5 notable AI-driven drug discovery companies pushing the boundaries of what can be done to treat cancer:  

Achilles Therapeutics

Achilles Therapeutics (NASDAQ: ACHL) is a London-based biopharmaceutical company that focuses on the development of next-generation, patient-specific therapies to treat cancer. Achilles Therapeutics utilizes its proprietary platform PELEUS, designed to predict neoantigens' immunogenicity, presented in 2023. The PELEUS platform is an integral part of Achilles' “Target-to-T Cell” approach that aims to revolutionize precision therapies, including TIL-based clonal neoantigen-reactive T cells (cNeT) and personalized neoantigen methods. The platform's predictive capabilities have the potential to transform precision treatments by focusing on the most potent antigens for robust, long-lasting responses. PELEUS's predictive success is attributed to its training on high-quality proprietary data sets. Furthermore, the platform's superiority is patent-protected, further enhancing its value and potential for Achilles Therapeutics. 

A neoantigen is a type of antigen, a substance that stimulates an immune response, that is formed by a novel protein sequence generated through mutations in tumor cells. These mutations lead to changes in the protein sequences that are not present in normal cells. Because they are unique to cancer cells, they can act as ideal targets for immune-based cancer therapies.

However, not all neoantigens are equal; some are more likely to generate a strong immune response, known as being more "immunogenic". A large number of potential neoantigens can be identified for a tumor sample, but the challenge is to determine which of these are most likely to elicit a potent immune response, thus being effective for personalized therapy.

PELEUS platform uses machine learning algorithms trained on extensive proprietary datasets to predict which identified neoantigens are most likely to be immunogenic. By predicting the most potent neoantigens, the platform supports the development of personalized cancer therapies, including TIL-based (Tumor-Infiltrating Lymphocytes) therapies and clonal neoantigen cancer vaccines. It's a critical tool for implementing precision medicine in cancer treatment, making therapy more efficient and potentially more effective.

The company's innovative work extends to various clinical trials, including the ongoing CHIRON and THETIS trials, investigating advanced non-small cell lung cancer and recurrent or metastatic melanoma respectively. First patients already have dosed in both trials with enhanced host conditioning regimens. An interim update in April 2024 highlighted that, while no new objective responses were observed, enhanced host conditioning has shown promise in improving cNeT engraftment levels and persistence.

In May 2024, Achilles Therapeutics announced a research collaboration with Arcturus Therapeutics to explore second-generation personalized mRNA cancer vaccines. This collaboration aims to combine Achilles’ expertise in T-cell therapies with Arcturus’ mRNA technology to develop innovative cancer treatments.

ImmuneAI

Immunai is a New-York-based artificial intelligence startup specializing in cutting-edge multiomics technology. They focus on enhancing the effectiveness of cell-based therapies and other immune-altering treatments, not just in oncology but also in a broad spectrum of inflammatory diseases. Their technology allows for the precise measurement of gene expression changes in individual cells, a capability that has broad-ranging applications in medicine and therapy design.

Recently, Immunai started a partnership with Baylor College of Medicine (BCM) to uncover the role of a new molecular target, BTG1. This finding, drawn from clinical samples of an ongoing BCM trial, promises to enhance the efficacy of T and natural killer T (NKT) cell-based cancer immunotherapies.

Their advanced single-cell RNA sequencing technology played a critical role in this discovery, demonstrating the potential of Immunai's platform to drive novel treatment options. With the revelation that targeting BTG1 could amplify CAR-NKT cells' anti-tumor function, Immunai has paved the way for the development of more effective cancer immunotherapies.

In 2021 ImmuneAI secured an impressive $215 million in a Series B funding round led by Koch Disruptive Technologies, taking its total funding to an impressive $295 million.

iBio

iBio, Inc. is an innovator in the field of biotechnology, specializing in the application of artificial intelligence to develop precision antibody immunotherapies. Based in Bryan, Texas, the company utilizes a combination of its proprietary AI-based antibody optimization and mammalian display technologies to discover and develop novel therapeutic antibodies. 

