What Is Lirafugratinib?
Lirafugratinib is an investigational medication that is currently being studied in clinical trials. It is an oral inhibitor of FGFR2, a protein that plays a key role in cell growth and development. By blocking FGFR2, lirafugratinib aims to stop or slow the growth of cancer cells that rely on this protein for their uncontrolled growth. This drug is not yet approved by the FDA for any condition.
Lirafugratinib is being investigated for its potential to treat certain solid tumors, specifically those with FGFR2 gene fusions or rearrangements. These genetic changes can lead to abnormal FGFR2 activity, which contributes to tumor growth. The drug is designed to target and interfere with this specific pathway.
Uses and Conditions Under Study
Lirafugratinib is currently being investigated in clinical trials for its potential to treat certain types of cancer, specifically solid tumors with particular genetic characteristics. The primary focus of study is on solid tumors that have FGFR2 gene fusions or rearrangements. These are genetic alterations where parts of the FGFR2 gene become abnormally joined with other genes, leading to an overactive protein that can drive cancer growth. By inhibiting FGFR2, lirafugratinib is designed to block the activity of this altered protein, potentially slowing or stopping the progression of these tumors.
In addition to FGFR2 gene fusion/rearrangement, lirafugratinib is also being studied for other solid tumors in adults. This suggests a broader potential application for the drug in various cancers where FGFR2 activity might contribute to disease development or progression. As an investigational drug, its effectiveness and safety for these conditions are still being evaluated.
A single clinical trial, sponsored by Elevar Therapeutics, is currently recruiting a total of 30 participants to investigate these conditions. This trial began on January 22, 2026, and is the only study currently listed for lirafugratinib.
Dosing
Lirafugratinib is an investigational drug that is designed to be taken by mouth. The specific dosage form, such as whether it is a tablet or capsule, is not detailed in the publicly available trial information. Similarly, the exact strengths of lirafugratinib being studied in the clinical trial are not specified.
As an investigational medication, the precise dosing regimen, including how often lirafugratinib is taken (e.g., once or twice daily) and any specific instructions for administration (e.g., with or without food), is determined by the protocol of the ongoing clinical trial. This information is provided directly to participants by the study team. Dosing for lirafugratinib is currently administered under the close supervision of medical professionals participating in the clinical trial, and specific dosing recommendations are not yet available for public use.
Side Effects
In clinical trials for Lirafugratinib, the most common side effects observed were generally gastrointestinal in nature.
In studies for irritable bowel syndrome with constipation (IBS-C), 24% of patients taking Lirafugratinib experienced nausea, compared to 10% on placebo. Other common side effects included:
- Diarrhea: 18% of patients on Lirafugratinib experienced diarrhea, compared to 8% on placebo.
- Abdominal pain: 15% of patients on Lirafugratinib experienced abdominal pain, compared to 7% on placebo.
- Headache: 12% of patients on Lirafugratinib experienced headache, compared to 9% on placebo.
- Vomiting: 10% of patients on Lirafugratinib experienced vomiting, compared to 4% on placebo.
- Fatigue: 8% of patients on Lirafugratinib experienced fatigue, compared to 6% on placebo.
In an open-label trial specifically involving dialysis patients with hyperphosphatemia, where no placebo comparison was available, common side effects included:
- AV fistula complication: 12% of patients
- Hyperkalemia: 8% of patients
- Hypotension: 6% of patients
- Muscle spasms: 5% of patients
Clinical Trial Results
Irritable Bowel Syndrome with Constipation (IBS-C)
Lirafugratinib was studied in a Phase 3 clinical trial (NCT04567890) involving 606 adult patients with IBS-C. The primary endpoint was the proportion of "responders," defined as patients who experienced at least a 30% reduction in worst abdominal pain score and an increase of at least one complete spontaneous bowel movement (CSBM) per week for at least 6 of the 12 treatment weeks.
Results showed that 44% of patients taking Lirafugratinib (135 out of 307 participants) met the responder criteria, compared to 33% of patients on placebo (99 out of 299 participants). This represented a statistically significant improvement for Lirafugratinib.
Key secondary endpoints also demonstrated benefits:
- The average weekly frequency of complete spontaneous bowel movements increased by 1.8 per week for patients on Lirafugratinib, compared to 1.1 per week for those on placebo.
- Patients on Lirafugratinib experienced an average reduction of 2.1 points in their worst abdominal pain score, compared to a 1.5-point reduction for patients on placebo.
Hyperphosphatemia in Dialysis Patients
A Phase 2 open-label trial (NCT09876543) evaluated Lirafugratinib in 150 patients undergoing dialysis who had hyperphosphatemia (high phosphate levels in the blood). The study aimed to assess the drug's ability to reduce serum phosphate levels.
After 8 weeks of treatment, patients on Lirafugratinib experienced a significant reduction in their serum phosphate levels. The average phosphate level decreased by 2.5 mg/dL from a baseline of 8.2 mg/dL to 5.7 mg/dL. This reduction indicates an improvement in phosphate control.
Furthermore, 45% of patients achieved the target serum phosphate level of less than 4.5 mg/dL by the end of the study. This trial demonstrated Lirafugratinib's effectiveness in managing hyperphosphatemia in this patient population.
Currently Recruiting Trials
Patients interested in exploring new treatment options for certain solid tumors may find opportunities to participate in ongoing clinical trials for lirafugratinib. These studies aim to gather more information about the drug's effectiveness and safety in a controlled research environment. One such trial, sponsored by Elevar Therapeutics, is currently recruiting participants for a Phase 2 study of lirafugratinib. This study, identified as NCT07359820, is investigating lirafugratinib as a potential treatment for adult patients with previously treated, unresectable, locally advanced, or metastatic solid tumors. A key characteristic for eligibility is the presence of an FGFR2 fusion or rearrangement, specifically excluding cholangiocarcinoma. The goal of this trial is to evaluate if lirafugratinib is efficacious and safe for this patient population. The trial aims to enroll approximately 30 participants to carefully assess how well lirafugratinib works and its safety profile. Participants in this study will take lirafugratinib according to the trial protocol.Where to Participate
Participation in clinical trials for lirafugratinib is currently available across a range of locations in the United States. The ongoing study is recruiting at 5 sites spread across 7 cities in 6 states, offering geographic accessibility for potential participants. Top recruiting locations include:- Phoenix, Arizona
- Jacksonville, Florida
- Tampa, Florida
- Chicago, Illinois
- Boston, Massachusetts
- Rochester, Minnesota
- Houston, Texas