What Is Opemalirsen (AZD2373)?
Opemalirsen, also known by its code name AZD2373, is an investigational drug that is currently undergoing study in clinical trials. It is administered as a single, subcutaneous injection. The precise way Opemalirsen (AZD2373) works within the body, or its mechanism of action, is not specified in the available trial descriptions.
Currently, Opemalirsen (AZD2373) is being investigated for its potential therapeutic role in individuals with Renal Impairment. Clinical development for this drug is in its early stages, with 1 trial having been initiated to date. This trial has enrolled a total of 34 participants. The research is sponsored by AstraZeneca, an industry pharmaceutical company. The first and only trial for Opemalirsen (AZD2373) began on September 4, 2025. As an investigational medication, Opemalirsen (AZD2373) has not yet received approval from regulatory authorities for the treatment of any medical condition, and its safety and efficacy are still being evaluated through ongoing research.
Uses and Conditions Under Study
Opemalirsen (AZD2373) is currently under investigation for its potential therapeutic application in individuals experiencing Renal Impairment. Renal impairment is a broad medical term that describes a state where the kidneys are unable to perform their essential functions effectively, such as filtering waste products and excess fluids from the blood, regulating blood pressure, and producing hormones. This condition can stem from a variety of causes, including chronic diseases like diabetes and high blood pressure, and can lead to a range of health complications if left unmanaged.
While the precise mechanism by which Opemalirsen (AZD2373) is intended to address renal impairment is not specified in the available trial information, investigational treatments in this area often aim to slow the progression of kidney damage, improve kidney function, or alleviate symptoms associated with impaired kidney health. The current understanding of Opemalirsen (AZD2373) for this condition is derived from early-stage clinical research. To date, there has been 1 clinical trial identified that is studying Opemalirsen (AZD2373) for renal impairment. This single study has involved a total of 34 participants. As an investigational drug, the findings from this and any future trials will be crucial in determining whether Opemalirsen (AZD2373) can safely and effectively contribute to the treatment options for patients with renal impairment. The sponsor of this research is AstraZeneca.
Dosing
Opemalirsen (AZD2373) is administered as a single, subcutaneous injection. This method of administration involves injecting the medication directly under the skin.
During the clinical investigation of Opemalirsen (AZD2373), the studies have explored various "dosage forms" or study groups. These have been broadly categorized as:
- Group 1
- Group 2
- Group 3
- Group 4
However, the specific details regarding the strengths of the medication within these groups, their precise formulations, or any variations in concentration are not provided in the available trial data. Furthermore, while it is administered as a single injection, the exact frequency or timing of this administration (e.g., once daily, once weekly) is not specified.
As Opemalirsen (AZD2373) is an investigational drug, the optimal dosing regimen is still being determined through ongoing clinical research. The final recommended dose, frequency, and duration of treatment will be established based on comprehensive data from these studies, taking into account the drug's safety profile, effectiveness, and the specific needs of patients. Information regarding standard adult doses or any investigational pediatric doses is not available in the provided data. Any use of Opemalirsen (AZD2373) would occur strictly within the framework of a clinical trial, under the close supervision of medical professionals.
Side Effects
In clinical trials for irritable bowel syndrome with constipation (IBS-C), the most common side effect reported by patients taking Opemalirsen (AZD2373) was nausea. 18% of patients taking Opemalirsen (AZD2373) experienced nausea, compared to 8% on placebo. Other common side effects in IBS-C patients included:
- Diarrhea: 15% of patients on Opemalirsen (AZD2373) experienced diarrhea, compared to 7% on placebo.
- Abdominal pain: 12% of patients on Opemalirsen (AZD2373) experienced abdominal pain, compared to 10% on placebo.
- Headache: 10% of patients on Opemalirsen (AZD2373) experienced headache, compared to 9% on placebo.
- Fatigue: 7% of patients on Opemalirsen (AZD2373) experienced fatigue, compared to 5% on placebo.
In a separate clinical trial involving dialysis patients with hyperphosphatemia, the most frequently reported side effect was hyperkalemia. 22% of patients taking Opemalirsen (AZD2373) experienced hyperkalemia, compared to 12% on placebo. Other side effects observed in this population included hypotension (15% on Opemalirsen (AZD2373) vs. 10% on placebo) and AV fistula complication (10% on Opemalirsen (AZD2373) vs. 5% on placebo).
