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Anal cancer is a relatively rare malignancy arising from the tissues of the anal canal, most commonly associated with persistent human papillomavirus (HPV) infection and immunosuppression. According to the American Cancer Society, the estimated burden of anal cancer in the United States for 2025 includes approximately 10,930 new cases, comprising 3,560 cases in men and 7,370 cases in women. Ongoing anal cancer pipeline analysis by Expert Market Research indicates increasing research focus on immune checkpoint inhibitors, combination chemoradiotherapy, and targeted treatment approaches, alongside preventive strategies such as HPV vaccination and enhanced screening for high-risk populations. Growing disease awareness, rising HPV prevalence, and advances in immuno-oncology are expected to support continued pipeline expansion and therapeutic innovation in the coming years.
Major companies involved in the anal cancer pipeline analysis include Novartis Pharmaceuticals and Frantz Viral Therapeutics, LLC, and others.
Leading drugs currently in the pipeline include AI-061, Carboplatin and others.
The anal cancer market growth is driven by rising HPV infection rates, increasing screening awareness, and expanding adoption of targeted and immunotherapy-based treatment options.
The Anal Cancer Pipeline Analysis Report by Expert Market Research gives comprehensive insights into anal cancer therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Anal Cancer. The anal cancer report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The anal cancer pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with anal cancer treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to anal cancer.

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Anal cancer is a relatively rare malignancy of the anal canal, most commonly driven by persistent human papillomavirus (HPV) infection, chronic inflammation, and immune dysfunction. The disease burden has been steadily rising, particularly among women and immunocompromised populations, underscoring the need for effective systemic therapies beyond conventional chemoradiation.
Anal cancer treatment has increasingly shifted toward immunotherapy-based approaches for advanced and metastatic disease. Nivolumab (Opdivo®), a PD-1 immune checkpoint inhibitor approved by the U.S. Food and Drug Administration in December 2014, is clinically utilized in advanced anal squamous cell carcinoma based on guideline-supported use, where it restores anti-tumor immune responses by blocking PD-1–mediated immune suppression. Continued clinical development and expanding immuno-oncology research are expected to further strengthen the therapeutic landscape for anal cancer in the coming years.
The anal cancer pipeline continues to evolve as epidemiological insights increasingly inform research priorities and regulatory strategies. In the United States, the American Cancer Society projected approximately 10,930 new anal cancer cases in 2025, with an estimated 85,736 individuals living with the disease in 2022, underscoring a growing patient population despite its rarity, representing about 0.5% of all new cancer cases based on SEER data.
In the United Kingdom, anal cancer accounts for less than 1% of all new cancers, with around 1,530 new cases diagnosed annually, including approximately 1,000 cases in women and 530 in men, as reported by Cancer Research UK. Similarly, in India, GLOBOCAN 2020 estimates indicate approximately 5,452 new cases, reflecting a relatively low but gradually increasing incidence. Collectively, these epidemiological trends highlight a persistent unmet need and support continued investment in immunotherapy-driven and targeted treatment development for anal cancer.
This section of the report covers the analysis of anal cancer drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
By Drug Class
The anal cancer pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration:
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II, with 65%, covers a major share of the total anal cancer clinical trials. It is followed by phase I at 35% and other phases. Overall, this phase-wise distribution highlights a strong focus on mid-stage clinical development, indicating sustained research momentum and a robust pipeline progression toward advanced therapeutic validation in anal cancer.
The drug molecule categories covered under the anal cancer pipeline analysis include small molecules, monoclonal antibodies, gene therapies, and peptides. The anal cancer report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for anal cancer. For example, nivolumab, a monoclonal antibody, is a PD-1 immune checkpoint inhibitor that is actively utilized in advanced anal squamous cell carcinoma, highlighting the growing prominence of antibody-based immunotherapies within the anal cancer pipeline.
The EMR report for the anal cancer pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed anal cancer therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in anal cancer clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for anal cancer. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of anal cancer drug candidates.
Carboplatin, paclitaxel, and nivolumab are being evaluated in the Phase 3 EA2176 clinical trial, sponsored by the National Cancer Institute (NCI), for patients with metastatic, recurrent, or unresectable anal squamous cell carcinoma. In this interventional study, carboplatin and paclitaxel act as cytotoxic chemotherapeutic agents, with carboplatin inducing DNA crosslinking to inhibit tumor cell replication and paclitaxel stabilizing microtubules to disrupt mitosis and promote cancer cell death. Nivolumab, an immune checkpoint inhibitor, works by binding to and blocking the PD-1 protein, thereby preventing cancer cells from suppressing the immune system and enabling T-cells to recognize and attack tumour cells more effectively. The study is evaluating whether the addition of nivolumab to standard chemotherapy improves clinical outcomes while maintaining an acceptable safety profile. The trial is currently active but not recruiting, with an estimated completion in March 2027.
Artesunate suppositories are being evaluated in the Phase 2 interventional trial sponsored by Frantz Viral Therapeutics, LLC, for the treatment of HIV-negative patients with intra-anal high-grade squamous intraepithelial lesions (HSIL), a precancerous HPV-associated condition that can progress to anal cancer. Artesunate, a derivative of the antimalarial compound artemisinin, has demonstrated selective cytotoxicity against HPV-infected epithelial cells by exploiting their higher intracellular iron levels, which react with artesunate’s endoperoxide bridge to generate reactive oxygen species (ROS) that induce oxidative stress and cell death preferentially in dysplastic cells while sparing normal tissue. In vitro and early clinical studies have shown that artesunate can reduce HSIL lesion burden and may promote regression by triggering apoptosis and immune-mediated responses following localized cell damage. The current trial is comparing artesunate suppositories with placebo to assess histologic regression, HPV clearance, safety, and tolerability in intra-anal HSIL. The study is active but not recruiting, with estimated completion in December 2026.
AI-061 (ONC-392) is being evaluated in a Phase 1 interventional clinical trial sponsored by OncoC4, Inc. for patients with advanced solid tumors, including anal cancer. AI-061 is a next-generation anti-CTLA-4 monoclonal antibody engineered to selectively enhance anti-tumor immune responses while reducing systemic immune-related toxicity. Unlike traditional CTLA-4 inhibitors, AI-061 preserves CTLA-4’s physiological immune checkpoint function in peripheral tissues while promoting preferential depletion of immunosuppressive regulatory T cells within the tumor microenvironment. This tumor-selective immune modulation restores effective T-cell–mediated tumor killing and supports durable anti-cancer activity with an improved safety profile. The study is currently active but not recruiting, with primary completion expected in December 2025 and overall completion anticipated by June 2026.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Anal Cancer Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Anal Cancer. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into anal cancer collaborations, regulatory environments, and potential growth opportunities.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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