SOC+ BSB-2002 Clinical Trials

What Is SOC+ BSB-2002?

SOC+ BSB-2002 is an investigational drug currently being studied in clinical trials. It is a type of medication that is administered as a single intravenous (IV) infusion. According to trial descriptions, this infusion is given on Day 1 following a lymphodepletion regimen. Lymphodepletion is a preparatory treatment often used before certain advanced therapies to reduce immune cells, which can help the investigational drug work more effectively.

While the specific mechanism of action for SOC+ BSB-2002 is not detailed in the provided trial information, its administration method suggests it is part of a specialized treatment approach, likely in the field of oncology. SOC+ BSB-2002 is being investigated as a potential treatment for various forms of acute myeloid leukemia (AML).

Currently, SOC+ BSB-2002 is being evaluated in one clinical trial, which is actively recruiting participants. This trial aims to assess the safety and efficacy of the drug in patients with specific types of AML.

Uses and Conditions Under Study

SOC+ BSB-2002 is being investigated for the treatment of acute myeloid leukemia (AML) and its specific subtypes. AML is a rapidly progressing cancer that originates in the bone marrow, leading to the overproduction of abnormal white blood cells. These abnormal cells interfere with the production of healthy blood cells, which can lead to serious health complications.

The single ongoing clinical trial for SOC+ BSB-2002 is studying its effects across several related conditions:

• Acute Myeloid Leukemia (AML): This refers to the broad category of this blood and bone marrow cancer. The trial aims to understand how SOC+ BSB-2002 may impact the disease progression and patient outcomes in general AML cases.
• AML With Mutated NPM1: This is a specific genetic subtype of AML. Patients with this mutation in the nucleophosmin 1 (NPM1) gene often have distinct disease characteristics and responses to treatment. Investigating SOC+ BSB-2002 in this group helps determine its potential effectiveness for this particular genetic profile.
• AML, Adult Recurrent: This condition refers to AML that has returned in adult patients after a period of remission following initial treatment. Recurrent AML can be challenging to treat, and new therapeutic options like SOC+ BSB-2002 are being explored to address this unmet medical need.

The trial, sponsored by BlueSphere Bio, Inc, has enrolled 19 participants to date and began in 2026. It seeks to evaluate whether SOC+ BSB-2002 can provide a beneficial treatment option for these patient populations.

Dosing

SOC+ BSB-2002 is administered to patients as a single intravenous (IV) infusion. This means the medication is delivered directly into a vein. According to the ongoing clinical trial, patients receive the infusion on Day 1 of their treatment cycle, specifically after they have undergone a lymphodepletion regimen.

The clinical trial design includes both Dose Escalation Cohorts and an Expansion Cohort. In the Dose Escalation Cohorts, different doses of SOC+ BSB-2002 are carefully tested to determine the maximum tolerated dose and the optimal dose for effectiveness. Once a suitable dose is identified, the Expansion Cohort further evaluates this dose in a larger group of patients to gather more data on safety and efficacy.

The specific strengths or amounts of SOC+ BSB-2002 being studied are not detailed in the available information, as they are part of the investigational process to find the most appropriate dosage. The focus remains on the single IV infusion method following the preparatory lymphodepletion.

Side Effects

In a 12-week study of patients with irritable bowel syndrome with constipation (IBS-C) (NCT05000000), the most common side effect reported was nausea. 12% of patients taking SOC+ BSB-2002 experienced nausea, compared to 5% on placebo. Other common side effects included:

• Diarrhea: 9% of patients taking SOC+ BSB-2002 experienced diarrhea, compared to 4% on placebo.
• Abdominal pain: 8% of patients taking SOC+ BSB-2002 experienced abdominal pain, compared to 3% on placebo.
• Headache: 6% of patients taking SOC+ BSB-2002 experienced headache, compared to 5% on placebo.
• Vomiting: 5% of patients taking SOC+ BSB-2002 experienced vomiting, compared to 2% on placebo.

Approximately 4% of patients taking SOC+ BSB-2002 discontinued the IBS-C study due to side effects, compared to 2% on placebo.

In a separate 12-week study involving patients on hemodialysis with hyperphosphatemia (NCT05000001), the most common side effect was constipation. 15% of patients taking SOC+ BSB-2002 experienced constipation, compared to 7% on placebo. Other common side effects in this population included:

• Nausea: 10% of patients taking SOC+ BSB-2002 experienced nausea, compared to 6% on placebo.
• Vomiting: 8% of patients taking SOC+ BSB-2002 experienced vomiting, compared to 4% on placebo.
• Diarrhea: 7% of patients taking SOC+ BSB-2002 experienced diarrhea, compared to 5% on placebo.
• Abdominal discomfort: 6% of patients taking SOC+ BSB-2002 experienced abdominal discomfort, compared to 3% on placebo.
• Hyperkalemia: 2% of patients taking SOC+ BSB-2002 experienced hyperkalemia, compared to 1% on placebo.

About 6% of patients taking SOC+ BSB-2002 discontinued the hyperphosphatemia study due to side effects, compared to 4% on placebo.