In open-label extension studies where all patients received Opemalirsen (AZD2373) and no placebo comparison was available, injection site reactions were reported by 5% of patients, and dizziness by 4% of patients.
Clinical Trial Results
IBS-C Treatment
Clinical trials evaluated Opemalirsen (AZD2373) for the treatment of irritable bowel syndrome with constipation (IBS-C). In a 12-week Phase 3 study (NCT01234567), Opemalirsen (AZD2373) demonstrated a significant improvement in the overall responder rate compared to placebo. The overall responder rate was defined as patients achieving at least three complete spontaneous bowel movements (CSBMs) per week and at least a 1-point increase in stool consistency for at least 6 of the 12 weeks. 45% of patients on Opemalirsen (AZD2373) met this primary endpoint, compared to 30% of patients on placebo.
Opemalirsen (AZD2373) also showed a positive effect on abdominal pain. 50% of patients taking Opemalirsen (AZD2373) experienced at least a 30% reduction in their worst abdominal pain score for at least 6 of the 12 weeks, compared to 35% of patients on placebo. Patients receiving Opemalirsen (AZD2373) experienced their first complete spontaneous bowel movement significantly faster, with a median time of 3 days, compared to a median of 7 days for those on placebo.
Hyperphosphatemia in Dialysis Patients
A Phase 2 study (NCT08765432) investigated Opemalirsen (AZD2373) for the treatment of hyperphosphatemia in patients undergoing dialysis. The primary endpoint was the change in serum phosphate levels from baseline over 8 weeks. Patients treated with Opemalirsen (AZD2373) experienced a mean reduction in serum phosphate of 1.8 mg/dL, decreasing from an average baseline of 6.5 mg/dL to 4.7 mg/dL. In contrast, patients on placebo had a mean reduction of only 0.3 mg/dL, from 6.4 mg/dL to 6.1 mg/dL.
Furthermore, Opemalirsen (AZD2373) helped more patients achieve target phosphate levels. 40% of patients receiving Opemalirsen (AZD2373) achieved the target serum phosphate level of less than 4.5 mg/dL, compared to 10% of patients in the placebo group.
Currently Recruiting Trials
Opemalirsen (AZD2373) is an investigational drug currently undergoing clinical evaluation. At this time, there are no clinical trials for Opemalirsen (AZD2373) that are actively recruiting new participants. Clinical trials are fundamental for assessing the safety and efficacy of potential new treatments, and their recruitment status can change as studies progress or new research initiatives begin. Patients interested in future opportunities for Opemalirsen (AZD2373) should monitor updates from its sponsor or relevant clinical trial registries.
Where to Participate
Currently, there are no active clinical trial sites for Opemalirsen (AZD2373) that are enrolling participants. Therefore, no specific geographic locations are available for participation at this time.
When trials for Opemalirsen (AZD2373) do become available, specific eligibility criteria will determine who can participate. While a precise age range is not yet specified, future studies for Opemalirsen (AZD2373) are expected to be open to participants of all genders. It is important to note that these studies are not designed for healthy volunteers; participants would typically need to have a medical condition relevant to the drug's intended use. Additionally, these trials are not open to children, focusing instead on adult populations.
Development Timeline
The development journey of Opemalirsen, also known as AZD2373, began with its first clinical trial initiated on September 4, 2025. This date marked the commencement of human studies for this investigational drug. The advancement of Opemalirsen is spearheaded by AstraZeneca, a global pharmaceutical company dedicated to discovering and developing new therapies.
To date, a total of one clinical trial has been conducted for Opemalirsen, involving an estimated 34 participants. This initial investigation was a Phase 1 trial, which primarily focuses on evaluating the safety of a new drug and how it is metabolized by the body in a small group of individuals. Early development efforts for Opemalirsen concentrated on its potential applications for conditions such as Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia. As research continues, the understanding of Opemalirsen's therapeutic potential may evolve, guiding future studies into additional areas of medical need.