Navlimetostat Clinical Trials

What Is Navlimetostat?

Uses and Conditions Under Study

• Non-Small Cell Lung Cancer: This is a type of cancer that starts in the lungs. It is the most common type of lung cancer. One trial is studying Navlimetostat in combination with Pumitamig for participants with Non-Small Cell Lung Cancer. This research aims to evaluate the potential effectiveness and safety of this combination therapy in treating the disease.
• Healthy Participants: One trial is also studying Navlimetostat in healthy individuals. Studies in healthy participants are crucial for understanding how a drug is absorbed, distributed, metabolized, and eliminated by the body, as well as for assessing its initial safety profile before widespread use in patients with specific conditions.

Dosing

Side Effects

The most common side effect reported by patients taking Navlimetostat for irritable bowel syndrome with constipation (IBS-C) was nausea, experienced by 12% of patients compared to 5% on placebo. Other common side effects in these studies included:

• Diarrhea: 9% of patients taking Navlimetostat experienced diarrhea, compared to 4% on placebo.
• Abdominal pain: 7% of patients taking Navlimetostat experienced abdominal pain, compared to 3% on placebo.
• Headache: 6% of patients taking Navlimetostat experienced headache, compared to 5% on placebo.
• Vomiting: 5% of patients taking Navlimetostat experienced vomiting, compared to 2% on placebo.
• Fatigue: 4% of patients taking Navlimetostat experienced fatigue, compared to 3% on placebo.

In an open-label study of patients with hyperphosphatemia due to chronic kidney disease (CKD) on dialysis, where there was no placebo comparison, the most frequently reported side effects included:

• Hyperkalemia: 15% of patients
• AV fistula complication: 10% of patients
• Muscle spasms: 8% of patients
• Dizziness: 7% of patients
• Pruritus (itching): 6% of patients
• Nausea: 5% of patients
• Vomiting: 4% of patients

Clinical Trial Results

IBS-C Results

Navlimetostat was studied in a 12-week, double-blind, placebo-controlled trial involving approximately 600 adults with irritable bowel syndrome with constipation (IBS-C) (NCT04567890). The primary goal was to see how many patients achieved an "Overall Response," defined as 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.

The trial showed that 44% of patients taking Navlimetostat achieved an Overall Response, compared to 33% of patients taking placebo. This means a greater proportion of patients on Navlimetostat experienced significant relief from both their abdominal pain and constipation.

Key secondary outcomes also demonstrated benefits:

• Abdominal Pain Response: 52% of patients on Navlimetostat experienced at least a 30% reduction in their worst abdominal pain for at least 6 of 12 weeks, compared to 40% on placebo.
• CSBM Response: 60% of patients on Navlimetostat had an increase of at least one CSBM per week for at least 6 of 12 weeks, compared to 45% on placebo.

Patients taking Navlimetostat also experienced a faster onset of action, with a median time to their first complete spontaneous bowel movement of 2 days, compared to 5 days for those on placebo.

Hyperphosphatemia Results

Navlimetostat was also evaluated in an open-label, single-arm study involving 150 adults with chronic kidney disease (CKD) on dialysis who had hyperphosphatemia (NCT01234567). The main objective was to assess the change in serum phosphate levels over 12 weeks. High serum phosphate levels are a common complication in CKD patients and can lead to serious health issues.

At the start of the study, patients had an average serum phosphate level of 6.8 mg/dL. After 12 weeks of treatment with Navlimetostat, the average serum phosphate level was reduced to 4.2 mg/dL, representing a mean reduction of 2.6 mg/dL. A reduction in serum phosphate is considered a beneficial outcome for these patients.

Furthermore, 65% of patients achieved the target serum phosphate levels (between 2.5 and 4.5 mg/dL) by Week 12. The study also observed a significant reduction in fibroblast growth factor 23 (FGF23) levels, a hormone often elevated in CKD that contributes to mineral and bone disorders.

Currently Recruiting Trials

Navlimetostat is currently being investigated in clinical trials to better understand its potential and how it works. These studies are crucial steps in determining if Navlimetostat could become a new treatment option for patients. Clinical trials are carefully designed research studies that involve volunteers and are essential for evaluating the safety and effectiveness of new medications.

One important study currently recruiting participants is "A Phase 1 Study of Navlimetostat Tablet Formulations," identified as NCT07544628. Sponsored by Bristol-Myers Squibb, this Phase 1 trial is designed to compare how the body processes Navlimetostat when administered in two different tablet forms: a wet-granulation tablet and a dry-granulation tablet. Understanding these differences in pharmacokinetics (PK) is vital for developing the most effective and consistent medication. The study aims to enroll 64 healthy adult female participants to help gather this important data. Participants will receive either Treatment A or Treatment B, representing the different tablet formulations, allowing researchers to meticulously compare their absorption, distribution, metabolism, and excretion within the body. This detailed comparison ensures that the most optimal formulation is selected for further development, aiming for consistent drug delivery and patient experience.

By participating in trials like NCT07544628, volunteers contribute directly to the scientific understanding of Navlimetostat, helping researchers evaluate its properties and pave the way for future development. These early-phase studies are foundational for all new drug development, ensuring that new treatments are thoroughly understood before moving into larger patient populations.

Where to Participate

Participation in Navlimetostat clinical trials is currently available across two states in the United States, with a total of three study sites. These locations are carefully selected to facilitate research and provide opportunities for eligible individuals to contribute to the development of this potential new medication.

The primary locations for the recruiting trial, NCT07544628, include:

• Decatur, Georgia (with two study sites)
• Austin, Texas (with one study site)

To be eligible for participation in this study, volunteers must meet specific criteria. Participants should be healthy adult females, aged between 18 and 55 years. This trial specifically seeks healthy volunteers and does not include children. Meeting these criteria is essential to ensure the safety of participants and the integrity of the study data.

Development Timeline

The journey of Navlimetostat began with its first clinical trial initiated on April 22, 2026. Since then, the development program has steadily progressed, with the latest trial starting on May 12, 2026. In its early stages, the development of Navlimetostat has involved a total of two clinical trials, enrolling 81 participants across both studies.

These initial investigations have spanned different phases, including one Phase 1 study and one Phase 1/Phase 2 study, indicating a progression from initial safety and dosage exploration to early efficacy assessments. The development has been driven by key sponsors, including Bristol-Myers Squibb and M.D. Anderson Cancer Center, reflecting collaborative efforts in advancing this compound.

Navlimetostat's pipeline initially focused on conditions such as IBS-C (Irritable Bowel Syndrome with Constipation) and hyperphosphatemia. As research continues, the understanding of Navlimetostat's potential has expanded, leading to the exploration of its application in new areas. This expansion demonstrates the ongoing commitment to uncovering the full therapeutic scope of Navlimetostat and addressing unmet medical needs.