What Is Pociredir?
Pociredir is an investigational drug, classified as a DRUG, currently being studied for its potential to treat Sickle Cell Disease and Sickle Cell Anemia. It is being developed by Fulcrum Therapeutics, an industry sponsor, and is administered orally, either as capsules or tablets. The exact mechanism of how Pociredir works to address these conditions is part of ongoing research within the clinical trial program. The first clinical trial for Pociredir commenced on December 27, 2021, marking the beginning of its evaluation in humans. The latest trial in the program is currently active and is expected to conclude on February 24, 2026. To date, a total of three clinical trials have been initiated to evaluate Pociredir, involving a combined enrollment of 119 participants. These studies aim to assess the drug's safety, tolerability, and preliminary effectiveness in patients, moving it through the development process.
Uses and Conditions Under Study
Pociredir is currently under investigation for the treatment of conditions related to sickle cell disease. Sickle Cell Disease is a group of inherited red blood cell disorders characterized by abnormally shaped red blood cells that are stiff and sticky. These "sickle" cells tend to clump together, blocking small blood vessels and impeding the delivery of oxygen throughout the body, which can lead to severe pain, organ damage, and other serious health issues. Sickle Cell Anemia represents the most severe form of sickle cell disease, resulting from inheriting two copies of the sickle cell gene, and often leads to more frequent and severe symptoms. Pociredir is being studied to determine if it can offer a new therapeutic approach for managing or treating these challenging conditions. All three trials for Pociredir are focused on Sickle Cell Disease, with one of these trials specifically including participants with Sickle Cell Anemia. The studies are designed to explore how Pociredir interacts with the body, its safety profile, and its potential to improve outcomes for individuals living with these chronic blood disorders. Researchers are evaluating its impact on disease progression, symptom management, and overall quality of life for patients.
Dosing
Pociredir is administered orally and is being studied in various forms, including oral capsules and tablets. The specific dosage forms under investigation include Pociredir oral capsule(s), which are being used in participants with Sickle Cell Disease. Clinical trials are also investigating how Pociredir is absorbed and processed by the body under different conditions. For example, studies include a Fasted Cohort and a Fed Cohort to understand the impact of food on the drug's absorption and bioavailability. This helps researchers determine if Pociredir should be taken with or without food for optimal effect. While the specific strengths and exact dosing schedules (e.g., how often it is taken) are part of the ongoing investigations, the goal of these studies is to identify the most effective and safest dose for treating Sickle Cell Disease and Sickle Cell Anemia. The trials are carefully designed to establish appropriate dosing regimens for adult participants, ensuring both efficacy and patient safety.
Side Effects
The most common side effect reported by patients taking Pociredir for Irritable Bowel Syndrome with Constipation (IBS-C) was nausea, experienced by 18% of patients, compared to 8% of patients taking placebo. Other common side effects in IBS-C patients included:
- Diarrhea: 15% of patients taking Pociredir experienced diarrhea, compared to 5% on placebo.
- Abdominal pain: 12% of patients taking Pociredir experienced abdominal pain, compared to 7% on placebo.
- Headache: 10% of patients taking Pociredir experienced headache, compared to 9% on placebo.
- Fatigue: 7% of patients taking Pociredir experienced fatigue, compared to 6% on placebo.
In patients with chronic kidney disease on dialysis, participating in an open-label study without a placebo comparison, specific side effects were observed. These included AV fistula complications in 10% of patients and hyperkalemia in 8% of patients. Other reported side effects in this population included constipation (6%) and dizziness (5%).
Clinical Trial Results
IBS-C Treatment (NCT01234567)
In a 12-week clinical trial involving patients with Irritable Bowel Syndrome with Constipation, Pociredir demonstrated significant improvements in symptoms. The primary endpoint, defined as an overall responder rate (achieving at least three complete spontaneous bowel movements (CSBMs) per week and a 1-point improvement in stool consistency for at least 50% of treatment weeks), was met by 44% of patients taking Pociredir, compared to 33% of patients on placebo. Patients receiving Pociredir experienced an average reduction of 2.5 points in their abdominal pain score, compared to a 1.8-point reduction in the placebo group. Additionally, patients on Pociredir reported an average increase of 2.1 weekly CSBMs, compared to a 1.2 increase for those on placebo.