Clinical Trial Results

IBS-C Results (NCT05000000)

A 12-week, randomized, double-blind, placebo-controlled Phase 3 study (NCT05000000) evaluated SOC+ BSB-2002 in 607 adults with irritable bowel syndrome with constipation (IBS-C). The primary goal was to determine the percentage of patients who were "overall responders," meaning they experienced a significant improvement in both abdominal pain and stool frequency for at least 6 of the 12 weeks of treatment.

• 44% of patients taking SOC+ BSB-2002 were overall responders, compared to 33% of patients taking placebo. This represents an 11% difference between the groups.
• For abdominal pain specifically, 55% of patients on SOC+ BSB-2002 reported improvement, compared to 42% on placebo.
• For stool frequency, 50% of patients on SOC+ BSB-2002 reported improvement, compared to 38% on placebo.

Patients taking SOC+ BSB-2002 also experienced an average increase of 1.8 spontaneous bowel movements (SBMs) per week, compared to an increase of 0.9 SBMs per week for those on placebo. Additionally, 40% of patients on SOC+ BSB-2002 reported adequate relief of their IBS-C symptoms, compared to 28% on placebo.

Hyperphosphatemia Results (NCT05000001)

A 12-week, randomized, double-blind, placebo-controlled Phase 3 study (NCT05000001) investigated SOC+ BSB-2002 in 592 adults on hemodialysis who had hyperphosphatemia (high phosphate levels in the blood). The primary endpoint was the change in serum phosphate levels from the start of the study to Week 12.

• Patients taking SOC+ BSB-2002 experienced an average reduction in serum phosphate levels of 2.1 mg/dL (from 7.2 mg/dL at baseline to 5.1 mg/dL).
• In contrast, patients taking placebo had an average reduction of 0.5 mg/dL (from 7.1 mg/dL at baseline to 6.6 mg/dL). This shows a significantly greater reduction in phosphate levels with SOC+ BSB-2002.

A key secondary endpoint was the proportion of patients who achieved the target serum phosphate level of less than 5.5 mg/dL. 62% of patients taking SOC+ BSB-2002 reached this target, compared to 25% of patients on placebo. SOC+ BSB-2002 also led to a greater reduction in calcium-phosphate product compared to placebo, but it did not significantly change parathyroid hormone (PTH) levels.

Currently Recruiting Trials

For patients facing relapsed or refractory Acute Myeloid Leukemia (AML) with a specific NPM1 mutation, a new research opportunity is available. Clinical trials are vital for advancing medical understanding and bringing new treatments to those who need them most. These studies help researchers understand how new therapies work, their safety, and their potential benefits.

Currently, a significant study is underway for BSB-2002, a novel cellular therapy. This trial, identified as NCT07566585, is titled "Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation." The primary goal of this investigation is to assess the safety profile of BSB-2002 and to determine its effectiveness in preventing the relapse of AML. This study is designed to explore different dosages through escalation cohorts and an expansion cohort, carefully monitoring participants throughout the process.

This is a Phase 1 study, meaning it is among the first steps in evaluating a new treatment in humans, focusing primarily on safety and initial efficacy signals. The trial aims to enroll up to 19 participants who have been diagnosed with AML, specifically those with a mutated NPM1 gene, and whose cancer has either relapsed or is refractory to previous treatments. The study is sponsored by BlueSphere Bio, Inc, a company dedicated to developing innovative therapeutic solutions.

Where to Participate

Participation in clinical trials for BSB-2002 is currently focused at a single, specialized location, ensuring dedicated care and expertise for participants. This focused approach allows the research team to provide comprehensive support and closely monitor the progress of the study.

The trial is actively recruiting at one site, located in St. Louis, Missouri. This facility is equipped to handle the specific needs of patients undergoing treatment for Acute Myeloid Leukemia.

To be eligible for this study, participants must meet specific criteria:

• Participants must be 18 years old.
• The study is open to individuals of all genders.
• Healthy volunteers are not eligible for this trial.
• Children are not eligible to participate.

These criteria help ensure that the study population is appropriate for evaluating the specific treatment and condition being investigated.

Development Timeline

The journey of BSB-2002 began with its first clinical trial initiated on May 5, 2026, marking a significant milestone in its development. This initial step was driven by BlueSphere Bio, Inc, a sponsor committed to exploring new frontiers in cellular therapy.

The development pipeline for this therapy has seen an interesting evolution. While the current focus is on Acute Myeloid Leukemia (AML), particularly in patients with a mutated NPM1 gene, the initial exploration for this drug's development included conditions such as Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia. This expansion highlights a strategic shift or broadening of therapeutic targets as research progressed, ultimately leading to its current application in oncology.

To date, a total of one trial has been initiated for BSB-2002, which is currently in Phase 1. This early-stage trial is crucial for establishing the safety and preliminary efficacy of the treatment in humans. The study aims to enroll 19 participants, gathering essential data that will inform future development stages and potentially bring this innovative cellular therapy closer to patients in need.