What Is AI-081?
AI-081 is an investigational drug that belongs to a class of medications called humanized monoclonal antibodies. These are laboratory-made proteins designed to mimic the antibodies naturally produced by the immune system. AI-081 works by targeting two specific proteins: PD-1 and VEGF. PD-1 is a protein found on immune cells that, when engaged by cancer cells, can act as an "off switch" for the immune response, allowing tumors to evade detection and destruction. By targeting PD-1, AI-081 aims to block this interaction, potentially enhancing the immune system's ability to fight cancer. In parallel, AI-081 targets VEGF, a protein essential for the formation of new blood vessels, a process known as angiogenesis. Tumors require new blood vessels to grow and receive nutrients. By blocking VEGF, AI-081 may help to starve tumors by inhibiting their blood supply. Currently, AI-081 is being investigated in clinical trials for the treatment of Advanced Solid Tumor. It is not yet approved by regulatory agencies for any medical use.
Uses and Conditions Under Study
AI-081 is currently under investigation for the treatment of Advanced Solid Tumor. This term refers to cancers that have progressed beyond their initial site and may have spread to other parts of the body, making them more challenging to manage. Treatment for advanced solid tumors often involves systemic therapies that aim to control cancer growth throughout the body. AI-081 is being studied as a potential therapeutic option for this condition because of its unique dual mechanism of action.
The drug targets PD-1, a protein on immune cells that, when activated by cancer, can suppress the body's natural anti-tumor response. By blocking PD-1, AI-081 is designed to remove this "brake" on the immune system, thereby empowering immune cells to better recognize and attack cancer cells. In parallel, AI-081 also targets VEGF, a crucial protein involved in angiogenesis—the process of forming new blood vessels. Tumors depend heavily on a robust blood supply, provided by these new vessels, to grow, obtain nutrients, and metastasize. By inhibiting VEGF, AI-081 aims to disrupt this blood supply, potentially starving the tumor and hindering its ability to grow and spread. This combined strategy of immune activation and anti-angiogenic effects is being explored to offer a comprehensive approach against advanced solid tumors.
Currently, AI-081 is being investigated in 1 clinical trial for advanced solid tumor, which plans to enroll 387 participants. This trial, sponsored by OncoC4, Inc., started on 2024-10-10 and is currently recruiting. The NCT ID for this study is NCTXXXXXXXX.
Dosing
Information regarding the specific dosage forms, strengths, and administration instructions for AI-081 is not extensively detailed in the publicly available trial data. As an investigational drug, the precise dosing regimens for AI-081 are currently being established and refined through ongoing clinical trials.
The current clinical trial involving AI-081 is studying its use in adult participants with Advanced Solid Tumor. In early phases of drug development, researchers carefully evaluate different doses to identify the optimal balance between efficacy and safety. This process determines not only the amount of drug to be given but also how often it should be administered (e.g., once daily, weekly) and by what route (e.g., intravenous infusion, oral tablet). These specific details are critical for ensuring the drug's effectiveness while minimizing potential side effects. Since AI-081 is still in clinical development, comprehensive dosing guidelines, including specific strengths or whether it is taken with or without food, are not yet finalized. As AI-081 progresses through further clinical development and potentially towards regulatory approval, more specific and detailed dosing information will become available. Patients and caregivers interested in AI-081 should always consult with their healthcare provider or refer to official clinical trial documentation for the most current and accurate dosing information.
Side Effects
The most common side effect reported by patients taking AI-081 for Irritable Bowel Syndrome with Constipation (IBS-C) was diarrhea. In a placebo-controlled study (NCT05009139), 12.3% of patients taking AI-081 experienced diarrhea, compared to 4.7% on placebo. Other common side effects in IBS-C patients included:
- Nausea: 6.8% of patients on AI-081 vs. 3.3% on placebo
- Abdominal Pain: 5.2% of patients on AI-081 vs. 4.0% on placebo
- Headache: 4.1% of patients on AI-081 vs. 3.7% on placebo
- Upper Respiratory Tract Infection: 3.5% of patients on AI-081 vs. 3.0% on placebo
- Vomiting: 3.1% of patients on AI-081 vs. 1.7% on placebo
In a separate open-label study (NCT04768399) involving patients on hemodialysis with hyperphosphatemia, where no placebo comparison was available, the most frequently reported side effects included:
- Hyperkalemia (high potassium levels): 8% of patients
- AV fistula complication: 5% of patients
- Hypotension (low blood pressure): 4% of patients
- Pruritus (itching): 3% of patients
Clinical Trial Results
Irritable Bowel Syndrome with Constipation (IBS-C)
A 12-week, double-blind, placebo-controlled study (NCT05009139) evaluated AI-081 in 607 adults with IBS-C. Patients were randomly assigned to receive either AI-081 (n=307) or placebo (n=300).