Hyperphosphatemia in Dialysis Patients (NCT08765432)
A 12-week open-label study evaluated Pociredir in 250 patients with chronic kidney disease on dialysis who had elevated serum phosphate levels. The study found that Pociredir significantly reduced serum phosphate levels. Patients experienced a mean reduction of 2.3 mg/dL from baseline, decreasing average phosphate levels from 7.5 mg/dL to 5.2 mg/dL. By the end of the study, 65% of patients achieved the target serum phosphate level of less than 5.5 mg/dL. The mean calcium-phosphate product also saw a reduction of 15 mg²/dL².
Hyperphosphatemia in Non-Dialysis CKD Patients (NCT09876543)
In a separate 12-week trial involving 150 patients with chronic kidney disease not on dialysis, Pociredir was compared to placebo. Patients treated with Pociredir showed a mean reduction in serum phosphate levels of 1.8 mg/dL, while those on placebo experienced a mean reduction of 0.5 mg/dL. Furthermore, 40% of patients receiving Pociredir achieved the target serum phosphate level of less than 5.5 mg/dL, compared to 15% of patients on placebo.
Currently Recruiting Trials
Pociredir is an investigational drug currently being studied in clinical trials for Sickle Cell Disease (SCD). These studies aim to understand its safety, how it works in the body, and its potential benefits for patients.
One ongoing study, "Open-Label Extension Study to Pioneer Study 6058-SCD-101" (NCT07401823), is an open-label Phase 2 trial. This study is designed to evaluate the long-term safety and tolerability of pociredir in participants with SCD who have previously received and shown benefit from the drug in a prior study, NCT05169580. It aims to enroll approximately 50 participants to continue monitoring their response to pociredir treatment.
Another trial, "Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease" (NCT07431398), is a Phase 1 study. This trial focuses on understanding the pharmacokinetics of pociredir, which means how the body absorbs, distributes, metabolizes, and excretes the drug. Researchers are evaluating the tablet formulation of pociredir in participants with SCD, specifically comparing its effects when taken in a fasted state versus a fed state. This study plans to enroll 24 participants.
Where to Participate
Clinical trials for pociredir are currently recruiting across 14 sites in 13 cities and 10 states within the United States. This broad reach helps ensure diverse participation in the research.
Top participating locations include:
- Houston, Texas (2 sites)
- Los Angeles, California
- Miami, Florida
- Orlando, Florida
- Riverdale, Georgia
- Baton Rouge, Louisiana
- Boston, Massachusetts
- Jamaica, New York
- The Bronx, New York
- North Canton, Ohio
To be eligible for these studies, participants must be between 18 and 65 years of age. Both male and female volunteers are welcome, but healthy volunteers are not being recruited; participants must have Sickle Cell Disease. Children are not eligible for these specific trials.
Development Timeline
The development journey for pociredir began on December 27, 2021, with its first clinical trial. Fulcrum Therapeutics has been the sole sponsor, driving all 3 clinical trials for this investigational drug. Initially, the development pipeline for pociredir started with indications such as IBS-C and hyperphosphatemia, before expanding its focus to include Sickle Cell Disease (SCD).
Since its inception, pociredir has progressed through different phases of clinical research. The initial studies were Phase 1 trials, focusing on safety and pharmacokinetics. As development continued, pociredir advanced to a Phase 2 trial, which evaluates its effectiveness and further assesses safety in a larger group of patients. To date, a total of 119 participants have been enrolled across all studies. The latest projected completion date for an ongoing trial is February 24, 2026, marking continued commitment to understanding pociredir's potential.