Balstilimab for Oropharyngeal Cancer

Overseen By Luana Guimaraes de Sousa, MD

Age: 18+

Sex: Female

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Must not be taking: Steroids, Glucocorticoids

Disqualifiers: Uncontrolled hypertension, Active infections, others

Stay on Your Current Meds You can continue your current medications while participating

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Balstilimab for individuals with HPV-linked oropharyngeal cancer. The researchers aim to determine if Balstilimab can clear any remaining virus after the main cancer treatment and prevent the cancer from returning. Suitable participants have undergone treatment for oropharyngeal cancer but still show signs of the virus without visible cancer. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to important findings.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that Balstilimab is likely to be safe for humans?

Research has shown that balstilimab has been tested with another drug, botensilimab, in several studies. Safety data indicates that 58% of patients experienced immune-related side effects, most of which were mild and manageable.

In these studies, the most common side effect was a reaction to the treatment itself, though specific percentages for each side effect were not provided. As balstilimab remains under investigation, researchers have gathered some evidence of its safety for humans, but they continue to explore all its safety aspects.

Overall, despite the presence of side effects, the treatment appears to be generally well-tolerated. Monitoring for any adverse events remains crucial as more people receive the treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for oropharyngeal cancer, which often include surgery, radiation, and chemotherapy, Balstilimab offers a fresh approach by targeting the immune system. Balstilimab is a type of immunotherapy known as a PD-1 inhibitor, which works by blocking a protein that prevents the immune system from attacking cancer cells. Researchers are excited because this mechanism might enable the body to better recognize and fight cancer, potentially leading to more effective and longer-lasting results with fewer side effects compared to traditional therapies.

What evidence suggests that Balstilimab might be an effective treatment for oropharyngeal cancer?

Research has shown that balstilimab may help treat various solid tumors. In earlier studies, patients who received balstilimab lived for a median of 17.2 months, with about 39% still alive two years after starting treatment. These results suggest that balstilimab might improve survival rates. In this trial, participants will receive balstilimab, which strengthens the immune system to better fight cancer cells—an important mechanism for cancers like oropharyngeal cancer linked to HPV. These findings highlight balstilimab's potential to help the body more effectively manage and clear cancer. ^{5 6 7 8 9}

Who Is on the Research Team?

Luana Guimaraes de Sousa, MD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for adults over 18 with HPV+ oropharyngeal cancer confirmed by biopsy, who've completed treatment but still have detectable HPV. They must be in good health otherwise, with proper organ function and non-smokers or light smokers. Women of childbearing age must not be pregnant, breastfeeding, and agree to use contraception.

Inclusion Criteria

I still have HPV DNA in my blood after treatment, but no visible cancer.

My cancer is HPV positive or I've smoked less than 10 pack-years if HPV status is unknown.

I am a man who can father children and will use birth control during the study.

Exclusion Criteria

I am on systemic treatment for an autoimmune or rheumatological condition.

I have never been treated with anti-PD-1 or anti-PD-L1 therapies.

Judgment by the investigator that the patient should not participate in the study if the patient is unlikely to comply with study procedures, restrictions and requirement

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Definitive Therapy

Participants complete definitive therapy for HPV+ OPC

Not specified

Adjuvant Immune Therapy

Participants receive Balstilimab to evaluate viral clearance

Every 2 weeks

Visits on Days 1 and 15 of each cycle

Follow-up

Participants are monitored for safety, viral clearance, and long-term disease control

2 years

What Are the Treatments Tested in This Trial?

Interventions

• Balstilimab

Trial Overview The study tests Balstilimab's ability to clear persistent HPV infection after standard cancer treatment in patients without visible disease. It aims to understand if clearing the virus can help control the cancer long-term.

How Is the Trial Designed?

Group I: Balstilimab Experimental Treatment 1 Intervention

Participants will receive Balstilimab by vein over about 30 minutes on Days 1 and 15 of each cycle (every 2 weeks)

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Agenus Inc.

Industry Sponsor

Trials

58

Recruited

4,900+

Citations

1. investor.agenusbio.com investor.agenusbio.com/news/news-details/2025/Agenus-Reports-39-of-Patients-Alive-at-Two-Years-with-BOTBAL-Across-Multiple-Refractory-Solid-Tumors-at-ESMO-2025/default.aspx

Agenus Reports 39% of Patients Alive at Two-Years with ... Agenus Reports 39% of Patients Alive at Two-Years with BOT/BAL Across Multiple Refractory Solid Tumors at ESMO 2025 · Cervical cancer: Results ...

2. targetedonc.com targetedonc.com/view/botensilimab-and-balstilimab-prove-notable-os-in-patients-with-advanced-solid-tumors

Botensilimab and Balstilimab Prove Notable OS in Patients ... Botensilimab and balstilimab showed promising survival outcomes in advanced solid tumors, with a median OS of 17.2 months and 39% 24-month ...

3. investor.agenusbio.com investor.agenusbio.com/news/news-details/2025/Agenus-BOTBAL-Neoadjuvant-Pan-Cancer-Data-from-the-NEOASIS-Study-Presented-in-an-Oral-Session-at-AACR/default.aspx

Agenus' BOT/BAL Neoadjuvant Pan-Cancer Data from the ... "These initial results from the NEOASIS study indicate that botensilimab and balstilimab can induce pathological responses in patients with a ...

4. onclive.com onclive.com/view/dr-schlechter-on-updated-data-for-botensilimab-plus-balstilimab-in-mss-mcrc

Dr Schlechter on Updated Data for Botensilimab Plus ... Median progression-free survival (PFS) was 4.0 months (95% CI, 2.8-4.1), and the median overall survival (OS) reached 20.9 months (95% CI, 16.2- ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11974637/

Botensilimab (Fc-enhanced anti–cytotoxic lymphocyte ... In the metastatic setting, overall survival (OS) with treatment remains poor, with 5-year survival rates approximately 16%, thus novel treatments are urgently ...

6. onclive.com onclive.com/view/dr-schlechter-on-the-safety-of-botensilimab-plus-balstilimab-in-pretreated-mss-crc

Dr Schlechter on the Safety of Botensilimab Plus ... Additional safety data from C-800-01 revealed that any-grade imAEs occurred in 58% of patients in the overall population; 29% of these were ...

7. investor.agenusbio.com investor.agenusbio.com/news/news-details/2025/Agenus-to-Highlight-Emerging-Survival-Plateaus-with-BotensilimabBalstilimab-in-Oral-Presentation-of-Study-in-Refractory-Patients-Across-Five-Tumor-Types-at-ESMO-2025/default.aspx

Press Release Details The highlight is an oral presentation that will feature emerging survival plateaus from a study of botensilimab plus balstilimab in 343 ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT02694822

AGEN1884, an Anti-CTLA-4 Human Monoclonal Antibody ... This is an open-label, Phase 1/2, multicenter study to evaluate the safety, pharmacokinetics, and pharmacodynamics of an anti-cytotoxic T ...

9. agenusbio.com agenusbio.com/wp-content/uploads/2023/11/13Oct2023_Wilky_Bot-Bal-Sarcoma-Oral_CTOS-2023_FINAL.pdf

efficacy and safety of botensilimab plus balstilimab *Nine additional patients were treated as of 06 April 2023 but had not received the first 6-week tumor assessment (ITT/Safety population=50). Data cutoff: 17- ...