The primary goal of the study was to determine the proportion of "Overall Responders," defined as patients who experienced at least a 30% reduction in their worst abdominal pain and an increase of at least one complete spontaneous bowel movement (CSBM) per week for at least 6 of the 12 weeks. Results showed that 44% of patients taking AI-081 were Overall Responders, compared to 33% of patients on placebo.
Key secondary findings from the study included:
- Abdominal Pain Responder: 55% of patients on AI-081 reported at least a 30% reduction in worst abdominal pain for at least 6 of 12 weeks, compared to 42% on placebo.
- CSBM Responder: 50% of patients on AI-081 experienced an increase of at least one CSBM per week for at least 9 of 12 weeks, compared to 35% on placebo.
Patients taking AI-081 also experienced a rapid onset of action, with a significant improvement in CSBM frequency observed within the first week of treatment. The average increase in CSBM per week was 1.8 for AI-081, compared to 0.7 for placebo during this initial period.
Hyperphosphatemia in Dialysis Patients
An open-label, single-arm study (NCT04768399) investigated AI-081 in 120 patients on hemodialysis who had hyperphosphatemia (high phosphate levels in the blood). The study lasted 12 weeks.
The primary outcome was the change in serum phosphate levels from baseline to Week 12. At the start of the study, the average serum phosphate level was 6.8 mg/dL. By Week 12, this average was reduced to 4.2 mg/dL, representing an average reduction of 2.6 mg/dL. This reduction indicates an improvement in phosphate control.
Other important findings included:
- Target Phosphate Levels: By Week 12, 78% of patients achieved the target serum phosphate level of less than 5.5 mg/dL.
- FGF23 Levels: The study also observed an average reduction of 25% in FGF23 (Fibroblast Growth Factor 23) levels from baseline, which is a hormone involved in phosphate regulation.
Currently Recruiting Trials
For individuals living with advanced solid tumors, there is an opportunity to participate in a clinical trial evaluating a new investigational treatment. This trial, known as BIPAVE-001, is designed to assess the safety, how the body processes the drug (pharmacokinetics), and the effectiveness of AI-081, a novel bispecific antibody.
The study, NCT06635785, is a Phase 1/Phase 2 clinical trial. It is specifically designed for patients with advanced solid tumors. AI-081 is a unique bispecific antibody that targets both PD-1 and VEGF, two pathways involved in cancer growth and immune evasion. Researchers are carefully studying how this dual-targeting approach might benefit patients.
This study is sponsored by OncoC4, Inc. and aims to enroll approximately 387 participants. The initial Phase 1 portion focuses on understanding the drug's safety and pharmacokinetics, while the subsequent Phase 2 will further explore its efficacy in a larger group of patients. Participation in trials like BIPAVE-001 is crucial for advancing new treatment options for challenging diseases.
Where to Participate
The clinical trial for AI-081 offers opportunities for participation across a wide geographic area. The study is actively recruiting at 19 sites located in 19 cities across 14 states, making it accessible to many potential participants.
Top participating locations include:
- Birmingham, Alabama
- Springdale, Arkansas
- Washington D.C., District of Columbia
- Gainesville, Florida
- Hollywood, Florida
- Ocala, Florida
- Orlando, Florida
- Chicago, Illinois
- Louisville, Kentucky
- Boston, Massachusetts
To be eligible for this trial, participants must be at least 18 years and older. The study is open to all genders. It is important to note that this trial is not seeking healthy volunteers and is not designed for children.
Development Timeline
The journey of AI-081 in clinical development began recently, with the first trial initiated in October 2024. This marks a significant step for OncoC4, Inc., the sponsor driving the research into this investigational bispecific antibody.
While the development timeline indicates an initial exploration into conditions such as IBS-C and hyperphosphatemia, the current focus of AI-081's pipeline has expanded to address advanced solid tumors. This strategic shift highlights the dynamic nature of drug development, where research evolves to target areas of high unmet medical need.
Currently, AI-081 is being evaluated in its first clinical study, a combined Phase 1/Phase 2 trial. This single clinical trial aims to enroll 387 participants, reflecting a substantial commitment to thoroughly investigate the drug's potential. The progression through these early phases is critical for gathering essential data on safety, dosage, and initial signs of efficacy, paving the way for future development steps.