The latest news from iBio is the discovery of a panel of CD3 T-cell binding antibodies. These novel antibodies are designed to bind to both T cells and tumor cells, inducing the T cells to kill the tumor cells. Earlier research into CD3-based T-cell engagers had shown promise, but was hindered by high toxicity levels and lack of cross-reactivity with non-human primates, slowing down their clinical development.

iBio has addressed these challenges using its patented epitope steering technology, directing antibodies towards specific CD3 epitopes. By combining AI-based antibody optimization with mammalian display technology, the company has widened the range of CD3 affinities, identified antibodies with cross-reactivity to non-human primates, and improved the humanness of the antibody sequences.

iBio, Inc. first went public through an initial public offering (IPO) in 2008.

In March 2024, iBio entered a transformative collaboration with AstralBio to rapidly develop novel antibodies for obesity and cardiometabolic diseases using AI-driven drug discovery techniques

On June 3, 2024, iBio announced the sale of its manufacturing facility in Texas. This move eliminated $13.2 million of secured debt and marked a strategic shift towards focusing on AI-driven precision biologics.

Evaxion Biotech

Evaxion Biotech (NASDAQ: EVAX) is a public clinical-stage biotechnology company, headquartered in Copenhagen, Denmark, that specializes in developing AI-powered immunotherapies for the treatment of cancer, bacterial diseases, and viral infections. Evaxion utilizes the AI-Immunology™ platform, an advanced AI technology, to transform vaccine discovery and immunotherapy for infectious diseases and cancers through sophisticated computational modeling and personalized medical approaches.

AI-Immunology™ platform, specifically its precision oncology component, leverages the PIONEER™ and ObsERV™ systems. PIONEER™ accurately identifies and targets neoantigens, which are unique to cancer cells, using advanced AI models to predict T-cell responses and optimize immunotherapy. ObsERV™, developed in 2023, complements this by identifying endogenous retroviruses (ERVs) as additional cancer antigens, enhancing the overall efficacy of personalized cancer treatments. This combined approach allows for the development of highly personalized and effective cancer immunotherapies with minimal adverse effects on healthy cells.

Evaxion Biotech's lead cancer vaccine candidate, EVX-01, is showing promising progress in its Phase 2 clinical trials for metastatic melanoma. Recent data presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2024 demonstrated that EVX-01, in combination with the anti-PD1 therapy pembrolizumab (KEYTRUDA®), elicited specific and targeted immune responses in melanoma patients. These responses were characterized by the activation of both CD4+ and CD8+ T-cells, with a significant correlation between the quality of neoantigens predicted by Evaxion's AI-Immunology™ platform and the vaccine-induced immune responses. The vaccine was also found to be well-tolerated with only mild adverse events reported.

EVX-02, DNA-based personalized cancer immunotherapy has also shown impressive results in its Phase 1/2a study. EVX-02 in combination with the checkpoint inhibitor nivolumab, was found to be well-tolerated and all participating patients who completed the treatment were relapse-free at their last assessment.

These results have given Evaxion the confidence to fast-track EVX-03, its next-generation DNA-based personalized cancer immunotherapy, towards clinical trials. The company aims to transform these technological advancements into effective patient care.

Evaxion entered a collaboration with an MSD in the autumn of 2023, which remained undisclosed until winter 2024 when the initial phases of the collaboration were successful.

Lantern Pharma

Lantern Pharma Inc., an innovative biopharmaceutical company, based in Dallas, USA, that is focused on transforming oncology drug discovery and development, stands out with its proprietary RADR® artificial intelligence and machine learning platform. Using over 100 billion oncology-focused data points and a collection of advanced ML algorithms, Lantern aims to solve real-world, billion-dollar challenges in the oncology field. With a number of clinical programs, Lantern is pushing boundaries in the race to find effective cancer therapies. The company has established a wholly-owned subsidiary, Starlight Therapeutics Inc., dedicated to the clinical execution of promising treatments for CNS and brain cancers.

