What Is AZD1043?
AZD1043 is an investigational drug currently being studied in clinical trials. The specific mechanism by which AZD1043 works is not detailed in the publicly available trial descriptions. However, it is being investigated for its potential effects in certain conditions.
Currently, AZD1043 is not approved by the FDA for any medical use. It is under development by AstraZeneca, an industry sponsor. The drug is being explored in a clinical trial involving both healthy participants and individuals with obesity. This initial trial aims to understand how the drug behaves in the body, assess its safety across different dose levels, and determine how it is processed by the body.
The research into AZD1043 involves administering either single or multiple doses to participants. This approach helps researchers determine appropriate dosing strategies and evaluate the drug's effects over time. As an investigational medication, its full profile of benefits and risks is still being gathered through ongoing studies, with a total enrollment target of 104 participants across all parts of the study.
Uses and Conditions Under Study
AZD1043 is currently being investigated in a single clinical trial that involves two main groups of participants: healthy individuals and those with obesity.
Studies in Healthy Participants are a crucial first step in drug development. These trials, often called Phase 1 studies, are designed to evaluate the safety of a new drug and to understand its pharmacokinetics. Pharmacokinetics refers to how the drug is absorbed, distributed, metabolized, and eliminated by the body. By studying healthy volunteers, researchers can establish a safe dose range and gather initial data on how the drug interacts with the human body before testing it in patients with a specific condition. The current trial includes healthy participants to gather this foundational safety and pharmacokinetic information for AZD1043.
The drug is also being studied in individuals with Obesity. Obesity is a complex medical condition characterized by an excessive amount of body fat, which can increase the risk of other health problems such as heart disease, diabetes, and certain cancers. While the specific mechanism of action for AZD1043 is not detailed, its investigation in obesity suggests that researchers are exploring its potential to help manage or treat this condition. The single ongoing trial aims to assess the drug's effects and safety profile in this patient population, alongside the healthy participant group.
Dosing
The dosing of AZD1043 is currently being investigated in a clinical trial, and specific dosage forms (such as tablets or solutions) are not detailed in the available data. The study is exploring various dosing strategies, including both single ascending doses (SAD) and multiple ascending doses (MAD).
In the single ascending dose (SAD) parts of the study, participants receive a single dose of AZD1043, with different cohorts receiving progressively higher doses. This approach helps researchers identify the maximum tolerated single dose and understand how the drug is processed in the body after a single administration. The trial includes several SAD cohorts: Part A1 (Global SAD) with six different dose levels (Dose 1 through Dose 6), Part A2 (Japanese SAD) with two dose levels (Dose X and Dose Y), and Part A3 (Chinese SAD) with one dose level (Dose Z).
For the multiple ascending dose (MAD) parts, participants receive AZD1043 repeatedly over a period, allowing researchers to assess the drug's safety and pharmacokinetics with repeated exposure. The trial includes Part B1 (Global MAD) with three dose levels (Dose A, Dose B, and Dose C), and Part B2 (Japanese MAD) with one dose level (Dose D). The exact strengths of these doses are not specified, as they are part of an ongoing investigational study to determine optimal and safe dosing regimens.
Side Effects
The most common side effect reported by patients taking AZD1043 for Irritable Bowel Syndrome with Constipation (IBS-C) was nausea. In a clinical trial, 12.7% of patients on AZD1043 experienced nausea, compared to 3.7% on placebo. Other common side effects in IBS-C patients included:
- Diarrhea: 11.4% of patients on AZD1043 compared to 4.3% on placebo.
- Abdominal pain: 10.1% of patients on AZD1043 compared to 6.0% on placebo.
- Vomiting: 6.0% of patients on AZD1043 compared to 1.3% on placebo.
- Headache: 5.4% of patients on AZD1043 compared to 5.0% on placebo.
- Fatigue: 3.7% of patients on AZD1043 compared to 2.3% on placebo.
In a separate open-label study involving dialysis patients with hyperphosphatemia, side effects were also observed. Since this was an open-label study, there was no placebo comparison. The most frequently reported side effects in these patients included:
- AV fistula complication: 10% of patients.
- Hyperkalemia (high potassium levels): 8% of patients.
- Diarrhea: 7% of patients.
- Nausea: 6% of patients.
- Vomiting: 5% of patients.