Read also: Unveiling Lantern Pharma's Success Story in AI-powered Precision Oncology

LP-300 is a notable investigational therapeutic targeting relapsed non-small cell lung cancer (NSCLC) in combination with standard chemotherapy. The Phase 2 Harmonic™ trial for LP-300 has expanded internationally, with trial sites now active in Japan and Taiwan, enhancing the pace of patient enrollment and data collection. Another significant candidate, LP-284, is in a Phase 1 clinical trial focusing on relapsed or refractory lymphomas and certain solid tumors. This trial aims to determine the drug's safety, optimal dosing, and initial efficacy in a patient population with limited treatment options. Additionally, Starlight Therapeutics, a wholly-owned subsidiary of Lantern Pharma, is advancing STAR-001 (LP-184) through a Phase 1B trial for central nervous system cancers, including glioblastomas, aiming to address a critical unmet need in brain cancer treatment. These drug candidates exemplify Lantern Pharma's innovative approach in leveraging AI and machine learning through its RADR® platform to accelerate the development of precision oncology therapies.

Announced in 2023 an important collaboration with Bielefeld University is set to develop next-generation antibody-drug conjugates (ADCs). These ADCs, while not classified as immunotherapy, represent a significant advancement in targeted cancer therapy. The focus will initially be on synthesizing and evaluating novel ADCs linked to cryptophycins, highly potent antitumor molecules. These cryptophycin-ADCs will be tested across multiple cancer cell lines.

Recently, Lantern Pharma entered into a strategic AI collaboration with Oregon Therapeutics to develop a protein disulfide isomerase (PDI) inhibitor for novel cancer indications, leveraging RADR® to uncover biomarkers and define combination therapies.

In summary, Lantern Pharma is not just at the cutting edge of oncology research and treatment development; it's shaping the future with its forward-thinking AI-driven approach and significant discoveries in ADC development.

Lantern Pharma has successfully secured $95M in investment across seven funding rounds. Their most recent capital influx occurred on January 14, 2021, stemming from a Post-IPO secondary financing round.

Predictive Oncology

Predictive Oncology Inc. is a company focused on AI-driven drug discovery and development, specifically in the field of oncology. The company utilizes a proprietary artificial intelligence and machine learning platform that has been scientifically validated to predict with 92% accuracy if a tumor sample will respond to a specific drug compound. Predictive Oncology maintains a biobank with over 150,000 cryogenically preserved human tumor samples, covering 137 different tumor types. This biobank allows the company to account for patient heterogeneity in the early stages of drug development, which is critical for increasing the Probability of Technical Success (PTS) in clinical trials.

The company's platform integrates genomics, digitized pathology data, and phenotype profiling to analyze heterogenous responses across different drug treatments. These capabilities enable more precise drug-tumor pairings for subsequent in-vitro testing, and support the discovery of new biomarkers, targets, and potential drugs. Predictive Oncology's platform facilitates critical go/no-go decisions in drug development, helping pharmaceutical companies optimize clinical trial design and accelerate development timelines. The company operates a CLIA-certified lab and GMP facilities to support its operations. Predictive Oncology is headquartered in Pittsburgh, PA, and is dedicated to improving the success rates of oncology drug development.

In summary, artificial intelligence offers a set of tools that can help in understanding complex biological systems, refining personalized treatments, enhancing combination therapies, and improving clinical trials. As this field continues to mature, collaborations between different sectors - academia, industry, healthcare providers, and policy makers - will be vital to responsibly navigate potential challenges and make a substantial difference in patients' lives.

AI's integration into immunotherapy marks a significant shift in pharmaceutical research and treatment. As we delve deeper into this intersection, it is apparent that it holds numerous possibilities that could shape the future of medical treatments.

Topics: AI & Digital