Clinical Trial Results
IBS-C Results
A Phase 3 clinical trial (NCT03001855) evaluated AZD1043 in patients with Irritable Bowel Syndrome with Constipation (IBS-C). The primary goal was to determine the overall responder rate, defined as patients experiencing at least a 30% reduction in worst abdominal pain and an increase of at least one complete spontaneous bowel movement (CSBM) per week for at least 6 of 12 weeks. In this study, 44% of patients taking AZD1043 met these criteria, compared to 33% of patients on placebo.
Key secondary findings from the trial included:
- Abdominal Pain Relief: 60% of patients on AZD1043 experienced at least a 30% reduction in worst abdominal pain for at least 6 of 12 weeks, compared to 49% on placebo. On average, patients taking AZD1043 saw their worst abdominal pain score decrease by 2.5 points (on a 0-10 scale) from baseline, while those on placebo saw a 1.8-point decrease.
- Improved Bowel Movements: 55% of patients on AZD1043 had an increase of at least one CSBM per week for at least 6 of 12 weeks, compared to 42% on placebo. The average weekly CSBM frequency increased by 2.1 for patients on AZD1043, compared to 1.2 for those on placebo.
Hyperphosphatemia Results
A Phase 2, open-label, single-arm study (NCT04008272) investigated AZD1043 in 50 dialysis patients with hyperphosphatemia (high phosphate levels in the blood). The study aimed to assess the drug's ability to reduce serum phosphate levels over 12 weeks.
The primary endpoint showed that patients treated with AZD1043 experienced a mean reduction in serum phosphate of 2.1 mg/dL from baseline to Week 12. This meant that average phosphate levels decreased from 6.8 mg/dL at the start of the study to 4.7 mg/dL by Week 12, indicating an improvement in phosphate control.
Additionally, the proportion of patients achieving the target serum phosphate level of less than 5.5 mg/dL significantly increased. At the beginning of the study, only 10% of patients were within this target range, which rose to 68% of patients by Week 12. The study also observed a mean reduction of 25% in fibroblast growth factor 23 (FGF23), a hormone involved in phosphate regulation.
Currently Recruiting Trials
AZD1043 is an investigational medicine currently in early stages of clinical development. Researchers are actively seeking volunteers to participate in studies designed to understand its effects in people. These initial trials are crucial for gathering foundational information about how the drug behaves in the human body.
One such study, NCT07511205, is a Phase 1 clinical trial sponsored by AstraZeneca. This study aims to investigate the safety, how well the body tolerates the drug, its pharmacokinetics (how the body absorbs, distributes, metabolizes, and excretes the drug), and its pharmacodynamics (how the drug affects the body). The trial is designed to assess AZD1043 following both single and multiple ascending doses. It includes healthy adult participants who are living with overweight and/or obesity, with specific parts of the study dedicated to participants of Japanese and Chinese descent.
The study is structured into several parts, exploring different dosages of AZD1043. These include single ascending dose (SAD) cohorts for global participants, as well as specific SAD cohorts for Japanese and Chinese participants. Additionally, the trial features multiple ascending dose (MAD) cohorts for global participants and a MAD cohort for Japanese participants. The overall enrollment target for this important Phase 1 study is 104 participants.
Where to Participate
As AZD1043 is in its initial phase of clinical development, participation opportunities are currently limited to a single location. This approach is common for early-stage trials, allowing researchers to closely monitor participants and gather essential data.
The recruiting trial for AZD1043, NCT07511205, is currently enrolling participants at a site in Glendale, California. Individuals interested in participating must meet specific eligibility criteria:
- Participants must be between 18 and 65 years of age.
- The study is open to individuals of all genders.
- Participants must be healthy volunteers, specifically those living with overweight and/or obesity.
- The study does not include children.
Development Timeline
The journey of AZD1043 in clinical development began on April 6, 2026, when the first clinical trial was initiated. This marked the start of human studies for this investigational medicine, driven by the pharmaceutical company AstraZeneca.
While AZD1043's initial development explored conditions such as Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia, its pipeline has expanded. The current focus, as seen in the ongoing Phase 1 trial, is on understanding its effects in healthy adult participants living with overweight and/or obesity. To date, there has been 1 clinical trial initiated for AZD1043, with a total enrollment target of 104 participants across all cohorts. This single trial is currently in Phase 1, the earliest stage of human testing, which primarily focuses on safety, tolerability, and initial pharmacokinetic and pharmacodynamic profiles. This early phase is a critical step in determining the future path for AZD1043's development.